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What changed in Ocugen, Inc.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of Ocugen, Inc.'s 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.

+554 added−513 removedSource: 10-K (2026-03-04) vs 10-K (2025-03-05)

Top changes in Ocugen, Inc.'s 2025 10-K

554 paragraphs added · 513 removed · 395 edited across 8 sections

Item 1. Business

Business — how the company describes what it does

136 edited+66 added−65 removed351 unchanged
Biggest changeAt the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access, sometimes establishing Prescription Drug Affordability Boards (or similar entities) to review high-cost drugs, and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
Biggest changeAt the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access, sometimes establishing Prescription Drug Affordability Boards (or similar entities) to review high-cost drugs, and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. 31 Table of Contents We expect that additional federal, state, and foreign healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in limited coverage and reimbursement and reduced demand for our products, once approved, or additional pricing pressures.
CanSinoBIO is responsible for the CMC development and manufacture of clinical supplies of such product candidates and is responsible for the costs associated with such activities.
CanSinoBIO is responsible for the CMC development and manufacture of clinical supplies of such product candidates and is responsible for the costs associated with such activities.
The process required by the FDA before product candidates may be marketed in the United States generally involves the following: • completion of preclinical laboratory tests, animal studies, and formulation studies in compliance with the FDA's Good Laboratory Practice ("GLP") regulations, applicable requirements for the human use of laboratory animals, such as the Animal Welfare Act ("AWA"), or other applicable regulations; • submission to the FDA of an IND application, which must become effective before human clinical trials may begin at United States clinical trial sites; • approval by an Institutional Review Board ("IRB") for each clinical site, or centrally, before a clinical trial may be initiated at that site; • adequate and well-controlled human clinical trials to establish the safety and efficacy, in the case of a drug product candidate, or safety, purity, and potency, in the case of a biological product candidate, for its intended use, performed in accordance with Good Clinical Practice ("GCP") and additional requirements for the protection of human research subjects and their health information; • development of manufacturing processes to ensure the product candidate's identity, strength, quality, purity, and potency in compliance with current GMP; • submission to the FDA of a New Drug Application ("NDA"), in the case of a drug product candidate, or a BLA, in the case of a biological product candidate, including results of preclinical testing, detailed information about the CMC, and proposed labeling and packaging for the product candidate; • satisfactory completion of an FDA advisory committee review, if applicable; • satisfactory completion of an FDA inspection of the manufacturing facility or facilities at which the products are produced to assess compliance with current GMP, and to assure that the facilities, methods, and controls are adequate to preserve the therapeutics' identity, strength, quality, purity, and potency as well as satisfactory completion of an FDA inspection of selected clinical sites, selected clinical investigators to determine GCP compliance, and payment of user fees; and • FDA review and approval of the NDA, or licensure of a BLA, to permit commercial marketing for particular indications for use, including agreement on post-marketing commitments, if applicable.
The process required by the FDA before product candidates may be marketed in the United States generally involves the following: 19 Table of Contents • completion of preclinical laboratory tests, animal studies, and formulation studies in compliance with the FDA's Good Laboratory Practice ("GLP") regulations, applicable requirements for the human use of laboratory animals, such as the Animal Welfare Act ("AWA"), or other applicable regulations; • submission to the FDA of an IND application, which must become effective before human clinical trials may begin at United States clinical trial sites; • approval by an Institutional Review Board ("IRB") for each clinical site, or centrally, before a clinical trial may be initiated at that site; • adequate and well-controlled human clinical trials to establish the safety and efficacy, in the case of a drug product candidate, or safety, purity, and potency, in the case of a biological product candidate, for its intended use, performed in accordance with Good Clinical Practice ("GCP") and additional requirements for the protection of human research subjects and their health information; • development of manufacturing processes to ensure the product candidate's identity, strength, quality, purity, and potency in compliance with current GMP; • submission to the FDA of a New Drug Application ("NDA"), in the case of a drug product candidate, or a BLA, in the case of a biological product candidate, including results of preclinical testing, detailed information about the CMC, and proposed labeling and packaging for the product candidate; • satisfactory completion of an FDA advisory committee review, if applicable; • satisfactory completion of an FDA inspection of the manufacturing facility or facilities at which the products are produced to assess compliance with current GMP, and to assure that the facilities, methods, and controls are adequate to preserve the therapeutics' identity, strength, quality, purity, and potency as well as satisfactory completion of an FDA inspection of selected clinical sites, selected clinical investigators to determine GCP compliance, and payment of user fees; and • FDA review and approval of the NDA, or licensure of a BLA, to permit commercial marketing for particular indications for use, including agreement on post-marketing commitments, if applicable.
We believe these broad ODD, RMAT, and OMPD designations demonstrate that OCU400 has the potential to be a broad-spectrum therapeutic to treat RP. These ODD, RMAT, and OMPD designations represent gene-agnostic broad coverage for RP and is not mutation-specific designations. Figure 9: Mechanism of our modifier gene therapy. Figure 9 demonstrates the mechanism of our modifier gene therapy.
We believe these broad ODD, RMAT, and OMPD designations demonstrate that OCU400 has the potential to be a broad-spectrum therapeutic to treat RP. These ODD, RMAT, and OMPD designations represent gene-agnostic broad coverage for RP and is not mutation-specific designations. Figure 9: Mechanism of our modifier gene therapy.
Under the Food and Drug Omnibus Reform Act of 2022 ("FDORA"), the FDA is now permitted to require, as appropriate, that confirmatory trials be underway prior to approval or within a specific time period after the date of approval for a product granted accelerated approval.
Under the Food and Drug Omnibus Reform Act of 2022 ("FDORA"), the FDA is permitted to require, as appropriate, that confirmatory trials be underway prior to approval or within a specific time period after the date of approval for a product granted accelerated approval.
If possible, Phase 1 trials may also be used to gain an initial indication of product effectiveness. 20 Table of Contents • Phase 2 — Controlled studies are conducted in larger but still limited subject populations with a specified disease or condition to evaluate preliminary efficacy, identify optimal dosages, dosage tolerance and schedule, possible adverse effects and safety risks, and expanded evidence of safety. • Phase 3 — These adequate and well-controlled clinical trials are undertaken in expanded subject populations (e.g., several hundred to several thousand patients), generally at geographically dispersed clinical trial sites, to generate enough data to provide statistically significant evidence of clinical efficacy and safety of the product candidate for approval, to establish the overall risk-benefit profile of the product candidate, and to provide adequate information for the labeling of the product candidate.
If possible, Phase 1 trials may also be used to gain an initial indication of product effectiveness. • Phase 2 — Controlled studies are conducted in larger but still limited subject populations with a specified disease or condition to evaluate preliminary efficacy, identify optimal dosages, dosage tolerance and schedule, possible adverse effects and safety risks, and expanded evidence of safety. • Phase 3 — These adequate and well-controlled clinical trials are undertaken in expanded subject populations (e.g., several hundred to several thousand patients), generally at geographically dispersed clinical trial sites, to generate enough data to provide statistically significant evidence of clinical efficacy and safety of the product candidate for approval, to establish the overall risk-benefit profile of the product candidate, and to provide adequate information for the labeling of the product candidate.
OCU410 and OCU410ST for the Treatment of Dry AMD and Stargardt Disease We are developing OCU410 and OCU410ST for the treatment of dAMD and Stargardt disease, respectively. OCU410 and OCU410ST utilize an AAV delivery platform for the retinal delivery of the RORA gene. RORA regulated gene networks are relevant in the treatment of dAMD and Stargardt disease.
OCU410 and OCU410ST for the Treatment of Dry AMD and Stargardt Disease We are developing OCU410 and OCU410ST for the treatment of dAMD and ST, respectively. OCU410 and OCU410ST utilize an AAV delivery platform for the retinal delivery of the RORA gene. RORA regulated gene networks are relevant in the treatment of dAMD and ST.
Even if the FDA approves a product, it may limit the approved indications or populations for use of the product, require that contraindications, warnings, or precautions be included in the product labeling, including a boxed warning, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess a product's safety and efficacy after approval, require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, including distribution restrictions or other risk management mechanisms under a REMS, which can materially affect the potential market and profitability of the product.
Even if the FDA approves a product, it may limit the approved indications or populations for use of the product, require that contraindications, warnings, or precautions be included in the product labeling, including a boxed warning, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess a product's safety and efficacy after approval, 22 Table of Contents require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, including distribution restrictions or other risk management mechanisms under a REMS, which can materially affect the potential market and profitability of the product.
As of February 24, 2025, we had exclusive rights or owned rights to: (i) two issued United States patents, one pending United States patent applications, and one pending foreign patent applications related to OCU400; (ii) one pending United States patent applications, and three pending foreign patent applications related to OCU410 and OCU410ST; (iii) 15 issued United States patents; six pending United States patent applications, 20 issued or registered foreign patents, and nine pending foreign patent applications related to NeoCart; (iv) two pending United States patent applications and seven pending foreign patent applications related to OCU500, OCU510 and OCU520; and (v) one issued United States patent and 25 issued or registered foreign patents related to OCU200.
As of February 24, 2026, we had exclusive rights or owned rights to: (i) two issued United States patents, one pending United States patent applications, and one pending foreign patent applications related to OCU400; (ii) one pending United States patent applications, and three pending foreign patent applications related to OCU410 and OCU410ST; (iii) 15 issued United States patents; six pending United States patent applications, 20 issued or registered foreign patents, and nine pending foreign patent applications related to NeoCart; (iv) two pending United States patent applications and seven pending foreign patent applications related to OCU500, OCU510 and OCU520; and (v) one issued United States patent and 25 issued or registered foreign patents related to OCU200.
Further, we have assumed primary responsibility for all patent activities, including all costs associated with the perfection and maintenance of the patents for OCU200. INTELLECTUAL PROPERTY Our success depends in part upon our ability to protect our core technologies and intellectual products. We have applied, obtained, and licensed patent protection for our product candidates.
Further, we have assumed primary responsibility for all patent activities, including all costs associated with the prosecution and maintenance of the patents for OCU200. INTELLECTUAL PROPERTY Our success depends in part upon our ability to protect our core technologies and intellectual products. We have applied, obtained, and licensed patent protection for our product candidates.
NeoCart License Agreement with Purpose In December 2005, Histogenics entered into an exclusive agreement (the "Purpose Agreement") to sublicense certain technology from Purpose, which we assumed as a result of our reverse merger with Histogenics. Purpose entered into the original license agreement ("BWH-Purpose Agreement") with Brigham and Women’s Hospital, Inc. ("BWH") in August 2001.
NeoCart License Agreement with Purpose In December 2005, Histogenics entered into an exclusive agreement (the "Purpose Agreement") to sublicense certain technology from Purpose, which we assumed as a result of our reverse merger with Histogenics. Purpose entered into the original license agreement ("BWH-Purpose Agreement") with Brigham and Women’s Hospital, Inc.
A copy of our Corporate Governance Guidelines, Code of Business Conduct and Ethics and the charters of the Audit Committee, Compensation Committee and Nominating and Corporate Governance Committee are posted on our website, www.ocugen.com, under “Investors”. Any amendments to Code of Business Conduct and Ethics are also being posted on our website, www.ocugen.com, under “Investors”. 34 Table of Contents
A copy of our Corporate Governance Guidelines, Code of Business Conduct and Ethics and the charters of the Audit Committee, Compensation Committee and Nominating and Corporate Governance Committee are posted on our website, www.ocugen.com, under “Investors”. Any amendments to Code of Business Conduct and Ethics are also being posted on our website, www.ocugen.com, under “Investors”. 35 Table of Contents
In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission.
In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which can be the basis of a BLA submission.
Our issued or registered patents and pending patent applications include those licensed from SERI, Purpose, Washington University, and CU. Certain issued patents and pending patent applications cover multiple of our product candidates. Our intellectual property includes compositions of matter, methods of use, product candidates, and other proprietary technology.
Our issued or registered patents and pending patent applications include those licensed from SERI, Purpose and CU. Certain issued patents and pending patent applications cover multiple of our product candidates. Our intellectual property includes compositions of matter, methods of use, product candidates, and other proprietary technology.
The civil FCA provides for treble damages and a civil penalty for each false claim, such as an invoice or pharmacy claim for reimbursement, which can aggregate into millions of dollars, and potential exclusion from federal health care programs. The civil monetary penalties statute is another statute under which biotechnological companies may be subject to enforcement.
The civil FCA provides for treble damages and a civil penalty for each false claim, such as an invoice or pharmacy claim for reimbursement, which can aggregate into millions of dollars, and potential exclusion from federal health care programs. 26 Table of Contents The civil monetary penalties statute is another statute under which biotechnological companies may be subject to enforcement.
If a manufacturer fails to pay the required rebate amount or report pricing data on a timely basis, it may be subject to civil monetary penalties and/or termination of its Medicaid Drug Rebate program agreement, in which case federal payments may not be available under Medicaid or Medicare Part B for its covered outpatient drugs.
If a manufacturer fails to pay the required rebate amount or report pricing data on a timely basis, it may be subject to civil monetary penalties and/or termination 30 Table of Contents of its Medicaid Drug Rebate program agreement, in which case federal payments may not be available under Medicaid or Medicare Part B for its covered outpatient drugs.
GA involves several biological factors and pathways, including lipid metabolism, oxidative stress, inflammation, and activation of the complement system. The currently approved treatments for GA only focus on the complement system with limited treatment benefits. Similarly, Stargardt disease is a rare genetic eye disorder that causes retinal degeneration and ultimately leads to loss of central vision.
GA involves several biological factors and pathways, including lipid metabolism, oxidative stress, inflammation, and activation of the complement system. The currently approved treatments for GA only focus on the complement system with limited treatment benefits. Similarly, ST is a rare genetic eye disorder that causes retinal degeneration and ultimately leads to loss of central vision.
RORA reduces oxidative stress, limits lipofuscin deposits, reduces chronic inflammation, regulates complement activation, and improves choroidal blood flow. Gene variants of the ABCA4 gene are associated with both AMD and Stargardt disease. Stargardt disease is usually caused by mutations in the ABCA4 gene. This gene transports oxidized retinol compounds from photoreceptors to RPE cells for detoxification.
RORA reduces oxidative stress, limits lipofuscin deposits, reduces chronic inflammation, regulates complement activation, and improves choroidal blood flow. Gene variants of the ABCA4 gene are associated with both AMD and ST. ST is usually caused by mutations in the ABCA4 gene. This gene transports oxidized retinol compounds from photoreceptors to RPE cells for detoxification.
We believe that the experience of our management team, our scientific advisory board members, business advisory board, and our broad network of relationships with leaders within the industry and the medical community provides us with insight into the identification of product candidate opportunities as well as supports us in advancing the development and commercialization of our product candidates. • Key Partnerships and Internal Capabilities.
We believe that the experience of our management team, our scientific advisory board members, business advisory board, and our broad network of relationships with leaders within the industry and the medical 5 Table of Contents community provides us with insight into the identification of product candidate opportunities as well as supports us in advancing the development and commercialization of our product candidates. • Key Partnerships and Internal Capabilities.
There is a significant need to develop a novel, differentiated therapeutic to treat DR and DME. Additionally, current therapies target only one pathway associated with DR and DME, either angiogenesis (development of new blood vessels) with anti-VEGF therapy, such as Ranibizumab or Aflibercept, or inflammation in case of corticosteroid therapy, such as Dexamethasone or Fluocinolone.
There is a significant need to develop a novel, differentiated therapeutic to treat DR and DME. Additionally, current therapies target only one pathway associated with DR and DME, either angiogenesis (development of new blood vessels) with anti-VEGF therapy, such as Ranibizumab or Aflibercept, or inflammation in case of corticosteroid therapy, 10 Table of Contents such as Dexamethasone or Fluocinolone.
The photoreceptor cells convert light into electrical signals, which are then sent to the brain where they are processed to create the images we see. Stargardt disease is usually caused by mutations in the ABCA4 gene and is inherited in an autosomal recessive manner. This gene affects how a person's body uses vitamin A.
The photoreceptor cells convert light into electrical signals, which are then sent to the brain where they are processed to create the images we see. ST is usually caused by mutations in the ABCA4 gene and is inherited in an autosomal recessive manner. This gene affects how a person's body uses vitamin A.
Developing a custom gene therapy for each of the more than 100 mutated genes linked to RP would not only be expensive but also may not be possible due to size, class, or localization that will impact delivery of the gene. Not all genes and disease expressions are amenable to gene therapy.
Developing a custom gene therapy for each of the more than 100 mutated genes linked to RP would not only be expensive but also may not be possible due to size, class, or localization that will impact 6 Table of Contents delivery of the gene. Not all genes and disease expressions are amenable to gene therapy.
It is the most common form of inherited macular degeneration, affecting approximately 0.1 million people in the United States and Europe. Stargardt disease happens when lipofuscin, a fatty yellow pigment, accumulates on the macula, which leads to the degeneration of the photoreceptor cells in the macula and ultimately leads to progressive central vision loss.
It is the most common form of inherited macular degeneration, affecting approximately 0.1 million people in the United States and Europe. ST happens when lipofuscin, a fatty yellow pigment, accumulates on the macula, which leads to the degeneration of the photoreceptor cells in the macula and ultimately leads to progressive central vision loss.
Before approving a product candidate for which no active ingredient (including any ester or salt of an active ingredient) has previously been approved by the FDA, the FDA must either refer that product candidate to an external advisory committee or provide in an action letter a summary of the reasons 21 Table of Contents why the FDA did not refer the product candidate to an advisory committee.
Before approving a product candidate for which no active ingredient (including any ester or salt of an active ingredient) has previously been approved by the FDA, the FDA must either refer that product candidate to an external advisory committee or provide in an action letter a summary of the reasons why the FDA did not refer the product candidate to an advisory committee.
The PHSA also provides authority to the FDA to immediately suspend licenses in situations where there exists a danger to public health, to prepare or procure products in the event of shortages and critical public health needs, and to authorize the creation and enforcement of regulations to prevent the introduction or spread of communicable diseases in the United States and between states.
The PHSA also provides 25 Table of Contents authority to the FDA to immediately suspend licenses in situations where there exists a danger to public health, to prepare or procure products in the event of shortages and critical public health needs, and to authorize the creation and enforcement of regulations to prevent the introduction or spread of communicable diseases in the United States and between states.
Like the 26 Table of Contents Medicaid rebate amount, the FCP includes an inflation penalty. A Department of Defense statute and regulation requires labelers to provide this discount on therapeutics dispensed by retail pharmacies when paid by the TRICARE Program, the health care program for military personnel, retirees, and related beneficiaries.
Like the Medicaid rebate amount, the FCP includes an inflation penalty. A Department of Defense statute and regulation requires labelers to provide this discount on therapeutics dispensed by retail pharmacies when paid by the TRICARE Program, the health care program for military personnel, retirees, and related beneficiaries.
In addition, newly discovered or developed safety or efficacy data may require changes to a product's approved labeling, including the addition of new warnings and contraindications. 24 Table of Contents In addition, the distribution of prescription drug and biological products is subject to the Prescription Drug Marketing Act ("PDMA"), which regulates the distribution of samples at the federal level.
In addition, newly discovered or developed safety or efficacy data may require changes to a product's approved labeling, including the addition of new warnings and contraindications. In addition, the distribution of prescription drug and biological products is subject to the Prescription Drug Marketing Act ("PDMA"), which regulates the distribution of samples at the federal level.
This study will be a randomized, controlled clinical trial designed to evaluate the efficacy and safety of NeoCart in comparison to the current standard of care, chondroplasty, in subjects with articular cartilage defects. We intend to initiate the Phase 3 trial contingent on adequate availability of funding.
This study will be a randomized, controlled clinical trial designed to evaluate the efficacy and safety of NeoCart in comparison to the current standard of care, chondroplasty, in subjects with articular cartilage defects. We intend to initiate the Phase 3 trial contingent on adequate availability of funding to support clinical trial and manufacturing of NeoCart product.
The seasonal flu causes illnesses that range in severity and may lead to hospitalization and death in certain cases. The WHO estimates the seasonal flu results in three to five million cases of severe illness, and approximately 0.3 million to 0.7 million respiratory deaths annually. 13 Table of Contents The seasonal flu spreads easily and rapidly transmits in crowded areas.
The seasonal flu causes illnesses that range in severity and may lead to hospitalization and death in certain cases. The WHO estimates the seasonal flu results in three to five million cases of severe illness, and approximately 0.3 million to 0.7 million respiratory deaths annually. The seasonal flu spreads easily and rapidly transmits in crowded areas.
We are aware of several companies focusing on gene therapies for various ophthalmic indications including Applied Genetic Technologies Corporation, as acquired by Syncona Limited, Astellas Pharma Inc., MeiraGTx Holdings plc in partnership with Janssen Pharmaceuticals, Inc., Nanoscope Therapeutics Inc., REGENXBIO Inc., Novartis AG, F.
We are aware of several companies focusing on gene therapies for various ophthalmic indications including Applied Genetic Technologies Corporation, as acquired by Beacon Therapeutics, Astellas Pharma Inc., MeiraGTx Holdings plc in partnership with Janssen Pharmaceuticals, Inc., Nanoscope Therapeutics Inc., REGENXBIO Inc., Novartis AG, F.
Preclinical Studies and IND Submission The testing and approval process of product candidates requires substantial time, effort, and financial resources. Satisfaction of FDA pre-market approval requirements typically takes many years, and the actual time required may vary substantially based 19 Table of Contents upon the type, complexity, and novelty of the product or disease.
Preclinical Studies and IND Submission The testing and approval process of product candidates requires substantial time, effort, and financial resources. Satisfaction of FDA pre-market approval requirements typically takes many years, and the actual time required may vary substantially based upon the type, complexity, and novelty of the product or disease.
The main characteristics of the CTR include: a streamlined application procedure via a single-entry point through the Clinical Trials Information System, or CTIS; a single set of documents to be prepared and submitted for the application as well as simplified reporting procedures for clinical trial sponsors; and a harmonized procedure for the assessment of applications for clinical trials.
The main characteristics of the CTR include: a streamlined application procedure via a single-entry point through the Clinical Trials Information System, or CTIS; a single set of documents to be prepared and submitted for the application as well as simplified reporting procedures for clinical trial sponsors; and a harmonized procedure for the 32 Table of Contents assessment of applications for clinical trials.
The body uses vitamin A to make cells in the retina. Common symptoms of Stargardt disease include gray, black, or hazy spots in one's central vision, sensitivity to light, increased time for eyes to adjust between light and dark places, color blindness, and gradual central vision loss in both eyes.
The body uses vitamin A to make cells in the retina. Common symptoms of ST include gray, black, or hazy spots in one's central vision, sensitivity to light, increased time for eyes to adjust between light and dark places, color blindness, and gradual central vision loss in both eyes.
The BWH-Purpose Agreement granted Purpose an exclusive, royalty-bearing, worldwide, sublicensable license, under its rights in licensed patents and patent applications co-owned by BWH and Purpose to make, use, and sell (1) an apparatus for cultivating a cell or tissue, (2) cell or tissue products made using such apparatus, (3) cell or tissue products made using processes for cultivating a cell or tissue as disclosed in the licensed patents and patent applications, and (4) any apparatus that cultivates cells or tissues using such processes, in each case, whose manufacture, use, or sale is covered by a valid claim of the licensed patents and patent applications, only for therapeutic use.
("BWH") in August 2001. 17 Table of Contents The BWH-Purpose Agreement granted Purpose an exclusive, royalty-bearing, worldwide, sublicensable license, under its rights in licensed patents and patent applications co-owned by BWH and Purpose to make, use, and sell (1) an apparatus for cultivating a cell or tissue, (2) cell or tissue products made using such apparatus, (3) cell or tissue products made using processes for cultivating a cell or tissue as disclosed in the licensed patents and patent applications, and (4) any apparatus that cultivates cells or tissues using such processes, in each case, whose manufacture, use, or sale is covered by a valid claim of the licensed patents and patent applications, only for therapeutic use.
We plan to compete in the segments of pharmaceutical, biotechnological, and other related markets with therapeutics, regenerative medicines, and vaccines that have an acceptable safety profile and target commercially attractive indications. 14 Table of Contents The development and commercialization of gene therapies is highly competitive.
