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

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Paragraph-level year-over-year comparison of Intellia Therapeutics, 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.

+523 added−480 removedSource: 10-K (2026-02-26) vs 10-K (2025-02-27)

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

523 paragraphs added · 480 removed · 389 edited across 8 sections

Item 1. Business

Business — how the company describes what it does

171 edited+65 added−56 removed136 unchanged
Biggest changeA reconciliation of the federal statutory income tax rate and the Company’s effective income tax rate is as follows: Year Ended December 31, 2024 2023 2022 Federal statutory income tax rate ( 21.0 )% ( 21.0 )% ( 21.0 )% State income taxes ( 7.9 ) ( 8.6 ) ( 8.3 ) Research and development tax credits ( 7.4 ) ( 6.9 ) ( 5.3 ) Stock-based compensation 2.0 1.6 ( 0.1 ) Non-deductible officers' compensation 0.1 - 0.1 In-process research and development - - 2.5 Change in valuation allowance 34.2 34.9 32.1 Effective income tax rate — % — % — % The Company’s net deferred tax assets (liabilities) consisted of the following: December 31, 2024 2023 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 296,766 $ 258,664 Research and development credit carryforwards 200,816 151,333 Section 174 capitalized research 216,469 150,993 Operating lease liability 57,438 31,183 Deferred revenue 10,634 9,633 Stock-based compensation 34,827 30,408 Accruals and allowances 5,468 6,244 Prepaid rent 4,410 1,190 Equity investment adjustments 16,560 7,580 Intangibles, including in-process research and development 920 1,017 Capitalized start-up costs 211 249 Gross deferred tax assets 844,519 648,494 Deferred tax asset valuation allowance ( 783,211 ) ( 616,082 ) Total deferred tax assets 61,308 32,412 Deferred tax liabilities: Fixed assets ( 1,385 ) ( 1,221 ) Operating lease right-of-use assets ( 59,923 ) ( 31,191 ) Total deferred tax liabilities ( 61,308 ) ( 32,412 ) Net deferred tax asset (liability) $ - $ - The Tax Cuts and Jobs Act (“TCJA” ) requires taxpayers to capitalize and amortize, rather than deduct, research and development expenditures under section 174 for tax years beginning after December 31, 2021.
Biggest changeThe following table is a reconciliation of the U.S. federal statutory rate of 21 % to the Company’s effective income tax rate for the year ended December 31, 2025 in accordance with the guidance in ASU 2023-09: Year Ended December 31, In thousands, except percentages 2025 Provision for income taxes at U.S. federal statutory rate ( 21.0 )% $ ( 86,666 ) Tax credits: Research and development credits ( 2.3 ) ( 9,547 ) Orphan drug credits ( 6.5 ) ( 26,678 ) Changes in valuation allowances 25.1 103,507 Non-taxable or non-deductible items: Stock-based compensation 4.5 18,718 Other reconciling items 0.2 666 Total tax provision and effective tax rate — % $ - The following table is a reconciliation of the U.S. federal statutory rate of 21 % to the Company’s effective rate for the years ended December 31, 2024 and 2023 in accordance with the guidance prior to the adoption of ASU 2023-09: 2024 2023 Federal statutory income tax rate ( 21.0 )% ( 21.0 )% State income taxes ( 7.9 ) ( 8.6 ) Research and development tax credits ( 7.4 ) ( 6.9 ) Stock-based compensation 2.0 1.6 Non-deductible officers' compensation 0.1 - Change in valuation allowance 34.2 34.9 Effective income tax rate — % — % F- 17 The Company’s net deferred tax assets (liabilities) consisted of the following: December 31, 2025 2024 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 457,009 $ 296,766 Research and development credit carryforwards 243,725 200,816 Section 174 capitalized research 168,503 216,469 Operating lease liability 25,502 57,438 Deferred revenue 1,562 10,634 Stock-based compensation 26,036 34,827 Accruals and allowances 5,642 5,468 Prepaid rent 899 4,410 Equity investment adjustments 14,743 16,560 Intangibles, including in-process research and development 773 920 Capitalized start-up costs 167 211 Gross deferred tax assets 944,561 844,519 Deferred tax asset valuation allowance ( 914,641 ) ( 783,211 ) Total deferred tax assets 29,920 61,308 Deferred tax liabilities: Fixed assets ( 968 ) ( 1,385 ) Operating lease right-of-use assets ( 28,952 ) ( 59,923 ) Total deferred tax liabilities ( 29,920 ) ( 61,308 ) Net deferred tax asset (liability) $ - $ - During the year ended December 31, 2025, the Company paid no federal income taxes, net of refunds.
F- 7 The effects of material revisions in estimates are reflected in the consolidated financial statements prospectively from the date of the change in estimate. Fair Value Measurements The Company’s financial instruments include cash equivalents, restricted cash equivalents, marketable securities, accounts receivable, non-marketable securities, accounts payable and accrued expenses.
The effects of material revisions in estimates are reflected in the consolidated financial statements prospectively from the date of the change in estimate. F- 7 Fair Value Measurements The Company’s financial instruments include cash equivalents, restricted cash equivalents, marketable securities, accounts receivable, non-marketable securities, accounts payable and accrued expenses.
In the absence of a readily determinable fair value, the Company measures the investment at cost less impairment, plus or minus observable changes, if any. These investments are included in “Investments and other assets” in the Company’s consolidated balance sheets. Refer to Note 10 for further information regarding the Company’s investments in equity securities.
In the absence of a readily determinable fair value, the Company measures the investment at cost less impairment, plus or minus observable changes, if any. These investments are included in “Investments and other assets” in the Company’s consolidated balance sheets. Refer to Note 10, “Investments and Other Assets”, for further information regarding the Company’s investments in equity securities.
(3) 10.13# Form of Employment Agreement for Executive Officers (14) 10.14† Consent to Assignments, Licensing and Common Ownership and Invention Management Agreement dated December 15, 2016 by and between the Registrant, CRISPR Therapeutics AG, The Regents of the University of California, University of Vienna, ERS Genomics Ltd., TRACR Hematology Ltd., Caribou Biosciences, Inc., and Dr.
(3) 10.13# Form of Employment Agreement for Executive Officers (13) 10.14† Consent to Assignments, Licensing and Common Ownership and Invention Management Agreement dated December 15, 2016 by and between the Registrant, CRISPR Therapeutics AG, The Regents of the University of California, University of Vienna, ERS Genomics Ltd., TRACR Hematology Ltd., Caribou Biosciences, Inc., and Dr.
The complaint alleges claims under Sections 10(b) and 20(a) and Rule 10b-5 of the Securities Exchange Act of 1934 (the “Exchange Act”) premised upon statements relating to the Company’s NTLA-3001 program and the demand for viral-based editing. The complaint seeks unspecified damages, interest, reasonable attorneys’ fees and other costs. The Company intends to defend vigorously against the claims.
The amended complaint alleges claims under Sections 10(b) and 20(a) and Rule 10b-5 of the Securities Exchange Act of 1934 (the “Exchange Act”) premised upon statements relating to the Company’s NTLA-3001 program and the demand for viral-based editing. The amended complaint seeks unspecified damages, interest, reasonable attorneys’ fees and other costs. The Company intends to defend vigorously against the claims.
Expected Dividend Yield. The Company has not paid cash dividends and has no intention to pay cash dividends in the future. Stock options generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining two years, unless they contain specific vesting provisions.
Expected Dividend Yield. The Company has not paid cash dividends and has no intention to pay cash dividends in the future. Stock options generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining two years, unless they contain other specific vesting provisions.
If a decline in the value of an equity method investment is determined to be other than temporary, a loss is recorded in earnings in the current period and the investment is written down to fair value. At December 31, 2024, the Company did not account for any of its investments under the equity method of accounting.
If a decline in the value of an equity method investment is determined to be other than temporary, a loss is recorded in earnings in the current period and the investment is written down to fair value. At December 31, 2025 and 2024, the Company did not account for any of its investments under the equity method of accounting.
Because ASC 808 does not provide recognition and measurement guidance for collaborative arrangements, the Company has analogized to ASC 606. Refer to Note 9 for additional information regarding the Company’s collaboration agreements. Research and Development Expenses Research and development costs are expensed as incurred.
Because ASC 808 does not provide recognition and measurement guidance for collaborative arrangements, the Company has analogized to ASC 606. Refer to Note 9, “Collaborations”, for additional information regarding the Company’s collaboration agreements. Research and Development Expenses Research and development costs are expensed as incurred.
In the fourth quarter of 2024, AvenCell completed a Series B financing, which represented an observable price change in the investment in AvenCell. The Company determined the fair value of the AvenCell investment using an option pricing model which requires the input of certain subjective assumptions.
In the fourth quarter of 2024, AvenCell completed a Series B financing, which represented an observable price change in the Company’s investment in AvenCell. The Company determined the fair value of the AvenCell investment using an option pricing model which requires the input of certain subjective assumptions.
If the Company is an active participant and is exposed to the significant risks and rewards with respect to the arrangement, the Company accounts for the arrangement under ASC 808 . Based on this consideration, accounting for the Company’s co-development agreements with Regeneron and AvenCell is under ASC 808.
If the Company is an active participant and is exposed to the significant risks and rewards with respect to the arrangement, the Company accounts for the arrangement under ASC 808 . Based on this consideration, accounting for the Company’s co-development agreements with Regeneron is under ASC 808.
Compensation Recovery Policy (16) 101.INS* Inline XBRL Instance Document. 101.SCH* Inline XBRL Taxonomy Extension Schema With Embedded Linkbase Documents. 104* Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101*) † Portions of this exhibit (indicated by asterisks) have been omitted pursuant to Item 601(b)(10) of Regulation S-K. * Filed herewith. # Indicates a management contract or any compensatory plan, contract or arrangement.
Compensation Recovery Policy (14) 101.INS* Inline XBRL Instance Document. 101.SCH* Inline XBRL Taxonomy Extension Schema With Embedded Linkbase Documents. 104* Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101*) † Portions of this exhibit (indicated by asterisks) have been omitted pursuant to Item 601(b)(10) of Regulation S-K. * Filed herewith. # Indicates a management contract or any compensatory plan, contract or arrangement.
As of December 31, 2024, the Company’s revenue recognized is solely related to collaboration agreements with third parties which are either within the scope of ASC 606, under which the Company licenses certain rights to its product candidates to third parties, or within the scope of ASC 808, Collaborative Arrangements (“ASC 808”) if it involves a joint operating activity pursuant to which the Company is an active participant and is exposed to significant risks and rewards with respect to the arrangement.
As of December 31, 2025, the Company’s revenue recognized is solely related to collaboration agreements with third parties which are either within the scope of ASC 606, under which the Company licenses certain rights to its product candidates to third parties, or within the scope of ASC 808, Collaborative Arrangements (“ASC 808”) if it involves a joint operating activity pursuant to which the Company is an active participant and is exposed to significant risks and rewards with respect to the arrangement.
During the year ended December 31, 2023, the Company issued 4,122,824 shares of its common stock, in a series of sales, at an average price of $ 30.57 per share, in accordance with the 2022 Sale Agreement for aggregate net proceeds of $ 121.9 million, after payment of cash commissions and legal, accounting and other fees in connection with the sales.
During the year ended December 31, 2023, the Company issued 4,122,824 shares of its common stock, in a series of sales, at an average price of $ 30.57 per share, in accordance with the 2022 Sale Agreement for aggregate net proceeds of $ 121.9 million, after commissions and legal, accounting and other fees in connection with the sales.
There were no material realized gains or losses in the years ended December 31, 2024 or 2023. The Company did not reclassify any amounts out of accumulated other comprehensive loss during these periods. The Company generally does not intend to sell any investments prior to recovery of their amortized cost basis for any investment in an unrealized loss position.
There were no material realized gains or losses in the years ended December 31, 2025 or 2024. The Company did not reclassify any amounts out of accumulated other comprehensive loss during these periods. The Company generally does not intend to sell any investments prior to recovery of their amortized cost basis for any investment in an unrealized loss position.
GAAP”) requires management to make estimates, judgments and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of revenues, research and development expenses, valuation and determination of impairment of equity and fair value method investments, contingent consideration and stock-based compensation expense.
GAAP”) requires management to make estimates, judgments and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of revenues, research and development expenses, valuation and determination of impairment of equity and fair value method investments and stock-based compensation expense.
Royalties: For arrangements that include sales-based royalties, including milestone payments based on levels of sales, if the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied).
Royalties: For arrangements that include sales-based royalties, including milestone payments based on levels of sales, if the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has F- 11 been satisfied (or partially satisfied).
However, a full valuation allowance has been provided against the deferred tax assets related to the Company’s net operating loss and tax credit carryforwards and, if an adjustment is required, this adjustment would be offset by an adjustment to the valuation allowance. As of December 31, 2024 , the Company had no t identified any unrecognized tax benefits.
However, a full valuation allowance has been provided against the deferred tax assets related to the Company’s net operating loss and tax credit carryforwards and, if an adjustment is required, this adjustment would be offset by an adjustment to the valuation allowance. As of December 31, 2025, the Company had no t identified any unrecognized tax benefits.
On February 21, 2025, the court substantially denied BlueAllele’s motion to dismiss, and granted the motion with respect to one counterclaim. At this stage, the Company is unable to determine the likelihood of an unfavorable outcome or estimate the amount or range of potential loss, if any. Gonzalez v. Intellia Therapeutics, Inc.
On February 21, 2025, the court substantially denied BlueAllele’s motion to dismiss, and granted the motion with respect to one counterclaim. At this stage, the Company is unable to determine the likelihood of an unfavorable outcome or estimate the amount or range of potential loss, if any. F- 19 Gonzalez v. Intellia Therapeutics, Inc.
At this stage, the Company is unable to determine the likelihood of an unfavorable outcome or estimate the amount or range of potential loss, if any. During the year ended December 31, 2024, except as noted above, there have been no material changes to any outstanding litigation, nor is the Company a party to any material new litigation.
At this stage, the Company is unable to determine the likelihood of an unfavorable outcome or estimate the amount or range of potential loss, if any. During the year ended December 31, 2025, except as noted above, there have been no material changes to any outstanding litigation, nor is the Company a party to any material new litigation.
The Company uses the simplified method because it does not have sufficient historical option exercise data to provide a reasonable basis upon which to estimate the expected term. Expected Volatility. Expected volatility is estimated based on actual movements in the Company’s stock price over the most recent historical periods, over the expected term of their stock option grants.
The Company F- 28 uses the simplified method because it does not have sufficient historical option exercise data to provide a reasonable basis upon which to estimate the expected term. Expected Volatility. Expected volatility is estimated based on actual movements in the Company’s stock price over the most recent historical periods, over the expected term of their stock option grants.
As of December 31, 2024 and 2023, cash equivalents consisted of interest-bearing money market accounts, U.S. Treasury bills and other government securities. Restricted Cash Equivalents The Company has restricted cash equivalents made up of money market funds held in collateral accounts that are restricted to secure letters of credit in accordance with certain of its leases.
As of December 31, 2025 and 2024, cash equivalents consisted of interest-bearing money market accounts, U.S. Treasury bills and other government securities. Restricted Cash Equivalents The Company has restricted cash equivalents made up of money market funds held in collateral accounts that are restricted to secure letters of credit in accordance with certain of its leases.
Lease expense for operating lease payments is recognized on a straight-line basis over the lease term. F- 9 The Company is required to pay fees for operating expenses in addition to monthly base rent for certain operating leases. The Company has elected the practical expedient which allows non-lease components to be combined with lease components for all asset classes.
Lease expense for operating lease payments is recognized on a straight-line basis over the lease term. The Company is required to pay fees for operating expenses in addition to monthly base rent for certain operating leases. The Company has elected the practical expedient which allows non-lease components to be combined with lease components for all asset classes.
Lease payments (including payments pertaining to lessor-owned leasehold improvements) made to the lessor prior to lease commencement are recorded as prepaid rent and included in “Prepaid expenses and other current assets” on the Company's consolidated balance sheets. The prepaid rent balance is reclassified to the right-of-use asset at lease commencement.
Lease payments (including F- 9 payments pertaining to lessor-owned leasehold improvements) made to the lessor prior to lease commencement are recorded as prepaid rent and included in “Prepaid expenses and other current assets” on the Company's consolidated balance sheets. The prepaid rent balance is reclassified to the right-of-use asset at lease commencement.
The Company expects that its cash, cash equivalents and marketable securities as of December 31, 2024 will enable the Company to fund its ongoing operating expenses and capital expenditure requirements for at least the twelve-month period following the issuance of these consolidated financial statements. 2.
The Company expects that its cash, cash equivalents and marketable securities as of December 31, 2025 will enable the Company to fund its ongoing operating expenses and capital expenditure requirements for at least the twelve-month period following the issuance of these consolidated financial statements. 2.
The analysis did not result in a material limitation to the Company’s tax attributes and the results of this analysis are reflected herein. The Company has not completed an analysis through December 31, 2024. To the extent there was a change in control during 2023 and 2024, the Company's tax attributes could be subject to limitation.
The analysis did not result in a material limitation to the Company’s tax attributes and the results of this analysis are reflected herein. The Company has not completed an analysis through December 31, 2025. To the extent there was a change in control during 2023 through 2025, the Company's tax attributes could be subject to limitation.
