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What changed in PRECISION BIOSCIENCES INC's 10-K2024 vs 2025

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Paragraph-level year-over-year comparison of PRECISION BIOSCIENCES 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.

+770 added657 removedSource: 10-K (2026-03-12) vs 10-K (2025-03-26)

Top changes in PRECISION BIOSCIENCES INC's 2025 10-K

770 paragraphs added · 657 removed · 238 edited across 7 sections

Item 1. Business

Business — how the company describes what it does

9 edited+450 added300 removed0 unchanged
Biggest changeCollaboration and License Agreement with Novartis On June 14, 2022, the Company entered into the Novartis Agreement, which became effective on June 15, 2022 (the “Novartis Effective Date”), to collaborate to discover and develop in vivo gene editing products incorporating its custom ARCUS nucleases for the purpose of seeking to research and develop potential treatments for certain diseases (collectively referred to as licensed products).
Biggest changeNovartis Pharma AG In June 2022, we and Novartis Pharma AG (“Novartis”) entered into a research and development collaboration and license agreement (the “Novartis Agreement”) incorporating our custom ARCUS nucleases for potential treatments for certain diseases, including certain hemoglobinopathies such as sickle cell disease and beta thalassemia.
Additionally, the Company entered into a license agreement with Imugene (the “Imugene License Agreement”) on the Closing Date, pursuant to which the Company granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic CAR T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement.
Additionally on August 15, 2023, we and Imugene US entered into a license agreement (the “Imugene License Agreement”), pursuant to which we granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic CAR T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement.
NOTE 2: COLLABORATION AND LICENSE AGREEMENTS TG Therapeutics On January 7, 2024, the Company entered into a license agreement (the “TG License Agreement”) with TG Cell Therapy, Inc. (“TG Subsidiary”) and its parent company TG Therapeutics, Inc.
TG Therapeutics On January 7, 2024, we entered into a license agreement (the “TG License Agreement”) with TG Cell Therapy, Inc. (“TG Subsidiary”) and its parent company TG Therapeutics, Inc.
Sale of Azer-cel CAR T Platform to Imugene On August 15, 2023 the Company entered into an asset purchase agreement (the “Imugene Purchase Agreement”) with Imugene Limited (“Imugene Limited”), and its wholly owned subsidiary Imugene (USA) Inc. (“Imugene US” and together with Imugene Limited, “Imugene”).
Imugene On August 15, 2023, we entered into an asset purchase agreement (the “Imugene Purchase Agreement”) with Imugene Limited (“Imugene Limited”), and its wholly owned subsidiary Imugene (USA) Inc. (“Imugene US” and together with Imugene Limited, “Imugene”).
Simultaneously with the entry into the iECURE DLA, the Company and iECURE entered into an equity issuance agreement (the “iECURE Equity Agreement”), pursuant to which iECURE issued the Company common stock in iECURE as additional consideration for the PCSK9 license.
In connection with the iECURE DLA, we and iECURE entered into an equity issuance agreement (the “iECURE Equity Agreement”), pursuant to which iECURE issued us common stock in iECURE as additional consideration for the license to use our PCSK9-directed ARCUS nuclease.
(“TG Parent” and, together with TG Subsidiary, “TG Therapeutics”), pursuant to which the Company granted TG Subsidiary certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer pursuant to the terms of the TG License Agreement.
(“TG Parent” and, together with TG Subsidiary, “TG Therapeutics”), pursuant to which we granted TG Subsidiary certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize azercabtagene zapreleucel (“azer-cel”) for autoimmune diseases and other indications outside of cancer. Refer to Part II. Item 7.
Prevail’s notice informed the Company that Prevail was exercising its right to terminate the Prevail Agreement in its entirety without cause upon 90 days’ prior written notice to the Company. The Company subsequently exercised its rights to the return of the three programs. The termination was effective on July 10, 2024.
On October 31, 2025, we received written notice from Novartis of its termination of the Novartis Agreement. The notice informed us that Novartis was exercising its right to terminate the Novartis Agreement in its entirety without cause upon 90 days’ prior written notice to us. The termination was effective as of January 30, 2026.
Either party may terminate the Imugene License Agreement (i) for material breach by the other party and a failure to cure such breach within the time period specified in the agreement or (ii) the other party’s insolvency.
Either party may terminate any of the licenses granted under the agreement (1) in the event of the other party’s material breach, subject to an opportunity to cure within the time period specified in the Cellectis License, or (2) if the other party directly or indirectly challenges a patent licensed to it by the other party.
The Company is a gene editing company dedicated to improving life by developing in vivo therapies for genetic and infectious diseases with the application of the Company’s wholly-owned proprietary ARCUS genome editing platform.
Our goal is to broadly translate the potential of genome editing into permanent genetic solutions for significant unmet medical needs by leveraging the ARCUS gene editing platform in genetic and infectious diseases.
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Item 1. Financi al Statements. PRECISION BIOSCIENCES, INC.
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Item 1. Business. We are a clinical stage gene editing company utilizing our novel proprietary ARCUS platform to concurrently develop two clinical-stage in vivo gene editing therapies. ARCUS differs from other technologies in the way it cuts, its smaller size, and its simpler structure.
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B ALANCE SHEETS (In thousands, except share and per share amounts) December 31, 2024 December 31, 2023 Assets Current assets: Cash and cash equivalents $ 85,899 $ 116,678 Accounts receivable 229 901 Marketable securities 413 — Prepaid expenses 6,441 5,977 Convertible note receivable — 11,897 Assets held for sale 169 487 Contract asset 1,469 — Other current assets 369 419 Total current assets 94,989 136,359 Restricted cash 22,569 — Property, equipment, and software—net 3,089 6,338 Intangible assets—net 622 400 Right-of-use assets—net 7,090 8,263 Investment in equity securities 3,206 3,206 Note receivable—net 4,602 4,990 Other assets 221 225 Total assets $ 136,388 $ 159,781 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 1,312 $ 2,968 Accrued compensation 5,182 4,978 Accrued research and development expenses 2,016 1,557 Deferred revenue 2,957 12,035 Loan payable—current portion — 22,412 Lease liabilities 1,320 1,133 Other current liabilities 989 2,391 Current liabilities of discontinued operations 1,204 2,513 Total current liabilities 14,980 49,987 Loan payable 22,321 — Deferred revenue 23,300 73,082 Lease liabilities 6,404 7,723 Warrant liability 2,796 — Contract liabilities 10,000 10,000 Other noncurrent liabilities 194 128 Total liabilities 79,995 140,920 Commitments and contingencies (Note 14) Stockholders’ equity: Preferred stock: $ 0.0001 par value— 10,000,000 shares authorized as of December 31, 2024 and December 31, 2023; no shares issued and outstanding as of December 31, 2024 and December 31, 2023 — — Common stock: $ 0.000005 par value— 200,000,000 shares authorized as of December 31, 2024 and December 31, 2023; 8,229,730 shares issued and 8,202,715 shares outstanding as of December 31, 2024; 4,191,053 shares issued and 4,164,038 shares outstanding as of December 31, 2023 1 1 Additional paid-in capital 539,808 509,443 Accumulated deficit ( 482,464 ) ( 489,631 ) Treasury stock ( 952 ) ( 952 ) Total stockholders’ equity 56,393 18,861 Total liabilities and stockholders’ equity $ 136,388 $ 159,781 See notes to financial statements F- 3 PRECISION BIOSCIENCES, INC.
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The ARCUS platform is used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome, e.g. viral DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single adeno-associated virus (“AAV”)).
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STATEM ENTS OF OPERATIONS (In thousands, except share and per share amounts) For the Years Ended December 31, 2024 2023 Revenue $ 68,696 $ 48,727 Operating expenses Research and development 59,559 53,375 General and administrative 35,299 39,088 Total operating expenses 94,858 92,463 Operating loss ( 26,162 ) ( 43,736 ) Other income (expense): Loss from equity method investment ( 1,084 ) ( 4,931 ) Gain on changes in fair value 258 1,145 Gain on change in fair value of warrant liability 29,610 — Interest expense ( 1,782 ) ( 2,230 ) Interest income 6,763 7,686 Loss on disposal of assets ( 436 ) ( 461 ) Total other income 33,329 1,209 Income (loss) from continuing operations $ 7,167 $ ( 42,527 ) Loss from discontinued operations (including gain on disposal of $ 8,446 during the year ended December 31, 2023) — ( 18,792 ) Net income (loss) $ 7,167 $ ( 61,319 ) Net income (loss) per share Basic $ 1.05 $ ( 15.96 ) Diluted $ 1.04 $ ( 15.96 ) Weighted-average shares of common stock outstanding Basic 6,832,982 3,841,405 Diluted 6,883,911 3,841,405 See notes to financial statements F- 4 PRE CISION BIOSCIENCES, INC.
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We believe that the ARCUS platform’s key capabilities and differentiating characteristics can be leveraged for effective and safe therapeutic outcomes. Overview of Genome Editing DNA carries the genetic instructions for all basic functions of a living cell. These instructions are encoded in four different molecules, called bases, which are strung together in specific sequences to form genes.
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STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY (In thousands, except share amounts) Common Stock Additional Paid-In Accumulated Treasury Total Stockholders’ Shares Amount Capital Deficit Stock Equity Balance- December 31, 2022 3,725,689 1 $ 489,696 $ ( 428,312 ) $ ( 952 ) $ 60,433 Stock option exercises 3,196 — 31 — — 31 Issuance of common stock under employee stock purchase plan 18,101 — 370 — — 370 Share-based compensation expense — — 14,040 — — 14,040 Restricted stock units vested 46,893 — — — — — Net proceeds from issuance of common stock 397,174 — 5,306 — — 5,306 Net loss — — — ( 61,319 ) — ( 61,319 ) Balance- December 31, 2023 4,191,053 $ 1 $ 509,443 $ ( 489,631 ) $ ( 952 ) $ 18,861 Issuance of common stock under employee stock purchase plan 23,831 — 249 — — 249 Share-based compensation expense — — 12,604 — — 12,604 Proceeds from issuance of common stock and warrants through underwritten offering, net of issuance costs 2,500,000 — 4,610 — — 4,610 Proceeds from issuance of common stock to collaboration partners and licensees 97,360 — 905 — — 905 Proceeds from issuance of common stock through ATM facility, net of issuance cost 1,290,354 — 11,697 — — 11,697 Proceeds from issuance of common stock, net of issuance cost 25,000 — 300 — — 300 Restricted stock units vested 102,132 — — — — — Net Income — — — 7,167 — 7,167 Balance- December 31, 2024 8,229,730 $ 1 $ 539,808 $ ( 482,464 ) $ ( 952 ) $ 56,393 See notes to financial statements F- 5 PRECISION BIOSCIENCES, INC.
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Each gene is responsible for a specific function in a cell, and the complete set of genes in a cell, which can consist of tens of thousands of genes and billions of individual bases, is known as a genome. The complete genome sequence has been determined for many organisms, including humans.
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STATEMENTS OF CASH FLOWS (In thousands) For the Years Ended December 31, 2024 2023 Cash flows from operating activities: Net Income (loss) $ 7,167 $ ( 61,319 ) Adjustments to reconcile net income (loss) to net cash used in operating activities: Depreciation and amortization 3,404 6,817 Share-based compensation 12,604 14,040 Loss on disposal of assets 436 563 Gain on disposal of business — ( 8,446 ) Non-cash interest expense 249 368 Amortization of right-of-use assets 1,173 1,438 Gain on changes in fair value ( 258 ) ( 1,145 ) Loss from equity method investment 1,084 4,931 Amortization of discount on note receivable ( 695 ) ( 515 ) Gain on change in fair value of warrant liability ( 29,610 ) — Impairment charges — 641 Changes in operating assets and liabilities: Prepaid expenses ( 464 ) 1,051 Marketable securities 1,990 — Convertible note receivable 9,750 — Accounts receivable 672 ( 181 ) Contract asset ( 1,469 ) — Other assets and other current assets ( 262 ) 1,752 Accounts payable ( 2,096 ) 1,508 Other liabilities and other current liabilities ( 2,128 ) ( 724 ) Deferred revenue ( 58,860 ) ( 43,947 ) Lease liabilities ( 1,132 ) ( 946 ) Net cash used in operating activities ( 58,445 ) ( 84,114 ) Cash flows from investing activities: Proceeds from disposal of business — 8,000 Purchases of property, equipment and software ( 250 ) ( 1,957 ) Purchases of intangibles assets ( 25 ) ( 321 ) Proceeds from sale of equipment 60 107 Net cash (used in) provided by investing activities ( 215 ) 5,829 Cash flows from financing activities: Proceeds from stock option exercises — 31 Proceeds from employee stock purchase plan 249 370 Proceeds from offering of common stock and warrants, net of issuance costs 49,333 4,986 Proceeds from offering of common stock to collaboration partners and licensees 905 — Repayment of revolving credit facility ( 22,505 ) — Borrowings from term loan debt facility, net of issuance costs paid to lender 22,468 — Net cash provided by financing activities 50,450 5,387 Net decrease in cash, cash equivalents and restricted cash ( 8,210 ) ( 72,898 ) Cash, cash equivalents, and restricted cash—beginning of period 116,678 189,576 Cash, cash equivalents, and restricted cash —end of period $ 108,468 $ 116,678 Reconciliation of cash, cash equivalents and restricted cash: Cash and cash equivalents $ 85,899 $ 116,678 Restricted cash $ 22,569 $ — Total of cash, cash equivalents and restricted cash $ 108,468 $ 116,678 Supplemental disclosures of noncash financing and investing activities: Property, equipment and software additions included in accounts payable, accrued expenses and other current liabilities $ 48 $ 14 Intangible asset additions included in accounts payable and accrued expenses and other current liabilities $ 250 $ — Cash paid for interest $ 1,738 $ 2,018 Unsettled at-the-market issuances of common stock included in other current assets $ — $ 320 See notes to financial statements F- 6 Precision BioSciences, Inc.
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This allows scientists to identify specific genes and determine how their unique sequences contribute to a particular cellular function. Studying variations in gene sequences further informs an understanding of why a cell behaves a certain way, which can greatly enhance understanding of what causes and how to treat aberrations that leads to disease.
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No tes to Financial Statements NOTE 1: DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Description of Business Precision BioSciences, Inc. (the “Company”) was incorporated on January 26, 2006 under the laws of the State of Delaware and is based in Durham, North Carolina.
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Genome editing is a biotechnology process that removes, inserts or repairs a portion of DNA at a specific location in a cell’s genome. Early applications of genome editing focused on advancing genetic research. As genome editing technologies have advanced, their application is moving beyond understanding disease to treating or preventing disease by editing DNA.
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Since its inception, the Company has devoted substantially all of its efforts to research and development activities, recruiting skilled personnel, establishing its intellectual property portfolio and providing general and administrative support for these operations. The Company is subject to a number of risks similar to those of other companies conducting early-stage research and development of product candidates.
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Genome editing is accomplished by delivering a DNA cutting enzyme, called an endonuclease, to a targeted segment of genetic code. There are several genome editing technologies, including ARCUS, zinc-finger nucleases (“ZFNs”), TAL-effector nucleases (“TALENs”), clustered regularly interspaced short palindromic repeats (“CRISPR”)-based editors including CRISPR-Cas9 and CRISPR-Cas12, base editors, and prime editors.
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Principal among these risks are the Company’s dependence on key individuals and intellectual property, competition from other products and companies, and the technical risks associated with the successful research, development and clinical manufacturing of its product candidates.
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These technologies differ from one another principally in the properties of the endonuclease that they each employ. The different endonucleases have fundamentally different mechanisms of recognizing and cutting their DNA targets, which gives each technology advantages and disadvantages depending on how each is used.
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The Company’s success is dependent upon its ability to continue to raise additional capital in order to fund ongoing research and development, obtain regulatory approval of its products, successfully commercialize its products, generate revenue, meet its obligations, and, ultimately, attain profitable operations.