We plan to compete in the segments of pharmaceutical, biotechnological, and other related markets with therapeutics, regenerative medicines, and vaccines that have an acceptable safety profile and target commercially attractive indications. The development and commercialization of gene therapies is highly competitive.
RP is the most common IRD involving photoreceptors and the retinal pigment epithelium ("RPE"). RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina. RP affects approximately 310,000 individuals in the 6 Table of Contents United States, Europe, and Canada.
RP is the most common IRD involving photoreceptors and the retinal pigment epithelium ("RPE"). RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina. RP affects approximately 310,000 individuals in the United States, Europe, and Canada.
The other options for cartilage repair include osteochondral autograft transplantation ("OAT"), osteochondral allograft resurfacing ("OCA"), and autologous chondrocyte implantation ("ACI"). During OAT, damaged cartilage is removed and replaced with healthy cartilage from a non-weight-bearing area of the joint.
The other options for cartilage repair include osteochondral autograft transplantation ("OAT"), osteochondral allograft resurfacing ("OCA"), and autologous chondrocyte implantation ("ACI"). During OAT, damaged cartilage is removed and 12 Table of Contents replaced with healthy cartilage from a non-weight-bearing area of the joint.
This approach represents a potential universal booster, regardless of previous COVID-19 vaccination. OCU520, our combination quadrivalent seasonal flu and COVID-19 vaccine, is designed to provide the unique ease of getting both an annual COVID-19 booster vaccine and an annual seasonal flu vaccine in one vaccine.
This approach represents a potential universal booster, regardless of previous COVID-19 vaccination. OCU520, 14 Table of Contents our combination quadrivalent seasonal flu and COVID-19 vaccine, is designed to provide the unique ease of getting both an annual COVID-19 booster vaccine and an annual seasonal flu vaccine in one vaccine.
Marketing Authorization 31 Table of Contents To obtain a marketing authorization for a product in the EU, an applicant must submit a MAA, either under a centralized procedure administered by the EMA or one of the procedures administered by competent authorities in the EU Member States (decentralized procedure, national procedure, or mutual recognition procedure).
Marketing Authorization To obtain a marketing authorization for a product in the EU, an applicant must submit a MAA, either under a centralized procedure administered by the EMA or one of the procedures administered by competent authorities in the EU Member States (decentralized procedure, national procedure, or mutual recognition procedure).
Some states have also created pharmacy assistance programs for individuals who do not qualify for federal programs. In the United States, private health insurers and other third-party payors often provide reimbursement for products and services based on the level at which the government provides reimbursement through the Medicare or Medicaid programs for such products and services.
Some states have also created pharmacy assistance programs for individuals who do not qualify for federal programs. In the United States, private 28 Table of Contents health insurers and other third-party payors often provide reimbursement for products and services based on the level at which the government provides reimbursement through the Medicare or Medicaid programs for such products and services.
NHRs play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, survival, and maintaining the cellular 7 Table of Contents and molecular homeostasis in retinal tissues. Our modifier gene therapy platform is designed to target NHRs to potentially provide therapeutic benefit to patients suffering from genetically diverse IRDs.
NHRs play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, survival, and maintaining the cellular and molecular homeostasis in retinal tissues. Our modifier gene therapy platform is designed to target NHRs to potentially provide therapeutic benefit to patients suffering from genetically diverse IRDs.
Given the above limitations of these existing treatments, we believe that a substantial unmet medical need exists for the treatment of DR, DME, and Wet AMD. 10 Table of Contents OCU200 for the Treatment of DR, DME, and Wet AMD OCU200 is a novel fusion protein containing parts of human transferrin and tumstatin, that are already present normally in retinal tissues.
Given the above limitations of these existing treatments, we believe that a substantial unmet medical need exists for the treatment of DR, DME, and Wet AMD. OCU200 for the Treatment of DR, DME, and Wet AMD OCU200 is a novel recombinant fusion protein containing parts of human transferrin and tumstatin, that are already present normally in retinal tissues.
These organizations also compete with us in recruiting and retaining qualified scientific and management personnel, establishing clinical trial sites, patient registration for clinical trials, licensing or acquiring technologies necessary for our programs, and in our commercialization efforts if our product candidates are approved.
These organizations also compete with us in recruiting and retaining qualified scientific and management personnel, establishing clinical trial sites, patient registration for clinical trials, licensing or acquiring technologies necessary for 15 Table of Contents our programs, and in our commercialization efforts if our product candidates are approved.
These designations demonstrate the potential broad-spectrum application of OCU400, through its use of NHRs, to treat the more than 125 genes associated with RP and LCA with one product rather than developing individual treatments for each gene mutation. OCU410ST has received ODD from the FDA for the treatment of ABCA4 -associated retinopathies, including Stargardt disease.
These designations demonstrate the potential broad-spectrum application of OCU400, through its use of NHRs, to treat the more than 100 genes associated with RP and LCA with one product rather than developing individual treatments for each gene mutation. OCU410ST has received ODD from the FDA for the treatment of ABCA4 -associated retinopathies, including ST.
Our business advisory board members have been selected based on their extensive professional backgrounds and proven track record of creating partnerships among the public and 4 Table of Contents private sector.
Our business advisory board members have been selected based on their extensive professional backgrounds and proven track record of creating partnerships among the public and private sector.
Under the CU Agreement, we must use commercially reasonable efforts to develop, manufacture, sublicense, market, and sell the licensed products and have assumed primary responsibility for preparing, filing, and prosecuting broad patent claims for OCU200 for CU's benefit.
Under the CU Agreement, we must use commercially 18 Table of Contents reasonable efforts to develop, manufacture, sublicense, market, and sell the licensed products and have assumed primary responsibility for preparing, filing, and prosecuting broad patent claims for OCU200 for CU's benefit.
In addition, an IRB at each study site participating in the clinical trial and/or a central IRB must review and approve the plan for any clinical trial, informed consent forms, and communications to study subjects before a study commences at that site.
In addition, an IRB at each study site participating in the clinical trial and/or a central IRB must review and approve the plan for 20 Table of Contents any clinical trial, informed consent forms, and communications to study subjects before a study commences at that site.
We 18 Table of Contents also seek to preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security of our information technology systems.
We also seek to preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security of our information technology systems.
Our technology pipeline includes: • Modifier Gene Therapy Platform — Based on the use of nuclear hormone receptors ("NHRs"), we believe our modifier gene therapy platform has the potential to address many retinal diseases, including rare genetic diseases such as RP (OCU400), with a gene-agnostic approach.
Our technology pipeline includes: Novel Modifier Gene Therapy Platform — • OCU400 - Based on the use of nuclear hormone receptors ("NHRs"), we believe our novel modifier gene therapy platform has the potential to address major blindness diseases, including rare genetic diseases such as RP (OCU400), with a gene-agnostic approach.
Orphan Designation and Exclusivity Regulation (EC) No 141/2000 and Regulation (EC) No. 847/2000 provide that a product can be designated as an orphan medicinal product by the EC if its sponsor can establish that: (1) the product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition, (2) either (i) such condition affects no more than five in ten thousand persons in the EU when the application is made, or (ii) without the benefits derived from orphan status, it is unlikely that the marketing of the product in the EU would generate sufficient return to justify the necessary investment in its development; (3) there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorized in the EU or, if such method exists, the product would be of significant benefit to those affected by that condition. 32 Table of Contents An orphan designation provides a number of benefits, including fee reductions, regulatory assistance and the possibility to apply for a centralized EU marketing authorization.
Orphan Designation and Exclusivity Regulation (EC) No 141/2000 and Regulation (EC) No. 847/2000 provide that a product can be designated as an orphan medicinal product by the EC if its sponsor can establish that: (1) the product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition, (2) either (i) such condition affects no more than five in ten thousand persons in the EU when the application is made, or (ii) without the benefits derived from orphan status, it is unlikely 33 Table of Contents that the marketing of the product in the EU would generate sufficient return to justify the necessary investment in its development; (3) there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorized in the EU or, if such method exists, the product would be of significant benefit to those affected by that condition.
We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. In January 2025, we announced that the Investigational New Drug (IND) application is in effect and the National Institute of Allergy and Infectious Diseases ("NIAID") intends to initiate a Phase 1 clinical trial in the second quarter of 2025.
We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. In January 2025, we announced that the Investigational New Drug ("IND") application is in effect, and the National Institute of Allergy and Infectious Diseases ("NIAID"), part of the National Institutes of Health ("NIH") intends to initiate a Phase 1 clinical trial for OCU500.
Currently no treatment options exist to address all four disease pathways related to dAMD or reverse or slow the progression of Stargardt disease, and accordingly, there remains a significant unmet medical need for these ocular diseases.
Currently no treatment options exist to address all four disease pathways 9 Table of Contents related to dAMD or reverse or slow the progression of ST, and accordingly, there remains a significant unmet medical need for these ocular diseases.
Key elements of the strategy we employ to accomplish this mission include: • Continuing to advance our modifier gene therapy platform into and through clinical development. We are developing our modifier gene therapy platform, inclusive of OCU400, OCU410, and OCU410ST, for the treatment of multiple IRDs, including RP, Stargardt disease and multifactorial diseases such as dAMD and GA.
Key elements of the strategy we employ to accomplish this mission include • Continuing to advance our modifier gene therapy platform through clinical development. We are developing our modifier early-to-advanced gene therapy platform, inclusive of OCU400, OCU410, and OCU410ST, for the treatment of multiple IRDs, including RP, ST and multifactorial diseases such as GA, the advanced form of dAMD.
As of February 24, 2025, our global intellectual property portfolio contains 63 patents and 30 pending patent applications related to composition of matter, pharmaceutical compositions, methods of use for our product candidates, and other proprietary technology including those under our licensing and development arrangements.
As of February 24, 2026, our global intellectual property portfolio contains 80 patents and 66 pending patent applications related to composition of matter, pharmaceutical compositions, methods of use for our product candidates, and other proprietary technology including those under our licensing and development arrangements.
These laws may affect our future sales, marketing, and other promotional activities by imposing administrative and compliance burdens. 27 Table of Contents If our operations are found to be in violation of any of the laws or regulations described above or any other applicable laws, we may be subject to penalties or other enforcement actions, including significant criminal, civil, and/or administrative monetary penalties, damages, fines, disgorgement, imprisonment, exclusion from participation in government healthcare programs, corporate integrity agreements, suspension and debarment from government contracts, and refusal of orders under existing government contracts, reputational harm, diminished profits and future earnings, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
If our operations are found to be in violation of any of the laws or regulations described above or any other applicable laws, we may be subject to penalties or other enforcement actions, including significant criminal, civil, and/or administrative monetary penalties, damages, fines, disgorgement, imprisonment, exclusion from participation in government healthcare programs, corporate integrity agreements, suspension and debarment from government contracts, and refusal of orders under existing government contracts, reputational harm, diminished profits and future earnings, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
A sponsor must submit a request for fast track designation, breakthrough therapy designation, or priority review, which may or may not be granted by the FDA.
A sponsor 23 Table of Contents must submit a request for fast track designation, breakthrough therapy designation, or priority review, which may or may not be granted by the FDA.
These licensing and development arrangements include the licensing agreement with The Schepens Eye Research Institute, Inc. ("SERI"), an affiliate of Harvard Medical School, through which we acquired the technology used in our modifier gene therapy platform as well as access to technologies for other NHR genes, the license agreement with Purpose Co., Ltd.
These licensing and development arrangements include the licensing agreement with The Schepens Eye Research Institute, Inc. ("SERI"), an affiliate of Harvard Medical School, through which we acquired the technology used in our modifier gene therapy platform as well as access to technologies for other NHR genes, the license agreement (as amended, the "WU License Agreement") with Washington University in St.
In March 2025, we announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU410 and OCU410ST ATMP classification. NOVEL BIOLOGIC PRODUCT CANDIDATE FOR RETINAL DISEASES We are developing OCU200, which is a novel fusion protein containing parts of human transferrin and tumstatin. OCU200 is designed to treat DME, DR, and Wet AMD.
In March 2025, we announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU410 and OCU410ST ATMP classification. OTHER PROGRAMS NOVEL BIOLOGIC PRODUCT CANDIDATE FOR RETINAL DISEASES We are developing OCU200, which is a novel fusion protein containing parts of human transferrin and tumstatin.
Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related serious adverse events ("SAEs"), reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity ("LLVA").
Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related serious adverse events ("SAEs"),reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low LLVA. In March 2025, OCU410 and OCU410ST received ATMP classification from the EMA.
In the Phase 1/2 OCU400 clinical trial a MLMT scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used, Luminance Dependent Navigation Assessment ("LDNA") that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.
For the Phase 3 OCU400 clinical trial, an updated mobility course will be used, Luminance Dependent Navigation Assessment ("LDNA") that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.
We have completed the technology transfer of manufacturing processes to our CDMO and have produced trial materials to initiate a Phase 1 trial. The Phase 1 clinical trial will assess the unilateral 9 Table of Contents intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME.
OCU200 is designed to treat DME, DR, and Wet AMD. We have completed the technology transfer of manufacturing processes to our CDMO and have produced trial materials to initiate a Phase 1 trial. The Phase 1 clinical trial will assess the unilateral intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME.
A priority review means that the goal for the FDA is to review an application within six months from the filing date, rather than the standard review of 10 months under current PDUFA guidelines. 23 Table of Contents Drug or biological products studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit over existing treatments may be developed and approved under the accelerated approval pathway, which means the FDA may approve the product candidate based upon a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments.
Drug or biological products studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit over existing treatments may be developed and approved under the accelerated approval pathway, which means the FDA may approve the product candidate based upon a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments.
We intend to explore strategic licensing, acquisition, and collaboration opportunities with qualified partners to maximize the potential benefit of our product candidates for patients globally and to expand our product candidate pipeline to support our future growth. • Obtaining government funding to advance our vaccine programs towards commercialization.
We intend to explore strategic licensing, acquisition, and collaboration opportunities with qualified partners to maximize the potential benefit of our product candidates for patients globally and to expand our product candidate pipeline to support our future growth.
Under the SERI Agreement, we may make, have made, use, offer to sell, and import licensed products, and must use commercially reasonable efforts to bring one or more licensed products to market as soon as reasonably practicable. SERI maintains control of patent preparation, filing, prosecution, and maintenance.
Under the SERI Agreement, we may make, have made, use, offer to sell, and import licensed products, and must use commercially reasonable efforts to bring one or more licensed products to market as soon as reasonably practicable.
OCU410 completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for the treatment of geographic atrophy ("GA"), an advanced form of dAMD.
The untreated eyes showed expected decline in atrophy. • OCU410 - We completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for OCU410 for the treatment of geographic atrophy ("GA"), an advanced form of dAMD.
The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x 10 11 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls.
The GARDian3 Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5x10 11 vector genomes(vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size.
Our cutting-edge modifier gene therapy platform provides a competitive advantage compared to traditional approaches for gene therapy, such as gene augmentation, replacement or editing. This platform technology utilizes NHR genes, master regulators inside cells, which promotes homeostasis – a balanced physiological state within cells.
COMPETITIVE STRENGTHS Our key competitive strengths include: • Platform Gene Therapy Technology. ◦ Our cutting-edge modifier gene therapy platform provides a competitive advantage compared to traditional approaches for gene therapy, such as gene augmentation, replacement or editing. This platform technology utilizes nuclear hormone receptor (NHR) genes, transcription factors, which promotes homeostasis – a balanced physiological state within cells.
Other legislative changes have been proposed and adopted since the passage of the ACA. The Budget Control Act of 2011, among other things, resulted in aggregate reductions to Medicare payments to healthcare providers of up to 2.0% per fiscal year, which went into effect in April 2013. Subsequent legislation extended sequestration to 2031.
The Budget Control Act of 2011, among other things, resulted in aggregate reductions to Medicare payments to healthcare providers of up to 2.0% per fiscal year, which went into effect in April 2013. Subsequent legislation extended sequestration through 2031.
HUMAN CAPITAL As of February 24, 2025, we had 95 employees, of which 74 employees are located in the United States, 20 are located in India and one is located in Canada. The majority of the employees are full-time. None of our employees are represented by a labor union or covered by a collective bargaining agreement.
HUMAN CAPITAL As of February 24, 2026, we had 116 employees, of which 79 employees are located in the United States, 34 are located in India and three are located in Canada. The majority of the employees are full-time. None of our employees are represented by a labor union or covered by a collective bargaining agreement.
Where the CHMP gives a positive opinion, the EMA provides the opinion together with supporting documentation to the EC, who makes the final decision to grant a marketing authorization, which is issued within 67 days of receipt of the EMA’s recommendation.
Clock stops may extend the timeframe of evaluation of an application considerably beyond 210 days. Where the CHMP gives a positive opinion, the EMA provides the opinion together with supporting documentation to the EC, who makes the final decision to grant a marketing authorization, which is issued within 67 days of receipt of the EMA’s recommendation.
As of February 24, 2025, our patent portfolio for our product candidates included a total of 18 issued patents in the United States, 45 issued or registered patents in foreign countries, 10 pending patent applications in the United States, and 20 pending patent applications in foreign countries.
As of February 24, 2026, our patent portfolio for our product candidates included a total of 24 issued patents in the United States, 56 issued or registered patents in foreign countries, 9 pending patent applications in the United States, and 57 pending patent applications in foreign countries.
For example, the Tax Cuts and Job Act of 2017, repealed the shared responsibility 29 Table of Contents payment for individuals who fail to maintain minimum essential coverage under section 5000A of the Internal Revenue Code of 1986, as amended, or the Code, commonly referred to as the individual mandate.
For example, the Tax Cuts and Job Act of 2017, repealed the shared responsibility payment for individuals who fail to maintain minimum essential coverage under section 5000A of the Internal Revenue Code of 1986, as amended, or the Code, commonly referred to as the individual mandate. Other legislative changes have been proposed and adopted since the passage of the ACA.
Other state laws and local ordinances require identification or licensing of sales representatives. Recently, states have enacted or are considering legislation intended to make drug prices more transparent and deter significant price increases, typically as consumer protection laws.
Other state laws and local ordinances require identification or licensing of sales representatives. Recently, states have enacted or are considering legislation intended to make drug prices more transparent and deter significant price increases, typically as consumer protection laws. These laws may affect our future sales, marketing, and other promotional activities by imposing administrative and compliance burdens.
Under the manufacturing agreement, our CDMO will manage all CMC and clinical manufacturing activities for OCU200. We have completed the technology transfer of manufacturing processes to our CDMO and have produced clinical trial materials to initiate the planned Phase 1 clinical trial.
We have completed the technology transfer of manufacturing processes to our CDMO and have produced clinical trial materials to initiate the planned Phase 1 clinical trial.
Marketing Application Submission, Review by the FDA, and Marketing Approval Assuming successful completion of the required clinical and preclinical testing, the results of product development, including CMC, non-clinical studies, and clinical trial results, including negative or ambiguous results, as well as positive findings, are all submitted to the FDA, along with the proposed labeling, as part of an NDA, in the case of a drug, or BLA, in the case of a biologic, requesting approval to market the product for one or more indications.
Additionally, appropriate packaging must be selected and tested, and stability studies must be conducted to demonstrate that the product candidate does not undergo unacceptable deterioration over its shelf life. 21 Table of Contents Marketing Application Submission, Review by the FDA, and Marketing Approval Assuming successful completion of the required clinical and preclinical testing, the results of product development, including CMC, non-clinical studies, and clinical trial results, including negative or ambiguous results, as well as positive findings, are all submitted to the FDA, along with the proposed labeling, as part of an NDA, in the case of a drug, or BLA, in the case of a biologic, requesting approval to market the product for one or more indications.
This procedure is performed through small incisions on the sides of the knee with the aid of an arthroscope. During this procedure, the damaged cartilage is trimmed, and the remaining surface is smoothed. Microfracture surgery is a frequently used procedure for severe cartilage damage which yields varying outcomes from patient to patient.
During this procedure, the damaged cartilage is trimmed, and the remaining surface is smoothed. Microfracture surgery is a frequently used procedure for severe cartilage damage which yields varying outcomes from patient to patient.
In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission.
In mice models, ABCA4 -/- displayed low levels of CD59. A cell-surface glycoprotein, CD59, prevents the formation of the complement membrane attack complex. In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission.
("Purpose") relating to NeoCart, the WU License Agreement with Washington University with respect to mucosal COVID-19 vaccines in the Mucosal Vaccine Territory, and the license agreement with the University of Colorado ("CU") pursuant to which we acquired rights to the transferrin-tumstatin fusion protein technology used in our OCU200 product candidate.
Louis ("Washington University") with respect to mucosal COVID-19 vaccines in the United States, Canada, Europe, Japan, South Korea, Australia, China, and Hong Kong (the "Mucosal Vaccine Territory"), and the license agreement with the University of Colorado ("CU") pursuant to which we acquired rights to the transferrin-tumstatin fusion protein technology used in our OCU200 product candidate.
Moreover, to the extent that we license the right to sell our product, if approved, to another entity under that entity's labeler code, the licensee would have regulatory responsibilities, including healthcare, reimbursement, pricing, and reporting regulatory responsibilities. 25 Table of Contents The federal Anti-Kickback Statute, prohibits, among other things, any person or entity, from knowingly and willfully offering, paying, soliciting, or receiving any remuneration, directly or indirectly, overtly or covertly, in cash or in kind, to induce or in return for purchasing, leasing, ordering, or arranging for or recommending the purchase, lease, or order, or the referral to another for the furnishing or arranging of any item or service reimbursable under Medicare, Medicaid, or other federal healthcare programs, in whole or in part.
The federal Anti-Kickback Statute, prohibits, among other things, any person or entity, from knowingly and willfully offering, paying, soliciting, or receiving any remuneration, directly or indirectly, overtly or covertly, in cash or in kind, to induce or in return for purchasing, leasing, ordering, or arranging for or recommending the purchase, lease, or order, or the referral to another for the furnishing or arranging of any item or service reimbursable under Medicare, Medicaid, or other federal healthcare programs, in whole or in part.
Our first product candidate based on this platform technology, NeoCart, is an autologous cartilage tissue–engineered implant designed for use in repair of articular cartilage injuries in the knee. In contrast to the approved cell-matrix based product, NeoCart showed rapid healing and durable benefit in clinical studies.
Our first product candidate based on this platform technology, NeoCart, is an autologous cartilage tissue–engineered implant designed for use in repair of articular cartilage injuries in the knee.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeWe may not be successful in establishing or maintaining collaborative relationships, any of which could adversely affect our ability to develop and commercialize our product candidates. • We may be unable to obtain and maintain patent protection for our technology and product candidates, or the scope of the patent protection obtained may not be sufficiently broad or enforceable, such that our competitors could develop and commercialize technology and products similar or identical to ours, and our ability to successfully commercialize our technology and product candidates may be impaired. • We may become involved in lawsuits to protect or enforce our patents or other intellectual property, which could be expensive, time-consuming, and unsuccessful. • Certain aspects of our product candidates are protected by patents exclusively licensed from other companies or institutions.
Biggest changeFurthermore, our ability to successfully commercialize our technology and product candidates may be impaired due to circumstances that may be out of our control. • We may become involved in lawsuits to protect or enforce our patents or other intellectual property, which could be expensive, time-consuming, and unsuccessful. • Certain aspects of our product candidates are protected by patents exclusively licensed from other companies or institutions.