Throughout the term of its leases, the Company is responsible for paying certain costs and expenses, in addition to the rent, as specified in the lease, including a proportionate share of applicable taxes, operating expenses and utilities. The variable portion of these costs are expensed as incurred and are disclosed as variable lease costs.
F- 24 Throughout the term of its leases, the Company is responsible for paying certain costs and expenses, in addition to the rent, as specified in the lease, including a proportionate share of applicable taxes, operating expenses and utilities. The variable portion of these costs are expensed as incurred and are disclosed as variable lease costs.
F- 34 SIGNATURES Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized. INTELLIA THERAPEUTICS, INC. By: /s/ John M. Leonard John M. Leonard, M.D.
SIGNATURES Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized. INTELLIA THERAPEUTICS, INC. By: /s/ John M. Leonard John M. Leonard, M.D.
To achieve this core principle, the Company applies the following five steps: (i) identify the contract with the customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) F- 10 allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when or as the Company satisfies a performance obligation.
To achieve this core principle, the Company applies the following five steps: (i) identify the contract with the 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 Company satisfies a performance obligation.
The Company concluded it is more likely than not that its net deferred tax assets would not be realized in the future; therefore, the Company has provided a full valuation allowance against its net deferred tax asset balance as of December 31, 2024 and 2023.
The Company concluded it is more likely than not that its net deferred tax assets would not be realized in the future; therefore, the Company has provided a full valuation allowance against its net deferred tax asset balance as of December 31, 2025 and 2024.
F- 22 SparingVision SAS In October 2021, the Company and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, entered into a license and collaboration agreement (the “SparingVision LCA”) to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.
SparingVision SAS In October 2021, the Company and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, entered into a license and collaboration agreement (the “SparingVision LCA”) to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.
Section 1350, as adopted pursuant to Section 906 of The Sarbanes-Oxley Act of 2002, by John M. Leonard, M.D., President and Chief Executive Officer of the Company, and Edward J. Dulac III, Executive Vice President, Chief Financial Officer of the Company (17) 97.1# Intellia Therapeutics, Inc.
Section 1350, as adopted pursuant to Section 906 of The Sarbanes-Oxley Act of 2002, by John M. Leonard, M.D., President and Chief Executive Officer of the Company, and Edward J. Dulac III, Executive Vice President, Chief Financial Officer of the Company (18) 97.1# Intellia Therapeutics, Inc.
Liquidity Since its inception through December 31, 2024, the Company has funded its operations through its initial public offering (“IPO”) and concurrent private placements, follow-on public offerings, at-the-market offerings and the sale of convertible preferred stock, as well as through its collaboration agreements.
Liquidity Since its inception through December 31, 2025, the Company has funded its operations through its initial public offering (“IPO”) and concurrent private placements, follow-on public offerings, at-the-market offerings and the sale of convertible preferred stock, as well as through its collaboration agreements.
Specifically, BlueAllele alleges that the Company’s experimentation, basic F- 19 research, identification, optimization, manufacturing and/or use of bi-directional insertion template technology infringes the asserted patents and seeks unspecified compensatory damages and an injunction against the alleged infringing activities.
Specifically, BlueAllele alleges that the Company’s experimentation, basic research, identification, optimization, manufacturing and/or use of bi-directional insertion template technology infringes the asserted patents and seeks unspecified compensatory damages and an injunction against the alleged infringing activities.
The Company recognized $ 2.3 million and $ 0.4 million of collaboration revenue in the years ended December 31, 2024 and 2023, respectively, in the consolidated statement of operations and comprehensive loss related to the 2023 Regeneron Amendment. ATTR and Hemophilia Co/Co Agreements: Accounting Analysis.
The Company recognized $ 2.3 million and $ 0.4 million of collaboration revenue in the years ended December 31, 2024 and 2023, respectively, in the consolidated statements of operations and comprehensive loss related to the 2023 Regeneron Amendment. ATTR Co/Co and Hemophilia Co/Co Agreements: Accounting Analysis.
Impairment of Long-Lived Assets The Company tests long-lived assets to be held and used, including property and equipment, for impairment whenever events or changes in circumstances indicate that the carrying amount of assets or asset groups may not be fully recoverable.
Impairment of Long-Lived Assets The Company tests long-lived assets to be held and used, including property and equipment and right-of-use assets, for impairment whenever events or changes in circumstances indicate that the carrying amount of assets or asset groups may not be fully recoverable.
Losses incurred prior to 2018 will generally be deductible to the extent of the lesser of a corporation’s net operating loss carryover or 100 % of a corporation’s taxable income and be available for twenty years from the period the loss F- 18 was generated.
Losses incurred prior to 2018 will generally be deductible to the extent of the lesser of a corporation’s net operating loss carryover or 100 % of a corporation’s taxable income and be available for twenty years from the period the loss was generated.
Investments in non-marketable securities are accounted for using the measurement alternative at cost minus impairment, adjusted for changes in observable prices. Refer to Note 4 for further information regarding the Company’s fair value measurements.
Investments in non-marketable securities are accounted for using the measurement alternative at cost minus impairment, adjusted for changes in observable prices. Refer to Note 4, “Fair Value Measurements” for further information regarding the Company’s fair value measurements.
If the Company has experienced a change of control, utilization of the net operating loss carryforwards or research and development tax credit carryforwards would be subject to an annual limitation under Section 382 and 383 of the Code.
If the Company has experienced a change of control, utilization of the net operating loss carryforwards or research and development tax credit carryforwards would be subject to an annual limitation under Sections 382 and 383 of the Code.
The maximum term of stock options granted under the 2015 Plan and the Inducement Plan is ten years.
The maximum term of stock options granted under the 2015 Plan, the 2025 Plan and the Inducement Plan is ten years.
Emmanuelle Charpentier (12) 10.15# Form of Amended and Restated Employment Agreement (6) 10.16† Letter Agreement, dated as of July 20, 2018, by and between the Company and Regeneron Pharmaceuticals, Inc. and the corresponding Form of Co-Development and Co-Promotion Agreement, by and between the Company and Regeneron Pharmaceuticals, Inc.
Emmanuelle Charpentier (11) 10.15# Form of Amended and Restated Employment Agreement (6) 10.16† Letter Agreement, dated as of July 20, 2018, by and between the Company and Regeneron Pharmaceuticals, Inc. and the corresponding Form of Co-Development and Co-Promotion Agreement, by and between the Company and Regeneron Pharmaceuticals, Inc.
Milestone payments: At the inception of each arrangement that includes development milestone payments, the Company evaluates the probability of reaching the milestones and estimates the amount to be included in the transaction price using the F- 11 most likely amount method.
Milestone payments: At the inception of each arrangement that includes development milestone payments, the Company evaluates the probability of reaching the milestones and estimates the amount to be included in the transaction price using the most likely amount method.
These restricted cash equivalents are long-term in nature and are included in “Investments and other assets” in the Company’s consolidated balance sheets. Marketable Securities The Company’s marketable securities are accounted for as available-for-sale and recorded at fair value with the related unrealized gains and losses included in accumulated other comprehensive (loss) income, a component of stockholders’ equity.
These restricted cash equivalents are long-term in nat ure and are included in “Investments and other assets” in the Company’s consolidated balance sheets. Marketable Securities The Company’s marketable securities are accounted for as available-for-sale and recorded at fair value with the related unrealized gains and losses included in accumulated other comprehensive (loss) income, a component of stockholders’ equity.
The Company is also eligible to earn royalties ranging from the high-single digits to low teens, in each case, on a per-product basis, which royalties are potentially subject to various reductions and offsets and incorporate the Company’s existing low- to mid-single-digit royalty obligations under a license agreement with Caribou.
The Company is also eligible to earn royalties ranging from the high-single digits to low teens, in each case, on a per-product basis, which royalties are potentially subject to various reductions and offsets and incorporate the Company’s existing low- to mid-single-digit royalty obligations under its in-license agreement with Caribou Biosciences, Inc.
To the extent the transaction price includes variable consideration, the Company estimates the amount of variable consideration that should be included in the transaction price utilizing either the expected value method or the most likely amount method, depending on the nature of the variable consideration.
To the extent the transaction price includes variable consideration, the Company estimates the amount of variable consideration that should be included in the transaction price utilizing either the expected value method F- 10 or the most likely amount method, depending on the nature of the variable consideration.
F- 12 For awards with service conditions only, the Company recognizes stock-based compensation expense on a straight-line basis over the requisite service period. For awards with performance or market-based conditions, the Company recognizes stock-based compensation expense using the accelerated attribution method over the requisite service period.
For awards with service conditions only, the Company recognizes stock-based compensation expense on a straight-line basis over the requisite service period. For awards with performance or market-based conditions, the Company recognizes stock-based compensation expense using the accelerated attribution method over the requisite service period.
(7) 10.17 First Amendment to Lease, dated as of April 5, 2019, by and between the Company and MIT 130 Brookline Leasehold LLC (8) 10.18# Seventh Amended and Restated Non-Employee Director Compensation Policy (14) 10.19 Lease, dated as of March 12, 2020, by and between the Company and 281-295 Albany Street Leasehold LLC (1) 10.20 Second Amendment to Lease, dated as of March 12, 2020, by and between the Company and MIT 130 Brookline Leasehold LLC (1) F- 32 10.21† Amendment No. 1, dated May 30, 2020, to the License and Collaboration Agreement, dated April 11, 2016, by and between the Company and Regeneron Pharmaceuticals, Inc.
(7) 10.17 First Amendment to Lease, dated as of April 5, 2019, by and between the Company and MIT 130 Brookline Leasehold LLC (8) 10.18# Seventh Amended and Restated Non-Employee Director Compensation Policy (13) 10.19 Lease, dated as of March 12, 2020, by and between the Company and 281-295 Albany Street Leasehold LLC (1) 10.20 Second Amendment to Lease, dated as of March 12, 2020, by and between the Company and MIT 130 Brookline Leasehold LLC (1) 10.21† Amendment No. 1, dated May 30, 2020, to the License and Collaboration Agreement, dated April 11, 2016, by and between the Company and Regeneron Pharmaceuticals, Inc.
(1) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on May 7, 2020 F- 33 (2) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on May 5, 2016 (3) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on April 27, 2016 (4) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on April 19, 2016 (5) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on April 11, 2016 (6) Incorporated by reference to the Registrant’s Current Report on Form 8-K (File No. 001-37766) filed with the Securities and Exchange Commission on April 17, 2018 (7) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on October 31, 2018 (8) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on May 2, 2019 (9) Incorporated by reference to the Registrant’s Annual Report on Form 10-K (File No. 001-37766) filed with the Securities and Exchange Commission on February 23, 2023 (10) Incorporated by reference to the Registrant’s Current Report on Form 8-K (File No. 001-37766) filed with the Securities and Exchange Commission on June 1, 2020 (11) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on August 3, 2023 (12) Incorporated by reference to the Registrant’s Annual Report on Form 10-K (File No. 001-37766) filed with the Securities and Exchange Commission on February 24, 2022 (13) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on November 9, 2023 (14) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on August 8, 2024 (15) Incorporated by reference to the Registrant’s Current Report on Form 8-K (File No. 001-37766) filed with the Securities and Exchange Commission on June 26, 2024 (16) Incorporated by reference to the Registrant’s Annual Report on Form 10-K (File No. 001-37766) filed with the Securities and Exchange Commission on February 22, 2024 (17) The certifications furnished in Exhibit 32.1 hereto are deemed to accompany this Annual Report on Form 10-K and will not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended.
(1) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on May 7, 2020 (2) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on May 5, 2016 (3) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on April 27, 2016 (4) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on April 19, 2016 (5) Incorporated by reference to the Registration Statement on Form S-1 (File No. 333-210689) filed with the Securities and Exchange Commission on April 11, 2016 (6) Incorporated by reference to the Registrant’s Current Report on Form 8-K (File No. 001-37766) filed with the Securities and Exchange Commission on April 17, 2018 (7) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on October 31, 2018 (8) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on May 2, 2019 (9) Incorporated by reference to the Registrant’s Current Report on Form 8-K (File No. 001-37766) filed with the Securities and Exchange Commission on June 1, 2020 (10) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on August 3, 2023 (11) Incorporated by reference to the Registrant’s Annual Report on Form 10-K (File No. 001-37766) filed with the Securities and Exchange Commission on February 24, 2022 (12) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on November 9, 2023 (13) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on August 8, 2024 (14) Incorporated by reference to the Registrant’s Annual Report on Form 10-K (File No. 001-37766) filed with the Securities and Exchange Commission on February 27, 2025 (15) Incorporated by reference to the Registrant’s Current Report on Form 8-K (File No. 001-37766) filed with the Securities and Exchange Commission on April 7, 2025 (16) Incorporated by reference to Registration Statement on Form S-8 (File No. 333-287959) filed with the Securities and Exchange Commission on June 11, 2025 (17) Incorporated by reference to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37766) filed with the Securities and Exchange Commission on August 7, 2025 (18) The certifications furnished in Exhibit 32.1 hereto are deemed to accompany this Annual Report on Form 10-K and will not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended.
Determining the transaction price requires significant judgment, which is discussed in further detail for each of the Company’s collaboration agreements in Note 9. In addition, none of the Company’s contracts as of December 31, 2024 or 2023 contained a significant financing component.
Determining the transaction price requires significant judgment, which is discussed in further detail for each of the Company’s collaboration agreements in Note 9, “Collaborations”. In addition, none of the Company’s contracts as of December 31, 2025 or 2024 contained a significant financing component.
As of December 31, 2024, the Company’s accounts receivable were related to its collaborations with Regeneron, AvenCell, SparingVision and ReCode, and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision.
As of December 31, 2025, the Company’s accounts receivable and contract liabilities were related to its collaboration with Regeneron. As of December 31, 2024, the Company’s accounts receivable were related to its collaborations with Regeneron, AvenCell, SparingVision and ReCode and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision.
Net Loss per Share The Company calculates basic net loss per share by dividing net loss for each respective period by the weighted average number of common shares outstanding for each respective period.
F- 12 Net Loss per Share The Company calculates basic net loss per share by dividing net loss for each respective period by the weighted average number of common shares outstanding for each respective period.
Weighted average assumptions used to apply this pricing model were as follows: Year Ended December 31, 2024 2023 2022 Risk-free interest rate 4.2 % 4.4 % 1.9 % Expected term of options 6.0 years 6.0 years 5.9 years Expected volatility of underlying stock 76.3 % 78.7 % 76.2 % Expected dividend yield 0.0 % 0.0 % 0.0 % Risk-free Interest Rate.
Weighted average assumptions used to apply this pricing model were as follows: Year Ended December 31, 2025 2024 2023 Risk-free interest rate 4.1 % 4.2 % 4.4 % Expected term of options 6.0 years 6.0 years 6.0 years Expected volatility of underlying stock 76.0 % 76.3 % 78.7 % Expected dividend yield 0.0 % 0.0 % 0.0 % Risk-free Interest Rate.
The Company is subject to examination by the Internal Revenue Service, Massachusetts taxing authorities and state taxing authorities for tax year 2021 through present.
The Company is subject to examination by the Internal Revenue Service, Massachusetts taxing authorities and state taxing authorities for tax year 2022 through present.
President and Chief Executive Officer Dated: February 27, 2025 Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed by the following persons on behalf of the registrant in the capacities and on the dates indicated. Name Title Date /s/ John M. Leonard President, Chief Executive Officer and Director February 27, 2025 John M.
President and Chief Executive Officer Dated: February 26, 2026 Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed by the following persons on behalf of the registrant in the capacities and on the dates indicated. Name Title Date /s/ John M. Leonard President, Chief Executive Officer and Director February 26, 2026 John M.
The Company will recognize interest and/or penalties related to uncertain tax benefits in income tax expense if they arise. The Company files income tax returns in the U.S. federal tax jurisdiction and Massachusetts and various other state tax jurisdictions.
The Company will recognize interest and/or penalties related to uncertain tax benefits in income tax e xpense if they arise. The Company files income tax returns in the U.S. federal tax jurisdiction and Massachusetts and various other state tax jurisdictions.
The accounting policies for the segment are the same as described those described in Note 2, “Summary of Significant Accounting Policies.” The CODM evaluates the performance of the operating segment and allocates resources based on net loss that also is reported on the consolidated statements of operations and comprehensive loss.
F- 30 16. Segment Information The accounting policies for the segment are the same as those described in Note 2, “Summary of Significant Accounting Policies.” The CODM evaluates the performance of the operating segment and allocates resources based on net loss that also is reported on the consolidated statements of operations and comprehensive loss.
In October 2023, Regeneron notified the Company that it was exercising its one-time option to extend the Technology Collaboration Term for an additional two years (the “2024 Technology Collaboration Extension”) , until April 2026 , in exchange for a nonrefundable payment of $ 30.0 million that was paid in April 2024. 2024 Technology Collaboration Extension: Accounting Analysis.
F- 21 In October 2023, Regeneron notified the Company that it was exercising its one-time option to extend the Technology Collaboration Term for an additional two years (the “2024 Technology Collaboration Extension”) , until April 2026 , in exchange for a nonrefundable payment of $ 30.0 million that was paid in April 2024.