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In addition to the importance of efficiency, or the percentage of cells that are edited on-target, we believe ARCUS is differentiated by the type of edit predictably driving a more defined outcome.
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Use of Estimates The preparation of financial statements in conformity with accounting principles generally accepted in the United States of America (“GAAP”) requires management to make estimates and assumptions that affect the amounts reported in the financial statements and disclosures made in the accompanying notes to the financial statements. Actual results could differ from those estimates.
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A defined outcome is a predictable, highly consistent, and intended therapeutic edit, as compared to a random outcome, a distribution of inconsistent edits which could potentially limit efficacy and the safety profile. Our ARCUS Genome Editing Platform ARCUS has three unique properties that can lead to defined outcomes: The Cut.
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Significant estimates include recording revenue for performance obligations recognized over time, determination of the fair value of share-based compensation grants, estimating services expended by third-party service providers used to recognize research and development expense and determination of the fair value of investments. Basis of Presentation These financial statements have been prepared in accordance with GAAP.
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ARCUS has a unique cut that was evolved to drive defined outcomes. As shown in Figure 1 below, ARCUS generates a staggered cut that produces a 4 base pair, single strand of DNA. The portions noted in pink and blue are critical to finding a matching sequence on a DNA template when inserting a gene.
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Additionally, the accompanying financial statements have been prepared on a going concern basis, which contemplates the realization of assets and the satisfaction of liabilities in the normal course of business. The accompanying financial statements have been recast for all periods presented to reflect the assets, liabilities and expenses related to discontinued operations (discussed below).
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These overhangs are also in the same direction as DNA replication, so once it finds the matching sequence, it can start DNA replication on the template and incorporate the intended edit into the genome. This process is known as homology directed repair (“HDR”). 7 Table of Contents Figure 1. The Size.
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The accompanying financial statements are generally presented in conformity with the Company’s historical format. Reverse Stock Split On February 13, 2024, the Company amended its amended and restated certificate of incorporation in order to effect a 1-for- 30 reverse stock split of its outstanding shares of capital stock (the “Reverse Stock Split”).
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Size affects the ease and versatility with which endonucleases can be delivered to cells for editing. ARCUS can use different delivery vehicles including lipid nanoparticles (“LNP”) for the liver and AAV, which have limited carrying capacity, to target diverse tissue types.
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As a result of the Reverse Stock Split, every 30 shares of the Company’s common stock issued or outstanding were automatically reclassified into one new share of common stock, subject to the treatment of fractional shares as described below, without any action on the part of the holders.
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Because of its small size relative to other genome editing endonucleases, ARCUS is uniquely able to include an insertion DNA template in the same AAV, which allows targeting in vivo gene insertion in various tissues. ARCUS has demonstrated editing in a breadth of diverse tissue types, including the liver, muscle, the central nervous system, hematopoietic stem cells, and the eye.
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All historical share and per-share amounts reflected throughout the accompanying financial statements and other financial information in this Annual Report on Form 10-K have been retroactively adjusted to reflect the 2024 Reverse Stock Split as if the split occurred as of the earliest period presented.
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The simplicity. ARCUS is the only single component editor. As a single protein with a DNA recognition motif and catalytic activity all in one, no guide RNA is required with ARCUS, unlike CRISPR-based editors, base editors, and prime editors.
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The Reverse Stock Split did not affect the number of authorized shares of common stock or the par value of the common stock. No fractional shares were issued in connection with the Reverse Stock Split.
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Because editing outcomes are not dependent on simultaneous delivery of multiple editor components in separate delivery vehicles, ARCUS may lead to higher efficiency with potentially lower AAV and LNP doses. Our Strategy We are dedicated to improving life.
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Stockholders who would otherwise have been entitled to receive fractional shares as a result of the Reverse Stock Split were entitled to a cash payment in lieu thereof at a price equal to the fraction to which the stockholder would otherwise be entitled multiplied by the closing sales price per share of the common stock (as adjusted to give effect to the Reverse Stock Split) on The Nasdaq Capital Market on February 13, 2024, the last trading day immediately preceding the effective time of the Reverse Stock Split.
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Our strategy is focused on progressing our gene editing portfolio, including programs under development internally and with partners, and differentiating ARCUS as a unique tool in the gene editing field. In Vivo Gene Editing Pipeline Wholly-Owned Programs PBGENE-HBV (Viral Elimination).
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F- 7 Summary of Significant Accounting Policies Cash and Cash Equivalents As of December 31, 2024 and December 31, 2023 , the Company held cash equivalents which are composed of money market funds.
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PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic hepatitis B infection.
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Restricted Cash Restricted cash includes a cash security account with Banc of California pursuant to the 2024 Loan and Security Agreement (as defined in Note 12, Debt , below). The balance is classified as long-term on the Company’s balance sheets as the maturity date under the 2024 Loan and Security Agreement is June 30, 2027.
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PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic hepatitis B, covalently closed circular DNA (“cccDNA”), while inactivating integrated hepatitis B virus (“HBV”) DNA.
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As of December 31, 2024 , the Company had a restricted cash balance of $ 22.6 million. There was no restricted cash as of December 31, 2023. Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash, cash equivalents, and notes receivable.
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The Phase 1/2a ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels across a number of administrations per dose level in patients with chronic hepatitis B. The ELIMINATE-B trial is enrolling patients at sites in Hong Kong, New Zealand, the United States and Moldova and expects to expand the study to sites in the U.K.
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All of the Company’s cash and cash equivalents are held at financial institutions that management believes to be of high credit quality. The Company may maintain cash deposits in financial institutions in excess of government insured limits. The Company regularly invests excess cash deposits in money market funds and repurchase agreements.
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Chronic HBV causes inflammation and damage to the liver, leading to chronic infection and increased risk of death from liver cancer or cirrhosis. There is no cure for chronic hepatitis B, and current treatments rarely result in a functional cure, primarily due to persistence of viral DNA in the liver.
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The Company believes that the credit risk arising from the holdings of these financial instruments is mitigated by the fact that these securities are of short duration, government backed and of high credit rating. The Company has not experienced any losses on cash and cash equivalents to date.
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In patients with chronic hepatitis B, genetic material of the virus is converted within infected liver cells into cccDNA that acts as the only template to make new infectious viral particles. Hepatitis B virus also inserts fragments of its DNA into the human genome of infected liver cells.
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Revenue from Prevail and TG Therapeutics accounted for 77 % and 12 % of revenue during the year ended December 31, 2024 , respectively. Revenue from Prevail and Novartis accounted for 62 % and 38 % of revenue during the year ended December 31, 2023, respectively. Novartis accounted for all of the Company’s deferred revenue as of December 31, 2024.
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These integrated fragments are viral replication incompetent and cannot produce new infectious virus. Both cccDNA and integrated HBV DNA produce the viral protein, hepatitis B surface antigen (“HBsAg”), which is secreted in the blood. Historically, the focus for drug development and regulatory approval of drugs for chronic hepatitis B has relied on the temporary suppression of HBsAg.
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In addition, the Company currently holds a $ 10.0 million promissory note payable from Elo (defined below), which exposes the Company to potential losses in the event of default. Counterparty credit risk is monitored through periodic reviews of financial records. As of December 31, 2024 , the Company considers the risk of counterparty default to be minimal.
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Achieving undetectable HBsAg may lead to a functional cure if there is no rebound in HBV DNA or HBsAg after drug treatment has been discontinued for at least six months, but this is achieved in less than three out of 100 patients treated with the current standard of care.
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Property, Equipment and Software Property, equipment and software (“PP&E”) are stated at cost, net of depreciation and amortization. Depreciation and amortization are calculated using the straight-line method over the estimated useful lives of the assets ranging from three to seven years.
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Since cccDNA is the only source of infectious particles (HBV DNA), elimination of cccDNA results in a cure of chronic hepatitis B. We believe sustained loss of HBV DNA alone as a result of cccDNA elimination is an approvable endpoint for the U.S.
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Leasehold improvements are amortized on a straight-line basis over the shorter of the lease term or estimated useful life of the asset.
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Food and Drug Administration (“FDA”) and highly relevant for PBGENE-HBV. 8 Table of Contents For information regarding our current clinical trials, please refer to Part II. Item 7 “Management’s Discussion and Analysis of Financial Condition and Results of Operations” of this Annual Report on Form 10-K. PBGENE-DMD (Excision).
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The depreciation and amortization periods for the Company’s significant PP&E categories are as follows: Laboratory equipment 5 to 7 years Furniture and fixtures and office equipment 3 to 5 years Leasehold improvements Lesser of remaining lease term or useful life Repairs and maintenance are charged to operations as incurred, and expenditures for additions and improvements that extend the useful life of the asset are capitalized.
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PBGENE-DMD is our development program for the treatment of Duchenne muscular dystrophy, or DMD. DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the United States alone.
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Intangible Assets Intangible assets primarily include in-licenses and capitalized patent costs. The Company capitalizes license fees paid to acquire access to proprietary technology if the technology is expected to have alternative future use in multiple research and development projects. The cost of licensed technology rights is amortized using the straight-line method over the estimated useful life of the technology.
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There are currently no approved therapies with curative intent that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to potentially improve function for approximately 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene.
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If the access to use the technology rights is one year or less, the cost is recorded as a prepaid expense and amortized over the period identified in the agreement. Amortization expense for licensed technology and capitalized patent costs is included in research and development expenses within the accompanying statement of operations.
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The aim of this approach is to restore a near full-length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated microdystrophin approaches with minimal functional benefit.
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F- 8 Impairment Charges Long-lived assets, such as PP&E, intangible assets, and long-term prepaid assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable.
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The Phase 1/2 FUNCTION-DMD study is expected to enroll ambulatory DMD patients with mutations between exons 45 and 55 impacting up to 60% of boys with DMD. The clinical trial will employ an immune modulation regimen and safety monitoring program to treat ambulatory patients at world class specialized DMD clinical sites.
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An impairment loss is assessed when future undiscounted cash flows are less than the assets’ carrying value and recognized when the carrying value of the asset exceeds fair value. Fair value is calculated by estimating the discounted future cash flows expected to be generated by the asset as well as other valuation techniques.
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Initial data from multiple patients is expected by year end 2026, with safety and early efficacy assessed by the percentage of near full-length dystrophin protein expression from muscle biopsies. The FDA granted PBGENE-DMD Rare Pediatric Disease designation in June 2025, Orphan Drug Designation in July 2025, and Fast-Track designation in February 2026.
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An impairment charge is recognized for the amount by which the carrying amount exceeds the fair value of the asset. Fair Value Measurements Fair value is defined as the price that would be received from selling an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date.
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The PBGENE-DMD program is eligible for a Priority Review Voucher (“PRV”) via the Rare Pediatric Disease PRV program. PBGENE-3243 (Mutant Mitochondrial Elimination). The PBGENE-3243 program is our wholly-owned potential treatment for m.3243 associated mitochondrial disease. Mitochondrial diseases are the most common hereditary metabolic disorder, affecting 1 in 4,300 people.
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When determining the fair value measurements for assets and liabilities, which are required to be recorded at fair value, we consider the principal or most advantageous market in which we would transact and the market-based risk measurements or assumptions that market participants would use in pricing the asset or liability, such as risks inherent in valuation techniques, transfer restrictions and credit risk.
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The highly specific mitochondria-targeted ARCUS nucleases are designed to shift heteroplasmy by editing and eliminating mutant mitochondrial DNA while allowing normal (wild-type) mitochondrial DNA to repopulate in the mitochondria, thus improving cellular function. We have paused development of PBGENE-3243 to prioritize our two lead programs, PBGENE-HBV and PBGENE-DMD. Partnered In Vivo Gene Editing Programs iECURE-OTC (Insertion).
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ASC 820, Fair Value Measurement , establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company’s assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from our independent sources.
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In partnership with iECURE, Inc. (“iECURE”), an ARCUS-mediated targeted gene insertion approach, ECUR-506, is being evaluated as a potential curative treatment option for neonatal onset ornithine transcarbamylase (“OTC”) deficiency. Recently, iECURE reached alignment with the FDA on the primary and key secondary efficacy endpoints, comparators and study size for the ongoing OTC-HOPE study which could support a Biologics License Application.
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Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the best information available in the circumstances.
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In addition, ECUR-506 was granted FDA Regenerative Medicine Advanced Therapy (“RMAT”) designation for neonatal onset OTC deficiency. The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain.
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The following fair value hierarchy is used to classify assets and liabilities based on the observable inputs and unobservable inputs used to value the assets and liabilities: • Level 1 - Observable inputs based on unadjusted quoted prices in active markets for identical assets or liabilities • Level 2 - Inputs, other than quoted prices in active markets, that are observable either directly or indirectly • Level 3 - Unobservable inputs for which there is little or no market date, which require the Company to develop its own assumptions To the extent that the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeIn addition to the submission of an IND to the FDA, before initiation of a clinical trial in the United States, certain human clinical trials subject to the NIH Guidelines are subject to review and oversight by an institutional biosafety committee (“IBC”), a local institutional committee that reviews and oversees research utilizing recombinant or synthetic nucleic acid molecules at that institution. 46 The IBC assesses the safety of the research and identifies any potential risk to public health or the environment, and such review may result in some delay before initiation of a clinical trial.
Biggest changeOur product candidates will need to meet safety, purity, and potency standards applicable to any new biologic under the regulatory framework administered by the FDA. 47 Table of Contents In addition to the submission of an IND to the FDA, before initiation of a clinical trial in the United States, certain human clinical trials subject to the NIH Guidelines are subject to review and oversight by an institutional biosafety committee (“IBC”), a local institutional committee that reviews and oversees research utilizing recombinant or synthetic nucleic acid molecules at that institution.
If we are unable to obtain sufficient funding on a timely basis or on favorable terms, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.
If we are unable to obtain sufficient funding on a timely basis or on favorable terms, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.
Among other things, these provisions include those establishing: 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 of directors; 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 of directors to elect a director to fill a vacancy created by the expansion of the board of directors or the resignation, death or removal of a director, which prevents stockholders from filling vacancies on our board of directors; the ability of our board of directors to authorize the issuance of shares of preferred stock and to determine the terms of those shares, including preferences and voting rights, without stockholder approval, which could be used to significantly dilute the ownership of a hostile acquirer; the ability of our board of directors to alter our amended and restated bylaws without obtaining stockholder approval; the required approval of the holders of at least two-thirds of the shares entitled to vote at an election of directors to adopt, amend or repeal our amended and restated bylaws or repeal the provisions of our amended and restated certificate of incorporation regarding the election and removal of directors; 84 a prohibition on stockholder action by written consent, which forces stockholder action to be taken at an annual or special meeting of our stockholders; the requirement that a special meeting of stockholders may be called only by the chairman of the board of directors, our chief executive officer (or our president, in the absence of a chief executive officer) or a majority of our board of directors, 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 of directors or to propose matters to be acted upon at a stockholders’ meeting, which may discourage or deter a potential acquirer from conducting a solicitation of proxies to elect the acquirer’s own slate of directors or otherwise attempting to obtain control of us.
Among other things, these provisions include those establishing: 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 of directors; 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 of directors to elect a director to fill a vacancy created by the expansion of the board of directors or the resignation, death or removal of a director, which prevents stockholders from filling vacancies on our board of directors; the ability of our board of directors to authorize the issuance of shares of preferred stock and to determine the terms of those shares, including preferences and voting rights, without stockholder approval, which could be used to significantly dilute the ownership of a hostile acquirer; the ability of our board of directors to alter our amended and restated bylaws without obtaining stockholder approval; the required approval of the holders of at least two-thirds of the shares entitled to vote at an election of directors to adopt, amend or repeal our amended and restated bylaws or repeal the provisions of our amended and restated certificate of incorporation regarding the election and removal of directors; a prohibition on stockholder action by written consent, which forces stockholder action to be taken at an annual or special meeting of our stockholders; the requirement that a special meeting of stockholders may be called only by the chairman of the board of directors, our chief executive officer (or our president, in the absence of a chief executive officer) or a majority of our board of directors, 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 of directors or to propose matters to be acted upon at a stockholders’ meeting, which may discourage or deter a potential acquirer from conducting a solicitation of proxies to elect the acquirer’s own slate of directors or otherwise attempting to obtain control of us.