We may not be successful in establishing or maintaining collaborative relationships, any of which could adversely affect our ability to develop and commercialize our product candidates. We are party to an agreement with CanSinoBIO for the development and commercialization of our modifier gene therapy platform.
We may not be successful in establishing or maintaining collaborative relationships, any of which could adversely affect our ability to develop and commercialize our product candidates. We are party to an agreement with CanSinoBIO for the development and commercialization of our modifier gene therapy platform product candidates.
If we are unable to obtain patent term extension or restoration, or the term of any such extension is less than we request, the period during which we will have the right to exclusively market our product will be shortened.
If we are unable to obtain patent term extension or restoration, or the term of any such extension is less than our request, the period during which we will have the right to exclusively market our product will be shortened.
Our own patent portfolio may have no deterrent effect on entities that do not use of practice their own patent. Moreover, we may become party to future adversarial proceedings or litigation regarding our patent portfolio or the patents of third parties.
Our own patent portfolio may have no deterrent effect on entities that do not use or practice their own patent. Moreover, we may become party to future adversarial proceedings or litigation regarding our patent portfolio or the patents of third parties.
We are and expect to continue to be a “smaller reporting company” as defined in the Exchange Act and have elected and expect to continue to elect to take advantage of certain of the scaled disclosures available to smaller reporting companies, including reduced disclosure obligations regarding executive compensation.
We are and expect to continue to be a “smaller reporting company” as defined in the Exchange Act and have elected and expect to continue to elect to take advantage of certain of the scaled disclosures available to smaller reporting companies, including reduced disclosure obligations regarding executive compensation.
We may experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receive marketing approval or commercialize our product candidates, including: • regulators, including the FDA and the NIH, or IRBs or IBCs may not authorize us or our investigators to commence or continue a clinical trial, conduct a clinical trial at a prospective trial site, or amend trial protocols, or regulators, IRBs, or IBCs may require that we modify or amend our clinical trial protocols; 44 Table of Contents • we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites and our CDMOs; • the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these clinical trials, or be lost to follow-up at a higher rate than we anticipate; • our third-party contractors may fail to comply with regulatory requirements or the clinical trial protocol, or meet their contractual obligations to us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring; • us, the regulators, IRBs, or IBCs may require the suspension or termination of clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate; • changes in marketing approval policies or regulations, or changes in or the enactment of additional statutes or regulations, during the development period rendering our data insufficient to obtain marketing approval and requiring us to conduct additional studies; • the cost of clinical trials of our product candidates may be greater than we anticipate or we may have insufficient funds for a clinical trial or to pay the substantial user fees required by the FDA upon the filing of a marketing application; • the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; • we may have delays in adding new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites; • patients that enroll in our studies may misrepresent their eligibility or may otherwise not comply with the clinical trial protocol, resulting in the need to drop the patients from the study, increase the needed enrollment size for the study, or extend the study’s duration; • the FDA or comparable foreign regulatory authorities may disagree with our study design, including endpoints, or our interpretation of data from preclinical studies and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks; • the FDA or comparable foreign regulatory authorities may not accept data from studies with clinical trial sites in foreign countries; • the FDA or comparable foreign regulatory authorities may disagree with our intended indications; • the FDA or comparable foreign regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes or our contract manufacturer’s manufacturing facility for clinical and future commercial supplies; • the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable foreign regulatory authorities to support the submission of a marketing application, or other comparable submissions in foreign jurisdictions, or to obtain regulatory approval in the United States or elsewhere; • the FDA or comparable foreign regulatory authorities may take longer than we anticipate to make a decision on our product candidates; and • we may not be able to demonstrate that a product candidate provides an advantage over current standards of care or current or future competitive therapies in development.
We may experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receive marketing approval or commercialize our product candidates, including: • regulators, including the FDA and the NIH, or IRBs or IBCs may not authorize us or our investigators to commence or continue a clinical trial, conduct a clinical trial at a prospective trial site, or amend trial protocols, or regulators, IRBs, or IBCs may require that we modify or amend our clinical trial protocols; • we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites and our CDMOs; • the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these clinical trials, or be lost to follow-up at a higher rate than we anticipate; • our third-party contractors may fail to comply with regulatory requirements or the clinical trial protocol, or meet their contractual obligations to us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring; • us, the regulators, IRBs, or IBCs may require the suspension or termination of clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate; • changes in marketing approval policies or regulations, or changes in or the enactment of additional statutes or regulations, during the development period rendering our data insufficient to obtain marketing approval and requiring us to conduct additional studies; • the cost of clinical trials of our product candidates may be greater than we anticipate or we may have insufficient funds for a clinical trial or to pay the substantial user fees required by the FDA upon the filing of a marketing application; • the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; • we may have delays in adding new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites; 45 Table of Contents • patients that enroll in our studies may misrepresent their eligibility or may otherwise not comply with the clinical trial protocol, resulting in the need to drop the patients from the study, increase the needed enrollment size for the study, or extend the study’s duration; • the FDA or comparable foreign regulatory authorities may disagree with our study design, including endpoints, or our interpretation of data from preclinical studies and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks; • the FDA or comparable foreign regulatory authorities may not accept data from studies with clinical trial sites in foreign countries; • the FDA or comparable foreign regulatory authorities may disagree with our intended indications; • the FDA or comparable foreign regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes or our contract manufacturer’s manufacturing facility for clinical and future commercial supplies; • the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable foreign regulatory authorities to support the submission of a marketing application, or other comparable submissions in foreign jurisdictions, or to obtain regulatory approval in the United States or elsewhere; • the FDA or comparable foreign regulatory authorities may take longer than we anticipate to make a decision on our product candidates; and • we may not be able to demonstrate that a product candidate provides an advantage over current standards of care or current or future competitive therapies in development.
Factors that may inhibit our efforts to commercialize our product candidates include: • the inability to recruit, train, manage, and retain adequate numbers of effective sales and marketing personnel; • the inability of sales personnel to obtain access to physicians or persuade adequate numbers of physicians to prescribe our product candidates; • our inability to effectively oversee a geographically dispersed sales and marketing team; • the costs associated with training sales and marketing personnel on legal and regulatory compliance matters and monitoring their actions; • an inability to secure adequate coverage and reimbursement by government and private health plans; 54 Table of Contents • reduced realization on government sales from mandatory discounts, rebates and fees, and from price concessions to private health plans and pharmacy benefit managers necessitated by competition for access to managed formularies; • the clinical indications for which the products are approved and the claims that we may make for the products; • limitations or warnings, including distribution or use restrictions, contained in the products' approved labeling; • any distribution and use restrictions imposed by the FDA or other foreign regulatory agencies, including those that we may agree to as part of a mandatory REMS or voluntary risk management plan; • liability for sales or marketing personnel who fail to comply with the applicable legal and regulatory requirements; • the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and • unforeseen costs and expenses associated with creating an independent sales and marketing organization or engaging a contract sales organization.
Factors that may inhibit our efforts to commercialize our product candidates include: • the inability to recruit, train, manage, and retain adequate numbers of effective sales and marketing personnel; • the inability of sales personnel to obtain access to physicians or persuade adequate numbers of physicians to prescribe our product candidates; • our inability to effectively oversee a geographically dispersed sales and marketing team; • the costs associated with training sales and marketing personnel on legal and regulatory compliance matters and monitoring their actions; • an inability to secure adequate coverage and reimbursement by government and private health plans; • reduced realization on government sales from mandatory discounts, rebates and fees, and from price concessions to private health plans and pharmacy benefit managers necessitated by competition for access to managed formularies; 55 Table of Contents • the clinical indications for which the products are approved and the claims that we may make for the products; • limitations or warnings, including distribution or use restrictions, contained in the products' approved labeling; • any distribution and use restrictions imposed by the FDA or other foreign regulatory agencies, including those that we may agree to as part of a mandatory REMS or voluntary risk management plan; • liability for sales or marketing personnel who fail to comply with the applicable legal and regulatory requirements; • the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and • unforeseen costs and expenses associated with creating an independent sales and marketing organization or engaging a contract sales organization.
Our current and future collaborations may pose a number of additional risks, including the following: • collaborators may not pursue development of product candidates and commercialization of any product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities; • collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product candidate for clinical testing; • collaborators could fail to make timely regulatory submissions for a product candidate; • collaborators may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements, which could subject them or us to regulatory enforcement actions; • collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; • product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; • a collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such product candidate or product; • disagreements with collaborators, including disagreements over proprietary rights, contract interpretation, or the preferred course of development, might cause delays or termination of the research, development, or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time consuming and expensive; 64 Table of Contents • collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation; • collaborators may infringe the intellectual property rights of third parties or fail to maintain intellectual property rights which they license to us, which may expose us to litigation and potential liability; and • collaborations may be terminated for the convenience of the collaborator and, if terminated, we could be required to raise additional capital to pursue further development or commercialization of the applicable product candidates.
Our current and future collaborations may pose a number of additional risks, including the following: • collaborators may not pursue development of product candidates and commercialization of any product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities; • collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product candidate for clinical testing; • collaborators could fail to make timely regulatory submissions for a product candidate; • collaborators may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements, which could subject them or us to regulatory enforcement actions; • collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; • product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; • a collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such product candidate or product; • disagreements with collaborators, including disagreements over proprietary rights, contract interpretation, or the preferred course of development, might cause delays or termination of the research, development, or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time consuming and expensive; • collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation; • collaborators may infringe the intellectual property rights of third parties or fail to maintain intellectual property rights which they license to us, which may expose us to litigation and potential liability; and • collaborations may be terminated for the convenience of the collaborator and, if terminated, we could be required to raise additional capital to pursue further development or commercialization of the applicable product candidates.
The success of our product candidates and our ability to generate revenues from our product candidates, if approved, will depend on many factors including our ability to: • complete and obtain favorable results from our clinical trials and preclinical studies with respect to our product candidates; • apply for and receive marketing approval from the applicable regulatory authorities; • receive regulatory approval for claims that are necessary or desirable for successful marketing; • receive approval for our manufacturing processes and facilities from the applicable regulatory authorities; • recruit and enroll qualified patients for clinical trials with respect to our product candidates in a timely manner; • expand and maintain a workforce of experienced scientists and others with experience in relevant technologies to continue to develop our product candidates; • hire, train, and deploy marketing and sales representatives or contract with a third-party for marketing and sales representatives to commercialize product candidates in the United States and key foreign markets; • launch and create market demand for our product candidates, if approved, through marketing and sales activities, and any other arrangements to promote these product candidates that we may otherwise establish; • achieve market acceptance of our product candidates by patients, the medical community, and third-party payors; • effectively compete with other therapies and establish a market share; • maintain a continued acceptable safety and efficacy profile of our product candidates, if approved, following commercial launch; 42 Table of Contents • achieve appropriate reimbursement, pricing, and payment coverage for our product candidates, if approved; • manufacture product candidates in sufficient quantities and at acceptable quality and manufacturing cost to meet commercial demand at launch and thereafter; • establish and maintain agreements with wholesalers, distributors, and group purchasing organizations on commercially reasonable terms; • pursue partnerships with, or offer licenses to, qualified third parties to promote and sell product candidates, if approved, in domestic and key foreign markets where we receive marketing approval; • develop our product candidates for additional indications or for use in broader patient populations; • maintain patent and trade secret protection and regulatory exclusivity for our product candidates; and • qualify for, identify, register, maintain, enforce, and defend intellectual property rights and claims covering our products and intellectual property portfolio; and not infringe on others’ intellectual property rights.
The success of our product candidates and our ability to generate revenues from our product candidates, if approved, will depend on many factors including our ability to: • complete and obtain favorable results from our clinical trials and preclinical studies with respect to our product candidates; • apply for and receive marketing approval from the applicable regulatory authorities; • receive regulatory approval for claims that are necessary or desirable for successful marketing; • receive approval for our manufacturing processes and facilities from the applicable regulatory authorities; • recruit and enroll qualified patients for clinical trials with respect to our product candidates in a timely manner; • expand and maintain a workforce of experienced scientists and others with experience in relevant technologies to continue to develop our product candidates; • hire, train, and deploy marketing and sales representatives or contract with a third-party for marketing and sales representatives to commercialize product candidates in the United States and key foreign markets; • launch and create market demand for our product candidates, if approved, through marketing and sales activities, and any other arrangements to promote these product candidates that we may otherwise establish; • achieve market acceptance of our product candidates by patients, the medical community, and third-party payors; • effectively compete with other therapies and establish a market share; • maintain a continued acceptable safety and efficacy profile of our product candidates, if approved, following commercial launch; • achieve appropriate reimbursement, pricing, and payment coverage for our product candidates, if approved; • manufacture product candidates in sufficient quantities and at acceptable quality and manufacturing cost to meet commercial demand at launch and thereafter; • establish and maintain agreements with wholesalers, distributors, and group purchasing organizations on commercially reasonable terms; • pursue partnerships with, or offer licenses to, qualified third parties to promote and sell product candidates, if approved, in domestic and key foreign markets where we receive marketing approval; • develop our product candidates for additional indications or for use in broader patient populations; • maintain patent and trade secret protection and regulatory exclusivity for our product candidates; and • qualify for, identify, register, maintain, enforce, and defend intellectual property rights and claims covering our products and intellectual property portfolio; and not infringe on others’ intellectual property rights.
The market price for our common stock may be influenced by those factors discussed in this "Risk Factors" section and many others, including: • our ability to enroll subjects in our ongoing and planned clinical trials; • the results of our clinical trials and preclinical studies, and the results of trials of our competitors or those of other companies in our market sector; • regulatory approval of our product candidates, or limitations to specific label indications or patient populations for use, or changes or delays in the regulatory review process; • the level of expenses related to any of our product candidates or clinical development programs; • regulatory developments in the United States and foreign countries; • reports of adverse events in any of our products, competing biologics, or gene therapy products; • changes in the structure of healthcare payment systems, especially in light of current reforms to the United States healthcare system; • the success or failure of our efforts to acquire, license, or develop additional product candidates; • innovations or new products developed by us or our competitors; • announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures, or capital commitments; • manufacturing, supply, or distribution delays or shortages; • any changes to our relationship with any manufacturers, suppliers, licensors, future collaborators, or other strategic partners; • achievement of expected product sales and profitability; • variations in our financial results or those of companies that are perceived to be similar to ours; • market conditions in the biopharmaceutical sector and issuance of securities analysts' reports or recommendations; • trading volume of our common stock; • an inability to obtain additional funding; • sales of our stock by insiders and stockholders or the perception that such sales could occur; • our ability to effectively manage our growth; • ineffectiveness of our internal control over financial reporting; • additions or departures of key personnel, including major changes in our board or management; • intellectual property, product liability, or other litigation against us; and • general economic, industry, market conditions, and other events or factors, many of which are beyond our control.
The market price for our common stock may be influenced by those factors discussed in this "Risk Factors" section and many others, including: • our ability to enroll subjects in our ongoing and planned clinical trials; • the results of our clinical trials and preclinical studies, and the results of trials of our competitors or those of other companies in our market sector; • regulatory approval of our product candidates, or limitations to specific label indications or patient populations for use, or changes or delays in the regulatory review process; • the level of expenses related to any of our product candidates or clinical development programs; • regulatory developments in the United States and foreign countries; • reports of adverse events in any of our products, competing biologics, or gene therapy products; • changes in the structure of healthcare payment systems, especially in light of current reforms to the United States healthcare system; • the success or failure of our efforts to acquire, license, or develop additional product candidates; • innovations or new products developed by us or our competitors; • announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures, or capital commitments; • manufacturing, supply, or distribution delays or shortages; • any changes to our relationship with any manufacturers, suppliers, licensors, future collaborators, or other strategic partners; • achievement of expected product sales and profitability; • variations in our financial results or those of companies that are perceived to be similar to ours; • market conditions in the biopharmaceutical sector and issuance of securities analysts' reports or recommendations; • trading volume of our common stock; • an inability to obtain additional funding; • sales of our stock by insiders and stockholders or the perception that such sales could occur; • our ability to effectively manage our growth; • ineffectiveness of our internal control over financial reporting; • additions or departures of key personnel, including major changes in our board or management; 78 Table of Contents • intellectual property, product liability, or other litigation against us; and • general economic, industry, market conditions, and other events or factors, many of which are beyond our control.
Our operating results will suffer if we fail to compete effectively. • If third-party payors do not reimburse patients for our products candidates, if approved, or if reimbursement levels are set too low for us to sell our product candidates at a profit, our ability to successfully commercialize our product candidates, if approved, and our results of operations will be harmed. • We rely, and expect to continue to rely, on third parties to conduct, supervise, and monitor our preclinical studies and clinical trials we may initiate, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials or failing to comply with regulatory requirements. • If we encounter difficulties in negotiating commercial manufacturing and supply agreements with third-party manufacturers and suppliers of our product candidates or any product components, our ability to commercialize our product candidates, if approved, would be impaired. 35 Table of Contents • If the manufacturers upon whom we rely on fail to produce our product candidates or product components pursuant to the terms of contractual arrangements with us or fail to comply with stringent regulations applicable to biotechnology manufacturers, we may face delays in the development and commercialization of, or be unable to meet demand for, our product candidates, if approved, and may lose potential revenues. • We may seek to collaborate with third parties for the development or commercialization of our product candidates.
Our operating results will suffer if we fail to compete effectively. • If third-party payors do not reimburse patients for our products candidates, if approved, or if reimbursement levels are set too low for us to sell our product candidates at a profit, our ability to successfully commercialize our product candidates, if approved, and our results of operations will be harmed. • We rely, and expect to continue to rely, on third parties to conduct, supervise, and monitor our preclinical studies and clinical trials we may initiate, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials or failing to comply with regulatory requirements. • If we encounter difficulties in negotiating commercial manufacturing and supply agreements with third-party manufacturers and suppliers of our product candidates or any product components, our ability to commercialize our product candidates, if approved, would be impaired. 36 Table of Contents • If the manufacturers upon whom we rely on fail to produce our product candidates or product components pursuant to the terms of contractual arrangements with us or fail to comply with stringent regulations applicable to biotechnology manufacturers, we may face delays in the development and commercialization of, or be unable to meet demand for, our product candidates, if approved, and may lose potential revenues. • We may seek to collaborate with third parties for the development or commercialization of our product candidates.
The provisions in our charter documents include the following: • a classified Board of Directors with three-year staggered terms, which may delay the ability of stockholders to change the membership of a majority of our Board; • no cumulative voting in the election of directors, which limits the ability of minority stockholders to elect director candidates; • the exclusive right of our Board, unless the Board grants such right to the stockholders, to elect a director to fill a vacancy created by the expansion of the Board or the resignation, death, or removal of a director, which prevents stockholders from being able to fill vacancies on our Board; • the prohibition on removal of directors without cause due to the classified Board; • the ability of our Board to authorize the issuance of shares of preferred stock and to determine the price and other terms of those shares, including preferences and voting rights, without stockholder approval, which could be used to significantly dilute the ownership of a hostile acquiror; • the ability of our Board of Directors to alter our amended and restated bylaws without obtaining stockholder approval; • the required approval of at least 66-2/3% of the shares entitled to vote to adopt, amend, or repeal our amended and restated bylaws or repeal certain provisions of our amended and restated certificate of incorporation; • a prohibition on stockholder action by written consent, which forces stockholder action to be taken at an annual or special meeting of our stockholders; • an exclusive forum provision providing that the Court of Chancery of the State of Delaware will be the exclusive forum for certain actions and proceedings; • the requirement that a special meeting of stockholders may be called only by the chairman of the Board of Directors, the Chief Executive Officer, or the Board, which may delay the ability of our stockholders to force consideration of a proposal or to take action, including the removal of directors; and • advance notice procedures that stockholders must comply with in order to nominate candidates to our Board or to propose matters to be acted upon at a stockholders’ meeting, which may discourage or deter a potential acquiror from 75 Table of Contents conducting a solicitation of proxies to elect the acquiror’s own slate of directors or otherwise attempting to obtain control of us.
The provisions in our charter documents include the following: • a classified Board of Directors with three-year staggered terms, which may delay the ability of stockholders to change the membership of a majority of our Board; 76 Table of Contents • no cumulative voting in the election of directors, which limits the ability of minority stockholders to elect director candidates; • the exclusive right of our Board, unless the Board grants such right to the stockholders, to elect a director to fill a vacancy created by the expansion of the Board or the resignation, death, or removal of a director, which prevents stockholders from being able to fill vacancies on our Board; • the prohibition on removal of directors without cause due to the classified Board; • the ability of our Board to authorize the issuance of shares of preferred stock and to determine the price and other terms of those shares, including preferences and voting rights, without stockholder approval, which could be used to significantly dilute the ownership of a hostile acquiror; • the ability of our Board of Directors to alter our amended and restated by laws without obtaining stockholder approval; • the required approval of at least 66-2/3% of the shares entitled to vote to adopt, amend, or repeal our amended and restated bylaws or repeal certain provisions of our amended and restated certificate of incorporation; • a prohibition on stockholder action by written consent, which forces stockholder action to be taken at an annual or special meeting of our stockholders; • an exclusive forum provision providing that the Court of Chancery of the State of Delaware will be the exclusive forum for certain actions and proceedings; • the requirement that a special meeting of stockholders may be called only by the chairman of the Board of Directors, the Chief Executive Officer, or the Board, which may delay the ability of our stockholders to force consideration of a proposal or to take action, including the removal of directors; and • advance notice procedures that stockholders must comply with in order to nominate candidates to our Board or to propose matters to be acted upon at a stockholders’ meeting, which may discourage or deter a potential acquiror from conducting a solicitation of proxies to elect the acquiror’s own slate of directors or otherwise attempting to obtain control of us.