(10) 10.23# Second Amended and Restated Corporate Bonus Plan, effective November 30, 2023 (16) 10.24† Agreement and Plan of Merger, by and among Intellia Therapeutics, Inc., Rewrite Therapeutics, Inc., RW Acquisition Corp., and Shareholder Representative Services, LLC, as securityholder representative, dated as of February 2, 2022 (12) 10.25 Lease Agreement by and between the Registrant and Are-Winter Street Property, LLC, dated as of February 22, 2022 (12) 10.26# Amended and Restated Retirement Policy for Equity Awards, effective December 6, 2022 (9) 10.27 Amendment to Lease Agreement by and between the Registrant and Are-Winter Street Property, LLC, dated as of June 20, 2023 (11) 10.28 Letter Agreement (Second Amendment) to License and Collaboration Agreement by and between Registrant and Regeneron Pharmaceuticals, Inc., dated November 22, 2022 (13) 10.29 Third Amendment to License and Collaboration Agreement by and between Registrant and Regeneron Pharmaceuticals, Inc., dated September 29, 2023 (13) 10.30# Intellia Therapeutics, Inc. 2024 Inducement Plan and forms of award agreements thereunder (15) 10.31†* Lease Agreement by and between the Registrant and ARE-Tech Square, LLC, dated as of February 18, 2025 10.32†* Second Amendment to Lease Agreement by and between the Registrant and ARE-Winter Street Property, LLC, dated as of February 18, 2025 10.33# Employment Agreement between Intellia Therapeutics, Inc. and Edward Dulac (15) 19.1†* Fourth Amended and Restated Insider Trading Policy 21.1* Subsidiaries of the Registrant 23.1* Consent of Deloitte & Touche LLP, Independent Registered Public Accounting Firm 31.1* Certification of Chief Executive Officer pursuant to Rules 13a-14(a) or 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 31.2* Certification of Chief Financial Officer pursuant to Rules 13a-14(a) or 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 32.1 Certifications pursuant to 18 U.S.C.
(9) 10.22# Third Amended and Restated Corporate Bonus Plan, effective June 25, 2025 (17) 10.23† Agreement and Plan of Merger, by and among Intellia Therapeutics, Inc., Rewrite Therapeutics, Inc., RW Acquisition Corp., and Shareholder Representative Services, LLC, as securityholder representative, dated as of February 2, 2022 (11) 10.24 Lease Agreement by and between the Registrant and Are-Winter Street Property, LLC, dated as of February 22, 2022 (11) 10.25#* Second Amended and Restated Retirement Policy for Equity Awards, effective December 27, 2025 10.26 Amendment to Lease Agreement by and between the Registrant and Are-Winter Street Property, LLC, dated as of June 20, 2023 (10) 10.27 Letter Agreement (Second Amendment) to License and Collaboration Agreement by and between Registrant and Regeneron Pharmaceuticals, Inc., dated November 22, 2022 (12) 10.28 Third Amendment to License and Collaboration Agreement by and between Registrant and Regeneron Pharmaceuticals, Inc., dated September 29, 2023 (12) 10.29#* Amendment No. 1 to the Intellia Therapeutics, Inc. 2024 Inducement Plan, effective as of December 4, 2025 10.30† Lease Agreement by and between the Registrant and ARE-Tech Square, LLC, dated as of February 18, 2025 (14) 10.31† Second Amendment to Lease Agreement by and between the Registrant and ARE-Winter Street Property, LLC, dated as of February 18, 2025 (14) 10.32 Amendment to Lease Agreement by and between the Registrant and ARE-Tech Square, LLC, dated as of July 2, 2025 (17) 19.1† Fourth Amended and Restated Insider Trading Policy (14) 21.1* Subsidiaries of the Registrant 23.1* Consent of Deloitte & Touche LLP, Independent Registered Public Accounting Firm 31.1* Certification of Chief Executive Officer pursuant to Rules 13a-14(a) or 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 31.2* Certification of Chief Financial Officer pursuant to Rules 13a-14(a) or 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 32.1 Certifications pursuant to 18 U.S.C.
During the year ended December 31, 2024 , the Company issued 7,004,370 shares of its common stock, in a series of sales, at an average price of $ 25.68 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $ 174.8 million , after payment of cash commissions and approximately $ 0.3 million related to legal, accounting and other fees in connection with the sales.
During the year ended December 31, 2024, the Company issued 7,004,370 shares of its common stock, in a series of sales, at an average price of $ 25.68 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $ 174.8 million, after commissions and legal, accounting and other fees in connection with the sales.
As of December 31, 2024 and 2023, these restricted cash equivalents amounted to $ 13.6 million . The letters of credit are required to be maintained throughout the term of the leases; in some cases, the Company is able to reduce the amounts held over time.
As of December 31, 2025 and 2024, these restricted cash equivalents amounted to $ 12.1 million and $ 13.6 million, respectively. The letters of credit are required to be maintained throughout the term of the leases; in some cases, the Company is able to reduce the amounts held over time.
The Company recognized $ 20.7 million, $ 11.7 million and $ 22.5 million of collaboration revenue in the years ended December 31, 2024, 2023 and 2022, respectively, in the consolidated statements of operations and comprehensive loss related to the 2016 Regeneron Agreement, the 2020 Regeneron Amendment and the 2024 Technology Collaboration Extension.
The Company recognized $ 20.8 million, $ 20.7 million and $ 11.7 million of collaboration revenue in the years ended December 31, 2025, 2024 and 2023, respectively, in the consolidated statements of operations and comprehensive loss related to the 2016 Regeneron Agreement, the 2020 Regeneron Amendment and the 2024 Technology Collaboration Extension.
Refer to Note 3 for further information regarding the Company’s marketable securities.
Refer to Note 3, “Marketable Securities”, for further information regarding the Company’s marketable securities.
Stock Options The weighted average grant date fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $ 21.21 , $ 28.92 and $ 57.23 per option for those options granted during the year ended December 31, 2024 , 2023 and 2022, respectively.
Stock Options The weighted average grant date fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $ 6.80 , $ 21.21 and $ 28.92 per option for those options granted during the year ended December 31, 2025 , 2024 and 2023, respectively.
Dube (Principal Accounting Officer) /s/ Muna Bhanji Director February 27, 2025 Muna Bhanji /s/ Bill Chase Director February 27, 2025 Bill Chase /s/ Fred Cohen Director February 27, 2025 Fred Cohen, M.D. /s/ Brian Goff Director February 27, 2025 Brian Goff /s/ Jesse Goodman Director February 27, 2025 Jesse Goodman, M.D. /s/ Georgia Keresty Director February 27, 2025 Georgia Keresty /s/ Frank Verwiel Director February 27, 2025 Frank Verwiel, M.D.
Dube (Principal Accounting Officer) /s/ Muna Bhanji Director February 26, 2026 Muna Bhanji /s/ Bill Chase Director February 26, 2026 Bill Chase /s/ Fred Cohen Director February 26, 2026 Fred Cohen, M.D. /s/ Brian Goff Director February 26, 2026 Brian Goff /s/ Jesse Goodman Director February 26, 2026 Jesse Goodman, M.D. /s/ Georgia Keresty Director February 26, 2026 Georgia Keresty /s/ Frank Verwiel Director February 26, 2026 Frank Verwiel, M.D.
In general, an ownership change, as defined by Sections 382 and 383 of the Internal Revenue Code of 1986, as amended (the “Code”), results from transactions increasing the ownership of certain shareholders or public groups in the stock of a corporation by more than 50% over a three-year period.
In general, an ownership change, as defined by Sections 382 and 383 of the Code, results from transactions increasing the ownership of certain shareholders or public groups in the stock of a corporation by more than 50% (by value) over a three-year period.
Unvested restricted stock units as of December 31, 2024 in the table above includes 468,277 RSUs that are market-based. Restricted Stock Units - Performance-Based Awards with TSR Multiplier Also in 2024, performance-based RSUs (“PSUs”) with a relative TSR modifier were granted to senior executives.
Unvested restricted stock units as of December 31, 2025 in the table above includes 596,138 RSUs that are market-based. Restricted Stock Units - Performance-Based Awards with TSR Multiplier In 2024, performance-based RSUs (“PSUs”) with a relative TSR modifier were granted to senior executives .
At December 31, 2024 and 2023, the Company did no t hold any marketable securities that matured beyond five years of the balance sheet date. Accrued interest on marketable securities is included in “Prepaid expenses and other current assets” on the Company's consolidated balance sheets. F- 15 4.
At December 31, 2025 and 2024, the Company did no t hold any marketable securities that matured beyond five years of the balance sheet date. Accrued interest on marketable securities is included in “Prepaid expenses and other current assets” on the Company’ s consolidated balance sheets. F- 14 4.
The following assumptions were used to determine the grant date fair value for the three years, respectively: risk free interest rate: 4.28 %, 4.60 % and 1.44 %; expected volatility: 77.2 %, 84.34 % and 82.53 %. The expected term for all grants was approximately 3.0 years; the expected dividend yield was 0.0 %.
The following assumptions were used to determine the grant date fair value for the three years, respectively: risk free interest rate: 3.99 %, 4.28 % and 4.60 %; expected volatility: 67.6 %, 77.2 % and 84.34 %. The expected term for all grants was approximately 3.0 years; the expected dividend yield was 0.0 %.
The Company’s financial assets recognized at fair value on a recurring basis consisted of the following: December 31, 2024 Total Level 1 Level 2 Level 3 (In thousands) Assets Cash equivalents and restricted cash equivalents $ 66,898 $ 66,898 $ - $ - Marketable securities: U.S.
The Company’s financial assets recognized at fair value on a recurring basis consisted of the following: December 31, 2025 Total Level 1 Level 2 Level 3 (In thousands) Assets Cash equivalents and restricted cash equivalents $ 92,263 $ 92,263 $ - $ - Marketable securities: U.S.
RSUs with a service condition granted to new and existing employees, and to non-employee directors upon appointment, under the 2015 Plan in 2024 and 2023 and the Inducement Plan in 2024 generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting annually over the remaining two years.
RSUs with a service condition granted to new and existing employees, and to non-employee directors upon appointment, generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting annually over the remaining two years.
In addition, the Tech Square Lease will expand, in two tranches, to include approximately 46,000 square feet of additional office and laboratory space at 400 Tech Square (the “Additional Tech Square Premises”) when each such space becomes available.
In addition, the Tech Square Lease would expand, in two tranches, to include approximately 46,000 square feet of additional office and laboratory space at 400 Tech Square representing the 5th and 7th floors (the “Additional Tech Square Premises”), when each such space becomes available.
Leonard, M.D. (Principal Executive Officer) /s/ Edward J. Dulac III Executive Vice President, Chief Financial Officer February 27, 2025 Edward J. Dulac III (Principal Financial Officer) /s/ Michael P. Dube Vice President, Chief Accounting Officer February 27, 2025 Michael P.
Leonard, M.D. (Principal Executive Officer) /s/ Edward J. Dulac III Executive Vice President, Chief Financial Officer February 26, 2026 Edward J. Dulac III (Principal Financial Officer) /s/ Michael P. Dube Vice President, Chief Accounting Officer February 26, 2026 Michael P.
The valuation allowance increased by $ 167.1 million in 2024, $ 161.3 million in 2023, and $ 150.0 million in 2022. Ownership changes may limit the amount of net operating loss carryforwards or research and development tax credit carryforwards that can be utilized to offset future taxable income or tax liability.
The valuation allowance increased by $ 131.4 m illion in 2025, $ 167.1 million in 2024, and $ 161.3 million in 2023. Ownership changes may limit the amount of net operating loss carryforwards or research and development tax credit carryforwards that can be utilized to offset future taxable income or tax liability.
Also, there will be no carryback for losses incurred after 2017. As of December 31, 2024 and 2023, the Company also had state net operating loss carryforwards of $ 1,079.2 million and $ 922.8 million, respectively, which may be available to offset future income tax liabilities and begin to expire in 2034 .
Also, there will be no carryback for losses incurred after 2017. As of December 31, 2025 and 2024, the Company also had state net operating loss carryforwards of $ 1,678.0 million and $ 1,079.2 million, respectively, which may be available to offset future income tax liabilities and begin to expire in 203 4 .
The fair value of shares under the 2016 Plan was estimated at the beginning of the offering period using a Black-Scholes option-pricing model with the following assumptions: Year Ended December 31, 2024 2023 2022 Risk-free interest rate 5.24 %- 5.37 % 4.7 %- 5.53 % 0.22 %- 2.52 % Expected term (in years) 0.5 years 0.5 years 0.5 years Expected volatility of underlying stock 56.7 %- 59.0 % 60.4 %- 69.2 % 63.6 %- 95.3 % Expected dividend yield 0.0 % 0.0 % 0.0 % 13.
The fair value of shares under the 2016 Plan was estimated at the beginning of the offering period using a Black-Scholes option-pricing model with the following assumptions: Year Ended December 31, 2025 2024 2023 Risk-free interest rate 4.24 %- 4.29 % 5.24 %- 5.37 % 4.7 %- 5.53 % Expected term (in years) 0.5 years 0.5 years 0.5 years Expected volatility of underlying stock 66.2 %- 92.16 % 56.7 %- 59.0 % 60.4 %- 69.2 % Expected dividend yield 0.0 % 0.0 % 0.0 % F- 29 13.
Any limitation may result in expiration of a portion of the net operating loss carryforwards or research and development tax credit carryforwards before utilization. During 2022, the Company completed an assessment of the available net operating loss carryforwards and other tax attributes under Section 382 that covered the period from inception through December 31, 2022.
Any limitation may result in expiration of a portion of the net operating loss carryforwards or research and development tax credit carryforwards before utilization. During 2022, the Company completed an assessment of the available net operating loss carryforwards and other tax attributes under Section 382.
The number of market-based RSUs granted in the year ended December 31, 2024 , 2023 and 2022 was 286,084 , 181,743 and 55,144 , respectively. The grant date fair value for the market-based RSUs, calculated using a Monte Carlo valuation model, was $ 51.12 , $ 68.55 and $ 126.49 , respectively.
The number of market-based RSUs granted in the year ended December 31, 2025 , 2024 and 2023 was 223,600 , 286,084 and 181,743 , respectively. The grant date fair value for the market-based RSUs, calculated using a Monte Carlo valuation model, was $ 15.76 , $ 51.12 and $ 68.55 , respectively.
Stock-Based Compensation Stock-based compensation expense is classified in the consolidated statements of operations and comprehensive loss as follows: Year Ended December 31, 2024 2023 2022 (In thousands) Research and development $ 94,230 $ 82,211 $ 56,279 General and administrative 60,043 51,839 35,121 Total $ 154,273 $ 134,050 $ 91,400 Stock Option and Incentive Plans In April 2016, the Company adopted the Amended and Restated 2015 Stock Option and Incentive Plan (the “2015 Plan”).
Stock-Based Compensation Stock-based compensation expense is classified in the consolidated statements of operations and comprehensive loss as follows: Year Ended December 31, 2025 2024 2023 (In thousands) Research and development $ 49,440 $ 94,230 $ 82,211 General and administrative 30,779 60,043 51,839 Total $ 80,219 $ 154,273 $ 134,050 Stock Option and Incentive Plans In April 2016, the Company adopted the Amended and Restated 2015 Stock Option and Incentive Plan (the “2015 Plan”).
Exhibit Index 3.1* Second Amended and Restated Certificate of Incorporation of the Registrant, as amended 3.2 Second Amended and Restated By-laws of the Registrant (1) 4.1* Description of Certain Registrant’s Securities 10.1# 2015 Amended and Restated Stock Option and Incentive Plan and forms of award agreements thereunder (3) 10.2# Senior Executive Cash Incentive Bonus Plan (5) 10.3† License Agreement dated as of July 16, 2014 by and between the Registrant (as successor in interest of Intellia Therapeutics, LLC) and Caribou Biosciences, Inc.
Exhibit Index 3.1* Second Amended and Restated Certificate of Incorporation of the Registrant, as amended 3.2 Third Amended and Restated By-laws of the Registrant (15) 4.1 Description of Certain Registrant’s Securities (14) 10.1# 2025 Equity Incentive Plan (16) 10.2# Senior Executive Cash Incentive Bonus Plan (5) 10.3† License Agreement dated as of July 16, 2014 by and between the Registrant (as successor in interest of Intellia Therapeutics, LLC) and Caribou Biosciences, Inc.
The Company will use a costs-incurred input method to recognize revenue, measuring the progress of the programs based on the costs incurred against budget, which in management's judgment is the best measure of progress towards satisfying the performance obligation.
The Company used a costs-incurred input method to recognize revenue, measuring the progress of the programs based on the costs incurred against budget, which in management's judgment is the best measure of progress towards satisfying the performance obligation. In October 2025, the Company terminated the SparingVision LCA.
The total fair value of RSUs vested (measured on the date of vesting) for the year ended December 31, 2024, 2023 and 2022 was $ 40.0 million , $ 24.9 million and $ 10.4 million, respectively. As of December 31, 2024, there was $ 106.5 million of unrecognized stock-based compensation expense related to all RSUs that are expected to vest.