Even if a therapeutic product candidate receives regulatory approval, future revenues for such product candidate will depend upon many factors, such as, as applicable, the size of any markets in which such product candidate is approved for sale, the market share captured by such product candidate, including as a result of the market acceptance of such product candidate and the 38 effectiveness of manufacturing, sales, marketing and distribution operations related to such product candidate, the terms of any collaboration, license, or other strategic arrangement we may have with respect to such product candidate and levels of reimbursement from third-party payors.
Even if a therapeutic product candidate receives regulatory approval, future revenues for such product candidate will depend upon many factors, such as, as applicable, the size of any markets in which such product candidate is approved for sale, the market share captured by such product candidate, including as a result of the market acceptance of such product candidate and the effectiveness of manufacturing, sales, marketing and distribution operations related to such product candidate, the terms of any collaboration, license, or other strategic arrangement we may have with respect to such product candidate and levels of reimbursement from third-party payors.
For example, California enacted the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act (collectively, the CCPA) requires covered businesses that process the personal information of California residents to, among other things: (i) provide certain disclosures to California residents regarding the business’s collection, use, and disclosure of their personal information; (ii) receive and respond to requests from California residents to access, delete, and correct their personal information, or to opt out of certain disclosures of their personal information; and (iii) enter into specific contractual provisions with service providers that process California resident personal information on the business’s behalf.
For example, California enacted the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act (collectively, the CCPA), which requires covered businesses that process the personal information of California residents to, among other things: (i) provide certain disclosures to California residents regarding the business’s collection, use, and disclosure of their personal information; (ii) receive and respond to requests from California residents to access, delete, and correct their personal information, or to opt out of certain disclosures of their personal information; and (iii) enter into specific contractual provisions with service providers that process California resident personal information on the business’s behalf.
Clinical trials have been and may in the future be delayed, suspended or terminated for a variety of reasons, including in connection with: the inability to generate sufficient preclinical, toxicology or other in vivo or in vitro data to support the initiation of clinical trials; applicable regulatory authorities disagreeing as to the design or implementation of the clinical trials; obtaining regulatory authorization to commence a trial; reaching an agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; obtaining IRB or ethics committee approval or positive opinion at each site; developing and validating the companion diagnostic to be used in a clinical trial, if applicable; insufficient or inadequate supply or quality of product candidates or other materials, including identification of lymphocyte donors meeting regulatory standards necessary for use in clinical trials, or delays in sufficiently developing, characterizing or controlling a manufacturing process suitable for clinical trials; recruiting and retaining enough suitable patients to participate in a trial; having enough patients complete a trial or return for post-treatment follow-up; adding a sufficient number of clinical trial sites; inspections of clinical trial sites or operations by applicable regulatory authorities, or the imposition of a clinical hold; clinical sites deviating from trial protocol or dropping out of a trial; the inability to demonstrate the efficacy and benefits of a product candidate; 49 discovering that product candidates have unforeseen safety issues, undesirable side effects or other unexpected characteristics; addressing patient safety concerns that arise during the course of a trial; receiving untimely or unfavorable feedback from applicable regulatory authorities regarding the trial or requests from regulatory authorities to modify the design of a trial; non-compliance with applicable regulatory requirements by us or third parties or changes in such regulations or administrative actions; suspensions or terminations by IRBs of the institutions at which such trials are being conducted, by the Data Safety Monitoring Board for such trial or by the FDA or other foreign regulatory authorities due to a number of factors, including those described above; third parties being unable or unwilling to satisfy their contractual obligations to us; competitive pressures and other market conditions; changes in our financial priorities, greater than anticipated costs of completing a trial or our inability to continue funding the trial; or unforeseen events, such as natural or manmade disasters, public health emergencies, such as natural catastrophic events.
Clinical trials have been and may in the future be delayed, suspended or terminated for a variety of reasons, including in connection with: the inability to generate sufficient preclinical, toxicology or other in vivo or in vitro data to support the initiation of clinical trials; applicable regulatory authorities disagreeing as to the design or implementation of the clinical trials; obtaining regulatory authorization to commence a trial; reaching an agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; obtaining IRB or ethics committee approval or positive opinion at each site; developing and validating the companion diagnostic to be used in a clinical trial, if applicable; insufficient or inadequate supply or quality of product candidates or other materials, including identification of lymphocyte donors meeting regulatory standards necessary for use in clinical trials, or delays in sufficiently developing, characterizing or controlling a manufacturing process suitable for clinical trials; recruiting and retaining enough suitable patients to participate in a trial; having enough patients complete a trial or return for post-treatment follow-up; adding a sufficient number of clinical trial sites; inspections of clinical trial sites or operations by applicable regulatory authorities, or the imposition of a clinical hold; clinical sites deviating from trial protocol or dropping out of a trial; the inability to demonstrate the efficacy and benefits of a product candidate; 50 Table of Contents discovering that product candidates have unforeseen safety issues, undesirable side effects or other unexpected characteristics; addressing patient safety concerns that arise during the course of a trial; receiving untimely or unfavorable feedback from applicable regulatory authorities regarding the trial or requests from regulatory authorities to modify the design of a trial; non-compliance with applicable regulatory requirements by us or third parties or changes in such regulations or administrative actions; suspensions or terminations by IRBs of the institutions at which such trials are being conducted, by the Data Safety Monitoring Board for such trial or by the FDA or other foreign regulatory authorities due to a number of factors, including those described above; third parties being unable or unwilling to satisfy their contractual obligations to us; competitive pressures and other market conditions; changes in our financial priorities, greater than anticipated costs of completing a trial or our inability to continue funding the trial; or unforeseen events, such as natural or manmade disasters, public health emergencies, such as natural catastrophic events.
While we do not believe that we have experienced any material impact to our business strategy, results of operations, or financial condition resulting from a system failure, accident or security breach to date, if such an event were to occur and cause interruptions in our or our critical third parties’ operations, it could result in delays and/or material disruptions of our research and development programs, our operations and ultimately, our financial results.
While we do not believe that we have experienced any material impact to our business strategy, results of operations, or financial condition resulting from a system failure, accident or security breach to date, if such an event were to occur and cause interruptions in our or our critical third parties’ operations, it could result in delays and/or material disruptions of our research and development programs, our operations and our financial results.
We expect to experience pricing pressures in connection with the sale of any products that may receive approval due to the trend toward managed health care, the increasing influence of health maintenance organizations and additional legislative changes. The downward pressure on healthcare costs in general, particularly 66 prescription drugs and biologics and surgical procedures and other treatments, has become intense.
We expect to experience pricing pressures in connection with the sale of any products that may receive approval due to the trend toward managed health care, the increasing influence of health maintenance organizations and additional legislative changes. The downward pressure on healthcare costs in general, particularly prescription drugs and biologics and surgical procedures and other treatments, has become intense.
Criminal or civil proceedings might be filed against us by patients, regulatory authorities, biopharmaceutical companies and any other third party using or marketing any product candidates or products that we develop alone or with collaborators. On occasion, 45 large judgments have been awarded in class action lawsuits based on products that had unanticipated adverse effects.
Criminal or civil proceedings might be filed against us by patients, regulatory authorities, biopharmaceutical companies and any other third party using or marketing any product candidates or products that we develop alone or with collaborators. On occasion, large judgments have been awarded in class action lawsuits based on products that had unanticipated adverse effects.
The number of patients in the United 65 States, Europe and elsewhere may turn out to be lower than expected, and patients may not be amenable to treatment with products that we may develop alone or with collaborators, or may become increasingly difficult to identify or gain access to, any of which would decrease our ability to realize revenue from any such products for such diseases.
The number of patients in the United States, Europe and elsewhere may turn out to be lower than expected, and patients may not be amenable to treatment with products that we may develop alone or with collaborators, or may become increasingly difficult to identify or gain access to, any of which would decrease our ability to realize revenue from any such products for such diseases.
If we are unable to enter into additional collaboration agreements, or to maintain existing collaborations, we may have to curtail the research and development of the product candidate or technology for which we are seeking to collaborate, reduce or delay 73 research and development programs, delay potential commercialization timelines, reduce the scope of any sales or marketing activities or undertake research, development or commercialization activities at our own expense.
If we are unable to enter into additional collaboration agreements, or to maintain existing collaborations, we may have to curtail the research and development of the product candidate or technology for which we are seeking to collaborate, reduce or delay research and development programs, delay potential commercialization timelines, reduce the scope of any sales or marketing activities or undertake research, development or commercialization activities at our own expense.
If the IRS or any other tax authorities were successful in challenging our positions, we may be liable for additional tax and penalties 71 and interest related thereto or other taxes, as applicable, in excess of any reserves established therefor, which may have a significant impact on our results and operations and future cash flow.
If the IRS or any other tax authorities were successful in challenging our positions, we may be liable for additional tax and penalties and interest related thereto or other taxes, as applicable, in excess of any reserves established therefor, which may have a significant impact on our results and operations and future cash flow.
We may not be able to protect our rights to our trademarks and trade names, which we need to build name recognition among potential collaborators or customers in our markets of interest. At times, competitors may adopt trade names or trademarks similar to ours, thereby impeding our ability to build brand identity and possibly leading to 83 market confusion.
We may not be able to protect our rights to our trademarks and trade names, which we need to build name recognition among potential collaborators or customers in our markets of interest. At times, competitors may adopt trade names or trademarks similar to ours, thereby impeding our ability to build brand identity and possibly leading to market confusion.
These current or future laws, regulations and permitting requirements may impair our research, development or production efforts. Failure to comply with these laws, regulations and permitting requirements, either by us 70 or by any third-party contract manufacturers and suppliers we engage, also may result in substantial fines, penalties or other sanctions or business disruption.
These current or future laws, regulations and permitting requirements may impair our research, development or production efforts. Failure to comply with these laws, regulations and permitting requirements, either by us or by any third-party contract manufacturers and suppliers we engage, also may result in substantial fines, penalties or other sanctions or business disruption.
With respect to the validity of patents, for example, we cannot be certain that there is no invalidating prior art of which we and the patent examiner were unaware during prosecution, but that an 80 adverse third party may identify and submit in support of such assertions of invalidity.
With respect to the validity of patents, for example, we cannot be certain that there is no invalidating prior art of which we and the patent examiner were unaware during prosecution, but that an adverse third party may identify and submit in support of such assertions of invalidity.
Resource allocation 72 and other developmental decisions made by our collaborators may result in the delay or termination of research programs, studies or trials, repetition of or initiation of new studies or trials or provision of insufficient funding or resources for the completion of studies or trials or the successful marketing and distribution of any product candidates that may receive approval.
Resource allocation and other developmental decisions made by our collaborators may result in the delay or termination of research programs, studies or trials, repetition of or initiation of new studies or trials or provision of insufficient funding or resources for the completion of studies or trials or the successful marketing and distribution of any product candidates that may receive approval.
For example, many other countries, including countries in the EU, have compulsory licensing laws under which a patent owner must grant licenses to third parties. In addition, many countries limit the 82 enforceability of patents against third parties, including government agencies or government contractors. In these countries, patents may provide limited or no benefit.
For example, many other countries, including countries in the EU, have compulsory licensing laws under which a patent owner must grant licenses to third parties. In addition, many countries limit the enforceability of patents against third parties, including government agencies or government contractors. In these countries, patents may provide limited or no benefit.
The market price of our common stock is likely to be highly volatile and may fluctuate substantially due to many factors, including: inconsistent trading volume levels of our common stock; announcements or expectations regarding debt or equity financing efforts; sales of common stock by us, our insiders or our other stockholders; actual or anticipated fluctuations in our financial condition and operating results; 86 failure to meet or exceed financial estimates and projections of the investment community or that we provide to the public; results from or delays in our studies or trials, or those of our collaborators, competitors or companies perceived to be similar to us; delay, failure or discontinuation of any of our product development and research programs, or those of our collaborators, competitors or companies perceived to be similar to us; announcements about new research programs or product candidates from us or our collaborators, our competitors or companies perceived to be similar to us; announcements by us, our collaborators, our competitors or companies perceived to be similar to us relating to significant acquisitions, strategic partnerships or alliances, joint ventures, collaborations or capital commitments; actual or anticipated changes in our growth rate relative to our competitors or companies perceived to be similar to us; fluctuations in the valuation of our collaborators, our competitors or companies perceived to be comparable to us; a lack of, limited or withdrawal of coverage by security analysts, or positive or negative recommendations by them; actual or expected changes in estimates as to financial results, development timelines or recommendations by securities analysts; publication of research reports about us, genome editing or the biopharmaceutical industries; developments or changing views regarding the use of genomic products, including those that involve genome editing; our ability to effectively manage our growth; the recruitment or departure of key personnel; the results of any efforts by us to identify, develop, acquire or in-license additional product candidates, products or technologies; unanticipated serious safety concerns related to the use of any of our product candidates, or those of our competitors or companies perceived to be similar to us; the termination of a collaboration agreement, licensing agreement or other strategic arrangement or the inability to establish additional strategic arrangements on favorable terms, or at all; regulatory actions with respect to any of our product candidates, or those of our competitors or companies perceived to be similar to us; developments or disputes concerning patent applications, issued patents or other proprietary rights; regulatory or legal developments in the United States and other countries; changes in physician, hospital, or healthcare provider practices that may make our or our collaborators’ products less useful; changes in the structure of healthcare payment systems; significant lawsuits, such as products liability, patent or stockholder litigation; short sales of our common stock; and 87 general economic, industry and market conditions.
The market price of our common stock is likely to be highly volatile and may fluctuate substantially due to many factors, including: inconsistent trading volume levels of our common stock; announcements or expectations regarding debt or equity financing efforts; sales of common stock by us, our insiders or our other stockholders; actual or anticipated fluctuations in our financial condition and operating results; failure to meet or exceed financial estimates and projections of the investment community or that we provide to the public; results from or delays in our studies or trials, or those of our collaborators, competitors or companies perceived to be similar to us; delay, failure or discontinuation of any of our product development and research programs, or those of our collaborators, competitors or companies perceived to be similar to us; announcements about new research programs or product candidates from us or our collaborators, our competitors or companies perceived to be similar to us; 90 Table of Contents announcements by us, our collaborators, our competitors or companies perceived to be similar to us relating to significant acquisitions, strategic partnerships or alliances, joint ventures, collaborations or capital commitments; actual or anticipated changes in our growth rate relative to our competitors or companies perceived to be similar to us; fluctuations in the valuation of our collaborators, our competitors or companies perceived to be comparable to us; a lack of, limited or withdrawal of coverage by security analysts, or positive or negative recommendations by them; actual or expected changes in estimates as to financial results, development timelines or recommendations by securities analysts; publication of research reports about us, genome editing or the biopharmaceutical industries; developments or changing views regarding the use of genomic products, including those that involve genome editing; our ability to effectively manage our growth; the recruitment or departure of key personnel; the results of any efforts by us to identify, develop, acquire or in-license additional product candidates, products or technologies; unanticipated serious safety concerns related to the use of any of our product candidates, or those of our competitors or companies perceived to be similar to us; the termination of a collaboration agreement, licensing agreement or other strategic arrangement or the inability to establish additional strategic arrangements on favorable terms, or at all; regulatory actions with respect to any of our product candidates, or those of our competitors or companies perceived to be similar to us; developments or disputes concerning patent applications, issued patents or other proprietary rights; regulatory or legal developments in the United States and other countries; changes in physician, hospital, or healthcare provider practices that may make our or our collaborators’ products less useful; changes in the structure of healthcare payment systems; significant lawsuits, such as products liability, patent or stockholder litigation; short sales of our common stock; and general economic, industry and market conditions.