In addition, later discovery of previously unknown adverse events or that the product is less effective than previously thought or other problems with our products, manufacturers, or manufacturing processes, or failure to comply with regulatory requirements both before and after approval, may yield various results, including: • restrictions on manufacturing, distribution, or marketing of such products; • restrictions on the labeling, including restrictions on the indication or approved patient population, and required additional warnings, such as black box warnings, contraindications, and precautions; • modifications to promotional pieces; • issuance of corrective information; • requirements to conduct post-marketing studies or other clinical trials; • clinical holds or termination of clinical trials; • requirements to establish or modify a REMS, or a comparable foreign authority may require that we establish or modify a similar strategy; • liability for harm caused to patients or subjects; • reputational harm; • warning, untitled, Form 483s, or cyber letters; • suspension of marketing or withdrawal or recall of the products from the market; • regulatory authority issuance of safety alerts, Dear Healthcare Provider letters, press releases, or other communications containing warnings or other safety information about the product; • refusal to approve pending applications or supplements to approved applications that we submit; • fines, restitution, or disgorgement of profits or revenues; • suspension or withdrawal of marketing approvals; • refusal to permit the import or export of our products; • product seizure or detention; • FDA debarment, suspension and debarment from government contracts, and refusal of orders under existing government contracts, exclusion from federal healthcare programs, consent decrees, or corporate integrity agreements; or • injunctions or the imposition of civil or criminal penalties, including imprisonment.
In addition, later discovery of previously unknown adverse events or that the product is less effective than previously thought or other problems with our products, manufacturers, or manufacturing processes, or failure to comply with regulatory requirements both before and after approval, may yield various results, including: • restrictions on manufacturing, distribution, or marketing of such products; • restrictions on the labeling, including restrictions on the indication or approved patient population, and required additional warnings, such as black box warnings, contraindications, and precautions; • modifications to promotional pieces; • issuance of corrective information; • requirements to conduct post-marketing studies or other clinical trials; • clinical holds or termination of clinical trials; • requirements to establish or modify a REMS, or a comparable foreign authority may require that we establish or modify a similar strategy; • liability for harm caused to patients or subjects; • reputational harm; • warning, untitled, Form 483s, or cyber letters; • suspension of marketing or withdrawal or recall of the products from the market; 52 Table of Contents • regulatory authority issuance of safety alerts, Dear Healthcare Provider letters, press releases, or other communications containing warnings or other safety information about the product; • refusal to approve pending applications or supplements to approved applications that we submit; • fines, restitution, or disgorgement of profits or revenues; • suspension or withdrawal of marketing approvals; • refusal to permit the import or export of our products; • product seizure or detention; • FDA debarment, suspension and debarment from government contracts, and refusal of orders under existing government contracts, exclusion from federal healthcare programs, consent decrees, or corporate integrity agreements; or • injunctions or the imposition of civil or criminal penalties, including imprisonment.
If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business and financial results, including, without limitation, the imposition of significant civil, criminal and administrative penalties, damages, monetary fines, disgorgements, possible exclusion from participation in Medicare, Medicaid, and other federal healthcare programs, individual imprisonment, contractual damages, reputational harm, diminished profits and future earnings, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of non-compliance with these laws, and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations. 67 Table of Contents We are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws, and other laws governing our operations.
If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business and financial results, including, without limitation, the imposition of significant civil, criminal and administrative penalties, damages, monetary fines, disgorgements, possible exclusion from participation in Medicare, Medicaid, and other federal healthcare programs, individual imprisonment, contractual damages, reputational harm, diminished profits and future earnings, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of non-compliance with these laws, and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations. 68 Table of Contents We are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws, and other laws governing our operations.
Additionally, our expenses will also increase if, and, as we: • initiate preclinical studies and clinical trials for any additional product candidates that we may pursue in the future, particularly if there are any delays in enrollment of patients in or completing our clinical trials or the development of our product candidates; • seek marketing approvals for product candidates that successfully complete clinical development; • establish sales, marketing, and distribution capabilities for our product candidates for which we obtain a regulatory approval; 37 Table of Contents • scale up our manufacturing processes and capabilities to support our clinical trials of our product candidates and commercialization of any of our product candidates for which we obtain a regulatory approval; • expand our operational, financial, and management systems and increase personnel, including personnel to support our clinical development, manufacturing, and commercialization efforts, and our operations as a public company; • acquire other companies, products, product candidates, or technologies, or in-license the rights to other products, product candidates, or technologies; and • develop, maintain, expand, and protect our intellectual property portfolio.
Additionally, our expenses will also increase if, and, as we: • initiate preclinical studies and clinical trials for any additional product candidates that we may pursue in the future, particularly if there are any delays in enrollment of patients in or completing our clinical trials or the development of our product candidates; • seek marketing approvals for product candidates that successfully complete clinical development; 38 Table of Contents • establish sales, marketing, and distribution capabilities for our product candidates for which we obtain a regulatory approval; • scale up our manufacturing processes and capabilities to support our clinical trials of our product candidates and commercialization of any of our product candidates for which we obtain a regulatory approval; • expand our operational, financial, and management systems and increase personnel, including personnel to support our clinical development, manufacturing, and commercialization efforts, and our operations as a public company; • acquire other companies, products, product candidates, or technologies, or in-license the rights to other products, product candidates, or technologies; and • develop, maintain, expand, and protect our intellectual property portfolio.
Patient enrollment is affected by other factors including: • the size and nature of the patient population (for instance, we are pursuing clinical trials for certain orphan indications, for which the size of the patient population is limited); • the severity of the disease under investigation; • the existence of current treatments for the indications for which we are conducting clinical trials; • the eligibility criteria for and design of the clinical trial in question, including factors such as frequency of required assessments, length of the study, and ongoing monitoring requirements; • the perceived risks and benefits of the product candidate, including the potential advantages or disadvantages of the product candidate being studied in relation to other available therapies; • competition in recruiting and enrolling patients in clinical trials; • efforts to facilitate timely enrollment in clinical trials; • patient referral practices of physicians; • effectiveness of publicity created by clinical trial sites regarding the trial; • patients’ ability to comply with the specific instructions related to the trial protocol, proper documentation, and use of the product candidate; • an inability to obtain or maintain patients' informed consents; • the risk that enrolled patients will drop out before completion or not return for post-treatment follow-up; • the ability to monitor patients adequately during and after treatment; • the ability to compensate patients for their time and effort; and • the proximity and availability of clinical trial sites for prospective patients.
Patient enrollment is affected by other factors including: • the size and nature of the patient population (for instance, we are pursuing clinical trials for certain orphan indications, for which the size of the patient population is limited); • the severity of the disease under investigation; • the existence of current treatments for the indications for which we are conducting clinical trials; • the eligibility criteria for and design of the clinical trial in question, including factors such as frequency of required assessments, length of the study, and ongoing monitoring requirements; • the perceived risks and benefits of the product candidate, including the potential advantages or disadvantages of the product candidate being studied in relation to other available therapies; • competition in recruiting and enrolling patients in clinical trials; • efforts to facilitate timely enrollment in clinical trials; • patient referral practices of physicians; • effectiveness of publicity created by clinical trial sites regarding the trial; • patients’ ability to comply with the specific instructions related to the trial protocol, proper documentation, and use of the product candidate; • an inability to obtain or maintain patients' informed consents; • the risk that enrolled patients will drop out before completion or not return for post-treatment follow-up; • the ability to monitor patients adequately during and after treatment; 49 Table of Contents • the ability to compensate patients for their time and effort; and • the proximity and availability of clinical trial sites for prospective patients.
Regardless of merit or eventual outcome, liability claims may result in: • loss of revenue from decreased demand for our products and/or product candidates; • impairment of our business reputation or financial stability; • costs of related litigation; • substantial monetary awards to patients or other claimants; • exhaustion of any available insurance and our capital resources; • diversion of management attention; • withdrawal of clinical trial participants and potential termination of clinical trial sites or entire clinical programs; • the inability to commercialize our product candidates; • significant negative media attention; 80 Table of Contents • decrease in our stock price; • initiation of investigations and enforcement actions by regulators; or • product recalls, withdrawals, revocation of approvals, or labeling, marketing, or promotional restrictions.
Regardless of merit or eventual outcome, liability claims may result in: • loss of revenue from decreased demand for our products and/or product candidates; 81 Table of Contents • impairment of our business reputation or financial stability; • costs of related litigation; • substantial monetary awards to patients or other claimants; • exhaustion of any available insurance and our capital resources; • diversion of management attention; • withdrawal of clinical trial participants and potential termination of clinical trial sites or entire clinical programs; • the inability to commercialize our product candidates; • significant negative media attention; • decrease in our stock price; • initiation of investigations and enforcement actions by regulators; or • product recalls, withdrawals, revocation of approvals, or labeling, marketing, or promotional restrictions.
It is unclear these safety events and resulting FDA action may adversely impact the FDA’s approach to regulating any product candidates we may develop based on the use of our modifier gene therapy platform, or may negatively impact patients’, providers’, or public perception of such product candidates.
It is unclear whether these safety events and resulting FDA action may adversely impact the FDA’s approach to regulating any product candidates we may develop based on the use of our modifier gene therapy platform, or may negatively impact patients’, providers’, or public perception of such product candidates.
The terms of our Loan and Security Agreement with Avenue Capital Management II, L.P. and the lenders listed therein as well as our loan agreement (the "EB-5 Loan Agreement") with EB5 Life Sciences, L.P. ("EB-5 Life Sciences") require us to meet certain operating covenants and place restrictions on our operating and financial flexibility.
The terms of our Loan and Security Agreement with Avenue Capital Management II, L.P. and the lenders listed therein as well as our loan agreement (the "EB-5 Loan Agreement") with EB-5 Life Sciences, L.P. ("EB-5 Life Sciences") require us to meet certain operating covenants and place restrictions on our operating and financial flexibility.
For example, we hold exclusive licenses for patent families relating to OCU400, OCU410, OCU410ST, and OCU200, and an exclusive license in the United States, Europe, Japan, South Korea, Australia, China, and Hong Kong with respect to a mucosal COVID-19 vaccine.
For example, we hold exclusive licenses for patent families relating to OCU400, OCU410, OCU410ST, and OCU200, and an exclusive license in the United States, Europe, Japan, South Korea, Australia, China, Hong Kong and Canada with respect to a mucosal COVID-19 vaccine.
The Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, which could have a material adverse effect on our business, financial condition, results of operations, and prospects.
The Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, which may have a material adverse effect on our business, financial condition, results of operations, and prospects.
Although use of a product directed by off-label prescriptions may infringe our method-of-treatment patents, the practice is common across medical specialties, particularly in the United States, and such infringement is difficult to detect, prevent, or prosecute. 70 Table of Contents Third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
Although use of a product directed by off-label prescriptions may infringe our method-of-treatment patents, the practice is common across medical specialties, particularly in the United States, and such infringement is difficult to detect, prevent, or prosecute. 71 Table of Contents Third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
Our vendors may in turn incorporate artificial intelligence tools into their offerings, and the providers of these artificial intelligence tools may not meet existing or rapidly evolving regulatory or industry standards, including with respect to privacy and data security.
Our vendors may in turn incorporate AI tools into their offerings, and the providers of these artificial intelligence tools may not meet existing or rapidly evolving regulatory or industry standards, including with respect to privacy and data security.
In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States, and the standards applied by the USPTO and foreign patent offices in granting patents are not always applied uniformly or predictably.
In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States, and because the standards applied by the USPTO and foreign patent offices in granting patents are not always applied uniformly or predictably.
Periodic maintenance, renewal, and annuity fees on any issued patent must be paid to the USPTO and foreign patent agencies in several stages or annually over the lifetime of our owned and licensed patents and patent applications.
Periodic maintenance, renewal, and annuity fees on any issued patent and pending patent applications must be paid to the USPTO and foreign patent agencies in several stages or annually over the lifetime of our owned and licensed patents and patent applications.
Termination of these agreements or reduction or elimination of our rights under these agreements may result in us having to negotiate new or reinstated agreements with less favorable terms or cause us to lose our rights under these agreements, including our rights to important intellectual property or technology.
Termination of these agreements or reduction or elimination of our rights under these agreements may result in us having to negotiate new or reinstated agreements with possibly less favorable terms or cause us to lose our rights under these agreements, including our rights to important intellectual property or technology.
If these Licensors terminate their agreements with us or fail to maintain or enforce the underlying patents or licenses thereto, or we otherwise lose our rights to these patents, our competitive position and our market share in the markets for any of our licensed and approved products will be harmed. 71 Table of Contents A substantial portion of our patent portfolio is in-licensed.
If these licensors terminate their agreements with us or fail to maintain or enforce the underlying patents or licenses thereto, or we otherwise lose our rights to these patents, our competitive position and our market share in the markets for any of our licensed and approved products will be harmed. 72 Table of Contents A substantial portion of our patent portfolio is in-licensed.
A failure to comply with the applicable regulatory requirements may result in regulatory enforcement actions against our manufacturers or us, including fines and civil and criminal penalties, including imprisonment, suspension or restrictions of production, injunctions, delays, withdrawal or denial of product approval or supplements to approved products, clinical holds or termination 62 Table of Contents of clinical studies, warning or untitled letters, Form 483s, regulatory authority communications warning the public about safety issues with the product, refusal to permit the import or export of the products, product seizure, detention, or recall, operating restrictions, suits under the civil FCA, corporate integrity agreements, or consent decrees.
A failure to comply with the applicable regulatory requirements may result in regulatory enforcement actions against our manufacturers or us, including fines and civil and criminal penalties, including imprisonment, suspension or restrictions of production, injunctions, delays, withdrawal or denial of product approval or supplements to approved products, clinical holds or termination of clinical studies, warning or untitled letters, Form 483s, regulatory authority communications warning the public about safety issues with the product, refusal to permit the import or export of the products, product seizure, detention, or recall, operating restrictions, suits under the civil FCA, corporate integrity agreements, or consent decrees.
For example, in November 2023, the FDA announced it was investigating the risk of T cell malignancies associated with currently approved BCMA- and CD19-directed autologous CAR T cell immunotherapies. The FDA subsequently announced class safety labeling changes to highlight these risks and recommend life-long monitoring of patients and clinical trial participants receiving treatment with these products.
For example, in November 2023, the FDA announced it was investigating the risk of T cell malignancies associated with currently approved BCMA- and CD19-directed autologous CAR T cell immunotherapies. The FDA subsequently announced class safety labeling changes to highlight these risks and recommended life-long monitoring of patients and clinical trial participants receiving treatment with these products.
Any failure by us or our service providers, collaborators, consultants, contractors or partners to detect, prevent, respond to or mitigate data breaches or cybersecurity incidents, compromises, or improper access to, use of, or inappropriate disclosure of any of this information or other confidential or sensitive information, including patients’ personal data, or the perception that any such failure has occurred, could result in claims, litigation, regulatory investigations and other proceedings, significant liability under state, federal and international law, and other financial, legal or reputational harm to us, including class action lawsuits from affected individuals.
Any failure by us or our service providers, collaborators, consultants, contractors or partners to detect, prevent, respond to or mitigate data breaches or cybersecurity incidents, compromises, or improper access to, use of, or inappropriate disclosure of any of this information or other confidential or sensitive information, including patients’ personal data, or the perception that any such failure has occurred, could result in claims, litigation, regulatory investigations and other proceedings, significant 82 Table of Contents liability under state, federal and international law, and other financial, legal or reputational harm to us, including class action lawsuits from affected individuals.
We anticipate that our expenses will increase in fiscal year 2025 compared to fiscal year 2024 as we continue to conduct clinical activities with respect to our product candidates, including the continuation of several clinical trials for our product candidates, as well as increased headcount, including management personnel to support our research and development, clinical, and business activities, and expanded infrastructure, among other factors.
We anticipate that our expenses will increase in fiscal year 2026 compared to fiscal year 2025 as we continue to conduct clinical activities with respect to our product candidates, including the continuation of several clinical trials for our product candidates, as well as increased headcount, including management personnel to support our research and development, clinical, and business activities, and expanded infrastructure, among other factors.
The availability of the PTAB as a lower-cost, faster, and potentially more potent tribunal for challenging patents could therefore increase the likelihood that our own patents will be challenged, thereby increasing the uncertainties and costs of maintaining, defending, and enforcing them. 69 Table of Contents If we are not able to obtain patent term extension in the United States under the Hatch-Waxman Act and in foreign countries under similar legislation, our business may be materially harmed.
The availability of the PTAB as a lower-cost, faster, and potentially more potent tribunal for challenging patents may therefore increase the likelihood that our own patents will be challenged, thereby increasing the uncertainties and costs of maintaining, defending, and enforcing them. 70 Table of Contents If we are not able to obtain patent term extension in the United States under the Hatch-Waxman Act and in foreign countries under similar legislation, our business may be materially harmed.
See the section of this Annual Report for the fiscal year ended December 31, 2024 entitled "Legal Proceedings." The complaints seek unspecified damages, interest, attorneys' fees, and other costs. We believe that these lawsuits are without merit and intend to vigorously defend against them.
See the section of this Annual Report for the fiscal year ended December 31, 2025 entitled "Legal Proceedings." The complaints seek unspecified damages, interest, attorneys' fees, and other costs. We believe that these lawsuits are without merit and intend to vigorously defend against them.
These include provisions that affect the way patent applications are prosecuted and may also affect patent litigation. The USPTO developed new regulations and procedures to govern administration of the Leahy-Smith Act, and many of the substantive changes to patent law associated with the Leahy-Smith Act, and in particular, the first to file provisions, became effective in 2013.
These include provisions that affect the way patent applications are prosecuted and may also affect patent litigation. The USPTO instituted new regulations and procedures to govern administration of the Leahy-Smith Act, and many of the substantive changes to patent law associated with the Leahy-Smith Act, and in particular, the first to file provisions, became effective in 2013.
Under the Bayh-Dole Act, the United States government also has the right to take title to inventions developed using a United States government funded program, if one fails to disclose the invention to the 72 Table of Contents government and fails to file an application to register the intellectual property within specified time limits.
Under the Bayh-Dole Act, the United States government also has the right to take title to 73 Table of Contents inventions developed using a United States government funded program, if one fails to disclose the invention to the government and fails to file an application to register the intellectual property within specified time limits.
In particular, during prosecution of any patent application, the issuance of any patents based on the application may depend upon our ability to generate additional preclinical or clinical data that support the patentability of our proposed claims. We may not be able to generate sufficient additional data on a timely basis, or at all.
During prosecution of any patent application, the issuance of any patents based on the application may depend upon our ability to generate additional preclinical or clinical data that support the patentability of our proposed claims. We may not be able to generate sufficient additional data on a timely basis, or at all.
This amount will not meet our capital requirements over the next 12 months. We estimate that our cash and cash equivalents will enable us to fund our operations into the first quarter of 2026. Based on this estimate, we will need to raise significant additional capital in order to fund our future operations.
This amount will not meet our capital requirements over the next 12 months. We estimate that our cash and cash equivalents will enable us to fund our operations into the fourth quarter of 2026. Based on this estimate, we will need to raise significant additional capital in order to fund our future operations.
Our success depends in large part on our ability to obtain and maintain patent protection in the United States and other countries, with respect to our proprietary technology and product candidates. We have sought to protect our proprietary position by filing in the United States and in certain foreign jurisdictions, patent applications related to our novel technologies and product candidates.
Our success depends in part on our ability to obtain and maintain patent protection in the United States and other countries, with respect to our proprietary technology and product candidates. We have sought to protect our proprietary position by filing in the United States and in certain foreign jurisdictions, patent applications related to our technologies and product candidates.
See the section of this Annual Report for the fiscal year ended December 31, 2024 entitled, "Legal Proceedings." If securities or industry analysts do not publish research or reports or publish unfavorable research or reports about our business, our stock price and trading volume could decline.
See the section of this Annual Report for the fiscal year ended December 31, 2025 entitled, "Legal Proceedings." If securities or industry analysts do not publish research or reports or publish unfavorable research or reports about our business, our stock price and trading volume could decline.
If the FDA or comparable foreign regulatory authorities become aware of new safety information after the approval of any of our product candidates, they may, among other actions, withdraw approval, require labeling changes or establishment of a REMS or similar strategy, impose significant restrictions on a 51 Table of Contents product’s indicated uses or marketing, or impose ongoing requirements for potentially costly post-approval studies or post-market surveillance.
If the FDA or comparable foreign regulatory authorities become aware of new safety information after the approval of any of our product candidates, they may, among other actions, withdraw approval, require labeling changes or establishment of a REMS or similar strategy, impose significant restrictions on a product’s indicated uses or marketing, or impose ongoing requirements for potentially costly post-approval studies or post-market surveillance.
Gene therapy clinical trials conducted at institutions that receive funding for recombinant deoxyribonucleic acid ("DNA") research from the NIH are also subject to review by the NIH Novel and Exceptional Technology and Research Advisory Committee ("NExTRAC"), formerly the Recombinant DNA Advisory Committee, which now focuses on emerging areas of research including, but not restricted to, technologies surrounding advances in recombinant or synthetic nucleic acid research.
Gene therapy clinical trials conducted at institutions that receive funding for recombinant deoxyribonucleic acid ("DNA") research from the NIH may also be subject to review by the NIH Novel and Exceptional Technology and Research Advisory Committee ("NExTRAC"), formerly the Recombinant DNA Advisory Committee, which now focuses on emerging areas of research including, but not restricted to, technologies surrounding advances in recombinant or synthetic nucleic acid research.