The total fair value of RSUs vested (measured on the date of vesting) for the years ended December 31, 2025, 2024 and 2023 was $ 22.9 million , $ 40.0 million and $ 24.9 million, respectively. As of December 31, 2025, there was $ 42.7 million of unrecognized stock-based compensation expense related to all RSUs that are expected to vest.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeGenome editing companies focused on CRISPR-based technologies include: Arbor Biotechnologies, Inc., Beam Therapeutics Inc., Caribou Biosciences, Inc., CRISPR Therapeutics AG, EdiGene, Inc., Editas Medicine, Inc., Emendo Biotherapeutics, Inc., Ensoma, Inc., Excision Biotherapeutics, Inc., Integra Therapeutics, S.L., Mammoth Biosciences, Inc., Metagenomi Technologies, LLC, Modalis Therapeutics Inc., nChroma Bio (formerly Chroma Medicine, Inc.), Prime Medicine, Inc., Scribe Therapeutics, Inc., Tessera Therapeutics, Inc., ToolGen, Inc., Tune Therapeutics, Inc., Verve Therapeutics, Inc. and YolTech Therapeutics.
Biggest changeCompanies focused on developing therapies using gene editing technologies include: AccurEdit Therapeutics, Allogene Therapeutics, Inc., Arbor Biotechnologies, Inc., Basecamp Research, Ltd., Beam Therapeutics Inc., Bendao Gene Technology Co., Ltd., Caribou Biosciences, Inc., Cellectis S.A., CorrectSequence Therapeutics Co., Ltd., Create Medicines (formerly Myeloid Therapeutics, Inc.), CRISPR Therapeutics AG, EdiGene, Inc., Editas Medicine, Inc., Emendo Biotherapeutics, Inc., Ensoma, Inc., Epicrispr Biotechnologies, Inc., Epigenic Therapeutics, Evox Therapeutics, Ltd., Excision Biotherapeutics, Inc., Integra Therapeutics, S.L., Life Edit Therapeutics (an ElevateBio Company), Mammoth Biosciences, Inc., Metagenomi Therapeutics, Inc., Modalis Therapeutics Inc., nChroma Bio (formerly Chroma Medicine, Inc.), Poseida Therapeutics, Inc.
If we are unable to complete the required studies satisfactorily, the FDA or other regulatory bodies could require that we exclude certain patient populations from clinical studies, place our clinical studies on hold, or require us to cease further clinical studies or deny approval of such product candidates.
If we are unable to complete the required studies satisfactorily, the FDA or other regulatory bodies could require that we exclude certain patient populations from clinical studies, place our other clinical studies on hold, or require us to cease further clinical studies or deny approval of such product candidates.
Among other things, the certificate of incorporation and by-laws: • permit the board of directors to issue up to 5,000,000 shares of preferred stock, with any rights, preferences and privileges as they may designate; • provide that the authorized number of directors may be changed only by resolution of the board of directors; • provide that all vacancies, including newly created directorships, may, except as otherwise required by law, be filled by the affirmative vote of a majority of directors then in office, even if less than a quorum; • divide the board of directors into three classes; • provide that a director may only be removed from the board of directors by the stockholders for cause; • require that any action to be taken by our stockholders must be effected at a duly called annual or special meeting of stockholders, and may not be taken by written consent; • provide that stockholders seeking to present proposals before a meeting of stockholders or to nominate candidates for election as directors at a meeting of stockholders must provide notice in writing in a timely manner, and meet specific requirements as to the form and content of a stockholder’s notice; • prevent cumulative voting rights (therefore allowing the holders of a plurality of the shares of common stock entitled to vote in any election of directors to elect all of the directors standing for election, if they should so choose); 73 • require that, to the fullest extent permitted by law, a stockholder reimburse us for all fees, costs and expenses incurred by us in connection with a proceeding initiated by such stockholder in which such stockholder does not obtain a judgment on the merits that substantially achieves the full remedy sought; • provide that special meetings of our stockholders may be called only by the chairman of the board, our chief executive officer (or president, in the absence of a chief executive officer) or by the board of directors; and • provide that stockholders will be permitted to amend the bylaws only upon receiving at least two-thirds of the total votes entitled to be cast by holders of all outstanding shares then entitled to vote generally in the election of directors, voting together as a single class.
Among other things, the certificate of incorporation and by-laws: • permit the board of directors to issue up to 5,000,000 shares of preferred stock, with any rights, preferences and privileges as they may designate; • provide that the authorized number of directors may be changed only by resolution of the board of directors; • provide that all vacancies, including newly created directorships, may, except as otherwise required by law, be filled by the affirmative vote of a majority of directors then in office, even if less than a quorum; • divide the board of directors into three classes; • provide that a director may only be removed from the board of directors by the stockholders for cause; • require that any action to be taken by our stockholders must be effected at a duly called annual or special meeting of stockholders, and may not be taken by written consent; • provide that stockholders seeking to present proposals before a meeting of stockholders or to nominate candidates for election as directors at a meeting of stockholders must provide notice in writing in a timely manner, and meet specific requirements as to the form and content of a stockholder’s notice; • prevent cumulative voting rights (therefore allowing the holders of a plurality of the shares of common stock entitled to vote in any election of directors to elect all of the directors standing for election, if they should so choose); • require that, to the fullest extent permitted by law, a stockholder reimburse us for all fees, costs and expenses incurred by us in connection with a proceeding initiated by such stockholder in which such stockholder does not obtain a judgment on the merits that substantially achieves the full remedy sought; • provide that special meetings of our stockholders may be called only by the chairman of the board, our chief executive officer (or president, in the absence of a chief executive officer) or by the board of directors; and 73 • provide that stockholders will be permitted to amend the bylaws only upon receiving at least two-thirds of the total votes entitled to be cast by holders of all outstanding shares then entitled to vote generally in the election of directors, voting together as a single class.
Our existing and future therapeutic collaborations may have a number of risks, including that collaborators: • have significant discretion in determining the efforts and resources that they will apply; • may not perform their obligations as expected; • may dispute the amounts of payments owed; • may not pursue development and commercialization of any product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs or license arrangements based on clinical trial results, changes in their strategic focus or available funding, or external factors, such as a strategic transaction that may divert resources or create competing priorities; • may delay, insufficiently fund, stop, initiate new or repeat clinical trials, reformulate a product candidate for clinical testing, or abandon a product candidate; • could develop independently, or with third parties, products that compete directly or indirectly with our products and product candidates; • may view product candidates discovered in our collaborations as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the development or commercialization of our product candidates; • may dispute ownership or rights in jointly developed technologies or intellectual property; • may fail to comply with applicable legal and regulatory requirements regarding the development, manufacture, sale, distribution or marketing of a product candidate or product; • with sales, marketing, manufacturing and distribution rights to our product candidates may not commit sufficient resources to the product’s sale, marketing, manufacturing and distribution; • may disagree with us about material issues, including proprietary rights, contract interpretation, payment obligations or the preferred course of discovery, development, sales or marketing, which might cause delays or terminations of the research, development or commercialization of product candidates, lead to additional and burdensome responsibilities for us with respect to product candidates, or result in litigation or arbitration, any of which would be time-consuming and expensive; • may not properly maintain or defend their or our relevant intellectual property rights or may use our proprietary information or sublicensed intellectual property rights in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation and liability; • may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; 60 • could become involved in a business combination or cessation that could cause them to deemphasize or terminate the development or commercialization of any product candidate licensed to it by us; and • may terminate our collaborations, which could require us to raise additional capital to develop or commercialize the applicable product candidates, or lose access to the collaborator’s intellectual property.
Our existing and future therapeutic collaborations may have a number of risks, including that collaborators: • have significant discretion in determining the efforts and resources that they will apply; • may not perform their obligations as expected; • may dispute the amounts of payments owed; • may not pursue development and commercialization of any product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs or license arrangements based on clinical trial results, changes in their strategic focus or available funding, or external factors, such as a strategic transaction that may divert resources or create competing priorities; • may delay, insufficiently fund, stop, initiate new or repeat clinical trials, reformulate a product candidate for clinical testing, or abandon a product candidate; • could develop independently, or with third parties, products that compete directly or indirectly with our products and product candidates; • may view product candidates discovered in our collaborations as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the development or commercialization of our product candidates; • may dispute ownership or rights in jointly developed technologies or intellectual property; • may fail to comply with applicable legal and regulatory requirements regarding the development, manufacture, sale, distribution or marketing of a product candidate or product; • with sales, marketing, manufacturing and distribution rights to our product candidates may not commit sufficient resources to the product’s sale, marketing, manufacturing and distribution; • may disagree with us about material issues, including proprietary rights, contract interpretation, payment obligations or the preferred course of discovery, development, sales or marketing, which might cause delays or terminations of the research, development or commercialization of product candidates, lead to additional and burdensome responsibilities for us with respect to product candidates, or result in litigation or arbitration, any of which would be time-consuming and expensive; • may not properly maintain or defend their or our relevant intellectual property rights or may use our proprietary information or sublicensed intellectual property rights in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation and liability; • may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; • could become involved in a business combination or cessation that could cause them to deemphasize or terminate the development or commercialization of any product candidate licensed to it by us; and • may terminate our collaborations, which could require us to raise additional capital to develop or commercialize the applicable product candidates, or lose access to the collaborator’s intellectual property.
Our ability to generate revenue, and achieve and retain profitability, depends significantly on our success in many areas, including: • obtaining regulatory approvals and marketing authorizations for our lead programs; • obtaining market acceptance of our product candidates as viable treatment options; • launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; • accurately assessing the size and addressability of potential patient populations; • addressing any competing technological and market developments; • maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and know-how; • avoiding infringement of or obtaining licenses to any valid intellectual property owned or controlled by third parties; • negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter or which may be necessary for us to develop, manufacture or commercialize our product candidates; • maintaining good relationships with our collaborators and licensors; • attracting, hiring and retaining qualified personnel; • developing a sustainable and scalable manufacturing process for product candidates, including establishing and maintaining commercially viable supply relationships with third parties, such as CMOs, and potentially establishing our own manufacturing capabilities and infrastructure; • successfully completing research, preclinical and clinical development of product candidates; • investing resources in developing commercial manufacturing and operational infrastructure prior to clinical evidence of safety and efficacy for a given product candidate; and • selecting commercially viable product candidates and effective delivery methods.
Our ability to generate revenue, and achieve and retain profitability, depends significantly on our success in many areas, including: • obtaining regulatory approvals and marketing authorizations for our lead programs; • obtaining market acceptance of our product candidates as viable treatment options; • launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; • accurately assessing the size and addressability of potential patient populations; • addressing any competing technological and market developments; • maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and know-how; • avoiding infringement of or obtaining licenses to any valid intellectual property owned or controlled by third parties; • negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter or which may be necessary for us to develop, manufacture or commercialize our product candidates; • maintaining good relationships with our collaborators and licensors; • attracting, hiring and retaining qualified personnel; • developing a sustainable and scalable manufacturing process for product candidates, including establishing and maintaining commercially viable supply relationships with third parties, such as CMOs, and potentially establishing our own manufacturing capabilities and infrastructure; • successfully completing research, preclinical and clinical development of product candidates; 49 • investing resources in developing commercial manufacturing and operational infrastructure prior to clinical evidence of safety and efficacy for a given product candidate; and • selecting commercially viable product candidates and effective delivery methods.
Our stock price is likely to continue to be volatile and subject to significant price and volume fluctuations in response to market and other factors, including: • the success of our products or technologies or competing products or technologies; • results of clinical trials of our product candidates or those of our competitors; • developments or disputes concerning issued patents, patent applications or other intellectual property rights; • regulatory or legal developments in the U.S. and other countries; • the recruitment or departure of key personnel; • the level of expenses related to any of our product candidates or clinical development programs; • the results of our efforts to discover, develop, manufacture, acquire or in-license our current and additional product candidates or products; • actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts; • variations in our financial results or the financial results of companies that are perceived to be similar to us; • sales of a substantial number of shares of our common stock in the public market, or the perception in the market that the holders of a large number of shares intend to sell shares; • changes in the structure of healthcare payment systems; • market conditions in the pharmaceutical and biotechnology sectors; • public perception of the safety of genome editing based therapeutics; • general economic, industry and market conditions; and • the other factors summarized and described in this Risk Factors section.
Our stock price is likely to continue to be volatile and subject to significant price and volume fluctuations in response to market and other factors, including: • the success of our products or technologies or competing products or technologies; • results of clinical trials of our product candidates or those of our competitors; • developments or disputes concerning issued patents, patent applications or other intellectual property rights; 70 • regulatory or legal developments in the U.S. and other countries; • the recruitment or departure of key personnel; • the level of expenses related to any of our product candidates or clinical development programs; • the results of our efforts to discover, develop, manufacture, acquire or in-license our current and additional product candidates or products; • actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts; • variations in our financial results or the financial results of companies that are perceived to be similar to us; • sales of a substantial number of shares of our common stock in the public market, or the perception in the market that the holders of a large number of shares intend to sell shares; • changes in the structure of healthcare payment systems; • market conditions in the pharmaceutical and biotechnology sectors; • public perception of the safety of genome editing based therapeutics; • general economic, industry and market conditions; and • the other factors summarized and described in this Risk Factors section.
However, we cannot predict: • if and when any patents will issue; • the scope, degree and range of protection any issued patents will afford us against competitors, including whether third parties will find ways to invalidate or otherwise circumvent our patents; 45 • whether others will apply for or obtain patents claiming aspects similar to those covered by our patents and patent applications; • whether certain governments will appropriate our intellectual property rights and allow competitors to use them; or • whether we will need to initiate litigation or administrative proceedings to assert or defend our patent rights, which may be costly whether we win or lose.
However, we cannot predict: • if and when any patents will issue; • the scope, degree and range of protection any issued patents will afford us against competitors, including whether third parties will find ways to invalidate or otherwise circumvent our patents; • whether others will apply for or obtain patents claiming aspects similar to those covered by our patents and patent applications; • whether certain governments will appropriate our intellectual property rights and allow competitors to use them; or • whether we will need to initiate litigation or administrative proceedings to assert or defend our patent rights, which may be costly whether we win or lose.
For example, through a license agreement between Caribou Biosciences, Inc. and us (the “Caribou License”), we sublicense the rights of the Regents of the University of California and the University of Vienna (collectively, “UC/Vienna”) to a worldwide patent portfolio that covers methods of use and compositions relating to engineered CRISPR/Cas9 systems for, among other things, cleaving or editing DNA and altering gene product expression in various organisms, including eukaryotic cells.
For example, through a license agreement between Caribou Biosciences, Inc. and us (the “Caribou License”), we sublicense the rights of the Regents of the University of California and the University of Vienna (collectively, “UC/Vienna”) to a worldwide patent portfolio that covers methods of use and compositions relating to engineered CRISPR systems for, among other things, cleaving or editing DNA and altering gene product expression in various organisms, including eukaryotic cells.
We also may experience numerous unforeseen events during, or as a result of, any current or future clinical trials that we conduct, which could delay or prevent our ability to receive marketing approval or commercialize our product candidates, including: • challenges in obtaining regulatory authorization or approval to conduct clinical trials in the U.S. from the FDA through an investigational new drug (“IND”) application or from other regulatory agencies outside the U.S., such as the United Kingdom (“U.K.”) Medicines and Healthcare products Regulatory Agency (“MHRA”) or the European Medicines Agency (“EMA”), through corresponding applications, such as a clinical trial application, a clinical trial notification or a clinical trial exemption, because these agencies have very limited or no experience with the clinical development of CRISPR/Cas9-based therapeutics, particularly in vivo therapeutics, which may require additional significant testing or data compared to more traditional therapies or otherwise delay the development of our product candidates; • successfully developing processes for the safe administration of these product candidates, including long-term follow-up for patients who receive treatment with any of our product candidates; • regulators, institutional review boards (“IRBs”) or ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial; • inability to reach, or delays in reaching, agreement on acceptable terms with trial sites and contract research organizations (“CROs”); • clinical trials of any product candidates may fail to show safety or efficacy, or could produce negative or inconclusive results, which could result in having to conduct additional preclinical studies or clinical trials or terminating the product development programs; • we may not be able to initiate or complete clinical trials of a product candidate if the required number of subjects is larger than we anticipated, the number of subjects willing to enroll is smaller than required, the pace of enrollment is slower than anticipated, or subjects drop out or fail to return for post-treatment follow-up at a higher rate than we anticipated; • we may need to educate medical personnel, including clinical investigators, and patients regarding the potential benefits and side effect profile of each of our product candidates; • regulatory agencies may require us to amend our INDs or equivalent regulatory filings, modify the design of our clinical trials or perform more extensive or lengthier preclinical or clinical testing compared to existing therapeutic modalities, any of which may delay the initiation or progression of any of our clinical trials; • animal models may not exist, or available animal models may be inadequate, for some of the human diseases we choose to pursue in our programs, or the preclinical studies we perform as part of our programs; • our third party contractors, clinical trial sites or investigators may fail to comply with regulatory requirements or meet their performance obligations to us in a timely manner, or at all, or may deviate from the clinical trial protocol or drop out of the trial, which may require that we add new clinical trial sites or investigators; • we may elect to, or regulators, IRBs or ethics committees may require that we or our investigators, suspend or terminate clinical research or trials for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks; • the cost of preclinical studies and clinical trials of any product candidates may be greater than we anticipate; • the supply or quality of our product candidates or other materials necessary to conduct preclinical studies and clinical trials of our product candidates may be insufficient or inadequate, or not available in a reasonable timeframe, and any transfer of manufacturing activities may require unforeseen manufacturing or formulation changes; • we may face challenges in sourcing preclinical, clinical and, if approved, commercial supplies for the materials used to manufacture and process our product candidates, which may include importing or exporting materials between different jurisdictions; 36 • our product candidates may have undesirable side effects or other unexpected characteristics, such as effects or characteristics resulting from their biodistribution or mechanism of action, causing us or our investigators, regulators, IRBs or ethics committees to suspend or terminate the trials, or reports may arise from preclinical or clinical testing of other gene therapies or genome editing-based therapies that raise safety or efficacy concerns about our product candidates; • the FDA or other regulatory authorities may require us to submit additional data, such as long-term toxicology studies, or impose other requirements, including submitting preclinical data earlier in clinical development compared to existing therapeutic modalities or requiring amendments to our regulatory filings, before permitting us to initiate or rely on a clinical trial; • we may face challenges in establishing sales and marketing capabilities in anticipation of, and after obtaining, any regulatory approval to gain market authorization; • the FDA or other regulatory authorities may revise the requirements for authorizing our clinical trials or approving our product candidates, or their interpretation of the authorization or approval requirements may not be what we anticipate or require us to adopt Risk Evaluation and Mitigation Strategy (“REMS”) as a condition of approval; and • we may not ultimately obtain regulatory approval for a BLA, or corresponding applications outside the U.S., such as a marketing authorization application in the U.K. and other similar regulatory authorities, which may have very limited or no experience with the clinical development of CRISPR/Cas9-based therapeutics, particularly in vivo therapeutics.