Our competitors also may obtain FDA 44 or other regulatory approval for their products more rapidly than we or our collaborators may obtain approval for any that we develop, which could result in our competitors establishing a strong market position before we are able to enter the market.
Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we or our collaborators may obtain approval for any that we develop, which could result in our competitors establishing a strong market position before we are able to enter the market.
Our business operations, as well as our current and anticipated future arrangements with investigators, healthcare professionals, consultants, third-party payors, customers and patients, expose or will expose us to broadly applicable foreign, federal, and state fraud 55 and abuse and other healthcare laws and regulations.
Our business operations, as well as our current and anticipated future arrangements with investigators, healthcare professionals, consultants, third-party payors, customers and patients, expose or will expose us to broadly applicable foreign, federal, and state fraud and abuse and other healthcare laws and regulations.
If key suppliers or manufacturers are lost, or if the supply of the materials is diminished or discontinued, we or our collaborators may not be able to develop, manufacture and market product 74 candidates in a timely and competitive manner, or at all.
If key suppliers or manufacturers are lost, or if the supply of the materials is diminished or discontinued, we or our collaborators may not be able to develop, manufacture and market product candidates in a timely and competitive manner, or at all.
Our ability to obtain and maintain patent protection for ARCUS and our product candidates is uncertain due to a number of factors, including that: we may not have been the first to invent the technology covered by our pending patent applications or issued patents; we may not be the first to file patent applications covering product candidates, including their compositions or methods of use, as patent applications in the United States and most other countries are confidential for a period of time after filing; our compositions and methods may not be patentable; our disclosures in patent applications may not be sufficient to meet the statutory requirements for patentability; any or all of our pending patent applications may not result in issued patents; others may independently develop identical, similar or alternative technologies, products or compositions or methods of use thereof; 75 others may design around our patent claims to produce competitive technologies or products that fall outside of the scope of our patents; we may fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection; we may not seek or obtain patent protection in countries that may eventually provide us a significant business opportunity; any patents issued to us may not provide a basis for commercially viable products, may not provide any competitive advantages or may be successfully challenged by third parties; others may identify prior art or other bases upon which to challenge and ultimately invalidate our patents or otherwise render them unenforceable; and the growing scientific and patent literature relating to engineered endonucleases, including our own patents and publications, may make it increasingly difficult or impossible to patent new engineered nucleases in the future.
Our ability to obtain and maintain patent protection for ARCUS and our product candidates is uncertain due to a number of factors, including that: we may not have been the first to invent the technology covered by our pending patent applications or issued patents; we may not be the first to file patent applications covering product candidates, including their compositions or methods of use, as patent applications in the United States and most other countries are confidential for a period of time after filing; our compositions and methods may not be patentable; our disclosures in patent applications may not be sufficient to meet the statutory requirements for patentability; any or all of our pending patent applications may not result in issued patents; others may independently develop identical, similar or alternative technologies, products or compositions or methods of use thereof; others may design around our patent claims to produce competitive technologies or products that fall outside of the scope of our patents; we may fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection; we may not seek or obtain patent protection in countries that may eventually provide us a significant business opportunity; any patents issued to us may not provide a basis for commercially viable products, may not provide any competitive advantages or may be successfully challenged by third parties; others may identify prior art or other bases upon which to challenge and ultimately invalidate our patents or otherwise render them unenforceable; and 78 Table of Contents the growing scientific and patent literature relating to engineered endonucleases, including our own patents and publications, may make it increasingly difficult or impossible to patent new engineered nucleases in the future.
If we or our collaborators fail to achieve announced milestones in the expected timeframes, 43 the commercialization of the product candidates may be delayed, our credibility may be undermined, our business and results of operations may be harmed, and the trading price of our common stock may decline.
If we or our collaborators fail to achieve announced milestones in the expected timeframes, the commercialization of the product candidates may be delayed, our credibility may be undermined, our business and results of operations may be harmed, and the trading price of our common stock may decline.
If microbial, viral or 51 other contaminations are discovered in our product candidates or in the manufacturing facilities in which such product candidates are made, such manufacturing facilities may need to be closed for an extended period of time to investigate and remedy the contamination.
If microbial, viral or other contaminations are discovered in our product candidates or in the manufacturing facilities in which such product candidates are made, such manufacturing facilities may need to be closed for an extended period of time to investigate and remedy the contamination.
Failure can occur at any time during the preclinical study or clinical trial process. Despite promising preclinical or clinical results, any 53 product candidate can unexpectedly fail at any stage of preclinical or clinical development. The historical failure rate for product candidates in our industry is high.
Failure can occur at any time during the preclinical study or clinical trial process. Despite promising preclinical or clinical results, any product candidate can unexpectedly fail at any stage of preclinical or clinical development. The historical failure rate for product candidates in our industry is high.
Patient enrollment may also be affected by many factors, including: severity and difficulty of diagnosing of the disease under investigation; the difficulty in recruiting and/or identifying eligible patients suffering from rare diseases being evaluated under our trials; size of the patient population and process for identifying subjects; eligibility and exclusion criteria for the trial in question, including unforeseen requirements by the FDA or other regulatory authorities that we restrict one or more entry criteria for the study for safety reasons; 52 our or our collaborators’ ability to recruit clinical trial investigators with the appropriate competencies and experience; design of the trial protocol; availability and efficacy of approved medications or therapies, or other clinical trials, for the disease or condition under investigation; perceived risks and benefits of the product candidate under trial or testing, or of the application of genome editing to human indications; availability of genetic testing for potential patients; efforts to facilitate timely enrollment in clinical trials; patient referral practices of physicians; ability to obtain and maintain subject consent; risk that enrolled subjects will drop out before completion of the trial; ability to monitor patients adequately during and after treatment; proximity and availability of clinical trial sites for prospective patients; and unforeseen events, such as natural or manmade disasters, public health emergencies may impact our operations, or other natural catastrophic events.
Patient enrollment may also be affected by many factors, including: severity and difficulty of diagnosing of the disease under investigation; the difficulty in recruiting and/or identifying eligible patients suffering from rare diseases being evaluated under our trials; size of the patient population and process for identifying subjects; eligibility and exclusion criteria for the trial in question, including unforeseen requirements by the FDA or other regulatory authorities that we restrict one or more entry criteria for the study for safety reasons; our or our collaborators’ ability to recruit clinical trial investigators with the appropriate competencies and experience; design of the trial protocol; 53 Table of Contents availability and efficacy of approved medications or therapies, or other clinical trials, for the disease or condition under investigation; perceived risks and benefits of the product candidate under trial or testing, or of the application of genome editing to human indications; availability of genetic testing for potential patients; efforts to facilitate timely enrollment in clinical trials; patient referral practices of physicians; ability to obtain and maintain subject consent; risk that enrolled subjects will drop out before completion of the trial; ability to monitor patients adequately during and after treatment; proximity and availability of clinical trial sites for prospective patients; and unforeseen events, such as natural or manmade disasters, public health emergencies may impact our operations, or other natural catastrophic events.
Moreover, there is a provision in each warrant under which we may be required to purchase the warrants from the holders by paying cash in an amount equal to the Black-Scholes value of the remaining unexercised portion of the warrants in certain specified situations involving a “fundamental transaction” (as defined in the warrants), which generally includes a merger with another person or entity, the sale, transfer or other disposition of all or substantially all of our assets, another person or entity becoming the beneficial owner of 50% of the outstanding shares of our common stock, any reclassification, reorganization or recapitalization of our common stock, any compulsory share exchange pursuant to which our common stock is effectively converted into or exchanged for other securities, cash or property, any plan or proposal for our voluntary or involuntary dissolution, liquidation or the winding up of our affairs, or if other conditions are met.
Moreover, there is a provision in each warrant under which we may be required to purchase the warrants from the holders by paying cash in an amount equal to the Black-Scholes value of the remaining unexercised portion of the warrants in certain specified situations involving a “fundamental transaction” (as defined in the warrants), which generally includes a merger with another person or entity, the sale, transfer or other disposition of all or substantially all of our assets, another person or entity becoming the beneficial owner of 50% of the outstanding shares of our common stock, any reclassification, reorganization or recapitalization of our common stock, any compulsory share 41 Table of Contents exchange pursuant to which our common stock is effectively converted into or exchanged for other securities, cash or property, any plan or proposal for our voluntary or involuntary dissolution, liquidation or the winding up of our affairs, or if other conditions are met.
Any time, effort and financial resources we expend on identifying and researching 41 new product candidates and product development platforms may divert our attention from, and adversely affect our ability to continue, development and commercialization of existing research programs, product candidates and product development platforms.
Any time, effort and financial resources we expend on identifying and researching new product candidates and product development platforms may divert our attention from, and adversely affect our ability to continue, development and commercialization of existing research programs, product candidates and product development platforms.
Defending against any such actions can be costly, time-consuming 56 and may require significant financial and personnel resources. Therefore, even if we are successful in defending against any such actions that may be brought against us, our business may be impaired.
Defending against any such actions can be costly, time-consuming and may require significant financial and personnel resources. Therefore, even if we are successful in defending against any such actions that may be brought against us, our business may be impaired.
There can be no assurance that we will have sufficient financial or other resources to file and pursue infringement 76 claims, which typically last for years before they are concluded.
There can be no assurance that we will have sufficient financial or other resources to file and pursue infringement claims, which typically last for years before they are concluded.
For example, the loss of trial data from completed, ongoing or planned trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data.
For example, the loss of trial data from completed, ongoing or planned trials could result in delays in our development and regulatory approval efforts and significantly increase our costs to recover or reproduce the data.
Our future capital requirements will depend on many factors, including: the timing, scope, progress, costs, results and analysis of results of research activities, preclinical studies and potential clinical trials for any of our product candidates; the costs of future activities, including product manufacturing, sales, marketing and distribution activities for any product candidates that receive regulatory approval; the success of our existing collaborative and other out-licensing relationships; the extent to which we exercise any development or commercialization rights under collaborative relationships; our ability to establish and maintain additional collaborative or other out-licensing relationships on favorable terms, or at all; the extent to which we expand our operations and the timing of such expansion, including with respect to facilities, employees and product development platforms; the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property and proprietary rights and defending intellectual property-related claims; 39 the extent to which we acquire or in-license other technologies or product candidates; the extent to which we acquire or invest in other businesses; the costs of continuing to operate as a public company; and the amount of revenues, if any, received from commercial sales of any products that we develop alone or with collaborators that receive regulatory approval.
Our future capital requirements will depend on many factors, including: the timing, scope, progress, costs, results and analysis of results of research activities, preclinical studies and potential clinical trials for any of our product candidates; the costs of future activities, including product manufacturing, sales, marketing and distribution activities for any product candidates that receive regulatory approval; the success of our existing or future collaborative and other out-licensing relationships; the extent to which we exercise any development or commercialization rights under collaborative relationships; our ability to establish and maintain additional collaborative or other out-licensing relationships on favorable terms, or at all; the extent to which we expand our operations and the timing of such expansion, including with respect to facilities, employees and product development platforms; the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property and proprietary rights and defending intellectual property-related claims; the extent to which we acquire or in-license other technologies or product candidates; the extent to which we acquire or invest in other businesses; the costs of continuing to operate as a public company; and 40 Table of Contents the amount of revenues, if any, received from commercial sales of any products that we develop alone or with collaborators that receive regulatory approval.
The delivery of healthcare in the EU, including the establishment and operation of health services and the pricing and reimbursement of medicines, is almost exclusively a matter for 61 national, rather than EU, law and policy.
The delivery of healthcare in the EU, including the establishment and operation of health services and the pricing and reimbursement of medicines, is almost exclusively a matter for national, rather than EU, law and policy.
If we fail to implement the requirements of Section 404 in the required timeframe, we may be 68 subject to sanctions or investigations by regulatory authorities, including the SEC and Nasdaq.
If we fail to implement the requirements of Section 404 in the required timeframe, we may be subject to sanctions or investigations by regulatory authorities, including the SEC and Nasdaq.
Moreover, addressing our personnel needs may lead to significant 67 costs and may divert our management and business development resources from other projects, such as the development of product candidates.
Moreover, addressing our personnel needs may lead to significant costs and may divert our management and business development resources from other projects, such as the development of product candidates.
If we or our collaborators fail to comply with applicable regulatory requirements following approval of any potential products we may develop, authorities may: issue an untitled enforcement letter or a warning letter asserting a violation of the law; 62 seek an injunction, impose civil and criminal penalties, and impose monetary fines, restitution or disgorgement of profits or revenues; suspend or withdraw regulatory approval; suspend or terminate any ongoing clinical trials or implement requirements to conduct post-marketing studies or clinical trials; refuse to approve a pending BLA or comparable foreign marketing application (or any supplements thereto) submitted by us or our collaborators; restrict the labeling, marketing, distribution, use or manufacturing of products; seize or detain products or otherwise require the withdrawal or recall of products from the market; refuse to approve pending applications or supplements to approved applications that we or our collaborators submit; refuse to permit the import or export of products; or refuse to allow us or our collaborators to enter into government contracts.
If we or our collaborators fail to comply with applicable regulatory requirements following approval of any potential products we may develop, authorities may: issue an untitled enforcement letter or a warning letter asserting a violation of the law; 65 Table of Contents seek an injunction, impose civil and criminal penalties, and impose monetary fines, restitution or disgorgement of profits or revenues; suspend or withdraw regulatory approval; suspend or terminate any ongoing clinical trials or implement requirements to conduct post-marketing studies or clinical trials; refuse to approve a pending BLA or comparable foreign marketing application (or any supplements thereto) submitted by us or our collaborators; restrict the labeling, marketing, distribution, use or manufacturing of products; seize or detain products or otherwise require the withdrawal or recall of products from the market; refuse to approve pending applications or supplements to approved applications that we or our collaborators submit; refuse to permit the import or export of products; or refuse to allow us or our collaborators to enter into government contracts.
It is critical that we do so in a secure manner to maintain the confidentiality and integrity of such Confidential Information.
It is critical that we do so in a secure manner to maintain the confidentiality, availability and integrity of such Confidential Information.
It is uncertain if and to what extent various states will conform to the TCJA or the CARES Act. As of December 31, 2024, we have a valuation allowance for the full amount of our net deferred tax assets as the realization of the net deferred tax assets is not determined to be more likely than not.
It is uncertain if and to what extent various states will conform to the TCJA or the CARES Act. As of December 31, 2025, we have a valuation allowance for the full amount of our net deferred tax assets as the realization of the net deferred tax assets is not determined to be more likely than not.
Companies that must comply with the GDPR face increased compliance obligations and risk, including more robust regulatory enforcement of data protection requirements, and potential fines for noncompliance of up to €20 million or 4% of the annual global revenues of the noncompliant company, whichever is greater.
Companies that must comply with the GDPR face increased compliance obligations and risk, including more robust regulatory enforcement of data protection requirements, and potential fines for noncompliance of up to €20 million / £17.5 million or 4% of the annual global revenues of the noncompliant company, whichever is greater.
Such laws include: the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under a U.S. healthcare program such as Medicare and Medicaid.
Such laws include: the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash 56 Table of Contents or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under a U.S. healthcare program such as Medicare and Medicaid.
If the patent applications we hold or have in-licensed with respect to our current and future research and development programs and product candidates fail to issue, if their validity, breadth or strength of protection is threatened, or if they fail to provide meaningful exclusivity for our technology or any products and product candidates that we or our collaborators may develop, it could dissuade companies from collaborating with us to develop product candidates, encourage competitors to develop competing products or technologies and threaten our or our collaborators’ ability to commercialize future product candidates.