Any problems or delays we experience in preparing for commercial-scale manufacturing of a product candidate or component, including manufacturing validation, may result in a delay in FDA approval or commercial launch, if approved, of the product candidate or may impair our ability to manufacture commercial quantities or such quantities at an acceptable cost, which could result in the delay, prevention, or impairment of commercialization of our product candidates and could adversely affect our business.
Any problems or delays we experience in preparing for commercial-scale manufacturing of a product candidate or component, including manufacturing validation, may result in a delay in FDA approval or commercial launch, if approved, of the product candidate or may impair our ability to manufacture commercial quantities or such quantities at an acceptable cost, which could result in the delay, prevention, or impairment of commercialization of our product candidates and could adversely affect our 63 Table of Contents business.
This is particularly true with respect to OCU200, our novel biologic product candidate, in the case of Wet AMD, which is most prevalent in persons over age 55. Government authorities and other third-party payors, such as private health insurers and health maintenance organizations, determine which medications they will cover and establish reimbursement levels.
This is particularly true with respect to OCU200, our novel biologic product candidate, in the case of Wet AMD, which is most prevalent in persons over age 55. Government authorities and other third-party payors, such as private health 59 Table of Contents insurers and health maintenance organizations, determine which medications they will cover and establish reimbursement levels.
In such instances, an appropriate replacement third-party relationship may not be readily available to us or on acceptable terms, which would cause additional delays and increased expense and may have a material adverse effect on our business. 63 Table of Contents We may seek to collaborate with third parties for the development or commercialization of our product candidates.
In such instances, an appropriate replacement third-party relationship may not be readily available to us or on acceptable terms, which would cause additional delays and increased expense and may have a material adverse effect on our business. We may seek to collaborate with third parties for the development or commercialization of our product candidates.
In some cases, we may not be able to or it may be difficult to timely identify patentable aspects of our research and development output to obtain patent protection. 68 Table of Contents The patent position of pharmaceutical and biotechnology companies generally is highly uncertain, involves complex legal and factual questions, and has in recent years been the subject of much litigation.
In some cases, we may not be able to or it may be difficult to timely identify patentable aspects of our research and development output to obtain patent protection. 69 Table of Contents The patent position of pharmaceutical and biotechnology companies generally is uncertain, involves complex legal and factual questions, and has in recent years been the subject of much litigation.
Proceedings to enforce our intellectual property and proprietary rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly, could put our patent applications at risk of not being issued, and could provoke third parties to assert claims against us.
Proceedings to enforce our intellectual property and proprietary rights in the U.S. and foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly, could put our patent applications at risk of not being issued, and could provoke third parties to assert claims against us.
The regulatory approval and successful commercialization of OCU400, OCU410, and OCU410ST depend on the successful development of this platform. There can be no assurance that any development problems we experience in the future related to our modifier gene therapy platform will not cause significant delays or unanticipated costs, or that such development problems can be solved.
The regulatory approval and successful commercialization of OCU400, OCU410, and OCU410ST depend on the successful development of this platform. There can be no assurance that any development problems we experience in the future related to our modifier gene therapy platform will not cause significant delays or unanticipated costs, or that such development problems 43 Table of Contents can be solved.
In the event that we are subject to or affected by privacy and data protection laws, including the CCPA, CPRA, or GDPR and other domestic or 82 Table of Contents international privacy and data protection laws, we may expend significant resources to comply with such laws, and any liability from failure to comply with the requirements of these laws could adversely affect our financial condition.
In the event that we are subject to or affected by privacy and data protection laws, including the CCPA, CPRA, or GDPR and other domestic or international privacy and data protection laws, we may expend significant resources to comply with such laws, and any liability from failure to comply with the requirements of these laws could adversely affect our financial condition.
We have based this estimate on assumptions that may prove to be wrong, and our operating and capital requirements may change as a result of many factors currently unknown to us. 38 Table of Contents Conducting preclinical testing and clinical trials is a time-consuming, expensive, and uncertain process that takes years to complete.
We have based this estimate on assumptions that may prove to be wrong, and our operating and capital requirements may change as a result of many factors currently unknown to us. Conducting preclinical testing and clinical trials is a time-consuming, expensive, and uncertain process that takes years to complete.
We have also received ODD and OMPD for OCU410ST for the treatment of ABCA4 -associated retinopathies including Stargardt, RP19 and CORD3 diseases. We may also seek ODD or OMPD for our other product candidates, as appropriate.
We have also received ODD and OMPD for OCU410ST for the treatment of ABCA4 -associated retinopathies including ST, RP19 and CORD3 diseases. We may also seek ODD or OMPD for our other product candidates, as appropriate.
We may also consider potential collaborative partnership opportunities for sales, marketing, distribution, development, or licensing or broader collaboration arrangements, including with mid-size and large pharmaceutical companies, regional and national pharmaceutical companies, and biotechnology companies. The success of our current and future collaboration arrangements will depend heavily on the efforts and activities of our collaborators.
We may also 64 Table of Contents consider potential collaborative partnership opportunities for sales, marketing, distribution, development, or licensing or broader collaboration arrangements, including with mid-size and large pharmaceutical companies, regional and national pharmaceutical companies, and biotechnology companies. The success of our current and future collaboration arrangements will depend heavily on the efforts and activities of our collaborators.
The existence of comprehensive privacy laws in different states in the country would make our compliance obligations more complex and costly and may increase the likelihood that we may be subject to enforcement actions or otherwise incur liability for noncompliance. There are also states that are specifically regulating health information.
The existence of comprehensive privacy laws in different states in the country would make our compliance obligations more complex and costly and may increase the likelihood that we may be subject to enforcement actions or otherwise incur liability for noncompliance. There are also states that are specifically 83 Table of Contents regulating health information.
We cannot predict when we will be able to generate the necessary data or results required to obtain regulatory approval of products with the market potential sufficient to enable us to achieve profitability, if ever. Accordingly, we may need to obtain substantial additional funding in connection with our continuing operations.
We cannot predict when we will be able to generate the necessary data or results required to obtain regulatory 39 Table of Contents approval of products with the market potential sufficient to enable us to achieve profitability, if ever. Accordingly, we may need to obtain substantial additional funding in connection with our continuing operations.
On November 6, 2024, we entered into a Loan and Security Agreement with Avenue Capital Management II, L.P., as administrative agent and collateral agent (the "Agent," together with Avenue I and Avenue II, "Avenue"), Avenue Venture Opportunities Fund II, L.P., as a lender ("Avenue 2"), and Avenue Venture Opportunities Fund, L.P., as a lender ("Avenue 1", and together with Avenue 2, the "Lenders") (the “Loan and Security Agreement”) that is secured by a lien on all of our assets.
On November 6, 2024, we entered into a Loan and Security Agreement with Avenue Venture Opportunities Fund II, L.P., as a lender ("Avenue 2"), Avenue Venture Opportunities Fund, L.P., as a lender ("Avenue 1", together with Avenue 2, the "Lenders") and Avenue Capital Management II, L.P., as administrative agent and collateral agent (the "Agent," together with the Lenders, "Avenue") that is secured by a lien on all of our assets (the "Loan and Security Agreement").
We will need to obtain FDA approval of any proposed product names, and any failure or delay associated with such approval may adversely affect our business. 52 Table of Contents Any name we intend to use for our product candidates will require approval from the FDA regardless of whether we have secured a formal trademark registration from the USPTO.
We will need to obtain FDA approval of any proposed product names, and any failure or delay associated with such approval may adversely affect our business. Any name we intend to use for our product candidates will require approval from the FDA regardless of whether we have secured a formal trademark registration from the USPTO.
The rapid evolution of artificial intelligence will require the application of significant resources to design, develop, test and maintain our products and services to help ensure that artificial intelligence is implemented in accordance with applicable law and regulation and in a socially responsible manner and to minimize any real or perceived unintended harmful impacts.
The rapid evolution of AI will require the application of significant resources to design, develop, test and maintain our products and services to help ensure that AI is implemented in accordance with applicable law and regulation and in a socially responsible manner and to minimize any real or perceived unintended harmful impacts.
These could prevent us from commercializing and generating revenues from the sale of our product candidates. Many compounds that initially showed promise in clinical or earlier stage testing have later been found to cause side effects that prevented further development of the compound.
These could prevent us from commercializing and generating revenues from the sale of our product candidates. 48 Table of Contents Many compounds that initially showed promise in clinical or earlier stage testing have later been found to cause side effects that prevented further development of the compound.
Further, for our licensed patents, we do not have the right to control prosecution, including filing with the USPTO, a petition for patent term extension under the Hatch-Waxman Act.
Further, for our licensed patents, we may not have the right to control prosecution, including filing with the USPTO, a petition for patent term extension under the Hatch-Waxman Act.
The delays associated with the verification of a new CDMO could negatively affect our ability to develop product candidates or commercialize our products in a timely manner or within budget. Furthermore, a CDMO may possess technology related to the manufacture of our product candidate that such CDMO owns independently.
The delays associated with the verification of a new CDMO could negatively affect our ability to 61 Table of Contents develop product candidates or commercialize our products in a timely manner or within budget. Furthermore, a CDMO may possess technology related to the manufacture of our product candidate that such CDMO owns independently.
If any collaborations do not result in the successful development and commercialization of our product candidates or if one of our collaborators subsequently terminates our agreement with us, we may not receive any future research funding, milestone, or royalty payments under the collaboration, as applicable.
If any collaborations do not result in the successful development and commercialization of our product candidates or if one of our collaborators subsequently terminates our agreement with us, we may not receive any future research 65 Table of Contents funding, milestone, or royalty payments under the collaboration, as applicable.
Further, the American Taxpayer Relief Act of 2012 among other things, reduced Medicare payments to several types of 66 Table of Contents providers, including hospitals, imaging centers and cancer treatment centers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years.
Further, the American Taxpayer Relief Act of 2012 among other things, reduced Medicare payments to several types of providers, including hospitals, imaging centers and cancer treatment centers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years.
If any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, from using that technology or information to compete with us.
If any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, 75 Table of Contents from using that technology or information to compete with us.
Our ability to successfully commercialize our product candidates, if approved, will depend in part on the extent to which coverage and adequate reimbursement for our product candidates will be available in a timely manner from third-party payors, 58 Table of Contents including governmental healthcare programs such as Medicare and Medicaid, commercial health insurers, and managed care organizations.
Our ability to successfully commercialize our product candidates, if approved, will depend in part on the extent to which coverage and adequate reimbursement for our product candidates will be available in a timely manner from third-party payors, including governmental healthcare programs such as Medicare and Medicaid, commercial health insurers, and managed care organizations.
These FCA lawsuits against sponsors of drugs and biologics have increased significantly in volume and breadth in recent years, leading to several substantial civil and criminal settlements, up to $3.0 billion, pertaining to certain sales practices and promoting off-label uses. In addition, FCA lawsuits may expose sponsors to follow-on claims by private payors based on fraudulent marketing practices.
These FCA lawsuits against sponsors of drugs and biologics have increased significantly in volume and breadth in recent years, leading to several substantial civil and criminal settlements pertaining to certain sales practices and promoting off-label uses. In addition, FCA lawsuits may expose sponsors to follow-on claims by private payors based on fraudulent marketing practices.
In such cases, our competitors will likely obtain approval of competing products sooner than we might have anticipated, and our revenue could be reduced.
In such cases, our competitors will likely obtain approval of competing products sooner than we might have anticipated, and our revenue could be impacted.
Any of these scenarios could compromise the commercial prospects for our product candidates. The development and manufacture of biologics is a complex process and entails particular risks. OCU200 is our novel biologic designed to treat retinal diseases.
Any of these scenarios could compromise the commercial prospects for our product candidates. 46 Table of Contents The development and manufacture of biologics is a complex process and entails particular risks. OCU200 is our novel biologic designed to treat retinal diseases.
As part of our annual review of the effectiveness of our internal control over financial reporting as of December 31, 2024, management has concluded that our internal control over financial reporting was effective.
As part of our annual review of the effectiveness of our internal control over financial reporting as of December 31, 2025, management has concluded that our internal control over financial reporting was effective.
For example, the IRA contains substantial drug pricing reforms, including the establishment of a drug price negotiation program within the United States Department of Health and Human Services that would require manufacturers to charge a negotiated "maximum fair price" for certain selected drugs administered or prescribed to Medicare beneficiaries or pay an excise tax for noncompliance; the establishment of rebate payment requirements on manufacturers of certain drugs payable under Medicare Parts B and D to penalize price increases that outpace inflation; and a requirement that manufacturers to provide discounts on Part D drugs.
In addition, the Inflation Reduction Act of 2022 ("IRA") contains substantial drug pricing reforms, including the establishment of a drug price negotiation program within the United States Department of Health and Human Services that would require manufacturers to charge a negotiated "maximum fair price" for certain selected drugs administered or prescribed to Medicare beneficiaries or pay an excise tax for noncompliance; the establishment of rebate payment requirements on manufacturers of certain drugs payable under Medicare Parts B and D to penalize price increases that outpace inflation; and a requirement that manufacturers to provide discounts on Part D drugs.
In order to satisfy our current and future debt service obligations, we will be required to raise funds from external sources. We may be unable to arrange for additional financing to pay the amounts due under our existing debt. Funds from external sources 40 Table of Contents may not be available on acceptable terms, if at all.
In order to satisfy our current and future debt service obligations, we will be required to raise funds from external sources. We may be unable to arrange for additional financing to pay the amounts due under our existing debt. Funds from external sources may not be available on acceptable terms, if at all.
The approval procedures vary among countries and the time required to obtain approval may differ substantially from that required to obtain FDA approval. The regulatory approval process outside the United States generally includes all of the risks associated with obtaining FDA approval.
The approval procedures vary among countries and the time required to obtain approval may differ substantially from that required to obtain FDA approval. The regulatory approval process outside the United States generally includes all of the risks associated with obtaining FDA 50 Table of Contents approval.
Our amended and restated certificate of incorporation and amended and restated bylaws contain provisions that could significantly reduce the value of our shares to a potential acquiror or delay or prevent changes in control or changes in our management without the consent of our Board.
Our amended and restated certificate of incorporation and amended and restated by laws contain provisions that could significantly reduce the value of our shares to a potential acquiror or delay or prevent changes in control or changes in our management without the consent of our Board.
If we raise additional funds through collaborations, strategic alliances, licensing arrangements, or marketing and distribution arrangements, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates, or grant licenses on terms that may not be favorable to us.
If we raise additional funds through collaborations, strategic alliances, licensing arrangements, or marketing and distribution arrangements, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or 40 Table of Contents product candidates, or grant licenses on terms that may not be favorable to us.
Significant political, trade, or regulatory developments in the jurisdictions in which we sell our products, if approved, such as those stemming from the change in U.S. federal administration, are difficult to predict and may have a material adverse effect on us.
Significant political, trade, or regulatory developments in the 53 Table of Contents jurisdictions in which we may sell our products, if approved, such as those stemming from the change in U.S. federal administration, are difficult to predict and may have a material adverse effect on us.
Additionally, we expect to see increasing government and supranational regulation related to artificial intelligence use and ethics, which may also significantly increase the burden and cost of research, development and compliance in this area.
Additionally, we expect to see increasing government and supranational regulation related to AI use and ethics, which may also significantly increase the burden and cost of research, development and compliance in this area.
Additionally, applicable laws and regulations relating to privacy, data protection or cybersecurity, external contractual commitments and internal privacy and security policies may require us to notify relevant stakeholders if there has been a cybersecurity incident or data breach, including affected 81 Table of Contents individuals, business partners and regulators.
Additionally, applicable laws and regulations relating to privacy, data protection or cybersecurity, external contractual commitments and internal privacy and security policies may require us to notify relevant stakeholders if there has been a cybersecurity incident or data breach, including affected individuals, business partners and regulators.
As of December 31, 2024, we had $30.0 million of outstanding principal borrowings under the Loan and Security Agreement.
As of December 31, 2025, we had $30.0 million of outstanding principal borrowings under the Loan and Security Agreement.
As a biotechnology company, we are subject to many federal and state healthcare laws, such as the federal Anti-Kickback Statute, the federal civil and criminal FCA, the civil monetary penalties statute, the Medicaid Drug Rebate statute and other price reporting requirements, the VHCA, HIPAA, the FCPA, the ACA, and similar state laws.
As a biotechnology company, we are subject to many federal and state healthcare laws, such as the federal Anti-Kickback Statute, the federal civil and criminal FCA, the civil monetary penalties statute, the Medicaid Drug Rebate statute and other 66 Table of Contents price reporting requirements, the VHCA, HIPAA, the FCPA, the ACA, and similar state laws.
While physicians may choose to prescribe products for uses that are not described in the product’s labeling and for uses that differ from those tested in clinical studies and approved by the regulatory 50 Table of Contents authorities, we are prohibited from marketing and promoting the products for indications and uses that are not specifically approved by the FDA.
While physicians may choose to prescribe products for uses that are not described in the product’s labeling and for uses that differ from those tested in clinical studies and approved by the regulatory authorities, we are prohibited from marketing and promoting the products for indications and uses that are not specifically approved by the FDA.
Additionally, as of December 31, 2024, we had $2.5 million of outstanding principal borrowings under the EB-5 Loan Agreement, which we are required to repay on the seventh anniversary of the date of the last disbursement under the EB-5 Loan Agreement (unless terminated earlier pursuant to the terms of the EB-5 Loan Agreement).
Additionally, as of December 31, 2025, we had $1.5 million of outstanding principal borrowings under the EB-5 Loan Agreement, which we are required to repay on the seventh anniversary of the date of the last disbursement under the EB-5 Loan Agreement (unless terminated earlier pursuant to the terms of the EB-5 Loan Agreement).
We have not generated significant revenue to date and have funded our operations to date through the sale of common stock, warrants to purchase common stock, the issuance of convertible notes and debt, and grant proceeds. We incurred net losses of approximately $54.1 million and $63.1 million for the years ended December 31, 2024 and 2023, respectively.
We have not generated significant revenue to date and have funded our operations to date through the sale of common stock, warrants to purchase common stock, the issuance of convertible notes and debt, and grant proceeds. We incurred net losses of approximately $67.8 million and $54.1 million for the years ended December 31, 2025 and 2024, respectively.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeIn order to monitor and appropriately escalate cybersecurity risks, our Associate Vice President of IT & Facilities receives reports on a monthly basis, and more frequently as appropriate, from our third-party cybersecurity vendor. 84 Table of Contents Our Board's role in risk oversight is consistent with our leadership structure, with management having day-to-day responsibility for assessing and managing our risk exposure and our Board actively overseeing the management of our risks both at the Board and Committee level.
Biggest changeOur Board's role in risk oversight is consistent with our leadership structure, with management having day-to-day responsibility for assessing and managing our risk exposure and our Board actively overseeing the management of our risks both at the Board and Committee level.
Risk factors” in this Annual Report, including “Our internal computer systems or those of our development collaborators, third-party CDMOs, or other contractors or consultants may fail or suffer cybersecurity incidents, data breaches, or other disruptions, which could result in a material disruption of our product development programs and cause our business and operations to suffer,” for additional discussion about cybersecurity-related risks.
Risk Factors” in this Annual Report, including the risk factor titled “Our internal computer systems or those of our development collaborators, third-party CDMOs, or other contractors or consultants may fail or suffer cybersecurity incidents, data breaches, or other disruptions, which could result in a material disruption of our product development programs and cause our business and operations to suffer,” for additional discussion of cybersecurity-related risks.
Our Associate Vice President of IT & Facilities briefs the Audit Committee on our cybersecurity risk management program on a quarterly basis, using risk assessment reports from our third-party cybersecurity vendor. The briefing includes discussion of management’s actions to identify and detect threats, as well as planned actions in the event of a response or recovery situation.
Our Associate Vice President of IT & Facilities briefs the Board of Directors on our cybersecurity risk management program on a quarterly basis, using risk assessment reports from our third-party cybersecurity vendor. The briefing includes discussion of management’s actions to identify and detect threats, as well as planned actions in the event of a response or recovery situation.
There have not been any risks from cybersecurity threats, including as a result of any cybersecurity incidents, which have materially affected or are reasonably likely to materially affect our Company, including our business strategy, results of operations, or financial condition. Refer to “Item 1A.
To date, we have not experienced any cybersecurity incidents that have materially affected, or are reasonably likely to materially affect, our company, including our business strategy, results of operations, or financial condition. Refer to “Item 1A.
Item 1C. Cybersecurity Cybersecurity Risk Management and Strategy We understand the importance of assessing, identifying, and managing risks associated with cybersecurity threats. Cybersecurity processes designed to assess, identify and manage risks from cybersecurity threats have been incorporated into our operations as a part of our overall risk assessment process.
Item 1C. Cybersecurity Cybersecurity Risk Management and Strategy We regularly assess, identify, and manage risks from cybersecurity threats as an integral part of our overall enterprise risk management (ERM) program.
Removed
To help us defend against, detect and respond to risks from cybersecurity threats, we engage a third-party cybersecurity firm to assist with aspects of our cybersecurity program including, but not limited to, network monitoring, cloud system monitoring, and employee cybersecurity awareness training.
Added
Our cybersecurity policies, processes, and practices include ongoing monitoring of information systems for vulnerabilities, periodic testing, in-house employee training and case discussions, and the use of advanced security tools designed to detect, prioritize, escalate, investigate, resolve, and recover from incidents in a timely manner.
Removed
Training topics include how to escalate suspicious activities including phishing, viruses, spams, insider threats, suspect human behaviors or safety issues. We also maintain cyber liability insurance. Our processes also include assessing cybersecurity risks associated with our use of third-party services providers in the normal course of business use.
Added
To evaluate how effective our cybersecurity prevention and response mechanisms are, we partner with external organizations such as cybersecurity assessors, consultants, and specialists. Their expertise enables us to pinpoint, confirm, and validate cybersecurity risks, as well as support the creation and implementation of mitigation strategies when required.
Removed
We assess cybersecurity considerations in the selection and oversight of our third-party services providers, including due diligence on the third parties that have access to our systems.
Added
By utilizing these systems and expert guidance, we can detect threats, determine their severity, and address possible repercussions by preventing breaches and relying on vendors who advise us on optimal risk management practices. These external parties and systems form a crucial part of our enterprise risk management framework for cybersecurity.
Added
Additionally, we have established a due diligence protocol to monitor and identify significant risks arising from our relationships with third-party vendors, including those providing cybersecurity services, to ensure we manage all threats related to their involvement appropriately.
Added
In order to monitor and appropriately escalate cybersecurity risks (including with respect to cybersecurity incidents), our Associate Vice President of IT & Facilities receives reports on a monthly basis, and more frequently as appropriate, from our third-party cybersecurity vendor.