We also may experience numerous unforeseen events during, or as a result of, any current or future clinical trials that we conduct, which could delay or prevent our ability to receive marketing approval or commercialize our product candidates, including: • challenges in obtaining regulatory authorization or approval to conduct clinical trials in the U.S. from the FDA through an investigational new drug (“IND”) application or from other regulatory agencies outside the U.S., such as the United Kingdom (“U.K.”) Medicines and Healthcare products Regulatory Agency (“MHRA”) or the European Medicines Agency (“EMA”), through corresponding applications, such as a clinical trial application, a clinical trial notification or a clinical trial exemption, because these agencies have very limited or no experience with the clinical development of CRISPR-based therapeutics, particularly in vivo therapeutics, which may require additional significant testing or data compared to more traditional therapies or otherwise delay the development of our product candidates; • successfully developing processes for the safe administration of these product candidates, including long-term follow-up for patients who receive treatment with any of our product candidates; • regulators, institutional review boards (“IRBs”) or ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial; • inability to reach, or delays in reaching, agreement on acceptable terms with trial sites and contract research organizations (“CROs”); • clinical trials of any product candidates may fail to show safety or efficacy, or could produce negative or inconclusive results, which could result in having to conduct additional preclinical studies or clinical trials or terminating the product development programs; • we may not be able to initiate or complete clinical trials of a product candidate if the required number of subjects is larger than we anticipated, the number of subjects willing to enroll is smaller than required, the pace of enrollment is slower than anticipated, or subjects drop out or fail to return for post-treatment follow-up at a higher rate than we anticipated; • we may need to educate medical personnel, including clinical investigators, and patients regarding the potential benefits and side effect profile of each of our product candidates; • regulatory agencies may require us to amend our IND applications or equivalent regulatory filings, modify the design of our clinical trials or perform more extensive or lengthier preclinical or clinical testing compared to existing therapeutic modalities, any of which may delay the initiation or progression of any of our clinical trials; • animal models may not exist, or available animal models may be inadequate, for some of the human diseases we choose to pursue in our programs, or the preclinical studies we perform as part of our programs; • our third party contractors, clinical trial sites or investigators may fail to comply with regulatory requirements or meet their performance obligations to us in a timely manner, or at all, or may deviate from the clinical trial protocol or drop out of the trial, which may require that we add new clinical trial sites or investigators; • we may elect to, or regulators, IRBs or ethics committees may require that we or our investigators, suspend or terminate clinical research or trials for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks; • the cost of preclinical studies and clinical trials of any product candidates may be greater than we anticipate; • the supply or quality of our product candidates or other materials necessary to conduct preclinical studies and clinical trials of our product candidates may be insufficient or inadequate, or not available in a reasonable timeframe, and any transfer of manufacturing activities may require unforeseen manufacturing or formulation changes; • we may face challenges in sourcing preclinical, clinical and, if approved, commercial supplies for the materials used to manufacture and process our product candidates, which may include importing or exporting materials between different jurisdictions; 34 • our product candidates may have undesirable side effects or other unexpected characteristics, such as effects or characteristics resulting from their biodistribution or mechanism of action, causing us or our investigators, regulators, IRBs or ethics committees to suspend or terminate the trials, or reports may arise from preclinical or clinical testing of other gene therapies or genome editing-based therapies that raise safety or efficacy concerns about our product candidates; • the FDA or other regulatory authorities may require us to submit additional data, such as long-term toxicology studies, or impose other requirements, including submitting preclinical data earlier in clinical development compared to existing therapeutic modalities or requiring amendments to our regulatory filings, before permitting us to initiate, continue or rely on a clinical trial; • we may face challenges in establishing sales and marketing capabilities in anticipation of, and after obtaining, any regulatory approval to gain market authorization; • the FDA or other regulatory authorities may revise the requirements for authorizing our clinical trials or approving our product candidates, or their interpretation of the authorization or approval requirements may not be what we anticipate or require us to adopt Risk Evaluation and Mitigation Strategy (“REMS”) as a condition of approval; and • we may not ultimately obtain regulatory approval for a BLA, or corresponding applications outside the U.S., such as a marketing authorization application in the U.K. and other similar regulatory authorities, which may have very limited or no experience with the clinical development of CRISPR-based therapeutics, particularly in vivo therapeutics.
Failure to maintain the security, confidentiality, accessibility or integrity of data stored on such systems could result in interruptions in our operations, damage our reputation in the market, increase our service costs, cause us to incur substantial costs, subject us to liability for damages and/or fines, and divert our resources from other tasks, any one of which could materially adversely affect our business, financial condition, results of operations and prospects.
Failure to maintain the security, confidentiality, accessibility or 63 integrity of data stored on such systems could result in interruptions in our operations, damage our reputation in the market, increase our service costs, cause us to incur substantial costs, subject us to liability for damages and/or fines, and divert our resources from other tasks, any one of which could materially adversely affect our business, financial condition, results of operations and prospects.
In addition, other third parties, such as Vilnius University (whose patents we have sublicensed under our Caribou Agreement) and Harvard University, filed patent applications claiming CRISPR/Cas9-related inventions around or within a year after the first patent application filed in the UC/Vienna/Charpentier patent family and allege (or may allege) that they invented one or more of the inventions claimed by UC/Vienna/Charpentier before UC/Vienna/Charpentier.
In addition, other third parties, such as Vilnius University (whose patents we have sublicensed under our Caribou Agreement) and Harvard University, filed patent applications claiming CRISPR-related inventions around or within a year after the first patent application filed in the UC/Vienna/Charpentier patent family and allege (or may allege) that they invented one or more of the inventions claimed by UC/Vienna/Charpentier before UC/Vienna/Charpentier.
If either the Broad Institute, ToolGen or Sigma-Aldrich were to succeed in any of their respective interferences, or if other third parties, such as Vilnius University or Harvard University, pursue claims and succeed in such claims, the prevailing party or parties could assert its issued patents against us based on our CRISPR/Cas9-based activities, including research, development, and commercialization of our product candidates or resulting products.
If either the Broad Institute, ToolGen or Sigma-Aldrich were to succeed in any of their respective interferences, or if other third parties, such as Vilnius University or Harvard University, pursue claims and succeed in such claims, the prevailing party or parties could assert its issued patents against us based on our CRISPR-based activities, including research, development, and commercialization of our product candidates or resulting products.
Our future success also is highly dependent on the successful development of CRISPR-based genome editing technologies, cellular delivery methods and therapeutic applications for the indications on which we have focused our ongoing research and development efforts. We may decide to alter or abandon these programs as new data become available and we gain experience in developing CRISPR/Cas9-based therapeutics.
Our future success also is highly dependent on the successful development of CRISPR-based genome editing technologies, cellular delivery methods and therapeutic applications for the indications on which we have focused our ongoing research and development efforts. We may decide to alter or abandon these programs as new data become available and we gain experience in developing CRISPR-based therapeutics.
In addition, the use of genome editing technology by third parties in areas that are not being pursued by us, such as for targeting and editing of embryonic cells, could adversely impact public and governmental perceptions regarding the ethics and risks of CRISPR/Cas9 technology and lead to social or legal changes that could limit our ability to apply the technology to develop human therapies addressing disease.
In addition, the use of genome editing technology by third parties in areas that are not being pursued by us, such as for targeting and editing of embryonic cells, could adversely impact public and governmental perceptions regarding the ethics and risks of CRISPR technology and lead to social or legal changes that could limit our ability to apply the technology to develop human therapies addressing disease.
Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our 46 owned and licensed patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our owned and licensed patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
New risk factors can emerge from time to time, and we cannot predict the impact that any factor or combination of factors may have on our business, prospects, financial condition and results of operations. Risks Related to Our Business Risks Related to Preclinical and Clinical Development CRISPR/Cas9 genome editing technology has only recently been clinically validated for human therapeutic use.
New risk factors can emerge from time to time, and we cannot predict the impact that any factor or combination of factors may have on our business, prospects, financial condition and results of operations. Risks Related to Our Business Risks Related to Preclinical and Clinical Development CRISPR genome editing technology has only recently been clinically validated for human therapeutic use.
It is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to 57 detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
It is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
Public perception may be influenced by claims that gene therapy or genome editing, including the use of CRISPR/Cas9, is unsafe or unethical, or carries an undue risk of side effects, such as improper modification of a gene sequence in a patient’s chromosome that could lead to disease such as cancer, and gene therapy or genome editing may not gain the acceptance of the public or the medical community.
Public perception may be influenced by claims that gene therapy or genome editing, including the use of CRISPR, is unsafe or unethical, or carries an undue risk of side effects, such as improper modification of a gene sequence in a patient’s chromosome that could lead to disease such as cancer, and gene therapy or genome editing may not gain the acceptance of the public or the medical community.
Our future capital requirements will depend on and could increase significantly as a result of many factors, including the scope, progress, results and costs of drug discovery, preclinical development, laboratory 51 testing and clinical trials for our current or future product candidates, including additional expenses attributable to adjusting our development plans (including any supply related matters).
Our future capital requirements will depend on and could increase significantly as a result of many factors, including the scope, progress, results and costs of drug discovery, preclinical development, laboratory testing and clinical trials for our current or future product candidates, including additional expenses attributable to adjusting our development plans (including any supply related matters).
There is a substantial amount of litigation as well as administrative proceedings for challenging patents, including interference, derivation, reexamination, and other post-grant proceedings before the USPTO and oppositions and other comparable proceedings in foreign jurisdictions, involving patents and other intellectual property rights in the biotechnology and pharmaceutical industries, and we expect this to be true for the CRISPR/Cas9 space as well.
There is a substantial amount of litigation as well as administrative proceedings for challenging patents, including interference, derivation, reexamination, and other post-grant proceedings before the USPTO and oppositions and other comparable proceedings in foreign jurisdictions, involving patents and other intellectual property rights in the biotechnology and pharmaceutical industries, and we expect this to be true for the CRISPR space as well.
We expect the novel nature of our product candidates to create challenges or raise questions from regulatory agencies in obtaining regulatory approval. For example, in the U.S., the FDA has not approved any in vivo gene editing-based therapeutic and has only approved one ex vivo CRISPR/Cas9 genome editing therapy for human therapeutic use.
We expect the novel nature of our product candidates to create 52 challenges or raise questions from regulatory agencies in obtaining regulatory approval. For example, in the U.S., the FDA has not approved any in vivo gene editing-based therapeutic and has only approved one ex vivo CRISPR/Cas9 genome editing therapy for human therapeutic use.
The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses and a company that is found to have improperly promoted off-label uses may be subject to significant liability. The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates.
The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses and a company that is found to have improperly promoted off-label uses may be subject to significant liability. 56 The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates.
These non-compete laws may negatively impact our ability to prevent employees from working with direct or indirect competitors in the future and may affect our ability to retain key talent in a competitive market. Our failure to comply with these and other related laws could expose us to civil and, in some cases, criminal liability, including fines and penalties.
These 58 non-compete laws may negatively impact our ability to prevent employees from working with direct or indirect competitors in the future and may affect our ability to retain key talent in a competitive market. Our failure to comply with these and other related laws could expose us to civil and, in some cases, criminal liability, including fines and penalties.
In addition, any material alteration, in an adverse manner, of any 59 material collaboration agreement, or dispute or litigation proceedings we may have related to a material collaboration in the future could delay development programs, create uncertainty as to ownership of or access to intellectual property rights, distract management from other business activities and generate substantial expense.
In addition, any material alteration, in an adverse manner, of any material collaboration agreement, or dispute or litigation proceedings we may have related to a material collaboration in the future could delay development programs, create uncertainty as to ownership of or access to intellectual property rights, distract management from other business activities and generate substantial expense.
All the therapeutic indications approved by the relevant authorities may not be covered or reimbursed. In addition, we cannot be sure that coverage and reimbursement will be available for, or accurately estimate the potential revenue from, our product candidates because they are novel treatments for diseases using a new technology and delivery approaches.
All the therapeutic indications approved by the relevant authorities may not be covered or reimbursed. In addition, we cannot be sure that coverage and reimbursement will be available for, or accurately estimate the potential revenue from, our product candidates because they are novel treatments for diseases using new technology and delivery approaches.
Although one CRISPR/Cas9-edited ex vivo therapy has been approved in the United States (“U.S.”) and European Union (“EU”), no genome editing in vivo therapy has been approved in the U.S., EU countries or other key jurisdictions, and the potential to successfully obtain approval for any of our CRISPR/Cas9 product candidates remains uncertain.
Although one CRISPR-edited ex vivo therapy has been approved in the United States (“U.S.”) and European Union (“EU”), no genome editing in vivo therapy has been approved in the U.S., EU countries or other key jurisdictions, and the potential to successfully obtain approval for any of our CRISPR product candidates remains uncertain.
Such an outcome could have a material adverse effect on our business. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees. We have limited foreign intellectual property rights and may not be able to protect our intellectual property rights throughout the world.
Such an outcome could have a material adverse effect on our business. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees. 46 We have limited foreign intellectual property rights and may not be able to protect our intellectual property rights throughout the world.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation or contractual litigation there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation or proceeding. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation or contractual litigation there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation or proceeding. In addition, there could be public announcements of the 47 results of hearings, motions or other interim proceedings or developments.
The size and complexity of our information technology systems, and those of third party vendors, service providers and collaborators, and the large amounts of confidential information stored on those systems, make such systems potentially vulnerable to service interruptions or systems failures, or to cybersecurity incidents, breaches or compromises from inadvertent or intentional actions by our employees, third party vendors, service providers, collaborators, and/or business partners, or from cyber-attacks by malicious third parties.
The size and complexity of our information technology systems, and those of third party vendors, service providers and collaborators, and the large amounts of confidential information stored on those systems, make such systems potentially vulnerable to service interruptions or systems failures, or to cybersecurity incidents, breaches or compromises from inadvertent or intentional actions by our employees, third party vendors, service providers, collaborators, and/or business partners, or from cybersecurity attacks by malicious third parties.
We are focused on developing novel therapeutics utilizing CRISPR/Cas9 genome editing technology, including in vivo therapies and ex vivo engineered cell therapies. Although there have been significant advances in recent years in the fields of gene therapy and genome editing, in vivo CRISPR-based genome editing technologies are relatively new and their therapeutic utility is largely unproven.
We are focused on developing novel therapeutics utilizing CRISPR genome editing technology, including in vivo therapies and ex vivo engineered cell therapies. Although there have been significant advances in recent years in the fields of gene therapy and genome editing, in vivo CRISPR-based genome editing technologies are relatively new and their therapeutic utility is largely unproven.
If such third parties were found to have rights to patents or patent applications covering our licensed technology (such as CRISPR/Cas9 technology), they could assert that we infringe such patents and, as discussed above, we may be required to obtain rights from such parties (e.g., by obtaining a license) or cease our development and commercialization efforts.
If such third parties were found to have rights to patents or patent applications covering our licensed technology (such as CRISPR technology), they could assert that we infringe such patents and, as discussed above, we may be required to obtain rights from such parties (e.g., by obtaining a license) or cease our development and commercialization efforts.
For example, various patent applications within the UC/Vienna/Charpentier patent family and the Broad Institute patent family have been involved in patent interferences and other patent challenges in the U.S. and other jurisdictions, in which the respective owners have alleged, and are alleging, that they invented and/or own intellectual property claiming overlapping aspects of CRISPR/Cas9 systems.