If the patent applications we hold or have in-licensed with respect to our current and future research and development programs and product candidates fail to issue, if their validity, breadth or strength of protection is threatened, or if they fail to provide meaningful exclusivity for our technology or any products and product candidates that we or our collaborators may develop, it could dissuade companies from collaborating with us to develop product candidates, 82 Table of Contents encourage competitors to develop competing products or technologies and threaten our or our collaborators’ ability to commercialize future product candidates.
While we are not aware of any downgrades, material losses or other significant deterioration in the fair value of our cash equivalents since December 31, 2024, deterioration of the global credit and financial markets could negatively impact our current portfolio of cash equivalents or our ability to meet our financing objectives.
While we are not aware of any downgrades, material losses or other significant deterioration in the fair value of our cash equivalents since December 31, 2025, deterioration of the global credit and financial markets could negatively impact our current portfolio of cash equivalents or our ability to meet our financing objectives.
Additionally, for the year ended December 31, 2024, we had federal Orphan Drug credits of $13.5 million, which begin to expire in 2038. Changes in tax laws or regulations may adversely impact our ability to utilize all, or any, of our NOL carryforwards.
Additionally, for the year ended December 31, 2025, we had federal Orphan Drug credits of $13.5 million, which begin to expire in 2038. Changes in tax laws or regulations may adversely impact our ability to utilize all, or any, of our NOL carryforwards.
Our current and future product candidates may never be approved. Failure to successfully identify and develop new product candidates and obtain regulatory approvals for our products would have a material adverse effect on our business and financial condition and could cause us to cease operations.
Our current development programs and future product candidates may never be approved. Failure to successfully identify and develop new product candidates and obtain regulatory approvals for our products would have a material adverse effect on our business and financial condition and could cause us to cease operations.
We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could have a material adverse effect on our business, and such an event could disrupt our operations, cause us to incur remediation costs, damage our reputation and cause a loss of confidence in us and our or third parties’ ability to conduct clinical trials, which could adversely affect our reputation and delay our research and development programs.
We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could have a material adverse effect on our business, and such an event could disrupt our 89 Table of Contents operations, cause us to incur remediation costs, damage our reputation and cause a loss of confidence in us and our or third parties’ ability to conduct clinical trials, which could adversely affect our reputation and delay our research and development programs.
The developmental and commercial success of our current product candidates, or any that we develop alone or with collaborators in the future, will depend in part on public acceptance of the use of genome editing technology for the prevention or treatment of human diseases.
The developmental and commercial success of our current development programs of future product candidates, or any that we develop alone or with collaborators in the future, will depend in part on public acceptance of the use of genome editing technology for the prevention or treatment of human diseases.
A number of proposed and enacted federal, state and international laws and regulations obligate companies to notify individuals and other parties of security breaches involving particular types of information, which could result from breaches experienced by us or by third parties, including collaborators, vendors, contractors or other organizations with which we have formed relationships that involve the handling or processing of such information.
A number of proposed and enacted federal, state and international laws and regulations obligate companies to notify individuals and other parties of security breaches involving particular types of information, which could result from 60 Table of Contents breaches experienced by us or by third parties, including collaborators, vendors, contractors or other organizations with which we have formed relationships that involve the handling or processing of such information.
All of these milestones are based on a variety of assumptions, including assumptions regarding capital resources, constraints and priorities, progress of and results from development activities, participation of third parties including outside collaborators or vendors, the receipt of key regulatory approvals or actions, and other factors, any of which may cause the timing of achievement of the milestones to vary considerably from our estimates.
All of these milestones are based on a variety of assumptions, including assumptions regarding capital resources, constraints and priorities, progress of and results from development activities, participation of third parties including outside collaborators or vendors, the receipt of key regulatory approvals or actions, 44 Table of Contents and other factors, any of which may cause the timing of achievement of the milestones to vary considerably from our estimates.
However, the 10-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, for example, if the product is judged as sufficiently profitable not to justify maintenance of market exclusivity, or when the prevalence of the condition has increased above the orphan designation threshold.
However, the 10-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, for example, if the product is judged as sufficiently profitable not to justify maintenance of market exclusivity, or when the prevalence of the 61 Table of Contents condition has increased above the orphan designation threshold.
Our common stock was transferred to The Nasdaq Capital Market effective as of the open of business on October 26, 2023, and continues to trade under the symbol “DTIL.” The Nasdaq Capital Market operates in substantially the same manner as The Nasdaq Global Select Market, and listed companies must meet certain financial requirements and comply with Nasdaq’s corporate governance requirements.
Our common stock was transferred to The Nasdaq Capital Market effective as of the open of business on October 26, 2023, 91 Table of Contents and continues to trade under the symbol “DTIL.” The Nasdaq Capital Market operates in substantially the same manner as The Nasdaq Global Select Market, and listed companies must meet certain financial requirements and comply with Nasdaq’s corporate governance requirements.
Certain states have also adopted comparable privacy and security laws and regulations, which govern the privacy, processing, and protection of health-related and other personal information. Such laws and regulations will be subject to interpretation by various courts and other governmental authorities, thus creating potentially complex compliance issues for us and our future customers and strategic partners.
Certain states have also adopted comparable privacy and security laws and regulations, which govern the privacy, processing, and protection of health-related and other personal information. Such laws and regulations are subject to interpretation by various courts and other governmental authorities, thus creating potentially complex compliance issues for us and our future customers and strategic partners.
Such changes carry the risk that they will not achieve the intended objectives, and any of these changes could cause our product candidates to perform differently and affect the results of future clinical trials or our reliance on results of trials that have previously been conducted using the product candidate in its previous form.
Such changes carry the risk that they will not 52 Table of Contents achieve the intended objectives, and any of these changes could cause our product candidates to perform differently and affect the results of future clinical trials or our reliance on results of trials that have previously been conducted using the product candidate in its previous form.
If any of the above occur, it could adversely affect our ability to operate our business and our results of operations. Actual or perceived failures to comply with applicable data protection, privacy and security laws, regulations, standards and other requirements, and the increasing use of social media, could adversely affect our business, results of operations, and financial condition.
If any of the above occur, it could adversely affect our ability to operate our business and our results of operations. 57 Table of Contents Actual or perceived failures to comply with applicable data protection, privacy and security laws, regulations, standards and other requirements, and the increasing use of social media, could adversely affect our business, results of operations, and financial condition.
Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.
Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in even more resources being concentrated among a smaller 45 Table of Contents number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.
We do not carry specific biological or hazardous waste insurance coverage, and our property, casualty and general liability insurance policies (under which we currently have an aggregate of approximately $10 million in coverage) specifically exclude coverage for damages and fines arising from biological or hazardous waste exposure or contamination.
We do not carry specific biological or hazardous waste insurance coverage, and our property, casualty 72 Table of Contents and general liability insurance policies (under which we currently have an aggregate of approximately $10 million in coverage) specifically exclude coverage for damages and fines arising from biological or hazardous waste exposure or contamination.
If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business. We do not currently intend to pay dividends on our common stock. We do not intend to pay any dividends to holders of our common stock for the foreseeable future.
If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business. 87 Table of Contents We do not currently intend to pay dividends on our common stock. We do not intend to pay any dividends to holders of our common stock for the foreseeable future.
Coupled with ever-increasing EU and national regulatory burdens on those wishing to develop and market products, this could prevent or delay marketing approval of our product candidates, restrict or regulate post-approval activities and affect our ability to commercialize our product candidates, if approved.
Coupled with ever-increasing EU and national regulatory burdens on those wishing to develop and market products, this could prevent or delay marketing approval of our product candidates, restrict or regulate post-approval 64 Table of Contents activities and affect our ability to commercialize our product candidates, if approved.
If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could result in the imposition of any of the penalties discussed above and have a significant impact on our business and financial condition.
If any such actions are instituted against us, and we are not 73 Table of Contents successful in defending ourselves or asserting our rights, those actions could result in the imposition of any of the penalties discussed above and have a significant impact on our business and financial condition.
For example, we have granted exclusive rights or options to Novartis for certain targets, and during the term of our collaboration agreements we will be restricted from granting rights to other parties to use our ARCUS technology to pursue potential products that address those targets.
For example, we have granted exclusive rights or options to iECURE for certain targets, and during the term of our collaboration and license agreements we will be restricted from granting rights to other parties to use our ARCUS technology to pursue potential products that address those targets.
In the future, we may choose to build a focused sales, marketing and commercial support infrastructure to sell, or participate in sales activities with our collaborators for, certain product candidates if and when they are approved. There are risks involved with both establishing our own commercial capabilities and entering into arrangements with third parties to perform these services.
In the future, we may choose to build a focused sales, marketing and commercial support infrastructure to sell, or participate in sales activities with our collaborators for, certain product candidates if and when they are approved. 67 Table of Contents There are risks involved with both establishing our own commercial capabilities and entering into arrangements with third parties to perform these services.
The Regulation entered into force in January 2022 and has been applicable since January 2025, with phased implementation based on the type of product, i.e. oncology and advanced therapy medicinal products as of 2025, orphan medicinal products as of 2028, and all other medicinal products by 2030.
The Regulation entered into force in January 2022 and has been applicable since January 2025, with phased implementation based on the type of product, i.e. oncology and ATMPs as of 2025, orphan medicinal products as of 2028, and all other medicinal products by 2030.
If we are not able to effectively manage our operations, it may result in weaknesses in our infrastructure, increase our expenses more than expected, or give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity.
If we are not able to effectively manage our 70 Table of Contents operations, it may result in weaknesses in our infrastructure, increase our expenses more than expected, or give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity.
As of December 31, 2024, we had 108 full-time employees. Our future financial performance, ability to develop and commercialize product candidates alone or with collaborators and ability to compete effectively will depend in part on our ability to effectively manage the then applicable needs of our business. We may have difficulty identifying, hiring and integrating new personnel.
As of December 31, 2025, we had 68 full-time employees. Our future financial performance, ability to develop and commercialize product candidates alone or with collaborators and ability to compete effectively will depend in part on our ability to effectively manage the then applicable needs of our business. We may have difficulty identifying, hiring and integrating new personnel.
We also face the risk of potential unauthorized disclosure or misappropriation of our intellectual property by CROs or other third parties, which may reduce our trade secret protection and allow our potential competitors to access and exploit our proprietary technology.
We also face the 76 Table of Contents risk of potential unauthorized disclosure or misappropriation of our intellectual property by CROs or other third parties, which may reduce our trade secret protection and allow our potential competitors to access and exploit our proprietary technology.
Clinical trials of any of our product candidates may never commence despite the expenditure of significant resources in pursuit of their development, and our spending on current and future research and development programs, product candidates and product development platforms may not yield any commercially viable products.
Clinical trials of any of our product candidates may never commence despite the expenditure of significant resources in pursuit of their development, and our spending 42 Table of Contents on current and future research and development programs, product candidates and product development platforms may not yield any commercially viable products.
In many countries, the prices of medical products are subject to varying price control mechanisms as part of national health systems. Other countries allow companies to fix their own prices for medical products, but monitor and control company profits.
In many countries, the prices of medical products are subject to varying price control mechanisms as part of national health systems. Other countries allow companies to fix their own prices for medical 69 Table of Contents products, but monitor and control company profits.
The ability to achieve acceptable levels of coverage and reimbursement for any potential products that may be approved by governmental authorities will have an effect on our and our collaborators’ ability to successfully commercialize such products.
The ability to achieve acceptable levels of coverage and reimbursement for any potential products that may be approved by governmental 68 Table of Contents authorities will have an effect on our and our collaborators’ ability to successfully commercialize such products.
Exclusive marketing rights in the 59 United States may be limited if we or our collaborators or licensees seek approval for a disease or condition broader than the orphan designated disease or condition and may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product to meet the needs of patients with the rare disease or condition.
Exclusive marketing rights in the United States may be limited if we or our collaborators or licensees seek approval for a disease or condition broader than the orphan designated disease or condition and may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product to meet the needs relating to the approved indication or use of patients with the rare disease or condition.
If we are unable to develop and commercialize one or more product candidates either alone or with collaborators, or if revenues from any product candidate that receives marketing approval or is commercialized are insufficient, we may not achieve profitability. Even if we do achieve profitability, we may not be able to sustain or increase profitability.
If we are unable to develop and commercialize one or more product candidates either alone or with collaborators, or if revenues from any product candidate that receives marketing approval or is commercialized are insufficient, we may not achieve 39 Table of Contents profitability. Even if we do achieve profitability, we may not be able to sustain or increase profitability.
In addition to serious adverse events or side effects caused by product candidates we develop alone or with collaborators, the administration process or related procedures may also cause undesirable side effects. Further, any side effects may not be appropriately recognized or managed by the treating medical staff.
In addition to serious adverse events or side effects caused by product candidates we develop alone or with collaborators, the administration process or related procedures may also cause undesirable side effects. 55 Table of Contents Further, any side effects may not be appropriately recognized or managed by the treating medical staff.
The FDA or comparable foreign regulatory authorities may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or otherwise affected interpretation of the study.
The FDA or comparable foreign regulatory authorities may conclude that a financial relationship between us and a principal investigator has 51 Table of Contents created a conflict of interest or otherwise affected interpretation of the study.
Among other requirements, the GDPR regulates transfers of personal data subject to the GDPR to third countries that have not been found to provide adequate protection to such personal data, including the United States, and the efficacy and longevity of current transfer 57 mechanisms between the EEA, and the United States remains uncertain.
Among other requirements, the GDPR regulates transfers of personal data subject to the GDPR to third countries that have not been found to provide adequate protection to such personal data, including the United States, and the efficacy and 58 Table of Contents longevity of current transfer mechanisms between the EEA, and the United States remains uncertain.
If a product that has orphan drug designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications, including a BLA, to market the same biologic for the same disease or condition for seven years, except in limited circumstances such as a showing of clinical superiority to the product with orphan product exclusivity or if FDA finds that the holder of the orphan drug exclusivity has not shown that it can ensure the availability of sufficient quantities of the orphan drug to meet the needs of patients with the disease or condition for which the drug was designated.
If a product that has orphan drug designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications, including a BLA, to market the same biologic for the same approved indication or use within such disease or condition for seven years, except in limited circumstances such as a showing of clinical superiority to the product with orphan product exclusivity with respect to the relevant indication or use or if FDA finds that the holder of the orphan drug exclusivity has not shown that it can ensure the availability of sufficient quantities of the orphan drug to meet the needs relating to the approved indication or use of patients with the disease or condition for which the drug was designated.
There can also be no assurance that our and our third-party service providers’, strategic partners’, contractors’, 58 consultants’, CROs’ and collaborators’ cybersecurity risk management program and processes, including policies, controls or procedures, will be fully implemented, complied with or effective in protecting our systems, networks and Confidential Information.
There can also be no assurance that our and our third-party service providers’, strategic partners’, contractors’, consultants’, CROs’ and collaborators’ 59 Table of Contents cybersecurity risk management program and processes, including policies, controls or procedures, will be fully implemented, complied with or effective in protecting our systems, networks and Confidential Information.
In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of our patents and patent applications.
In addition to increasing uncertainty with 84 Table of Contents regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of our patents and patent applications.
In addition, there is a risk that one or more of our current service providers, manufacturers or others with whom we have strategic relationships may not survive any difficult economic times, which could directly affect our ability to attain our operating goals. As of December 31, 2024, we had cash, cash equivalents, and restricted cash of $108.5 million.
In addition, there is a risk that one or more of our current service providers, manufacturers or others with whom we have strategic relationships may not survive any difficult economic times, which could directly affect our ability to attain our operating goals. As of December 31, 2025, we had cash, cash equivalents, and restricted cash of $137.2 million.
Moreover, non-clinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that believed their product candidates performed satisfactorily in preclinical studies and clinical trials nonetheless failed to obtain regulatory approval.
Moreover, non-clinical and clinical data are often susceptible to 54 Table of Contents varying interpretations and analyses, and many companies that believed their product candidates performed satisfactorily in preclinical studies and clinical trials nonetheless failed to obtain regulatory approval.