Item 2. Properties

Properties — owned and leased real estate

3 edited+1 added−0 removed1 unchanged
Biggest changeWe lease two other general use facilities within the United States, which have initial terms of two to three years and contain no option to extend. We believe that our existing facilities are adequate to meet our current needs, and that suitable additional space will be available as and when needed.
Biggest changeWe believe that our existing facilities are adequate to meet our current needs, and that suitable additional space will be available as and when needed. 86 Table of Contents
Our current GMP facility has an initial term of seven years and includes an option to extend the lease for up to five years, which we have elected to account for since it is reasonably certain that we will exercise such option.
Our current GMP facility has a lease term of seven years and includes an option to extend the lease for up to five years, which we have elected to account for since it is reasonably certain that we will exercise such option.
Our corporate headquarters has an initial term of approximately seven years and includes options to extend the leases for up to 10 years, which we have not elected to account for since it is not reasonably certain that we will exercise such option.
Our corporate headquarters has a lease term of approximately seven years and includes options to extend the lease for up to 10 years, which we have not elected to account for since it is not reasonably certain that we will exercise such option.
Added
We lease three other general use facilities within the United States, which have initial terms of two to three years and contain no option to extend. The Company also has leases in Canada and India which have terms of four to five years and contain no option to extend.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeRegardless of the outcome, litigation can have an adverse impact on us because of defense and settlement costs, diversion of management resources and other factors. For a discussion of legal proceedings, see Note 14 in the notes to the consolidated financial statements included elsewhere in this Annual Report. This discussion is incorporated herein by reference. Item 4. Mine Safety Disclosures.
Biggest changeRegardless of the outcome, litigation can have an adverse impact on us because of defense and settlement costs, diversion of management resources and other factors. For a discussion of legal proceedings, see Note 16 in the notes to the consolidated financial statements included elsewhere in this Annual Report. This discussion is incorporated herein by reference. Item 4. Mine Safety Disclosures.
Not applicable. 85 Table of Contents PART II
Not applicable. 87 Table of Contents PART II

Item 4. Mine Safety Disclosures

Mine Safety Disclosures — required of mining issuers

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Biggest changeItem 4. Mine Safety Disclosures 85 Part II Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities 86 Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations 87 Item 7A . Quantitative and Qualitative Disclosures About Market Risk 97 Item 8. Financial Statements and Supplementary Data 97
Biggest changeItem 4. Mine Safety Disclosures 87 Part II Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities 88 Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations 89 Item 7A . Quantitative and Qualitative Disclosures About Market Risk 102 Item 8. Financial Statements and Supplementary Data 102

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

4 edited+0 added−0 removed2 unchanged
Biggest changeFurthermore, our ability to pay cash dividends is currently restricted by the terms of our Loan and Security Agreement with Avenue 1 and Avenue 2. As a result, we anticipate that only the appreciation of the price of our common stock, if any, will provide a return to investors for at least the foreseeable future.
Biggest changeFurthermore, our ability to pay cash dividends is currently restricted by the terms of our Loan and Security Agreement. As a result, we anticipate that only the appreciation of the price of our common stock, if any, will provide a return to investors for at least the foreseeable future. Unregistered Sales of Equity Securities and Use of Proceeds None.
Securities Authorized for Issuance Under Equity Compensation Plans The information required by Item 5 of Form 10-K regarding equity compensation plans is incorporated herein by reference to Item 12 of Part III of this Annual Report. 86 Table of Contents
Securities Authorized for Issuance Under Equity Compensation Plans The information required by Item 5 of Form 10-K regarding equity compensation plans is incorporated herein by reference to Item 12 of Part III of this Annual Report. 88 Table of Contents
Item 5. Market for Registrant's Common Equity, Related Shareholder Matters and Issuer Purchases of Equity Securities. Market Information Our common stock is traded on the Nasdaq under the symbol "OCGN." Holders As of February 26, 2025, we had 292.0 million shares of common stock outstanding held by approximately 26 stockholders of record.
Item 5. Market for Registrant's Common Equity, Related Shareholder Matters and Issuer Purchases of Equity Securities. Market Information Our common stock is traded on the Nasdaq under the symbol "OCGN." Holders As of February 24, 2026, we had 327.9 million shares of common stock outstanding held by approximately 27 stockholders of record.
Unregistered Sales of Equity Securities and Use of Proceeds None. Issuer Purchases of Equity Securities During the quarter ended December 31, 2024, we did not repurchase any shares of our common stock.
Issuer Purchases of Equity Securities During the quarter ended December 31, 2025, we did not repurchase any shares of our common stock.