For example, various patent applications within the UC/Vienna/Charpentier patent family and the Broad Institute patent family have been involved in patent interferences and other patent challenges in the U.S. and other jurisdictions, in which the respective owners have alleged, and are alleging, that they invented and/or own intellectual property claiming overlapping aspects of CRISPR systems.
If we are not able to generate revenue from the sale of any approved products, we may never become profitable. 50 Our operating history may make difficult the evaluation of our business’s success to date and assessment of our future viability. We are a clinical-stage company. We were founded and commenced operations in mid-2014.
If we are not able to generate revenue from the sale of any approved products, we may never become profitable. Our operating history may make difficult the evaluation of our business’s success to date and assessment of our future viability. We are a clinical-stage company. We were founded and commenced operations in mid-2014.
For example, the State of Washington recently passed a law, effective as of March 31, 2024, that regulates health and medical information that is not subject to HIPAA. Similar laws have been passed in Connecticut and Nevada. Additionally, a small number of states have enacted laws that specifically target the collection and use of biometric information.
For example, the State of Washington passed a law, effective as of March 31, 2024, that regulates health and medical information that is not subject to HIPAA. Similar laws have been passed in Connecticut and Nevada. Additionally, a small number of states have enacted laws that specifically target the collection and use of biometric information.
Supreme Court overruled the Chevron doctrine, which gave deference to regulatory agencies’ statutory interpretations in litigation against federal government agencies, such as the FDA and Centers for Medicare & Medicaid Services (“CMS”), agencies within the U.S. Department of Health and Human Services, where the law is ambiguous.
Supreme Court overruled the Chevron doctrine, which gave deference to regulatory agencies’ statutory interpretations in litigation against federal government agencies, such as the FDA and Centers for Medicare & Medicaid Services (“CMS”), agencies within the U.S. Department of Health and Human Services (“HHS”), where the law is ambiguous.
In addition, adverse publicity due to the ethical and social controversies surrounding the therapeutic in vivo use of CRISPR/Cas9, genome edited modified cells, or other therapeutics mediums, such as viral vectors that we may use in our clinical trials may limit market acceptance of our product candidates.
In addition, adverse publicity due to the ethical and social controversies surrounding the therapeutic in vivo use of CRISPR, genome edited modified cells, or other therapeutics mediums, such as viral vectors that we may use in our clinical trials may limit market acceptance of our product candidates.
If the FDA or any comparable foreign regulatory authority does not accept such data, it would result in the need for additional trials, which could be costly and time-consuming, and which may result in product candidates that we may develop not receiving approval for commercialization in the applicable jurisdiction.
If the FDA or any 53 comparable foreign regulatory authority does not accept such data, it would result in the need for additional trials, which could be costly and time-consuming, and which may result in product candidates that we may develop not receiving approval for commercialization in the applicable jurisdiction.
For more information on coverage and reimbursement see the section entitled “ Business – Government Regulation and Product Approval – Coverage and Reimbursement. ” 67 In the U.S. and some other jurisdictions, patients generally rely on third party payors to reimburse all or part of the costs associated with their treatment.
For more information on coverage and reimbursement see the section entitled “ Business – Government Regulation and Product Approval – Coverage and Reimbursement. ” In the U.S. and some other jurisdictions, patients generally rely on third party payors to reimburse all or part of the costs associated with their treatment.
Patent and Trademark Office (“USPTO”) declared an interference between the UC/Vienna/Charpentier eukaryotic patent family and the Broad Institute patent family to determine which research group first invented the use of the CRISPR/Cas9 technology in eukaryotic cells and, therefore, is entitled to the U.S. patents covering that invention.
Patent and Trademark Office (“USPTO”) declared an interference between the UC/Vienna/Charpentier eukaryotic patent family and the Broad Institute patent family to determine which research group first invented the use of the CRISPR technology in eukaryotic cells and, therefore, is entitled to the U.S. patents covering that invention.
Before obtaining marketing approval from regulatory authorities for the sale of any product candidate, we must complete preclinical development, manufacture (or have manufactured) product candidates and components, and then conduct extensive clinical trials to demonstrate the safety and efficacy of any of our future product candidates in humans.
Before obtaining marketing approval from regulatory authorities for the sale of any product candidate, we must complete preclinical development, 50 manufacture (or have manufactured) product candidates and components, and then conduct extensive clinical trials to demonstrate the safety and efficacy of any of our future product candidates in humans.
For example, in October 2023, we announced an expanded research collaboration with Regeneron to develop therapies for the treatment of neurological and muscular diseases. These current and future therapeutic-focused collaborations could provide us with important technologies and/or funding for our programs and technology.
For example, in October 2023, we announced an expanded research collaboration with Regeneron to develop therapies for the treatment of neurological and 59 muscular diseases. These current and future therapeutic-focused collaborations could provide us with important technologies and/or funding for our programs and technology.
Obtaining and maintaining regulatory approval of our product candidates in one jurisdiction does not guarantee that we will be able to obtain or maintain regulatory approval in any other jurisdiction, but a failure or delay in obtaining regulatory approval in 55 one jurisdiction may have a negative effect on the regulatory approval process in others.
Obtaining and maintaining regulatory approval of our product candidates in one jurisdiction does not guarantee that we will be able to obtain or maintain regulatory approval in any other jurisdiction, but a failure or delay in obtaining regulatory approval in one jurisdiction may have a negative effect on the regulatory approval process in others.
These stockholders may have the ability to influence us through their ownership positions. These stockholders may be able to determine all matters requiring stockholder approval. For example, these stockholders, acting together, may be able to control elections of directors or approval of any merger, sale of assets or other major corporate transaction.
These stockholders may have the ability to influence us through their ownership positions. These stockholders may be able to determine all matters requiring stockholder approval. For example, these stockholders, acting 71 together, may be able to control elections of directors or approval of any merger, sale of assets or other major corporate transaction.
Our approach to developing therapies centers on using CRISPR/Cas9 technology to alter, introduce or remove genetic information in vivo to treat various disorders, or to engineer human cells ex vivo to create therapeutic cells that can be introduced into the human body to address the underlying disease.
Our approach to developing therapies centers on using CRISPR technology to alter, introduce or remove genetic information in vivo to treat various disorders, or to engineer human cells ex vivo to create therapeutic cells that can be introduced into the human body to address the underlying disease.
Because the patent applications involved in these interferences also purport to cover the use of CRISPR/Cas9 for gene editing in eukaryotic cells, the PTAB seeks to determine between the various groups which one invented first and is entitled to the resulting U.S. patents.
Because the patent applications involved in these interferences also purport to cover the use of CRISPR for gene editing in eukaryotic cells, the PTAB seeks to determine between the various groups which one invented first and is entitled to the resulting U.S. patents.
Even if we are not a party to legal actions or other disputes involving our licensed intellectual property, an adverse outcome could harm our business because it might prevent us from continuing to license intellectual property that we may need to operate our business.
Even if we are not a party to legal actions or other disputes involving our licensed intellectual property, an adverse outcome could harm our business because it might prevent us from continuing to license intellectual property that we may need to operate 43 our business.
Further, patients may choose to travel to countries in which we do not have intellectual property rights or which do not enforce these rights to obtain the products or treatment from competitors in such countries. 47 Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
Further, patients may choose to travel to countries in which we do not have intellectual property rights or which do not enforce these rights to obtain the products or treatment from competitors in such countries. Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
In addition, if the FDA or a comparable foreign regulatory authority approves our product 56 candidates, we will have to comply with their respective legal or regulatory requirements including submissions of safety and other post-marketing information and reports and registration.
In addition, if the FDA or a comparable foreign regulatory authority approves our product candidates, we will have to comply with their respective legal or regulatory requirements including submissions of safety and other post-marketing information and reports and registration.
Even if we are successful in selecting and developing any product candidates, in order to compete successfully we may need to be first-to-market or demonstrate that our CRISPR/Cas9-based products are superior to therapies based on the same or different treatment methods.
Even if we are successful in selecting and developing any product candidates, in order to compete successfully we may need to be first-to-market or demonstrate that our CRISPR-based products are superior to therapies based on the same or different treatment methods.
Pursuant to Section 404 of the Sarbanes-Oxley Act of 2002 (“Section 404”), we are required to furnish a report by our management on our internal control over financial reporting, including an attestation report on internal control over financial 72 reporting issued by our independent registered public accounting firm.
Pursuant to Section 404 of the Sarbanes-Oxley Act of 2002 (“Section 404”), we are required to furnish a report by our management on our internal control over financial reporting, including an attestation report on internal control over financial reporting issued by our independent registered public accounting firm.
We seek to protect our proprietary processes, in part, by entering into confidentiality agreements with our employees, consultants, outside scientific advisors, contractors (including CMOs and CROs), and collaborators, and we also rely on federal and state laws requiring our directors, employees, consultants, contractors (including CMOs and CROs), and collaborators to protect our proprietary information.
We seek to protect our proprietary processes, in part, by entering into confidentiality agreements with our employees, consultants, outside scientific advisors, contractors (including CMOs and CROs), and collaborators, and we also rely on federal and state laws requiring our directors, employees, consultants, contractors (including CMOs and CROs), and collaborators to protect our proprietary 48 information.
Even if we obtain coverage for a given product, the resulting reimbursement payment rates might be insufficient or may require co-insurance or co-payments that patients find unacceptably high, which may prevent us from achieving or sustaining profitability.
Even if we obtain coverage for a given product, the resulting reimbursement payment rates might be insufficient or may require co-insurance or co-payments 67 that patients find unacceptably high, which may prevent us from achieving or sustaining profitability.
These different laws 68 governing the privacy and security of health and other personal information often differ from each other in significant ways and may not have the same effective requirements, thus complicating efforts to comply with their respective provisions.
These different laws governing the privacy and security of health and other personal information often differ from each other in significant ways and may not have the same effective requirements, thus complicating efforts to comply with their respective provisions.
For this reason, we will employ multiple steps to control the manufacturing process to ensure that the process results in product candidates that meet their specifications, but complications 52 at any one step could adversely impact our manufacturing of products.
For this reason, we will employ multiple steps to control the manufacturing process to ensure that the process results in product candidates that meet their specifications, but complications at any one step could adversely impact our manufacturing of products.
There is no certainty that the FDA or other similar agencies will continue to apply to all our CRISPR/Cas9 product candidates the same regulatory pathway and requirements it is applying to other in vivo therapies or ex vivo engineered therapeutics.
There is no certainty that the FDA or other similar agencies will continue to apply to all our CRISPR product candidates the same regulatory pathway and requirements it is applying to other in vivo therapies or ex vivo engineered therapeutics.
We may also need to rely on multiple third parties, 69 such as partners and service providers, to meet these legal requirements, which could result in additional liability for us if they do not comply.
We may also need to rely on multiple third parties, such as partners and service providers, to meet these legal requirements, which could result in additional liability for us if they do not comply.
Our trade secrets and other confidential information of ours may also be exposed through cybersecurity attacks, ransomware attacks, and other hacking attempts directed at our information technology systems and those 49 of our employees, consultants, outside scientific advisors, contractors, vendors and collaborators.
Our trade secrets and other confidential information of ours may also be exposed through cybersecurity attacks, ransomware attacks, and other hacking attempts directed at our information technology systems and those of our employees, consultants, outside scientific advisors, contractors, vendors and collaborators.
Further, many third parties, including the third parties described above, have also filed patent applications and obtained patents covering aspects of the CRISPR/Cas9 technology in other key jurisdictions, including the EU members, the U.K., China and Japan.
Further, many third parties, including the third parties described above, have also filed patent applications and obtained patents covering aspects of the CRISPR technology in other key jurisdictions, including the EU members, the U.K., China and Japan.
The GDPR also confers a private right of action on data subjects and consumer 62 associations to lodge complaints with supervisory authorities, seek judicial remedies, and obtain compensation for damages resulting from violations.
The GDPR also confers a private right of action on data subjects and consumer associations to lodge complaints with supervisory authorities, seek judicial remedies, and obtain compensation for damages resulting from violations.
If we cannot replicate positive results from any of our preclinical or clinical activities and studies, we may be unable to successfully develop, obtain regulatory approval for and commercialize any potential product candidate.
If we cannot replicate the positive results from any of our preclinical or clinical activities and studies, we may be unable to successfully develop, obtain regulatory approval for and commercialize any potential product candidate.
The use of the CRISPR/Cas9 system to create genome editing-based therapies is a recent development and may not become broadly accepted by patients, healthcare providers, third party payors and other stakeholders.
The use of the CRISPR system to create genome editing-based therapies is a recent development and may not become broadly accepted by patients, healthcare providers, third party payors and other stakeholders.
If we or our CMOs cannot successfully manufacture material that conforms to 61 our specifications and the strict relevant regulatory requirements, we and our CMOs will not be able to secure or maintain regulatory approval for our respective manufacturing facilities.
If we or our CMOs cannot successfully manufacture material that conforms to our specifications and the strict relevant regulatory requirements, we and our CMOs will not be able to secure or maintain regulatory approval for our respective manufacturing facilities.
Cybersecurity incidents, breaches, compromises, insider threats and other inappropriate access can be difficult to detect, and any delay in identifying them may lead to increased harm of the types summarized and described above.
Cybersecurity incidents, data breaches, compromises, insider threats and other inappropriate access can be difficult to detect, and any delay in identifying them may lead to increased harm of the types summarized and described above.
The scope of 64 requirements depends on legal and risk determinations that rely on novel legal provisions that have not yet been interpreted by courts or regulators, and non-compliance can lead to significant fines.
The scope of requirements depends on legal and risk determinations that rely on novel legal provisions that have not yet been interpreted by courts or regulators, and non-compliance can lead to significant fines.
Any inactive trading market for our common stock may also impair our ability to 70 raise capital to continue to fund our operations by selling shares and may impair our ability to acquire other companies or technologies by using our shares as consideration.
Any inactive trading market for our common stock may also impair our ability to raise capital to continue to fund our operations by selling shares and may impair our ability to acquire other companies or technologies by using our shares as consideration.
Therapeutic applications of genome editing technologies, and CRISPR/Cas9 in particular, for both in vivo products and ex vivo products, are uncertain and must undergo rigorous clinical trials and regulatory review before receiving marketing authorization.
Therapeutic applications of genome editing technologies, and CRISPR in particular, for both in vivo products and ex vivo products, are uncertain and must undergo rigorous clinical trials and regulatory review before receiving marketing authorization.
Specifically, certain patent applications within the UC/Vienna/Charpentier patent family, licensed to us under the Caribou License, covering certain aspects of CRISPR/Cas9 systems to edit genes in eukaryotic cells, including human cells (collectively, the “UC/Vienna/Charpentier eukaryotic patent family”) have been the subject of patent interferences involving patents and patent application co-owned by the Broad Institute, Massachusetts Institute of Technology, the President and Fellows of Harvard College and the Rockefeller University (collectively, the “Broad Institute”) that also claim CRISPR/Cas9 systems to edit genes in eukaryotic cells (collectively, the “Broad Institute patent family”).
Specifically, certain patent applications within the UC/Vienna/Charpentier patent family, licensed to us under the Caribou License, covering certain aspects of CRISPR systems to edit genes in eukaryotic cells, including human cells (collectively, the “UC/Vienna/Charpentier eukaryotic patent family”) have been the subject of patent interferences involving patents and patent applications co-owned by the Broad Institute, Massachusetts Institute of Technology, the President and Fellows of Harvard College and the Rockefeller University (collectively, the “Broad Institute”) that also claim CRISPR systems to edit genes in eukaryotic cells (collectively, the “Broad Institute patent family”).
We may not meet the requirements of regulatory authorities, such as the FDA, for initiating later phase clinical trials for our product candidates, which could delay the development of our product candidates, including the submission of a BLA or comparable marketing application. 35 Because these are new therapeutic approaches, discovering, developing, manufacturing and commercializing our product candidates may subject us to a number of challenges or delays in completing our preclinical studies and initiating or completing clinical trials.
We may not meet the requirements of regulatory authorities, such as the FDA, for initiating later phase clinical trials for our product candidates, which could delay the development of our product candidates, including the submission of a BLA or comparable marketing application. 33 Because these are new therapeutic approaches, discovering, developing, manufacturing and commercializing our product candidates may subject us to a number of challenges or delays in completing our preclinical studies and initiating or completing clinical trials.
As discussed above, such allegations or litigation may affect our ability to market and sell CRISPR/Cas9-based human therapeutics and may result in significant costs or delays to our programs.
As discussed above, such allegations or litigation may affect our ability to market and sell CRISPR-based human therapeutics and may result in significant costs or delays to our programs.
Dube, our Chief Accounting Officer, David Lebwohl, our Executive Vice President and Chief Medical Officer and Birgit Schultes, our Executive Vice President and Chief Scientific Officer, as well as the other principal members of our management, scientific and clinical teams.
Dube, our Chief Accounting Officer, David Lebwohl, our Executive Vice President and Chief Medical Officer and Birgit Schultes, our Executive Vice President and Chief Scientific Officer, as well as the other principal members of our management, scientific and clinical 66 teams.