Additionally, in March 2024, we entered into an underwriting agreement relating to the offering, issuance and sale of an aggregate of 2,500,000 shares of our common stock and warrants to purchase up to an aggregate of 2,500,000 shares of our common stock at a combined offering price of $16.00 per share which resulted in dilution to our existing stockholders.
Additionally, in March 2024, we entered into an underwriting agreement relating to the offering, issuance and sale of an aggregate of 2,500,000 shares of our common stock and warrants to purchase up to an aggregate of 2,500,000 shares of our common stock at a combined offering price of $16.00 per share.
We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded to us, if any, may not be commercially meaningful, while the damages and other remedies we may be ordered to pay such third parties may be significant.
We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded to us, if any, may not be commercially meaningful, while the damages and other remedies we may be ordered to pay such third parties 86 Table of Contents may be significant.
Therefore, we cannot be certain that 77 these patents and patent applications will be prepared, filed, prosecuted, maintained and defended in a manner consistent with the best interests of our business.
Therefore, we cannot be certain that these 80 Table of Contents patents and patent applications will be prepared, filed, prosecuted, maintained and defended in a manner consistent with the best interests of our business.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeThe Audit Committee receives quarterly and, if necessary, ad-hoc reports from the Vice President of Operation & Information Technology and the Head of Information Technology on our cybersecurity and information technology risks, including data security incidents (material and those with lesser impact potential), annual risk assessments, risk identification and evaluation, and targeted remediation plans and related execution/delivery. 89 Our management team, including our Vice President of Operations and Information Technology and Head of Information Technology, has 19+ years of experience in cybersecurity and data risk administration, governance and technical operations, information technology enterprise architecture, and information technology management.
Biggest changeThe Audit Committee receives quarterly and, if necessary, ad-hoc reports from the Vice President of Operation & Information Technology and the Head of Information Technology on our cybersecurity and information technology risks, including data security incidents (material and those with lesser impact potential), annual risk assessments, risk identification and evaluation, and targeted remediation plans and related execution/delivery.
The Audit Committee reports to the full board of directors regarding its activities, including those related to cybersecurity.
The Audit Committee reports to the full board of directors regarding its activities, including those related to cybersecurity. 93 Table of Contents
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Our management team, including our Vice President of Operations and Information Technology and Head of Information Technology, has 20+ years of experience in cybersecurity and data risk administration, governance and technical operations, information technology enterprise architecture, and information technology management.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeItem 3. Legal Proceedings. From time to time we may be involved in claims and proceedings arising in the course of our business. The outcome of any such claims or proceedings, regardless of the merits, is inherently uncertain. We are not currently party to any material legal proceedings. Item 4. Mine Safe ty Disclosures. Not applicable. 90 PART II
Biggest changeItem 3. Legal Proceedings. From time to time we may be involved in claims and proceedings arising in the course of our business. The outcome of any such claims or proceedings, regardless of the merits, is inherently uncertain. We are not currently party to any material legal proceedings. Item 4. Mine Safety Disclosures.
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Not applicable. 94 Table of Contents PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeItem 5. Market for Registrant’s Common Equity, Related Stock holder Matters and Issuer Purchases of Equity Securities. Our common stock trades on The Nasdaq Capital Market under the symbol “DTIL.” Holders of Common Stock As of March 20, 2025, there were approximately 29 holders of record of our common stock.
Biggest changeItem 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities. Our common stock trades on The Nasdaq Capital Market under the symbol “DTIL.” Holders of Common Stock As of March 5, 2026, there were approximately 30 holders of record of our common stock.
Any future determination related to our dividend policy will be made at the discretion of our board of directors after considering our financial condition, results of operations, capital requirements, business prospects and other factors our board of directors deems relevant, and subject to any restrictions applicable to us contained in any future financing instruments. Item 6. [Reserved] 91
Any future determination related to our dividend policy will be made at the discretion of our board of directors after considering our financial condition, results of operations, capital requirements, business prospects and other factors our board of directors deems relevant, and subject to any restrictions applicable to us contained in any future financing instruments.
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Unregistered Sale of Common Stock On October 1, 2025, we issued 47,564 shares of our common stock to LifeSci Advisors, LLC (“LifeSci”) for compensatory purposes. The unregistered shares of our common stock were issued to LifeSci in exchange for investor relations services provided to us by LifeSci.
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The issuance of the shares to LifeSci was not registered under the Securities Act in reliance upon the exemption from registration provided by Section 4(a)(2) thereunder. Item 6. [Reserved] 95 Table of Contents

Item 7. Management's Discussion & Analysis

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

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Biggest changeLoss from Discontinued Operations Loss from discontinued operations represents the gain on the sale of our CAR T infrastructure to Imugene and the results of operations of our historical cell therapy operations. 99 Results of Operations Comparison of the Years Ended December 31, 2024 and December 31, 2023 The following table summarizes our results of operations for the years ended December 31, 2024 and December 31, 2023, together with the changes in those items in dollars: Years ended December 31, (in thousands) 2024 2023 Change Revenue $ 68,696 $ 48,727 $ 19,969 Operating expenses: Research and development 59,559 53,375 6,184 General and administrative 35,299 39,088 (3,789 ) Total operating expenses 94,858 92,463 2,395 Loss from operations (26,162 ) (43,736 ) 17,574 Other income (expense), net: Loss from equity method investment (1,084 ) (4,931 ) 3,847 Gain on changes in fair value 258 1,145 (887 ) Gain on change in fair value of warrant liability 29,610 29,610 Interest expense (1,782 ) (2,230 ) 448 Interest income 6,763 7,686 (923 ) Loss on disposal of assets (436 ) (461 ) 25 Total other income, net 33,329 1,209 32,120 Income (loss) from continuing operations 7,167 (42,527 ) 49,694 Loss from discontinued operations (including gain on disposal of $8,446 during the year ended December 31, 2023) (18,792 ) 18,792 Net income (loss) $ 7,167 $ (61,319 ) $ 68,486 Revenue Revenue for the year ended December 31, 2024 was $68.7 million, compared to $48.7 million for the year ended December 31, 2023.
Biggest changeResults of Operations Comparison of the Years Ended December 31, 2025 and December 31, 2024 The following table summarizes our results of operations for the years ended December 31, 2025 and December 31, 2024, together with the changes in those items: For the Years Ended December 31, (in thousands) 2025 2024 Change Revenue $ 34,264 $ 68,696 $ (34,432) Operating expenses Research and development 54,172 59,559 (5,387) General and administrative 32,240 35,299 (3,059) Total operating expenses 86,412 94,858 (8,446) Operating loss (52,148) (26,162) (25,986) Other income (expense): Loss from equity method investment (5,284) (1,084) (4,200) (Loss) gain on changes in other fair value adjustments (2,666) 258 (2,924) Gain on change in fair value of warrant liability 11,129 29,610 (18,481) Interest expense (1,422) (1,782) 360 Interest income 4,239 6,763 (2,524) Loss on disposal of assets (421) (436) 15 Impairment charges (36) (36) Total other income 5,539 33,329 (27,790) (Loss) income from continuing operations $ (46,609) $ 7,167 $ (53,776) Gain from discontinued operations 885 885 Net (loss) income $ (45,724) $ 7,167 $ (52,891) Revenue Revenue for the year ended December 31, 2025 was $34.3 million, compared to $68.7 million for the year ended December 31, 2024.
For example, if the FDA or another regulatory authority were to require us to conduct clinical trials beyond those that we anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant delays in our clinical trials due to slower than expected 98 patient enrollment or other reasons, we would be required to expend significant additional financial resources and time on the completion of clinical development.
For example, if the FDA or another regulatory authority were to require us to conduct clinical trials beyond those that we anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant delays in our clinical trials due to slower than expected patient enrollment or other reasons, we would be required to expend significant additional financial resources and time on the completion of clinical development.
If we are unable to raise additional funds through equity or debt financings when needed, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to 104 develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.
If we are unable to raise additional funds through equity or debt financings when needed, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.
For up to three additional research programs to be developed by Imugene, we are eligible for up to $145 million in milestone payments and, if licensed products are approved and sold, tiered royalties ranging from the mid-single digit to low-double digit percentages on net sales of such licensed products.
For up to three additional research programs to be developed by Imugene, we are eligible for up to $145.0 million in milestone payments and, if licensed products are approved and sold, tiered royalties ranging from the mid-single digit to low-double digit percentages on net sales of such licensed products.
Income Taxes Since our inception in 2006, we have generated cumulative federal and state NOL and R&D credit carryforwards for which we have not recorded any net tax benefit due to the uncertainty around utilizing these tax attributes within their respective carryforward 101 periods.
Income Taxes Since our inception in 2006, we have generated cumulative federal and state NOL and R&D credit carryforwards for which we have not recorded any net tax benefit due to the uncertainty around utilizing these tax attributes within their respective carryforward periods.
In consideration for the Acquired Assets, Imugene US assumed certain liabilities, paid us $8 million in cash, and issued us convertible notes pursuant to the terms and conditions set forth in a convertible note subscription deed (collectively, the “Imugene Convertible Note”) in an aggregate principal amount of $13 million.
In consideration for the Acquired Assets, Imugene US assumed certain liabilities, paid us $8.0 million in cash, and issued us convertible notes pursuant to the terms and conditions set forth in a convertible note subscription deed (collectively, the “Imugene Convertible Note”) in an aggregate principal amount of $13.0 million.
Our future funding requirements will depend on many factors, including: the ability to collaborate and partner with third parties to fund any or all of our programs; the progress, costs and results of our additional research and preclinical development programs including our in vivo pipeline and our planned IND or CTA submissions and potential biologics license application (“BLA”) submissions; the outcome, timing and cost of meeting regulatory requirements established by the FDA and other comparable foreign regulatory authorities; our ability to establish and maintain strategic collaborations, licensing or other agreements and the financial terms of such agreements; the scope, progress, results and costs of any product candidates that we may derive from ARCUS or any other product candidates we may develop alone or with collaborators; the extent to which we in-license or acquire rights to other products, product candidates or technologies; the costs and timing of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending against any intellectual property-related claims; and the costs and timing of future commercialization activities, including product manufacturing, marketing, sales and distribution, for any product candidates for which we or our collaborators obtain marketing approval.
Our future funding requirements will depend on many factors, including: the ability to collaborate and partner with third parties to fund any or all of our programs; the progress, costs and results of our additional research and preclinical development programs including our in vivo pipeline and our planned IND or CTA submissions and potential biologics license application (“BLA”) submissions; 106 Table of Contents the outcome, timing and cost of meeting regulatory requirements established by the FDA and other comparable foreign regulatory authorities; our ability to establish and maintain strategic collaborations, licensing or other agreements and the financial terms of such agreements; the scope, progress, results and costs of any product candidates that we may derive from ARCUS or any other product candidates we may develop alone or with collaborators; the extent to which we in-license or acquire rights to other products, product candidates or technologies; the costs and timing of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending against any intellectual property-related claims; and the costs and timing of future commercialization activities, including product manufacturing, marketing, sales and distribution, for any product candidates for which we or our collaborators obtain marketing approval.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations. The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our Financial Statements and the related notes to those statements included elsewhere in this Annual Report on Form 10-K.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations. The following discussion and analysis of our financial condition, results of operations, and cash flows should be read in conjunction with our Financial Statements and the related notes to those statements included elsewhere in this Annual Report on Form 10-K.
Under the terms of the 2024 Loan and Security Agreement, we granted Banc of California a security interest in a cash security account at Banc of California (the “Cash Security Account”), and Banc of California agreed to terminate all other security interests it had in our assets.
Debt Obligations Under the terms of the 2024 Loan and Security Agreement, we granted Banc of California a security interest in a cash security account at Banc of California (the “Cash Security Account”), and Banc of California agreed to terminate all other security interests it had in our assets.
Pursuant to and simultaneously with the execution of the Imugene Purchase Agreement, Imugene US acquired our manufacturing infrastructure used in the development and manufacture of azer-cel, including assuming the lease to our manufacturing facility and certain contracts with respect to our manufacturing facility, and related equipment, supplies, azer-cel clinical trial inventory and other assets related to our CAR T cell therapy platform (the “Acquired Assets”).
Pursuant to and simultaneously with the execution of the Imugene Purchase Agreement, Imugene US acquired our manufacturing infrastructure used in the development and manufacture of azer-cel, including assuming the lease to our manufacturing facility and certain contracts with respect to our manufacturing facility, and 98 Table of Contents related equipment, supplies, azer-cel clinical trial inventory and other assets related to our CAR T cell therapy platform (the “Acquired Assets”).
Derivative warrant liabilities are classified as non-current liabilities as their liquidation is not reasonably expected to require the use of current assets or require the creation of current liabilities. Accrued Research and Development Expenses As part of the process of preparing our financial statements, we are required to make certain estimates and judgments in our accrued research and development expenses.
Derivative warrant liabilities are classified as non-current liabilities as their liquidation is not reasonably expected to require the use of current assets or require the creation of current liabilities. 109 Table of Contents Accrued Research and Development Expenses As part of the process of preparing our financial statements, we are required to make certain estimates and judgments in our accrued research and development expenses.
We will continue to be a smaller reporting company if either (i) the market value of our stock held by non-affiliates is less than $250 million or (ii) our annual revenue is less than $100 million during the most recently completed fiscal year and the market value of our stock held by non-affiliates is less than $700 million.
We will continue to be a smaller reporting company if either (i) the market value of our stock held by non-affiliates is less than $250 million or (ii) our annual revenue is less than $100 million during the most recently completed fiscal year and the market value of 110 Table of Contents our stock held by non-affiliates is less than $700 million.
The ordinary shares of Imugene Limited were determined using a conversion price based on the 10-day volume weighted average price of Imugene Limited’s ordinary shares prior to the date of conversion.
The ordinary shares of Imugene Limited were determined using a conversion price based on the 10 days volume weighted average price of Imugene Limited’s ordinary shares prior to the date of conversion.
Gain on Change in Fair Value of Warrant Liability The change in fair value of warrant liability represents the mark-to-market fair value adjustments to the outstanding warrants issued in connection with the March 2024 Public Offering. Interest Expense Interest expense consists of interest payments incurred and discount amortization on debt outstanding.
Change in Fair Value of Warrant Liability The change in fair value of warrant liability represents the mark-to-market fair value adjustments to the outstanding warrants issued in connection with the March 2024 Public Offering and November 2025 Public Offering. Interest Expense Interest expense consists of interest payments incurred and discount amortization on debt outstanding.
Interest Income Interest income consists of interest income earned on our cash and cash equivalents and note receivable from Elo. Loss on Disposal of Assets Loss on disposal of assets represents the remaining net book value of disposed assets at the time of their disposal and impairment recognized on assets held for sale.
Interest Income Interest income consists of interest income earned on our cash and cash equivalents and note receivable from Elo. 101 Table of Contents Loss on Disposal of Assets Loss on disposal of assets represents the remaining net book value of disposed assets at the time of their disposal and impairment recognized on assets held for sale.
Interest Expense Interest expense was $1.8 million and $2.2 million for the years ended December 31, 2024 and 2023, respectively. The $0.4 million decrease in interest expense was the result of lower interest rates during the year ended December 31, 2024 as compared to the year ended December 31, 2023.
Interest Expense Interest expense was $1.4 million and $1.8 million for the years ended December 31, 2025 and 2024, respectively. The $0.4 million decrease in interest expense was the result of lower interest rates during the year ended December 31, 2025 as compared to the year ended December 31, 2024.
We cannot determine with certainty the duration and costs of future clinical trials for our product candidates we may develop or if, when or to what extent we will generate revenue from the commercialization and sale of any product candidate for which we obtain marketing approval. We may never succeed in obtaining marketing approval for any product candidate.
We cannot determine with certainty the duration and costs of future clinical trials for our product candidates we may develop or if, when or to what extent we will generate revenue from the commercialization and sale of any product candidate for which we obtain marketing approval.