Item 7. Management's Discussion & Analysis

Management's Discussion & Analysis (MD&A) — revenue / margin commentary

63 edited+44 added−19 removed71 unchanged
Biggest changeIn addition, as of December 31, 2024, we had accounts payable and accrued expenses and other current liabilities of $19.7 million, lease liability of $3.8 million, and indebtedness of $28.7 million. 92 Table of Contents The following table shows a summary of our cash flows for the year ended December 31, 2024 and the year ended December 31, 2023 (in thousands): Year ended December 31, 2024 2023 Net cash used in operating activities $ (42,142) $ (62,054) Net cash (used in) provided by investing activities (3,385) 3,077 Net cash provided by financing activities 64,858 20,881 Effect of changes in exchange rate on cash and restricted cash 28 (5) Net (decrease) in cash and restricted cash $ 19,359 $ (38,101) Operating activities Cash used in operating activities was $42.1 million for the year ended December 31, 2024, and primarily consisted of a net loss of $54.1 million adjusted for non-cash items including stock-based compensation of $7.4 million, depreciation and amortization of $2.0 million, non-cash lease expense of $0.9 million, non-cash expense from collaborative arrangements, net of $2.2 million, non-cash interest expense of $0.1 million, and a change in net working capital of $3.7 million.
Biggest changeThe following table shows a summary of our cash flows for the year ended December 31, 2025 and the year ended December 31, 2024 (in thousands): Year ended December 31, 2025 2024 Net cash used in operating activities $ (56,964) $ (42,142) Net cash (used in) provided by investing activities (311) (3,385) Net cash provided by financing activities 17,328 64,858 Effect of changes in exchange rate on cash and restricted cash 13 28 Net (decrease) increase in cash and restricted cash $ (39,934) $ 19,359 Operating activities Cash used in operating activities was $57.0 million for the year ended December 31, 2025, and primarily consisted of a net loss of $67.8 million adjusted for non-cash items including stock-based compensation of $7.7 million, depreciation and amortization of $2.4 million, non-cash lease expense of $1.3 million, non-cash expense from collaborative arrangements, net of $2.5 million, non-cash interest expense of $0.1 million, and a change in net working capital of $(0.5) million.
Additionally, the Lenders have the right to convert an aggregate amount of up to $6.0 million of the outstanding principal amount into shares of our common stock at a conversion price per share equal to a 80% of the trading price on the date of conversion, which shall be at Lenders' option.
Additionally, the Lenders have the right to convert an aggregate amount of up to $6.0 million of the outstanding principal amount into shares of our common stock at a conversion price per share equal to 80% of the trading price on the date of conversion, which shall be at Lenders' option.
We also entered into the Loan and Security Agreement with Avenue that is secured by a lien on all of our assets. Pursuant to the Loan and Security Agreement entered into with Avenue, we have borrowed $30.0 million to date.
We also entered into the Loan and Security Agreement with Avenue that is secured by a lien on all of our assets. Pursuant to the Loan and Security Agreement, we have borrowed $30.0 million to date.
Segment Information As of December 31, 2024, we viewed our operations and managed our business as one operating segment consistent with how our chief operating decision-maker, our Chief Executive Officer, makes decisions regarding resource allocation and assessing performance. As of December 31, 2024, substantially all of our assets were located in the United States.
Segment Information As of December 31, 2025, we viewed our operations and managed our business as one operating segment consistent with how our chief operating decision-maker, our Chief Executive Officer, makes decisions regarding resource allocation and assessing performance. As of December 31, 2025, substantially all of our assets were located in the United States.
The estimation process involves identifying services that have been performed on our behalf by third-parties, estimating and accruing expenses in our consolidated financial statements based on the evaluation of the progress to completion of specific tasks and the facts and circumstances known to us at the time of the estimate, and assessing the accuracy of these estimates going forward to determine if adjustments are required.
The estimation process 99 Table of Contents involves identifying services that have been performed on our behalf by third-parties, estimating and accruing expenses in our consolidated financial statements based on the evaluation of the progress to completion of specific tasks and the facts and circumstances known to us at the time of the estimate, and assessing the accuracy of these estimates going forward to determine if adjustments are required.
In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission.
In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which can be the basis of a BLA submission.
We will continue to explore options to fund our operations through public and private placements of equity and/or debt, payments from potential strategic research and development arrangements, sales of assets, licensing and/or collaboration arrangements with 94 Table of Contents pharmaceutical companies or other institutions, funding from the government, particularly for the development of our novel inhaled mucosal vaccine platform, or funding from other third parties.
We will continue to explore options to fund our operations through public and private placements of equity and/or debt, payments from potential strategic research and development arrangements, sales of assets, licensing and/or collaboration arrangements with pharmaceutical companies or other institutions, funding from the government, particularly for the development of our novel inhaled mucosal vaccine platform, or funding from other third parties.
Due to the inherent uncertainty involved in making estimates and the risks associated with the research, development, and commercialization of biotechnology products, we have based this estimate on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us.
Due to the inherent uncertainty involved in making estimates and the risks associated with the research, 98 Table of Contents development, and commercialization of biotechnology products, we have based this estimate on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us.
See Note 8 in the notes to the consolidated financial statements included elsewhere in this Annual Report for information regarding our obligations under the EB-5 loan agreement and the Loan and Security Agreement.
See Note 10 in the notes to the consolidated financial statements included elsewhere in this Annual Report for information regarding our obligations under the EB-5 Loan agreement and the Loan and Security Agreement.
Expense related to stock-based compensation awards are recorded to research and development expense or general and administrative expense based on the 96 Table of Contents underlying function of the individual that was granted the stock-based compensation award. Shares issued upon stock option exercise, PSU and RSU vesting are newly issued common shares.
Expense related to stock-based compensation awards are recorded to research and development expense or general and administrative expense based on the underlying function of the individual that was granted the stock-based compensation award. Shares issued upon stock option exercise, PSU and RSU vesting are newly issued common shares.
See Note 3 in the notes to the consolidated financial statements included in elsewhere in this Annual Report for information regarding our obligations under licensing and development agreements. Lease Obligations We have obligations under our operating leases, which include leased office, laboratory, and future manufacturing space, located in Malvern, Pennsylvania and other locations.
See Note 3 in the notes to the consolidated financial statements included in elsewhere in this Annual Report for information regarding our obligations under licensing and development agreements. 97 Table of Contents Lease Obligations We have obligations under our operating leases, which include leased office, laboratory, and future manufacturing space, located in Malvern, Pennsylvania and other locations.
Liabilities represent the consideration received in excess of revenues recognized under collaborative arrangement . Stock-based compensation We account for our stock-based compensation awards in accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Codification ("ASC") Topic 718, Compensation—Stock Compensation ("ASC 718").
Liabilities represent the consideration received in excess of revenues recognized under collaborative arrangement . 100 Table of Contents Stock-based compensation We account for our stock-based compensation awards in accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Codification ("ASC") Topic 718, Compensation—Stock Compensation ("ASC 718").
We anticipate that our research and development expenses will be higher in fiscal year 2025 as compared to fiscal year 2024 due to an increase in clinical activities with respect to our product candidates as well as an increase in headcount.
We anticipate that our research and development expenses will be higher in fiscal year 2026 as compared to fiscal year 2025 due to an increase in BLA and clinical activities with respect to our product candidates as well as an increase in headcount.
Factors impacting our future funding requirements include, without limitation, the following: • the initiation, progress, timing, costs, and results of trials for our product candidates; • the preparation and submission of Investigational New Drug applications, or INDs, with the FDA for current and future product candidates; • the outcome, timing, and cost of the regulatory approval process for our product candidates; • the costs of manufacturing and commercialization; • the costs related to doing business internationally with respect to the development and commercialization of our product candidates; • the cost of filing, prosecuting, defending, and enforcing our patent claims and other intellectual property rights; • the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us; • the acquisition of or in-licensing of additional product candidates and technologies; • the costs of expanding infrastructure to support our development, commercialization, and business efforts, including the costs related to the development of a laboratory and manufacturing facility; • the costs involved in recruiting and retaining skilled personnel; • the extent to which we in-license or acquire other products, product candidates, or technologies and out-license our product candidates; and • the impact of geopolitical turmoil, macroeconomic conditions, social unrest, political instability, terrorism, or other acts of war.
Factors impacting our future funding requirements include, without limitation, the following: • the initiation, progress, timing, costs, and results of trials for our product candidates; • the preparation and submission of Investigational New Drug applications, or INDs, with the FDA for current and future product candidates; • the outcome, timing, and cost of the regulatory approval process for our product candidates; • the costs of manufacturing and commercialization; • the costs related to doing business internationally with respect to the development and commercialization of our product candidates; • the cost of filing, prosecuting, defending, and enforcing our patent claims and other intellectual property rights; • the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us; • the acquisition of or in-licensing of additional product candidates and technologies; • the costs of expanding infrastructure to support our development, commercialization, and business efforts, including the costs related to the development of a laboratory and manufacturing facility; • the costs involved in recruiting and retaining skilled personnel; • the extent to which we in-license or acquire other products, product candidates, or technologies and out-license our product candidates; • the impact of geopolitical turmoil, macroeconomic conditions, social unrest, political instability, terrorism, or other acts of war; and • the changes in tariffs and indirect trade restraints, including increased costs associated with global and retaliatory tariff polices.
Overview We are a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics and vaccines that improve health and offer hope for patients across the globe.
Overview We are a biotechnology company focused on discovering, developing, and commercializing novel gene therapies that improve health and offer hope for patients across the globe.
Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related SAEs, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of LLVA.
Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related serious adverse events ("SAEs"), reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity ("LLVA").
Investing activities Cash used in investing activities was $3.4 million for the year ended December 31, 2024, and primarily consisted of payments related to purchases of property and equipment.
Investing activities Cash used in investing activities was $0.3 million for the year ended December 31, 2025, and primarily consisted of payments of security deposits and purchases of property and equipment. Cash used in investing activities was $3.4 million for the year ended December 31, 2024, and primarily consisted of payments related to purchases of property and equipment.
In October 2023, OCU500 was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation and intranasal delivery. NIAID intends to initiate a Phase 1 clinical trial after IND clearance.
In October 2023, OCU500 was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation and intranasal delivery. The NIAID intends to initiate a Phase 1 clinical trial in the second quarter of 2026.
OCU200 possesses unique features which potentially enable it to treat vascular complications of DME, DR, and wet AMD. Tumstatin is the active component of OCU200 and binds to integrin receptors, which play a crucial role in disease pathogenesis.
OCU200 possesses unique features which potentially enable it to treat vascular complications of diabetic macular edema ("DME"), diabetic retinopathy ("DR"), and wet age-related macular degeneration ("AMD"). Tumstatin is the active component of OCU200 and binds to integrin receptors, which play a crucial role in disease pathogenesis.
As of December 31, 2024, we had cash of approximately $58.5 million. This amount will not meet our capital requirements over the next 12 months. We believe that our cash and cash equivalents will enable us to fund our operations into the first quarter of 2026.
As of December 31, 2025, we had cash of approximately $18.6 million. This amount will not meet our capital requirements over the next 12 months. We believe that our cash and cash equivalents will enable us to fund our operations into the fourth quarter of 2026.
Our technology pipeline includes: • Modifier Gene Therapy Platform — Based on the use of NHRs, we believe our modifier gene therapy platform has the potential to address many retinal diseases, including rare genetic diseases such as RP (OCU400), with a gene-agnostic approach.
Our technology pipeline includes: • Novel Modifier Gene Therapy Platform — OCU400 - Based on the use of nuclear hormone receptors ("NHRs"), we believe our novel modifier gene therapy platform has the potential to address major blindness diseases, including rare genetic diseases such as RP (OCU400), with a gene-agnostic approach.
Research and development expenses account for a significant portion of our operating expenses. We plan to incur research and development expenses for the foreseeable future as we expect to continue the development of our product candidates.
We plan to incur research and development expenses for the foreseeable future as we expect to continue the development of our product candidates.
We have completed renovating an existing facility into a GMP facility in accordance with the FDA's regulations in support of NeoCart manufacturing for personalized Phase 3 trial material. We intend to initiate the Phase 3 trial contingent on adequate availability of funding.
We have completed renovating an existing facility into a GMP facility in accordance with the FDA's regulations in support of NeoCart manufacturing for personalized Phase 3 trial material. We intend to initiate the Phase 3 trial contingent on adequate availability of funding. During 2025, we transferred the assets related to our NeoCart product candidate to OrthoCellix.
As of December 31, 2024, we had future minimum operating lease base rent payment obligations of $5.2 million, with $0.9 million payable within 12 months of December 31, 2024. See Note 6 in the 93 Table of Contents notes to the consolidated financial statements included elsewhere in this Annual Report for information regarding our obligations under lease obligations.
As of December 31, 2025, we had future minimum operating lease base rent payment obligations of $5.7 million, with $1.2 million payable within 12 months of December 31, 2025. See Note 8 in the notes to the consolidated financial statements included elsewhere in this Annual Report for information regarding our obligations under lease obligations.
The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x 10 11 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls.
The GARDian Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5x10 11 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size.
Since our inception and through December 31, 2024, we have raised an aggregate of $368.9 million to fund our operations, of which $325.1 million is from gross proceeds from the sale of our common stock and warrants, $10.3 million is from the issuance of convertible notes, $33.3 million is from the issuance of debt, and $0.2 million is from grant proceeds.
Since our inception and through December 31, 2025, we have raised an aggregate of $389.9 million to fund our operations, of which $345.2 million is from gross proceeds from the sale of our common stock and warrants, $10.3 million is from the issuance of convertible notes, $33.4 million is from the issuance of debt, $0.8 million was from the royalty agreement, and $0.2 million is from grant proceeds.
Indebtedness We have outstanding debt related to the funds borrowed from EB5 Life Sciences pursuant to the United States government's Immigrant Investor Program, commonly known as the EB-5 program. Pursuant to the loan agreement entered into with EB-5 Life Sciences, we have borrowed $2.5 million to date.
Indebtedness We have outstanding debt related to the funds borrowed from EB-5 Life Sciences pursuant to the United States government's foreign national investor program, commonly known as the EB-5 Program. Pursuant to the Loan agreement, we have borrowed $1.5 million to date.
We believe our estimates and assumptions are reasonable under the current conditions; however, actual results may differ from these estimates. Our research and development expenses are not currently tracked on a program-by-program basis for indirect and overhead costs. We use our personnel and infrastructure resources across multiple research and development programs directed toward identifying, developing, and commercializing product candidates.
We believe our estimates and assumptions are reasonable under the current conditions; however, actual results may differ from these estimates. Our research and development expenses are not currently tracked on a program-by-program basis for indirect and overhead costs.
Liquidity and Capital Resources As of December 31, 2024, we had $58.5 million in cash. We have not generated revenue from our product candidates to date, and have primarily funded our operations to date through the sale of common stock, warrants to purchase common stock, the issuance of convertible notes and debt, and grant proceeds.
We have not generated revenue from our product candidates to date, and have primarily funded our operations to date through the sale of common stock, warrants to purchase common stock, the issuance of convertible notes and debt, and grant proceeds.
Cash used in operating activities was $62.1 million for the year ended December 31, 2023, and primarily consisted of a net loss of $63.1 million adjusted for non-cash items including stock-based compensation of $9.2 million, impairment of advance for COVAXIN supply of $4.1 million, depreciation and amortization of $0.7 million, non-cash lease expense of $0.5 million, other non-cash items of $0.4 million, and a change in net working capital of $13.1 million.
Cash used in operating activities was $42.1 million for the year ended December 31, 2024, and primarily consisted of a net loss of $54.1 million adjusted for non-cash items including stock-based compensation of $7.4 million, depreciation and amortization of $2.0 million, non-cash lease expense of $0.9 million, non-cash expense from collaborative arrangements, net of $2.2 million, non-cash interest expense of $0.1 million, and a change in net working capital of $3.7 million.
For collaborative arrangements within the scope of ASC 808 we may analogize to ASC 606 for certain elements. 95 Table of Contents We identify the goods or services promised within each collaborative arrangement and assesses whether each promised good or service is distinct for the purpose of identifying the performance obligations in the contract.
We identify the goods or services promised within each collaborative arrangement and assesses whether each promised good or service is distinct for the purpose of identifying the performance obligations in the contract.
We received net proceeds of $34.7 million after deducting equity issuance costs. During the year ended December 31, 2023, we issued and sold 30.0 million shares of our common stock at a public offering price of $0.50 per share pursuant to an underwriting agreement (the "May 2023 Public Offering").
We received net proceeds of $18.5 million after deducting equity issuance costs. 96 Table of Contents During the year ended December 31, 2024, we issued and sold $32.7 million shares of our common stock at a public offering price of $1.15 per share pursuant to the July 2024 Public Offering.
Transferrin is expected to facilitate the targeted delivery of tumstatin into the retina and choroid and potentially help increase the interaction between tumstatin and integrin receptors.
Tumstatin is the active component of OCU200 and binds to integrin receptors,which play a crucial role in disease pathogenesis. Transferrin is expected to facilitate the targeted delivery of tumstatin into the retina and choroid and potentially help increase the interaction between tumstatin and integrin receptors.
The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and currently we are actively recruiting patients. • Regenerative Medicine Cell Therapy Platform — Our Phase 3-ready regenerative cell therapy platform technology, which includes NeoCart (autologous chondrocyte-derived neocartilage), is being developed for the repair of knee cartilage injuries in adults.
Regenerative Medicine Cell Therapy Platform — Our Phase 3-ready regenerative cell therapy platform technology, which includes NeoCart (autologous chondrocyte-derived neocartilage), is being developed for the repair of knee cartilage injuries in adults.
We expect to continue to incur net losses until our product candidates, if approved, are successfully commercialized. We 89 Table of Contents incurred net losses of approximately $54.1 million and $63.1 million for the years ended December 31, 2024 and 2023, respectively.