Any security incidents, compromises or breaches that lead to unauthorized access, use, or disclosure of personal information, including personal information regarding our employees or current or future clinical trial participants, could harm our reputation, require us to 63 comply with onerous legal requirements under laws and regulations that protect the privacy and security of personal information, and subject us to significant liability including fines, litigation, and loss of current and future business.
Any security incidents, compromises or data breaches that lead to unauthorized access, use, or disclosure of personal information, including personal information regarding our employees or current or future clinical trial participants, could harm our reputation, require us to comply with onerous legal requirements under laws and regulations that protect the privacy and security of personal information, and subject us to significant liability including fines, litigation, and loss of current and future business.
Additionally, because our in vivo technology potentially involves genome editing across multiple cell and tissue types, we are subject to many of the challenges and risks that other genome editing therapeutics and gene therapies face, including: • regulatory guidance for gene and genome editing therapy products has changed and may continue to change in the future, including, e.g., the finalized guidance document titled “Human Gene Therapy Products Incorporating Human Genome Editing” that the FDA issued in January 2024 and the draft guidance document titled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products” published in November 2024; • to date, only a limited number of products that involve in vivo gene transfer have been approved globally; • improper modulation of a gene sequence, including unintended editing events, insertion of a sequence into certain locations in a patient’s chromosome or other effects related to the biodistribution of our product candidates, could lead to cancer, other aberrantly functioning cells or other diseases, including death; • transient expression of the Cas9 protein or other genome editing components of our product candidates could lead to patients having an immunological reaction towards those modified cells, which could be severe or life-threatening; • corrective expression of a missing protein in patients’ cells could result in the protein being recognized as foreign, and lead to a sustained immunological reaction against the expressed protein or expressing cells, which could be severe or life-threatening; and • regulatory agencies may require extended follow-up observation periods of patients who receive treatment using genome editing products including, for example, the FDA’s recommended 15-year follow-up observation period for these patients, and we will need to adopt such observation periods for our product candidates if required by the relevant regulatory agency, which could vary by country or region. 37 Further, because our ex vivo product candidates involve editing human cells and then delivering modified cells to patients, we are subject to many of the challenges and risks that engineered cell therapies face.
Additionally, because our in vivo technology potentially involves genome editing across multiple cell and tissue types, we are subject to many of the challenges and risks that other genome editing therapeutics and gene therapies face, including: • regulatory guidance for gene and genome editing therapy products has changed and may continue to change in the future, including, e.g., the finalized guidance document titled “Human Gene Therapy Products Incorporating Human Genome Editing” that the FDA issued in January 2024 and the draft guidance document titled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products” published in November 2024; • to date, only a limited number of products that involve in vivo gene transfer have been approved globally; 35 • improper modulation of a gene sequence, including unintended editing events, insertion of a sequence into certain locations in a patient’s chromosome or other effects related to the biodistribution of our product candidates, could lead to cancer, other aberrantly functioning cells or other diseases, including death; • transient expression of the Cas9 protein or other genome editing components of our product candidates could lead to patients having an immunological reaction towards those modified cells, which could be severe or life-threatening; • corrective expression of a missing protein in patients’ cells could result in the protein being recognized as foreign, and lead to a sustained immunological reaction against the expressed protein or expressing cells, which could be severe or life-threatening; and • regulatory agencies may require extended follow-up observation periods of patients who receive treatment using genome editing products including, for example, the FDA’s recommended 15-year follow-up observation period for these patients, and we will need to adopt such observation periods for our product candidates if required by the relevant regulatory agency, which could vary by country or region.
In addition, we could be found liable for monetary damages, including treble damages and attorneys’ fees if we are found 41 to have willfully infringed a patent.
In addition, we could be found liable for monetary damages, including treble damages and attorneys’ fees if we are found to have willfully infringed a patent.
Specific to our nex-z program, we are aware of other companies that are currently commercializing or developing products and therapies used to treat ATTR amyloidosis, including Alnylam Pharmaceuticals, Inc., AstraZeneca Pharmaceuticals LP, BridgeBio Pharma Inc., Bayer AG, Ionis Pharmaceuticals, Inc., Metagenomi Technologies, LLC, Novo Nordisk A/S, Pfizer, Inc. and YolTech Therapeutics.
Specific to our nex-z program, we are aware of other companies that are currently commercializing or developing products and therapies used to treat ATTR amyloidosis, including Alnylam Pharmaceuticals, Inc., AstraZeneca Pharmaceuticals LP, BridgeBio Pharma Inc., Bayer AG, Ionis Pharmaceuticals, Inc., Metagenomi Therapeutics, Inc., Novo Nordisk A/S, Pfizer, Inc. and YolTech Therapeutics.
The approaches we are taking to discover and develop novel therapeutics using CRISPR/Cas9 systems are unproven and may never lead to marketable products.
The approaches we are taking to discover and develop novel therapeutics using CRISPR systems are unproven and may never lead to marketable products.
The interference involved 14 allowable patent applications from the UC/Vienna/Charpentier eukaryotic patent family 42 and 13 patents and one patent application from the Broad Institute patent family.
The interference involved 14 allowable patent applications from the UC/Vienna/Charpentier eukaryotic patent family and 13 patents and one patent application from the Broad Institute patent family.
GDPR and any other developments regulating the transfer of personal data between the U.K. and EU. For example, the U.K. government has introduced a Data Use and Access Bill (the “U.K. Bill”) into the U.K. legislative process. The aim of the U.K. Bill is to reform the U.K.’s data protection regime following Brexit.
GDPR and any other developments regulating the transfer of personal data between the U.K. and EU. For example, the U.K. government has introduced a Data Use and Access Bill (the “U.K. Bill”) into the U.K. legislative process, which has passed. The aim of the U.K. Bill is to reform the U.K.’s data protection regime following Brexit. The U.K.
U.S. law also provides for other procedures to challenge patents, including inter partes reviews, post-grant reviews and, in certain circumstances, interference or derivation proceedings, that add uncertainty to the possibility of challenge to our developed or licensed patents and patent applications in the future.
U.S. law also provides for other procedures to challenge patents, including inter partes reviews, post-grant reviews and, in certain circumstances, interference or derivation proceedings, which add uncertainty to the possibility of challenge to our developed or licensed patents and patent applications in the future.
We have limited intellectual property rights outside the U.S. Filing, prosecuting, maintaining and defending patents on product candidates in all countries throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the U.S. can have a different scope and strength than do those in the U.S.
Filing, prosecuting, maintaining and defending patents on product candidates in all countries throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the U.S. can have a different scope and strength than do those in the U.S.
As discussed above, a number of third parties have filed oppositions challenging the validity, and seeking the revocation, of several CRISPR/Cas9 genome editing patents granted to UC/Vienna/Charpentier by the European Patent Office (“EPO”).
As discussed above, a number of third parties have filed oppositions challenging the validity, and seeking the revocation, of several CRISPR genome editing patents granted to UC/Vienna/Charpentier by the European Patent 45 Office (“EPO”).

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeTo help the Company address these risks, we have implemented a cybersecurity risk management program that is informed by recognized industry standards and frameworks and incorporates elements of the same, including elements of the National Institute of Standards and Technology Cybersecurity Framework. Our cybersecurity risk management program is integrated within our enterprise risk management program.
Biggest changeTo help address these risks, we have implemented a cybersecurity risk management program that is informed by recognized industry standards and frameworks and incorporates elements of the same, including elements of the National Institute of Standards and Technology Cybersecurity Framework. Our cybersecurity risk management program is integrated within our enterprise risk management program.
The Head of IT role is currently held by an individual who has close to twenty years of professional IT management experience in the life sciences industry. Our Head of IT also provides regular updates on 75 our cybersecurity risk to our executive leadership team and other management committees responsible for IT and cybersecurity risk management.
The Head of IT role is currently held by an individual who has close to twenty years of professional IT management experience in the life sciences industry. Our Head of IT also provides regular updates on our cybersecurity risk to our executive leadership team and other management committees responsible for IT and cybersecurity risk management.
The Audit Committee, with assistance from our management, including our Head of Information Technology (“IT”), periodically reports to the full Board of Directors to inform them of potential cybersecurity risks and threats, the status of projects to further develop our information security systems, and the emerging cybersecurity threat landscape .
The Audit Committee, with assistance from our management, including our Head of Information Technology (“IT”), periodically reports to the full Board of Directors to inform them of potential 75 cybersecurity risks and threats, the status of projects to further develop our information security systems, and the emerging cybersecurity threat landscape .

Item 2. Properties

Properties — owned and leased real estate

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Biggest changeWe have subleased approximately 13,000 square feet of the property at 730 Main Street for office and laboratory use through March 2026. We also lease approximately 140,000 square feet of office, general laboratory and manufacturing space at 840 Winter Street, Waltham, Massachusetts. In February 2025, we entered into a Second Amendment to Lease (the “Winter Street Amendment”).
Biggest changeWe have executed subleases for two floors of the property at 730 Main Street for laboratory and office use, totaling approximately 25,000 square feet. These subleases expire in February 2026 and December 2028, respectively. We also lease approximately 158,000 square feet of office, general laboratory and manufacturing space at 840 Winter Street, Waltham, Massachusetts.
In addition, we lease approximately 15,200 square feet of office and laboratory space at 130 Brookline Street in Cambridge, Massachusetts, which expires in 2031, approximately 39,000 square feet of office and laboratory space at 281 Albany Street in Cambridge, Massachusetts, which expires in 2030 with an option to extend the lease for two successive five-year terms, approximately 62,000 square feet of office and laboratory space at 640 Memorial Drive, Cambridge, Massachusetts, which expires in 2027, approximately 14,000 square feet of office space at 17 Tudor Street in Cambridge, Massachusetts, which expires in 2025 and approximately 38,000 square feet of office and laboratory space at 730 Main Street, Cambridge, Massachusetts, which expires in 2032 with an option to extend the lease for one five-year term.
In addition, we lease approximately 15,200 square feet of office and laboratory space at 130 Brookline Street in Cambridge, Massachusetts, which expires in 2031, approximately 39,000 square feet of office and laboratory space at 281 Albany Street in Cambridge, Massachusetts, which expires in 2030 with an option to extend the lease for two successive five-year terms, approximately 62,000 square feet of office and laboratory space at 640 Memorial Drive, Cambridge, Massachusetts, which expires in 2027, and approximately 38,000 square feet of office and laboratory space at 730 Main Street, Cambridge, Massachusetts, which expires in 2032 with an option to extend the lease for one five-year term.
Our headquarters are located at 40 Erie Street in Cambridge, Massachusetts, where we occupy approximately 65,000 square feet of office and laboratory space, which expires in 2026, with an option to extend the term of the lease for an additional three years.
Item 2. Properties Our headquarters are located at 40 Erie Street in Cambridge, Massachusetts, where we occupy approximately 65,000 square feet of office and laboratory space, which expires in 2026.
Removed
Item 2. Properties In aggregate, the Company leases approximately 370,000 square feet of real estate, including office, laboratory and manufacturing space in Cambridge, Massachusetts and the surrounding areas.
Added
In February 2025, we entered into an amended agreement to terminate the 840 Winter Lease on or before June 30, 2028, as disclosed within Note 11, “Leases”, of the consolidated financial statements included in this Annual Report on Form 10-K.
Removed
Pursuant to the Winter Street Amendment, the 840 Winter Street lease will terminate on or before June 30, 2028, as described in Note 16, “Subsequent Events.”
Added
We entered into a lease agreement for approximately 125,000 square feet of office and laboratory space at 400 Technology Square in Cambridge, Massachusetts. This lease has not commenced as of December 31, 2025. The initial term of the Tech Square Lease is twelve years and three months beginning in September 2026.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeDistrict Court for the District of Massachusetts against the Company and certain of our officers on behalf of a putative class of stockholders who purchased Company shares from July 30, 2024 through January 8, 2025.
Biggest changeDistrict Court for the District of Massachusetts against the Company and certain of our officers (the “defendants”) asserting claims under Sections 10(b) and 20(a) and Rule 10b-5 of the Securities Exchange Act of 1934 (the “Exchange Act”) on behalf of a putative class of stockholders.
Removed
The complaint alleges claims under Sections 10(b) and 20(a) and Rule 10b-5 of the Securities Exchange Act of 1934 (the “Exchange Act”) premised upon statements relating to the Company’s NTLA-3001 program and the demand for viral-based editing. The complaint seeks unspecified damages, interest, reasonable attorneys’ fees and other costs. We intend to defend vigorously against the claims. Item 4.
Added
On May 26, 2025, the court entered an order 76 appointing co-lead plaintiffs and, on July 23, 2025, co-lead plaintiffs filed an amended complaint. The amended complaint alleges the defendants made certain false and/or misleading statements between January 4, 2024 and January 8, 2025, relating to the Company’s NTLA-3001 program and the demand for viral-based editing.
Removed
Mine Safety Disclosures Not applicable. 76 PART II
Added
The amended complaint seeks unspecified damages, interest, reasonable attorneys’ fees and other costs. On September 8, 2025, the defendants filed a motion to dismiss the amended complaint. On January 14, 2026, the court held a hearing on the motion to dismiss, which motion has been fully briefed and remains pending. Aiello v. Bhanji et al.
Added
On May 15, 2025, a purported stockholder of the Company filed a stockholder derivative lawsuit, captioned Aiello v. Bhanji et al. (C.A. No. 2025-0543-BWD), in the Court of Chancery of the State of Delaware against certain of the Company’s current and former directors (the “Individual Defendants”) and the Company as a nominal defendant.
Added
The complaint alleges claims for breach of fiduciary duty, unjust enrichment, and waste of corporate assets against the Individual Defendants based on, among other things, allegations of allegedly excessive compensation to the Company’s non-employee directors.
Added
The complaint seeks unspecified damages and restitution of compensation and other benefits from the Individual Defendants, corporate governance reforms from the Company, reasonable attorneys’ fees and other costs. On July 11, 2025, the Company and Individual Defendants filed answers to the complaint, and discovery commenced thereafter. Item 4. Mine Safety Disclosures Not applicable. 77 PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeStock Performance Graph The following graph shows a comparison from December 31, 2019 through December 31, 2024, of the cumulative total return on an assumed investment of $100.00 in cash in our common stock, the Nasdaq Composite Index and the Nasdaq Biotechnology Index. Such returns are based on historical results and are not intended to suggest future performance.
Biggest changeStock Performance Graph The following graph shows a comparison from December 31, 2020 through December 31, 2025, of the cumulative total return on an assumed investment of $100 in cash in our common stock, the Nasdaq Composite Index and the Nasdaq Biotechnology Index. Such returns are based on historical results and are not intended to suggest future performance.
Data for the Nasdaq Composite Index and the Nasdaq Biotechnology Index assume reinvestment of dividends. 77 The performance graph in this Item 5 is not deemed to be “soliciting material” or to be “filed” with the Securities and Exchange Commission for purposes of Section 18 of the Securities and Exchange Act of 1934, as amended, or otherwise subject to the liabilities under that Section, and shall not be deemed incorporated by reference into any of our filings under the Securities Act of 1933 or the Securities Exchange Act of 1934, except to the extent we specifically incorporate it by reference into such a filing.
Data for the Nasdaq Composite Index and the Nasdaq Biotechnology Index assume reinvestment of dividends. 78 The performance graph in this Item 5 is not deemed to be “soliciting material” or to be “filed” with the Securities and Exchange Commission for purposes of Section 18 of the Securities and Exchange Act of 1934, as amended, or otherwise subject to the liabilities under that Section, and shall not be deemed incorporated by reference into any of our filings under the Securities Act of 1933 or the Securities Exchange Act of 1934, except to the extent we specifically incorporate it by reference into such a filing.
Item 5. Market for the Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Our common stock is traded on the Nasdaq Global Market under the symbol “NTLA.” As of February 14, 2025, the number of holders of record of our common stock was 13.
Item 5. Market for the Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Our common stock is traded on the Nasdaq Global Market under the symbol “NTLA.” As of February 13, 2026, the number of holders of record of our common stock was 13.
Unregistered Sales of Equity Securities and Use of Proceeds None. Item 6. [Reserved]. 78
Unregistered Sales of Equity Securities and Use of Proceeds None. Item 6. [Reserved]. 79

Item 7. Management's Discussion & Analysis

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

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Biggest changeResearch and Development Research and development expenses increased by $31.2 million to $466.3 million during the year ended December 31, 2024, as compared to $435.1 million during the year ended December 31, 2023. 80 The following table summarizes our research and development expenses, together with the changes in those items in dollars and the respective percentages of change: Year Ended December 31, Period-to- Percent 2024 2023 Period Change Change (In thousands) External development expenses by program: Nex-z $ 69,793 $ 54,454 $ 15,339 28 % NTLA-2002 42,173 24,560 17,613 72 % NTLA-3001 8,709 17,312 (8,603 ) -50 % Unallocated research and development expenses: Employee-related expenses 127,383 136,628 (9,245 ) -7 % Research materials and contracted services 60,661 60,726 (65 ) 0 % Rewrite research milestone - 874 (874 ) -100 % Facility-related expenses 59,397 53,141 6,256 12 % Stock-based compensation 94,230 82,211 12,019 15 % Other 3,965 5,163 (1,198 ) -23 % Total research and development expenses $ 466,311 $ 435,069 $ 31,242 7 % The increase in research and development expenses for the year ended December 31, 2024 compared to the year ended December 31, 2023 was primarily attributable to: • a $15.3 million increase in external costs related to the development of nexiguran ziclumeran (“nex-z”, also referred to as NTLA-2001), one of our lead product candidates, primarily due to an increase in spend on contracted services and consulting fees, offset in part by a decrease in drug components; • a $17.6 million increase in external costs related to the development of NTLA-2002, primarily due to an increase in spend on drug components, contracted services and consulting fees; • an $8.6 million decrease in external costs related to NTLA-3001, primarily related to initial manufacturing activities in 2023, offset in part by an increase in spend on contracted services; • a $9.2 million decrease in employee-related expenses, primarily driven by a workforce reduction in January 2024; • a $6.3 million increase in facility-related expenses primarily related to depreciation, facility maintenance costs, technology expense allocated to research and development, and rent; and • a $12.0 million increase in stock-based compensation.