As of December 31, 2024, we also had federal and state R&D tax credits of $20.2 million and an amount less than $0.1 million, which begin to expire in 2029 and 2030, respectively. As of December 31, 2024 we had federal Orphan Drug credits of $13.5 million which begin to expire in 2038.
As of December 31, 2025, we also had federal and state R&D tax credits of $22.5 million and an amount less than $0.1 million, which begin to expire in 2029 and 2030, respectively. As of December 31, 2025, we had federal Orphan Drug credits of $13.5 million which begin to expire in 2038.
A discussion regarding our financial condition and results of operations, including liquidity and capital resources, for the year ended December 31, 2024 compared to the year ended December 31, 2023 is presented below.
A discussion regarding our financial condition, results of operations, and cash flows, including liquidity and capital resources, for the year ended December 31, 2025 compared to the year ended December 31, 2024 is presented below.
As of December 31, 2024, we had an accumulated deficit of $482.5 million. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the development of our product candidates.
As of December 31, 2025, we had an accumulated deficit of $528.2 million. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the development of our product candidates.
Cash (Used in) Provided by Investing Activities Cash used in investing activities primarily relates to cash expenditures to acquire leasehold additions, equipment, software and intangible assets. Net cash used in investing activities during the year ended December 31, 2024 was $0.2 million, compared to $5.8 million provided by investing activities in the year ended December 31, 2023.
Cash Used in Investing Activities Cash used in investing activities primarily relates to cash expenditures to acquire leasehold additions, equipment, software and intangible assets. Net cash used in investing activities during the year ended December 31, 2025 was $0.6 million, compared to $0.2 million in the year ended December 31, 2024.
Novartis Pharma AG On June 14, 2022, we entered into the Novartis Agreement, which became effective on June 15, 2022 (the “Novartis Effective Date”), to collaborate to discover and develop in vivo gene editing products incorporating our custom ARCUS nucleases for the purpose of seeking to research and develop potential treatments for certain diseases (collectively referred to as licensed products).
Novartis Pharma AG On June 14, 2022, we entered into the Novartis Agreement, which became effective on June 15, 2022 (the “Novartis Effective Date”), to collaborate to discover and develop in vivo gene editing products incorporating our custom ARCUS nucleases for the purpose of seeking to research and develop potential treatments for certain diseases, including sickle cell disease and beta thalassemia.
Cash Provided by Financing Activities Net cash provided by financing activities during the year ended December 31, 2024 was $50.5 million, compared to $5.4 million during the year ended December 31, 2023.
Cash Provided by Financing Activities Net cash provided by financing activities during the year ended December 31, 2025 was $95.2 million, compared to $50.5 million during the year ended December 31, 2024.
As of December 31, 2024, we had federal and state NOL carryforwards of $235.5 million and $212.0 million respectively, which may be available to offset future taxable income. The U.S. federal NOLs carryforward indefinitely. The state NOL carryforwards begin to expire in 2027.
As of December 31, 2025, we had federal and state NOL carryforwards of $340.2 million and $317.3 million respectively, which may be available to offset future taxable income. The U.S. federal NOLs carryforward indefinitely. The state NOL carryforwards begin to expire in 2027.
Loss from Equity Method Investment Loss from equity method investment represents our proportionate share of the net loss of our equity method investee, Elo Life Systems, Inc. (“Elo”). Gain on Changes in Fair Value The change in fair value represents the assessed changes in assets and liabilities carried at fair value.
Change in Equity Method Investment Changes in fair value on our equity method investment represents changes in the investment value of our equity method investee, Elo Life Systems, Inc. (“Elo”). Changes in Other Fair Value Adjustments The change in fair value represents the assessed changes in assets and liabilities carried at fair value.
Cash used in operating activities during the year ended December 31, 2024 was $58.4 million, compared to $84.1 million during the year ended December 31, 2023.
Cash used in operating activities during the year ended December 31, 2025 was $65.8 million, compared to $58.4 million during the year ended December 31, 2024.
The Upfront Payment of $10.0 million is comprised of (i) a $5.25 million cash payment that was paid to us on February 5, 2024, (ii) a $2.25 million cash payment that was paid to us on February 5, 2024 in exchange for 97,360 shares of our common stock, based on a price per share equal to a 100% premium to the VWAP of our common stock for the 30 trading days prior to the date of the TG License Agreement, and (iii) a deferred cash payment of $2.5 million that was paid to us on January 6, 2025 in exchange for 220,712 shares of our common stock, based on a price per share equal to the greater of (A) 100% premium to the VWAP of our common stock for the 30 trading days prior to the date of payment or (B) a minimum price of $11.1660 determined in accordance with Nasdaq Listing Rule 5635(d) (the “Minimum Price”). 94 The Initial Milestone Payment of $7.5 million, if payable, will consist of (i) a $5.25 million cash milestone payment and (ii) a $2.25 million cash payment payable in exchange for such number of shares of our common stock determined based on a price per share equal to the greater of (A) 100% premium to the VWAP of our common stock for the 30 trading days prior to the achievement of such milestone or (B) the Minimum Price.
The Upfront Payment of $10.0 million was comprised of (i) a $5.25 million cash payment that was paid to us on February 5, 2024, (ii) a $2.25 million cash payment that was paid to us on February 5, 2024 in exchange for 97,360 shares of our common stock, based on a price per share equal to a 100% premium to the VWAP of our common stock for the 30 trading days prior to the date of the TG License Agreement, and (iii) a deferred cash payment of $2.5 million that was paid to us on January 6, 2025 in exchange for 220,712 shares of our common stock, based on a price per share equal to the greater of (A) 100% premium to the VWAP of our common stock for the 30 trading days prior to the date of payment or (B) the Minimum Price.
For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, generally by analogy to ASC 606.
For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, generally by analogy to ASC 606. For those elements of the arrangement that are accounted for pursuant to ASC 606, we apply the five-step model described above.
The $45.1 million increase in cash provided by financing activities during the year ended December 31, 2024 was primarily due to a $44.4 million increase in net proceeds from offerings of common stock in the year ended December 31, 2024 as compared to the year ended December 31, 2023 and $0.9 million in net proceeds from the issuance of common stock to TG Therapeutics in the year ended December 31, 2024.
The $44.7 million increase in cash provided by financing activities during the year ended December 31, 2025 was primarily due to a $33.0 million increase in net proceeds from the underwritten offerings of common stock and warrants in the year ended December 31, 2025 as compared to the year ended December 31, 2024.
Critical Accounting Policies and Use of Estimates Our management’s discussion and analysis of financial condition and results of operations is based on our Financial Statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”).
The Company does not have any material capital expenditure commitments at December 31, 2025. 107 Table of Contents Critical Accounting Policies and Use of Estimates Our management’s discussion and analysis of financial condition and results of operations is based on our Financial Statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”).
As of December 31, 2024 management has constrained all variable consideration related to milestone payments in the Imugene License Agreement given the level of uncertainty associated with achievement of the milestone payments. Accordingly, no revenue was recognized under the Imugene License Agreement during the year ended December 31, 2024.
As of December 31, 2025 management has constrained all remaining variable consideration related to milestone payments in the Imugene License Agreement given the level of uncertainty associated with achievement of the milestone payments.
If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be required to raise additional capital, potentially on terms that are unfavorable to us, or we may be unable to continue our operations at planned levels and be forced to reduce or terminate operations. 102 Cash Flows Our cash, cash equivalents, and restricted cash totaled $108.5 million and $116.7 million as of December 31, 2024, and 2023, respectively.
If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be required to raise additional capital, potentially on terms that are unfavorable to us, or we may be unable to continue our operations at planned levels and be forced to reduce or terminate operations.
As described below, up to $10.0 million of the cash payments potentially payable us are payable in exchange for the issuance (the “Company Stock Issuances”) to TG Subsidiary of shares of our common stock.
We are entitled to additional payments upon the achievement of additional specified milestones of up to $288.6 million (the “Additional Milestone Payments”). As described below, up to $10.0 million of the cash payments received and potentially payable us are payable in exchange for the issuance (the “Company Stock Issuances”) to TG Subsidiary of shares of our common stock.
To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. 105 At contract inception, once the contract is determined to be within the scope of ASC 606, we evaluate the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determine whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract.
To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation.
Common Stock Offering In March 2024, we entered into an underwriting agreement relating to the offering, issuance and sale of an aggregate of 2,500,000 shares of our common stock and warrants to purchase up to an aggregate of 2,500,000 shares of our common stock at a combined offering price of $16.00 per share.
Common Stock Offering In November 2025, we entered into an underwriting agreement relating to the offering, issuance and sale of an aggregate of 10,815,000 shares of our common stock and accompanying one-half warrants to purchase up to an aggregate of 5,407,500 shares of our common stock at a combined offering price of $6.14 per share.
Additionally, we entered into a license agreement with Imugene (the “Imugene License Agreement”) on the Closing Date, pursuant to which we granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic CAR T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement. 95 In addition, under the License Agreement, we are eligible to receive milestone payments of up to an aggregate of $206 million for azer-cel, inclusive of a payment of $8 million in cash and equity upon successful completion of the Phase 1b dosing in the CAR T relapsed LBCL patient population.
Additionally, we entered into a license agreement with Imugene (the “Imugene License Agreement”) on the Closing Date, pursuant to which we granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic CAR T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement.
Expected dividend yield is based on the fact that we have never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future.
Expected dividend yield is based on the fact that we have never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. The fair value of each restricted stock unit is determined based on the closing market price of our common stock on the date of grant.
The Imugene Convertible Note matured on August 30, 2024 and resulted in payment to us in cash and ordinary shares of Imugene Limited. We adjust the carrying value of the ordinary shares issued under the Imugene Convertible Note to its fair value each reporting period with any changes in fair value recorded to other income (expense).
We adjust the carrying value of the ordinary shares issued from Imugene to its fair value each reporting period with any changes in fair value recorded to other income (expense).
The following table summarizes our sources and uses of cash for the periods presented: For the Years Ended December 31, (in thousands) 2024 2023 Components of cash used in operating activities: Net income (loss) 7,167 (61,319 ) Non-cash adjustments (11,613 ) 18,692 Changes in operating assets and liabilities (53,999 ) (41,487 ) Net cash used in operating activities $ (58,445 ) $ (84,114 ) Net cash (used in) provided by investing activities (215 ) 5,829 Net cash provided by financing activities 50,450 5,387 Decrease in cash, cash equivalents and restricted cash $ (8,210 ) $ (72,898 ) Cash Used in Operating Activities Our primary use of cash is to fund operating expenses, which consist primarily of research and development and general and administrative costs.
The following table summarizes our sources and uses of cash for the periods presented: For the Years Ended December 31, (in thousands) 2025 2024 Change Components of cash used in operating activities: Net (loss) income $ (45,724) $ 7,167 $ (52,891) Non-cash adjustments 9,125 (11,613) 20,738 Changes in operating assets and liabilities (29,244) (53,999) 24,755 Net cash used in operating activities $ (65,843) $ (58,445) $ (7,398) Net cash used in investing activities (634) (215) (419) Net cash provided by financing activities 95,162 50,450 44,712 Increase (decrease) in cash, cash equivalents and restricted cash $ 28,685 $ (8,210) $ 36,895 Cash Used in Operating Activities Our primary use of cash is to fund operating expenses, which consist primarily of research and development and general and administrative costs.
We are required to maintain an aggregate unencumbered balance in the Cash Security Account at least equal to the outstanding principal amount of the 2024 Term Loan then outstanding.
We are required to maintain an aggregate unencumbered balance in the Cash Security Account at least equal to the outstanding principal amount of the 2024 Term Loan then outstanding. As of December 31, 2025, the outstanding principal balance on the 2024 Term Loan was $22.5 million, the stated interest rate was 5.25% and the effective interest rate was 5.62%.
Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as noncurrent deferred revenue. Amounts recognized as revenue, but not yet invoiced are generally recognized as contract assets in the other current assets line item in our balance sheets.
Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as noncurrent deferred revenue.
The $12.5 million year over year decline in cash used in operating assets and liabilities is primarily driven by a decrease in net changes of deferred revenue as a result of the conclusion of the Prevail collaboration.
The change in cash used in operating assets and liabilities was primarily driven by the net changes of deferred revenue as a result of the conclusion of Novartis collaboration in the year ended December 31, 2025 and the conclusion of the Prevail collaboration in the year ended December 31, 2024.
The $25.7 million decrease in cash used in operating activities was primarily driven by a $68.5 million increase in net income, partially offset by decreases of $30.3 million in non-cash adjustments and $12.5 million in changes in operating assets and liabilities.
The $7.4 million increase in cash used in operating activities was primarily 105 Table of Contents driven by a $52.9 million decrease in net income, partially offset by increases of $20.7 million in non-cash adjustments and $24.8 million in changes in operating assets and liabilities.
Milestone Payments If an arrangement includes development and regulatory milestone payments, we evaluate whether the milestones are considered probable of being reached and estimate the amount to be included in the transaction price using the most likely amount method.
Amounts recognized as revenue, but not yet invoiced are generally recognized as contract assets in the other current assets line item in our balance sheets. 108 Table of Contents Milestone Payments If an arrangement includes development and regulatory milestone payments, we evaluate whether the milestones are considered probable of being reached and estimate the amount to be included in the transaction price using the most likely amount method.
The $6.0 million decrease in cash provided by investing activities was primarily the result of the $8.0 million proceeds from the disposal of CAR T infrastructure in 2023, partially offset by $2.0 million of property, equipment, and software purchases during the year ended December 31, 2023.
The increase in cash used in investing activities was primarily the result of a $0.6 million increase in intangible asset purchases during the year ended December 31, 2025, partially offset by a $0.2 million decrease in property, equipment, and software purchases between the comparison periods.
There was no change in the fair value of the IECURE equity during the year ended December 31, 2024.
During the year ended December 31, 2025, we recorded a $2.5 million decrease in the carrying value of our iECURE equity to adjust to fair value. There was no change in the fair value of the iECURE equity during the year ended December 31, 2024.
Common Stock Offering In March 2024, we entered into an underwriting agreement relating to the issuance and sale of an aggregate of 2,500,000 shares of our common stock and warrants to purchase 2,500,000 shares of our common stock at a combined offering price of $16.00 per share.
Common Stock Offering On November 10, 2025, we entered into an underwriting agreement relating to the issuance and sale of 10,815,000 shares of our common stock and accompanying one-half warrants to purchase up to 5,407,500 shares of our common stock at a combined price of $6.14 per share.
The $0.9 million decrease in interest income was the result of lower interest rates during the year ended December 31, 2024 as compared to the year ended December 31, 2023 Loss on Disposal of Assets Loss on disposal of assets was $0.4 million and $0.5 million during the years ended December 31, 2024 and 2023, respectively.
Interest Income Interest income was $4.2 million during the year ended December 31, 2025 compared to $6.8 million during the year ended December 31, 2024. The $2.6 million decrease in interest income was the result of lower interest rates during the year ended December 31, 2025 as compared to the year ended December 31, 2024.
If we obtain regulatory approval for any of our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. As a result, we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources.
As a result, we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources.
We expect our cash runway to be sufficient to achieve first-in-human Phase 1 clinical data for two of our wholly-owned in vivo gene editing programs. We have based these estimates on assumptions that may prove to be imprecise, and we could utilize our available capital resources sooner than we expect.
We expect our cash runway to be sufficient to fund PBGENE-HBV and PBGENE-DMD data milestones through 2028. We have based these estimates on assumptions that may prove to be imprecise, and we could utilize our available capital resources sooner than we expect.
General and Administrative Expenses General and administrative expenses were $35.3 million for the year ended December 31, 2024 compared to $39.1 million for the year ended December 31, 2023.
General and Administrative Expenses General and administrative expenses were $32.2 million for the year ended December 31, 2025 compared to $35.3 million for the year ended December 31, 2024. The decrease of $3.1 million was primarily due to a $2.2 million decrease in employee-related expenses and consulting services.