We expect to continue to incur net losses until our product candidates, if approved, are successfully commercialized. We incurred net losses of $67.8 million and $54.1 million for the years ended December 31, 2025 and 2024, respectively. As of December 31, 2025, we had an accumulated deficit of $408.1 million and a cash balance of $18.6 million.
We anticipate that our general and administrative expenses will increase in fiscal year 2025 as compared to fiscal year 2024 due to an increase in headcount. 90 Table of Contents Results of Operations The following table summarizes the results of our operations for the years ended December 31, 2024 and 2023 (in thousands): Year ended December 31, 2024 2023 Change Collaborative arrangement revenue $ 4,055 $ 6,036 $ (1,981) Total Revenue 4,055 6,036 (1,981) Operating expenses Research and development 32,126 39,573 (7,447) General and administrative 26,686 31,994 (5,308) Total operating expenses 58,812 71,567 (12,755) Loss from operations (54,757) (65,531) 10,774 Other income (expense), net 703 2,453 (1,750) Net loss $ (54,054) $ (63,078) $ 9,024 We believe the following table provides more transparency as to the type of research and development expenses incurred.
We anticipate that our general and administrative expenses will increase in fiscal year 2026 as compared to fiscal year 2025 due to an increase in headcount. 94 Table of Contents Results of Operations The following table summarizes the results of our operations for the years ended December 31, 2025 and 2024 (in thousands): Year ended December 31, 2025 2024 Change Collaborative arrangement revenue $ 4,413 $ 4,055 $ 358 Total Revenue 4,413 4,055 358 Operating expenses Research and development 39,750 32,126 7,624 General and administrative 27,579 26,686 893 Total operating expenses 67,329 58,812 8,517 Loss from operations (62,916) (54,757) (8,159) Other (expense) income : Interest income 922 1,251 (329) Interest expense (5,188) (688) (4,500) Other (expense) income, net (664) 140 (804) Total other (expense) income (4,930) 703 (5,633) Net loss $ (67,846) $ (54,054) $ (13,792) We believe the following table provides more transparency as to the type of research and development expenses incurred.
The following table summarizes our research and development expenses by product candidate for the years ended December 31, 2024 and 2023 (in thousands): Year ended December 31, 2024 2023 Change OCU400 $ 6,846 $ 7,318 $ (472) OCU410 and OCU410ST 3,653 4,029 (376) NeoCart 489 1,336 (847) COVAXIN 25 8,824 (8,799) Inhaled mucosal vaccine platform 2,464 629 1,835 OCU200 379 713 (334) Unallocated costs: Research and development personnel costs 12,992 13,868 (876) Facilities and other support costs 2,984 1,507 1,477 Other 2,294 1,349 945 Total research and development $ 32,126 $ 39,573 $ (7,447) Collaborative arrangement revenue Collaborative arrangement revenue decreased by $2.0 million for the year ended December 31, 2024 compared to the year ended December 31, 2023.
The following table summarizes our research and development expenses by product candidate for the years ended December 31, 2025 and 2024 (in thousands): Year ended December 31, 2025 2024 Change OCU400 $ 9,871 $ 6,846 $ 3,025 OCU410 and OCU410ST 5,465 3,653 1,812 NeoCart 295 489 (194) COVAXIN (2) 25 (27) Inhaled mucosal vaccine platform 417 2,464 (2,047) OCU200 756 379 377 Unallocated costs: Research and development personnel costs 16,786 12,992 3,794 Facilities and other support costs 3,581 2,984 597 Other 2,581 2,294 287 Total research and development $ 39,750 $ 32,126 $ 7,624 Collaborative arrangement revenue Collaborative arrangement revenue increased by $0.4 million for the year ended December 31, 2025 compared to the year ended December 31, 2024.
OCU410 targets multiple pathways associated with AMD pathogenesis, in contrast to products currently approved or under development that treat only one cause of GA, require multiple injections per year, and have safety considerations. OCU410ST has received ODD from the FDA for the treatment of ABCA4 -associated retinopathies, including Stargardt disease.
OCU410 is a potential one-time, curative therapy with a single sub-retinal injection that targets multiple pathways associated with AMD pathogenesis, in contrast to products currently approved or under development that treat only one cause of GA, require multiple injections per year, and have safety considerations.
As of December 31, 2024, we had an accumulated deficit of $340.2 million and a cash balance of $58.5 million. Substantially all of our net losses resulted from expenses incurred in connection with our research and development programs and from general and administrative costs associated with our operations.
Substantially all of our net losses resulted from expenses incurred in connection with our research and development programs and from general and administrative costs associated with our operations.
This assessment is performed throughout the life of the arrangements based on changes to the arrangements.
This assessment is performed throughout the life of the arrangements based on changes to the arrangements. For collaborative arrangements within the scope of ASC 808 we may analogize to ASC 606 for certain elements.
The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity as measured by best corrected visual acuity (BCVA) and LLVA compared to untreated controls. One-year data will be utilized for the BLA filing.
Secondary endpoints include visual acuity as measured by best corrected visual acuity and LLVA compared to untreated controls. One-year data will be utilized for the BLA filing. The Phase 2/3 pivotal confirmatory trial has adaptive design with sample size re-estimation. OCU410ST is intended for early to advanced cases of ST.
In February 2025, we announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU400 ATMP classification. OCU410 completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for the treatment of GA, an advanced form of dAMD.
OCU410 - We completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for OCU410 for the treatment of geographic atrophy ("GA"), an advanced form of dAMD.
This assumption is only used for the market-based PSUs. Stock-based compensation expense was $7.4 million and $9.2 million for the years ended December 31, 2024 and 2023, respectively. As of December 31, 2024, we had $5.4 million of unrecognized stock-based compensation expense, which is expected to be recognized over a remaining weighted-average period of 1.6 years.
This assumption is only used for the market-based PSUs. Stock-based compensation expense was $7.7 million and $7.4 million for the years ended December 31, 2025 and 2024, respectively.
We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. In January 2025, we announced that the IND application is in effect and NIAID intends to 87 Table of Contents initiate a Phase 1 clinical trial in the second quarter of 2025.
In January 2025, we announced that the Investigational New Drug ("IND") application is in effect, and the National Institute of Allergy and Infectious Diseases ("NIAID"), part of the National Institutes of Health ("NIH") intends to initiate a Phase 1 clinical trial for OCU500.
General and administrative expense 91 Table of Contents General and administrative expense decreased by $5.3 million for the year ended December 31, 2024 compared to the year ended December 31, 2023. The decrease was primarily due to $2.7 million in professional service fees and $2.4 million related to reduced headcount.
The increase was primarily due to $0.7 million increase in professional service fees. Interest income Interest income for the year ended December 31, 2025 decreased by $(0.3) million, compared to year ended December 31, 2024.
In February 2025, we announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU400 ATMP classification. ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life.
In February 2025, we announced that the European Commission ("EC") has provided a positive opinion from the European Medicines Agency's ("EMA") Committee for Advanced Therapies for OCU400 Advanced Therapy Medicinal Product ("ATMP") classification.
The Sales Agreement was terminated in February 2023. Since our inception, we have devoted substantial resources to research and development and have incurred significant net losses and may continue to incur net losses in the future. We incurred net losses of approximately $54.1 million and $63.1 million for the years ended December 31, 2024 and 2023, respectively.
We received net proceeds of $34.7 million after deducting equity issuance costs. Since our inception, we have devoted substantial resources to research and development and have incurred significant net losses and may continue to incur net losses in the future.
The decrease was due to the amount of co-development services provided by us to the business partner in the collaboration agreement. Research and development expense Research and development expense decreased by $7.4 million for the year ended December 31, 2024 compared to the year ended December 31, 2023.
The increase resulted from greater advancement in fulfilling the terms of the collaboration agreement. Research and development expense Research and development expense increased by $7.6 million for the year ended December 31, 2025 compared to the year ended December 31, 2024.
We intend to initiate the Phase 3 trial contingent on adequate availability of funding. • Inhaled Mucosal Vaccine Platform — Our next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine.
During 2025, we transferred the assets related to our NeoCart product candidate to OrthoCellix. Inhaled Mucosal Vaccine Platform — Our next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500.
We also granted the Lenders a negative pledge on our intellectual property. During the year ended December 31, 2024, we issued and sold 32.7 million shares of our common stock at a public offering price of $1.15 per share pursuant to the July 2024 Public Offering (as defined below).
During the year ended December 31, 2025, we issued and sold 20.0 million shares of our common stock in a registered direct offering at price of $1.00 per share and accompanying warrant.
Patient follow-up will take place up to three months after the last injection. Regenerative Medicine Cell Therapy Platform NeoCart is a Phase 3-ready, regenerative cell therapy technology that combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process.
The first subject was dosed in the OCU200 multicenter open label Phase 1 clinical trial in January 2025 and enrollment is expected to be completed during the first quarter of 2026. Regenerative Medicine Cell Therapy Platform NeoCart is a Phase 3-ready, regenerative cell therapy technology that combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process.
We are actively recruiting patients in the United States and Canada in the Phase 3 liMeliGhT clinical trial for OCU400 for the treatment of RP and are on track to complete enrollment in the first half of 2025. In January 2025, we announced positive two year data for multiple mutations from the Phase 1/2 clinical trial of OCU400.
OCU400 is intended for early to advanced cases of RP including clinical and/or genetic diagnosis with both syndromic and non-syndromic forms of the disease. In January 2025, we announced positive two-year data for multiple mutations from the Phase 1/2 clinical trial for OCU400.
Financing activities Cash provided by financing activities was $64.9 million for the year ended December 31, 2024 compared to $20.9 million for the year ended December 31, 2023.
Financing activities Cash inflows from financing activities totaled $17.3 million for the year ended December 31, 2025, a notable decline from the $64.9 million recorded for the year ended December 31, 2024.
Recent Accounting Pronouncements For a discussion of recent accounting pronouncements, see Note 2 in the notes to the consolidated financial statements included elsewhere in this Annual Report.
As of December 31, 2025, we had $10.9 million of unrecognized stock-based compensation expense, which is expected to be recognized over a remaining weighted-average period of 2.0 years. 101 Table of Contents Recent Accounting Pronouncements For a discussion of recent accounting pronouncements, see Note 2 in the notes to the consolidated financial statements included elsewhere in this Annual Report.
These broad ODD, RMAT, and OMPD designations further support the broad (gene-agnostic) therapeutic potential of OCU400 to treat RP associated with mutations in multiple genes. The OCU400 Phase 3 liMeliGhT clinical trial is currently underway, with enrollment on track to meet our target submission of a BLA and MAA in mid-2026.
These broad ODD, RMAT, and OMPD designations further support the broad (gene-agnostic) therapeutic potential of OCU400 to treat RP associated with mutations in multiple genes. OCU410 and OCU410ST are being developed utilizing the RORA (RAR Related Orphan Receptor A) gene for the treatment of GA secondary to dAMD and ST, respectively.
Contractual Obligations Licensing and Development Agreements We have obligations under certain license and development agreements for our product candidates including annual payments, payments upon the achievement of certain milestones, and royalty payments based on net sales of licensed products.
In comparison, the 2024 period saw its financing cash primarily generated from two sources: the July 2024 Public Offering, delivering $37.6 million in gross proceeds less $2.9 million in equity issuance costs, and a debt financing deal with Avenue, which contributed an additional $30.0 million in gross proceeds before deducting $0.8 million in related equity issuance expenses Contractual Obligations Licensing and Development Agreements We have obligations under certain license and development agreements for our product candidates including annual payments, payments upon the achievement of certain milestones, and royalty payments based on net sales of licensed products.
We are continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for our OCU510 and OCU520 platforms. Modifier Gene Therapy Platform We are developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, Stargardt disease; and multifactorial diseases such as dAMD.
The NIAID intends to initiate the OCU500 Phase 1 clinical trial in the second quarter of 2026. 90 Table of Contents Novel Modifier Gene Therapy Platform We are developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, ST; and multifactorial diseases such as dAMD.
Other income (expense), net Other income (expense), net decreased by $1.8 million for the year ended December 31, 2024 compared to the year ended December 31, 2023. The decrease was primarily due to $1.1 million in interest earned on our cash and restricted cash, which had a lower average balance compared to prior year.
The primary reason for this reduction was the lower average balances of cash and restricted cash, which resulted in less interest being generated compared to the previous year. Interest expense Interest expense for the year ended December 31, 2025 increased significantly by $4.5 million, compared to year ended December 31, 2024.
As of December 31, 2024, we had an accumulated deficit of $340.2 million.
We incurred net losses of approximately $67.8 million and $54.1 million for the years ended December 31, 2025 and 2024, respectively. As of December 31, 2025, we had an accumulated deficit of $408.1 million.
Additionally, the EMA granted OMPD for OCU410ST for the treatment of ABCA4 -associated retinopathies including Stargardt disease, RP19, and CORD3.
OCU410ST has received ODD from the FDA and OMPD from the EMA for the treatment of ABCA4 -associated retinopathies (>1200 mutations) including ST, RP19, and cone-rod dystrophy 3 (CORD3), and has the potential to be the first approved therapy to treat ST.
Both OCU410 and OCU410ST studies have completed the low, medium, and high dose cohorts in the Phase 1 part of the Phase 1/2 trials to date, with OCU410 completing dosing in the Phase 2 part of the Phase 1/2 trials. • Novel Biologic Therapy for Retinal Diseases — OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin.
In addition, OCU410 treatment demonstrated a 20.2% reduction in sqrt geographic atrophy lesion growth at 12 months compared to untreated fellow eyes. • Other Programs — Novel Biologic Therapy for Retinal Diseases — OCU200 is a novel recombinant fusion protein consisting of two human proteins, tumstatin and transferrin.
Novel Biologic Therapy for Retinal Diseases We are developing OCU200, which is a novel fusion protein containing parts of human transferrin and tumstatin. OCU200 is designed to treat DME, DR, and wet AMD. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and we are actively recruiting patients for the Phase 1 clinical trial.
Novel Biologic Therapy for Retinal Diseases OCU200 is a novel recombinant fusion protein consisting of two human proteins, tumstatin and transferrin. OCU200 possesses unique features which potentially enable it to treat vascular complications of diabetic macular edema ("DME"), diabetic retinopathy ("DR"), and wet age-related macular degeneration ("AMD").
We are continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for our OCU510 and OCU520 platforms. Financial Operations Overview We have not generated revenue from our product candidates to date and have incurred net losses in each year since inception.
Management has evaluated subsequent events through March 4th, 2026 and determined that this grant does not impact our financial position as of December 31, 2025, but is disclosed herein as a subsequent event. Financial Operations Overview We have not generated revenue from our product candidates to date and have incurred net losses in each year since inception.
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We also believe our modifier gene therapy platform has the potential to address multifactorial retinal diseases including dAMD, which affects millions of patients in the United States alone, using OCU410, and Stargardt disease, which is a rare genetic disease, using OCU410ST.
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We have completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400 and are on track to begin a rolling BLA submission in the third quarter of 2026. Positive long-term, 3-year Phase 1/2 durable, safety and tolerability data for OCU400 demonstrate sustained clinically meaningful, approximately 2-line LLVA gain, reinforcing durable gene-agnostic benefit.
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In October 2024, the DSMB for the OCU410ST GARDian clinical trial approved enrollment for the second phase of the Phase 1/2 clinical trial. In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission.
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Positive long-term, 3-year Phase 1/2 data for OCU400 were recently assessed in evaluable subjects and builds on prior 2-year results showing consistent clinically meaningful, approximately 2-line LLVA gain across mutations. OCU400 maintained a favorable durability, safety and tolerability profile with no new treatment-related serious adverse events or adverse events of interest emerged.
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In August 2024, we received notification from the FDA that we could begin our EAP for the treatment of adult patients with RP with OCU400. This program is available for patients with early, intermediate to advanced RP with at least minimal retinal preservation who may benefit from the mechanism of action of OCU400 prior to approval of the BLA.
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Additional data include: ◦ Visual function benefits were consistently observed over 3 years, with 88% (7/8) of evaluable treated subjects showing improvement or preservation versus untreated fellow eyes. ◦ Approximately 2-line gain (N=8) observed across multiple mutation types in treated eyes compared to untreated eyes at 3 years.
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We also received approval from Health Canada to initiate a Phase 3 trial for OCU400 for the treatment of RP. The Health Canada trial will run in parallel with the United States FDA trial, expediting the ability to potentially provide a gene-agnostic treatment option to approximately 110,000 patients in the United States and Canada .
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We are on track to begin a rolling BLA submission in the third quarter of 2026. Topline Phase 3 data expected in the first quarter of 2027, advancing OCU400 towards potential approval in 2027 as a treatment option for early- to late-stage RP. OCU410ST - We initiated dosing in GARDian3 pivotal confirmatory trial for OCU410ST in July 2025.
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In January 2025, we announced positive two-year long-term data across multiple mutations from the Phase 1/2 clinical trial of OCU400, which demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes. 100% (10/10) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes.
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The OCU410ST Phase 2/3 pivotal confirmatory trial represents our second late-stage clinical program. We plan to submit a BLA for OCU410ST in the first half of 2027 in alignment with our strategic goal of filing three BLAs over the next three years.
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Also, treated eyes with multiple mutations and RHO subjects demonstrated a statistically significant (p=0.005) improvement in visual function when compared to untreated eyes. Treated eyes in RHO subjects (who meet Intent-to-treat criteria) for OCU400 Phase 3 trial demonstrated a statistically significant improvement (p=0.01, N=4) with around two-line gain in visual function (LLVA) at two years when compared to untreated eyes.
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In November 2024, the EMA granted orphan medicinal product designation ("OMPD") for OCU410ST for the treatment of ABCA4-associated retinopathies (>1200 mutations) including ST, RP 19, and CORD3. In May 2025, we announced that the FDA granted Rare Pediatric Disease Designation ("RPDD") for OCU410ST for the treatment of ABCA4-associated retinopathies including ST, retinitis pigmentosa 19 ("RP19"), and cone-rod dystrophy 3 ("CORD3").
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OCU410 and OCU410ST are being developed utilizing the nuclear receptor gene RORA , for the treatment of GA secondary to dAMD and Stargardt disease, respectively. OCU410 is a potential one-time, curative therapy with a single sub-retinal injection.
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In June 2025, we announced that the FDA has cleared the Investigational New Drug ("IND") amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all ST ( ABCA4-associated retinopathies).
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OCU410ST has also received OMPD from the EMA for the treatment of ABCA4 -associated retinopathies, including Stargardt disease, RP19, and CORD3. In March 2025, we announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU410 and OCU410ST ATMP classification.
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In August 2025, we announced that the Committee for Medicinal Products for Human Use ("CHMP") of the EMA reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for ST and 89 Table of Contents provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application ("MAA").
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OCU410 has completed dosing in the Phase 2 of the Phase 1/2 ArMaDa clinical trial. OCU410ST has completed Phase 1 of the Phase 1/2 GARDian clinical trial, and the DSMB has approved enrollment for the second phase of the Phase 1/2 GARDian clinical trial.

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