Risk Factors, we believe that, as of the date of this Annual Report on Form 10-K, existing cash and cash equivalents, expected operational receipts, including upfront and potential near-term consideration to be received from our licensees, operational efficiencies gained from divestment of our historical CAR T operations, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2026.
Risk Factors ,” we believe that, as of the date of this Annual Report on Form 10-K, existing cash and cash equivalents, inclusive of the expected azer-cel milestone proceeds, continued fiscal and operating discipline, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements through 2028.
Gain on Change in Fair Value of Warrant Liability Gain from change in fair value of the warrant liability of $29.6 million for the year ended December 31, 2024 represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering. There was no warrant liability in the year ended December 31, 2023.
Gain on changes in fair value was $0.3 million for the year ended December 31, 2024, primarily due to an increase in the fair value of the Imugene Convertible Note and prior Imugene ordinary share holdings. 103 Table of Contents Gain on Change in Fair Value of Warrant Liability Gain from change in fair value of the warrant liability of $11.1 million for the year ended December 31, 2025 compared to a gain of $29.6 million for the year ended December 31, 2024, which represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 and November 2025 Public Offerings.
We record revenue from collaboration agreements, including amounts related to upfront payments, milestone payments, fees for licenses of our intellectual property and research and development funding. Research and Development Expenses Research and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts and the development of our product candidates.
Components of Our Results of Operations Revenue To date, we have not generated any revenue from product sales and do not expect to generate any revenue from product sales in the foreseeable future. We record revenue from collaboration agreements, including amounts related to upfront payments, milestone payments, fees for licenses of our intellectual property and research and development funding.
As of December 31, 2024, we also have federal contribution carryforwards of $0.1 million, which begin to expire in 2026. We have recorded a full valuation allowance against our net deferred tax assets at each balance sheet date.
As of December 31, 2025, we also have federal contribution carryforwards of $0.1 million, which begin to expire in 2026.
Loss from equity method investment was $4.9 million during the year ended December 31, 2023, which represented our proportionate share of Elo’s net loss for such periods.
Refer to Note 11, Elo Transaction , in the accompanying notes to the financial statements for further details. Loss from equity method investment was $1.1 million during the year ended December 31, 2024, which represented our proportionate share of Elo’s net loss for the period.
Prevail’s notice informed us that Prevail was exercising its right pursuant to Section 15.3.2 of the Prevail Agreement to terminate the Prevail Agreement in its entirety without cause upon 90 days’ prior written notice to us. We subsequently exercised our rights to the return of the three programs. The termination was effective on July 10, 2024.
The notice informed us that Novartis was exercising its right to terminate the Novartis Agreement in its entirety without cause upon 90 days’ prior written notice to us. The termination was effective on January 30, 2026.
We believe that, as of the date of this Annual Report on Form 10-K, existing cash and cash equivalents, expected operational receipts, including upfront and potential near-term consideration to be received from licensees, operational efficiencies gained from divestment of our historical CAR T operations, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2026.
We believe that, as of the date of this Annual Report on Form 10-K, existing cash and cash equivalents, inclusive of the expected azer-cel milestone proceeds, continued fiscal and operating discipline, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements through 2028.
The increase of $20.0 million in revenue during the year ended December 31, 2024 was primarily the result of a $26.7 million increase in revenue related to the Prevail Agreement as the remaining deferred revenue under the agreement was recognized during the year ended December 31, 2024 following conclusion of the collaboration in April 2024.
The decrease of $34.4 million in revenue during the year ended December 31, 2025 was primarily the result of $52.7 million of revenue recognized in the prior period related to the conclusion of our agreement with Prevail Therapeutics, which represented all remaining deferred revenue under that agreement.
Additionally, we are eligible to receive milestone and mid-single digit to low double digit royalty payments on sales of iECURE products developed with ARCUS. We adjust the carrying value of the iECURE equity to fair value each reporting period with any changes in fair value recorded to other income (expense).
We adjust the carrying value of the iECURE investment under the iECURE Equity Agreement to its fair value each reporting period with any changes in fair value recorded to other (expense) income.
For example, in in the year ended December 31, 2024, we did not record any cumulative catch-up adjustments on contracts. Warrant Liability The warrants issued in the March 2024 Public Offering were recognized as derivative warrant liabilities in accordance with ASC 815.
Warrant Liability The warrants issued in the March 2024 Public Offering as well as the warrants issued in the November 2025 Public Offering were recognized as derivative warrant liabilities in accordance with ASC 815.
For those elements of the arrangement that are accounted for pursuant to ASC 606, we apply the five-step model described above. 106 Revenue related to performance obligations satisfied over time could be materially impacted as a result of changes in the estimated research effort to satisfy performance obligations or changes in the transaction price related to variable consideration.
Revenue related to performance obligations satisfied over time could be materially impacted as a result of changes in the estimated research effort to satisfy performance obligations or changes in the transaction price related to variable consideration. For example, in in the year ended December 31, 2025, we did not record any cumulative catch-up adjustments on contracts.
Deferred revenue related to the Novartis Agreement amounted to $26.3 million and $32.4 million as of December 31, 2024 and December 31, 2023, respectively, of which $3.0 million and $7.4 million, respectively, was included in current liabilities within the balance sheets. Prevail Therapeutics, Inc.
The deferred revenue balance related to the Novartis agreement as of December 31, 2024 was $26.3 million, of which $3.0 million was included in current liabilities within the balance sheets. iECURE We adjust the carrying value of the iECURE equity to fair value each reporting period with any changes in fair value recorded to other income (expense).
We intend to use the net proceeds of the offering to fund ongoing and planned research and development, and for working capital and other general corporate purposes.
The offering was made pursuant to a registration statement on Form S-3. Gross proceeds from the transaction were $75.0 million before deducting underwriting discounts and commissions and offering expenses of approximately $5.0 million. We intend to use the net proceeds of the offering to fund ongoing and planned research and development, and for working capital and other general corporate purposes.
Under the TG License Agreement, we are entitled to receive an upfront cash payment of $10.0 million (the “Upfront Payment”), an additional cash payment of $7.5 million in the event that TG Therapeutics achieves a certain clinical milestone that is expected to be achieved in the near-term (the “Initial Milestone Payment”), and additional payments upon the achievement of additional specified milestones of up to $288.6 million (the “Additional Milestone Payments”).
The offering was made pursuant to a registration statement on Form S-3. 97 Table of Contents License and Collaboration Transactions TG Therapeutics Under the TG License Agreement, we received an upfront cash payment of $10.0 million (the “Upfront Payment”) and are entitled to receive an additional cash payment of $7.5 million as TG Therapeutics achieved a certain clinical milestone (the “Initial Milestone Payment”).
Gain on Changes in Fair Value Gain on changes in fair value was $0.3 million for the year ended December 31, 2024, which was primarily attributed to an increase during such period in the fair value of the Imugene Convertible Note and the ordinary shares issued thereunder and still held by us at the end of the period.
(Loss) Gain on Changes in Other Fair Value Adjustments Loss on changes in fair value was $2.7 million for the year ended December 31, 2025, driven by changes in the fair value of the iECURE investment and Imugene ordinary shares issued during the period.
Additionally, there was a $9.5 million increase in revenue in the year ended December 31, 2024 as a result of the TG License Agreement and the license agreement with Caribou.
As a result of the milestone payment received in October 2025, $8.0 million of revenue was recognized under the Imugene License Agreement during the year ended December 31, 2025. There was no revenue recognized under the Imugene License Agreement during the year ended December 31, 2024.
During the years ended December 31, 2024 and 2023 we recognized revenue under the Novartis Agreement of $6.4 million and $22.7 million, respectively.
Although we and Novartis have concluded work in the area of hemoglobinopathies, we and Novartis are continuing our research collaboration in other areas of undisclosed therapeutic focus. During the years ended December 31, 2025 and 2024, we recognized revenue under the Novartis Agreement of $26.3 million and 6.4 million, respectively.
Research and Development Expenses Years ended December 31, (in thousands) 2024 2023 Change Direct research and development expenses by product candidate: PBGENE-HBV external development costs 16,111 9,261 6,850 PBGENE-3243 external development costs 9,808 352 9,456 Platform development and early-stage research expenses: Employee-related costs (other than share-based compensation) 18,753 19,788 (1,035 ) Share-based compensation 2,560 3,642 (1,082 ) Laboratory supplies and services 3,016 5,741 (2,725 ) CMOs and outsourced research and development 1,083 4,753 (3,670 ) Facility-related costs, laboratory equipment, and maintenance 3,082 3,308 (226 ) Depreciation and amortization 2,718 4,022 (1,304 ) Licensing fees and other research and development costs 2,428 2,508 (80 ) Total research and development expenses $ 59,559 $ 53,375 $ 6,184 Research and development expenses for the year ended December 31, 2024 were $59.6 million, compared to $53.4 million for the year ended December 31, 2023.
Partially offsetting the decrease in revenue was a $19.9 million increase in revenue compared to the prior year under the Novartis Agreement as a result of the October 2025 conclusion of the Novartis Agreement and $8.0 million in revenue recognized under the Imugene License Agreement during the year ended December 31, 2025. 102 Table of Contents Research and Development Expenses For the Years Ended December 31, (in thousands) 2025 2024 Change Direct research and development expenses by product candidate: PBGENE-HBV external development costs 7,152 16,111 (8,959) PBGENE-DMD external development costs 15,298 1,230 14,068 PBGENE-3243 external development costs 3,103 9,808 (6,705) Platform development and early-stage research expenses: Employee-related costs (including share-based compensation) 19,939 21,313 (1,374) Laboratory supplies and services 1,798 2,621 (823) CMOs and outsourced research and development 504 247 257 Facility-related costs, laboratory equipment, and maintenance 2,841 3,082 (241) Depreciation and amortization 1,299 2,718 (1,419) Licensing fees and other research and development costs 2,238 2,429 (191) Total research and development expenses $ 54,172 $ 59,559 $ (5,387) Research and development expenses for the year ended December 31, 2025 were $54.2 million, compared to $59.6 million for the year ended December 31, 2024.
Smaller Reporting Company Status We are a “smaller reporting company” as defined under applicable regulations promulgated by the SEC.
Recent Accounting Pronouncements Refer to Note 1, Description of Business and Summary of Significant Accounting Policies, in the accompanying notes to the financial statements for a discussion of recent accounting pronouncements and new accounting pronouncements adopted during 2025. Smaller Reporting Company Status We are a “smaller reporting company” as defined under applicable regulations promulgated by the SEC.
The $30.3 million year over year decrease in non-cash adjustments was primarily driven by a $29.6 million decrease in the change in fair value of warrants which we did not have in the year ended December 31, 2023.
The increase in non-cash adjustments was primarily driven by the net change in fair value of warrants year over year as a result of the new warrants issued in the November 2025 Public Offering.
In January 2025, IECURE reported clinical efficacy and safety data in the first patient dosed with ECUR-506 in the Phase 1/2 OTC-HOPE study. iECURE expects to finish enrollment in 2025 and provide complete data for the program in the first half of 2026.
In January 2025, iECURE reported clinical results demonstrating complete clinical response in the first participant at the lowest dose level (1.3x10 13 GC/kg) of ECUR-506, as defined by the study protocol. iECURE expects to release additional patient data from the ongoing OTC-HOPE trial in the first half of 2026.
The decrease of $3.8 million was primarily due to a $1.0 million decrease in employee-related expenses, a $0.7 million decrease in consulting fees, and $0.5 million decreases each in information technology expenses and depreciation and amortization expense. Loss from Equity Method Investment Loss from equity method investment was $1.1 million during the year ended December 31, 2024.
The remainder of the decrease is attributable to decreases in other miscellaneous operating expenses. Loss from Equity Method Investment Loss from equity method investment was $5.3 million during the year ended December 31, 2025 as a result of the carrying amount adjustment on the Note Receivable.
The Additional Milestone Payments become due upon the achievement of certain milestones as specified in the TG License Agreement.
The Initial Milestone Payment of $7.5 million will consist of (i) a $5.25 million cash milestone payment and (ii) a $2.25 million cash payment in exchange for 201,504 shares of our common stock determined based on the Minimum Price. The Additional Milestone Payments become due upon the achievement of certain milestones as specified in the TG License Agreement.
These contracts are generally cancelable upon written notice. The Company does not have any material capital expenditure commitments at December 31, 2024.
These contracts are generally cancelable upon written notice.
As of December 31, 2024, we had cash and cash equivalents of $85.9 million and $22.6 million in restricted cash under the 2024 Term Loan, which replaced our Revolving Line on July 31, 2024.
As of December 31, 2025, we had cash and cash equivalents of $110.8 million and $26.3 million in restricted cash under the 2024 Term Loan and compensatory arrangements with certain of our officers, respectively. Refer to Note 14, Commitments and Contingencies , in the accompanying notes to the financial statements for more information on these compensatory arrangements.
Additionally, on the Novartis Effective Date, Novartis made an equity investment in our common stock pursuant to a stock purchase agreement (the “Novartis Stock Purchase Agreement”) pursuant to which, on the Novartis Effective Date, we issued and sold to Novartis 413,581 shares of our common stock (the “Novartis Shares”) in a private placement transaction for an aggregate purchase price of $25.0 million, or approximately $60.30 per share.
In July 2022, we received a $50.0 million upfront cash payment under the Novartis Agreement. Additionally, on the Novartis Effective Date, Novartis made an equity investment in our common stock pursuant to a stock purchase agreement. 99 Table of Contents On October 31, 2025, we received written notice from Novartis of its termination of the Novartis Agreement.
Removed
Business Updates Since mid-2023, we have solely focused on leveraging our proprietary ARCUS genome editing platform to advance in vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination, unlocking a broader potential for ARCUS in vivo gene editing in human therapeutics.
Added
Wholly-Owned Portfolio PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, ELIMINATE-B, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. In patients with chronic hepatitis B, cccDNA acts as the template to make new infectious viral particles.
Removed
In January 2024 we entered into a license agreement with TG Cell Therapy, Inc. (“TG Subsidiary”) and its parent company TG Therapeutics, Inc. (“TG Parent” and, together with TG Subsidiary, “TG Therapeutics”) for non-oncological applications of azercabtagene zapreleucel (“azer-cel”) (the “TG License Agreement”).

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Item 7A. Quantitative and Qualitative Disclosures About Market Risk

Market Risk — interest-rate, FX, commodity exposure

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Biggest changeWe had cash, cash equivalents, and restricted cash of $108.5 million, or 80% of our total assets, on December 31, 2024 and $116.7 million, or 73% of our total assets, on December 31, 2023. Interest income earned on our assets was $6.8 million and $7.7 million for the years ended December 31, 2024 and December 31, 2023, respectively.
Biggest changeWe had cash, cash equivalents, and restricted cash of $137.2 million, or 89% of our total assets, on December 31, 2025 and $108.5 million, or 80% of our total assets, on December 31, 2024. Interest income earned on our assets was 4.2 million and 6.8 million for the years ended December 31, 2025 and December 31, 2024, respectively.
A hypothetical 10% change in existing interest rates would not have had a material impact on the value of our cash and cash equivalents as of December 31, 2024.
A hypothetical 10% change in existing interest rates would not have had a material impact on the value of our cash and cash equivalents as of December 31, 2025.
Item 7A. Quantitative and Qualitat ive Disclosures About Market Risk. We are exposed to market risks in the ordinary course of our business. These risks primarily include interest rate sensitivities. Our interest-earning assets consist of cash, cash equivalents, and restricted cash which are denominated in U.S. dollars.
Item 7A. Quantitative and Qualitative Disclosures About Market Risk. We are exposed to market risks in the ordinary course of our business. These risks primarily include interest rate sensitivities. Our interest-earning assets consist of cash, cash equivalents, and restricted cash which are denominated in U.S. dollars.

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