Biggest changeThe following table summarizes our research and development expenses, together with the changes in those items in dollars and the respective percentages of change: Year Ended December 31, Period-to- Percent 2025 2024 Period Change Change (In thousands) External development expenses by program: Nex-z $ 90,458 $ 69,793 $ 20,665 30 % Lonvo-z 47,748 42,173 5,575 13 % Unallocated research and development expenses: Employee-related expenses 98,719 127,383 (28,664 ) -23 % Research materials and contracted services 34,356 69,370 (35,014 ) -50 % Facility-related expenses 64,409 59,397 5,012 8 % Stock-based compensation 49,440 94,230 (44,790 ) -48 % Other 3,731 3,965 (234 ) -6 % Total research and development expenses $ 388,861 $ 466,311 $ (77,450 ) -17 % 81 The decrease in research and development expenses for the year ended December 31, 2025 compared to the year ended December 31, 2024 was primarily attributable to: • a $20.7 million increase in external costs related to the development of nexiguran ziclumeran (“nex-z”, also referred to as NTLA-2001), one of our lead product candidates, primarily due to an increase in spend on contracted services and drug components; • a $5.6 million increase in external costs related to the development of lonvoguran ziclumeran (“lonvo-z”, also referred to as NTLA-2002), one of our lead product candidates, primarily due to an increase in spend on drug components and contracted services; • a $28.7 million decrease in employee-related expenses, primarily driven by a workforce reduction in January 2025; • a $35.0 million decrease in research materials and contracted services primarily driven by internal research and development expenses, contracted services and drug components. • a $5.0 million increase in facility-related expenses primarily related to rent expense; and • a $44.8 million decrease in stock-based compensation caused primarily by our reduced workforce and lower grant date fair values of share-based awards granted in the current period.
Net cash provided by financing activities Net cash provided by financing activities of $185.7 million during the year ended December 31, 2024 includes $176.9 million in net proceeds from at-the-market offerings, $5.9 million in cash received from the exercise of stock options and $3.0 million in cash received from the issuance of shares through our employee stock purchase plan.
Net cash provided by financing activities of $185.7 million during the year ended December 31, 2024 includes $176.9 million in net proceeds from at-the-market offerings, $5.9 million in cash received from the exercise of stock options and $3.0 million in cash received from the issuance of shares through our employee stock purchase plan.
Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our CRISPR/Cas9 technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance of our product candidates; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing, or other arrangements into which we may enter; maintaining good relationships with our 82 collaborators and licensors; maintaining, protecting, and expanding our portfolio of IP rights, including patents, trade secrets, and know-how; and attracting, hiring, and retaining qualified personnel.
Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our CRISPR/Cas9 technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance and pricing approvals for our product candidates; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing, or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, protecting, and expanding our portfolio of IP rights, including patents, trade secrets, and know-how; and attracting, hiring, and retaining qualified personnel.
If the consideration received in exchange for entering into a contract is in the form of noncash consideration, we are required to estimate the fair value of the noncash consideration received. If our estimates of the noncash consideration received are not appropriate it could impact the total amount of revenue recognized for the contract.
If the consideration received in exchange for entering into a contract is in the form of noncash consideration, we are required to estimate the fair value of the 85 noncash consideration received. If our estimates of the noncash consideration received are not appropriate it could impact the total amount of revenue recognized for the contract.
Outlook Based on our research and development plans and our expectations related to the progress of our programs, we expect that our cash, cash equivalents and marketable securities as of December 31, 2024, as well as research and cost reimbursement funding from our collaboration agreements, will enable us to fund our ongoing operating expenses and capital expenditure requirements into the first half of 2027, excluding any potential milestone payments or extension fees that could be earned and distributed under our collaboration agreements or any strategic use of capital not currently in the base case planning assumptions.
Outlook Based on our research and development plans and our expectations related to the progress of our programs, we expect that our cash, cash equivalents and marketable securities as of December 31, 2025, as well as research and cost reimbursement funding from our collaboration agreements, will enable us to fund our ongoing operating expenses and capital expenditure requirements into the second half of 2027, excluding any potential milestone payments or extension fees that could be earned and distributed under our collaboration agreements or any strategic use of capital not currently in the base case planning assumptions.
Information pertaining to fiscal year 2022 was included in our Annual Report on Form 10-K for the year ended December 31, 2023 under Part II, Item 7, “Management’s Discussion and Analysis of Financial Position and Results of Operations,” which was filed with the Securities and Exchange Commission (the “SEC”) on February 22, 2024. Management Overview Intellia Therapeutics, Inc.
Information pertaining to fiscal year 2023 was included in our Annual Report on Form 10-K for the year ended December 31, 2024 under Part II, Item 7, “Management’s Discussion and Analysis of Financial Position and Results of Operations,” which was filed with the Securities and Exchange Commission (the “SEC”) on February 27, 2025. Management Overview Intellia Therapeutics, Inc.
The increase in the year ended December 31, 2024 is primarily due to $131.3 million in marketable securities that matured (net of purchases), offset in part by $5.8 million in cash used for the purchase of property and equipment. During the year ended December 31, 2023 we used cash of $31.3 million in investing activities.
The increase in the year ended December 31, 2024 is primarily due to $131.3 million in marketable securities that matured (net of purchases), offset in part by $5.8 million in cash used for the purchase of property and equipment.
Forfeitures are recorded as they occur. 85 The fair value of market-based restricted stock units and performance-based restricted stock units with a Total Shareholder Return (“TSR”) multiplier are determined using a Monte Carlo simulation model, which uses multiple input variables to determine the probability of satisfying the market condition requirements.
The fair value of market-based restricted stock units and performance-based restricted stock units with a Total Shareholder Return (“TSR”) multiplier are determined using a Monte Carlo simulation model, which uses multiple input variables to determine the probability of satisfying the market condition requirements.
Estimates of stock-based compensation expense for an award with a performance condition are based on our assessment of the probability that the performance condition will be achieved, which requires significant judgment. Our stock price is a key input that will drive the grant date fair value of the equity awards.
Estimates of stock-based compensation expense for an award with a performance condition are based on our assessment of the probability that the performance condition will be achieved, which requires significant judgment. Our stock price is a key input in determining the grant date fair value of equity awards. Forfeitures are recorded as they occur.
As of December 31, 2024, we had $861.7 million in cash, cash equivalents and marketable securities. 81 At-the-Market Offering Programs 2022 Sale Agreement In 2022, we entered into an Open Market Sale Agreement (the “2022 Sale Agreement”) with Jefferies LLC (“Jefferies”), under which Jefferies is able to offer and sell, from time to time in “at-the-market” offerings, shares of our common stock having aggregate gross proceeds of up to $400.0 million.
At-the-Market Offering Programs 2022 Sale Agreement In 2022, we entered into an Open Market Sale Agreement (the “2022 Sale Agreement”) with Jefferies LLC (“Jefferies”), under which Jefferies is able to offer and sell, from time to time in “at-the-market” offerings, shares of our common stock having aggregate gross proceeds of up to $400.0 million.
To the extent that we raise additional capital through the future sale of equity, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders.
Except for these sources of funding, we will not have any committed external source of liquidity. To the extent that we raise additional capital through the future sale of equity, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders.
Liquidity and Capital Resources Since our inception through December 31, 2024, we have funded our operations through our initial public offering and concurrent private placements, follow-on public offerings, our collaboration agreements, at-the-market offerings and the sale of convertible preferred stock.
Liquidity and Capital Resources Since our inception through December 31, 2025, we have funded our operations through our initial public offering and concurrent private placements, follow-on public offerings, our collaboration agreements, at-the-market offerings and the sale of convertible preferred stock. As of December 31, 2025, we had $605.1 million in cash, cash equivalents and marketable securities.
Net cash provided by financing activities of $130.3 million during the year ended December 31, 2023 includes $119.8 million in net proceeds from at-the-market offerings, $6.6 million in cash received from the exercise of stock options and $3.9 million in cash received from the issuance of shares through our employee stock purchase plan.
Net cash provided by financing activities Net cash provided by financing activities of $131.5 million during the year ended December 31, 2025 includes $128.2 million in net proceeds from at-the-market offerings, $2.6 million in cash received from the issuance of shares through our employee stock purchase plan and $0.7 million in cash received from the exercise of stock options.
To date through December 31, 2024 we have issued 14,522,533 shares of our common stock under the 2022 Sale Agreement, as amended.
To date through December 31, 2025 we have issued 26,313,157 shares of our common stock under the 2022 Sale Agreement, as amended.
Cash Flows The following is a summary of cash flows: Year Ended December 31, 2024 2023 (In thousands) Net cash used in operating activities $ (348,880 ) $ (394,086 ) Net cash provided by (used in) investing activities 125,567 (31,347 ) Net cash provided by financing activities 185,747 130,323 Net cash used in operating activities Net cash used in operating activities of $348.9 million during the year ended December 31, 2024 primarily consists of a net loss of $519.0 million, further reduced by the non-cash recognition of $21.0 million of previously eliminated intra-entity profit recorded within “collaboration revenue” and accretion of investment discounts and premiums of $17.8 million.
Net cash used in operating activities of $348.9 million during the year ended December 31, 2024 primarily consists of a net loss of $519.0 million, further reduced by the non-cash recognition of $21.0 million of previously eliminated intra-entity profit recorded within “collaboration revenue” and accretion of investment discounts and premiums of $17.8 million.
Until such time as we can generate substantial product revenues, if ever, we expect to fund our ongoing cash needs through equity financings and collaboration arrangements. We receive cost reimbursements from Regeneron related to our collaboration agreements with them.
Until such time as we can generate substantial product revenues, if ever, we expect to fund our ongoing cash needs through equity financings and collaboration arrangements. We receive cost reimbursements from Regeneron related to our collaboration agreements with Regeneron. Additionally, we are eligible to earn milestone payments and royalties upon achievement of certain events under our collaboration agreements.
During 2025, we expect our expenses to decrease compared to prior periods as a result of our recently announced strategic reorganization in January 2025, as we focus resources on high value programs within our pipeline, such as NTLA-2002 and nex-z, to ensure efficient execution, achieve near-term clinical milestones, and prepare for commercial launch.
During 2026, we expect our research and development expenses to decrease compared to prior periods as we focus resources on high value programs within our pipeline, such as lonvo-z and nex-z, to ensure efficient execution, achieve near-term clinical milestones, and prepare for commercial launch.
Net cash provided by (used in) investing activities During the year ended December 31, 2024, we added $125.6 million of net cash through investing activities.
Net cash provided by investing activities During the year ended December 31, 2025, we added $228.0 million of net cash through investing activities.
Also included in general and administrative expenses are allocated facility-related costs not otherwise included in research and development expenses, travel expenses and professional fees for auditing, tax and legal services, including IP-related legal services, and other consulting fees and expenses. 79 Other Income (Expense), Net During the year ended December 31, 2024, other income (expense), net consists of interest income earned on our cash, cash equivalents, restricted cash equivalents and marketable securities and change in the fair value of our investments.
Also included in general and administrative expenses are allocated facility-related costs not otherwise included in research and development expenses, adjustments related to impairment or accelerated amortization of long-lived assets, ongoing expenses related to the buildout of our commercial infrastructure, travel expenses and professional fees for auditing, tax and legal services, including IP-related legal services, and other consulting fees and expenses. 80 Other Income, Net Other income, net consists of interest income earned on our cash, cash equivalents, restricted cash equivalents and marketable securities and change in the fair value of our investments.
Funding Requirements Our primary uses of capital are, and we expect will continue to be, research and development research materials and contracted services, clinical trial costs, compensation and related expenses, laboratory and office facilities, research supplies, legal and regulatory expenses, patent prosecution filing and maintenance costs for our licensed IP, milestone and royalty payments and general overhead costs.
As of December 31, 2025, $117.7 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended. 82 Funding Requirements Our primary uses of capital are, and we expect will continue to be, research and development research materials and contracted services, clinical trial costs, compensation and related expenses, laboratory and office facilities, research supplies, legal and regulatory expenses, patent prosecution filing and maintenance costs for our licensed IP, commercial launch capabilities, milestone and royalty payments and general overhead costs.
We only apply the five-step model to contracts when we determine that collection of substantially all consideration for goods and services that are transferred is probable based on the customer’s intent and ability to pay the promised consideration. 84 As of December 31, 2024, our revenue recognized is solely related to collaboration agreements with third parties which are either within the scope of ASC 606, under which we license certain rights to our product candidates to third parties, or within the scope of ASC 808, Collaborative Arrangements (“ASC 808”) if it involves a joint operating activity pursuant to which we are an active participant and are exposed to significant risks and rewards with respect to the arrangement.
As of December 31, 2025, our revenue is solely related to collaboration agreements with third parties which are either within the scope of ASC 606, under which we license certain rights to our product candidates to third parties, or within the scope of ASC 808, Collaborative Arrangements (“ASC 808”) if it involves a joint operating activity pursuant to which we are an active participant and are exposed to significant risks and rewards with respect to the arrangement.
During the year ended December 31, 2024, we issued 7,004,370 shares of our common stock, in a series of sales, at an average price of $25.68 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $174.8 million, after payment of cash commissions and approximately $0.3 million related to legal, accounting and other fees in connection with the sales.
During the year ended December 31, 2025, we issued 11,790,624 shares of our common stock, in a series of sales, at an average price of $11.15 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $128.2 million, after commissions.
For additional information on our leases and timing of future payments refer to Note 11 of the consolidated financial statements included in this Annual Report on Form 10-K. 83 Other Obligations We enter into contracts in the normal course of business with various third parties for clinical trials, preclinical research studies, supply manufacturing and other services and products for operating purposes.
Other Obligations We enter into contracts in the normal course of business with various third parties for clinical trials, preclinical research studies, supply manufacturing and other services and products for operating purposes.
Contractual Obligations We have entered into arrangements that contractually obligate us to make payments that will affect our liquidity and cash flows in future periods. Property Leases As of December 31, 2024, our total undiscounted future minimum lease payments for our property leases that have commenced were $294.8 million, which will be paid over the term of such leases.
Property Leases - Commenced As of December 31, 2025, our total undiscounted future minimum lease payments for our property leases that have commenced were $111.2 million, which will be paid over the term of such leases.
(“Regeneron”), offset by a $12.3 million decrease in revenue related to the AvenCell LCA. Refer to Note 9 to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K for further details.
(the “AvenCell LCA”) in the year ended December 31, 2024. Refer to Notes 9 and 10 to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K for further details.
Comparison of Year Ended December 31, 2024 and 2023 The following table summarizes our results of operations: Year Ended December 31, Period-to- 2024 2023 Period Change (In thousands) Collaboration revenue $ 57,877 $ 36,275 $ 21,602 Operating expenses: Research and development 466,311 435,069 31,242 General and administrative 125,829 116,497 9,332 Total operating expenses 592,140 551,566 40,574 Operating loss (534,263 ) (515,291 ) (18,972 ) Other income (expense), net: Interest income 47,807 49,832 (2,025 ) Change in fair value of investments, net (32,565 ) - (32,565 ) Loss from equity method investment - (15,633 ) 15,633 Change in fair value of contingent consideration - (100 ) 100 Total other income (expense), net 15,242 34,099 (18,857 ) Net loss $ (519,021 ) $ (481,192 ) $ (37,829 ) Collaboration Revenue Collaboration revenue increased by $21.6 million to $57.9 million during the year ended December 31, 2024, as compared to $36.3 million during the year ended December 31, 2023.
Comparison of Year Ended December 31, 2025 and 2024 The following table summarizes our results of operations: Year Ended December 31, Period-to- 2025 2024 Period Change (In thousands) Collaboration revenue $ 67,671 $ 57,877 $ 9,794 Operating expenses: Research and development 388,861 466,311 (77,450 ) General and administrative 119,800 125,829 (6,029 ) Total operating expenses 508,661 592,140 (83,479 ) Operating loss (440,990 ) (534,263 ) 93,273 Other income, net: Interest income 29,195 47,807 (18,612 ) Change in fair value of investments, net (899 ) (32,565 ) 31,666 Total other income, net 28,296 15,242 13,054 Net loss $ (412,694 ) $ (519,021 ) $ 106,327 Collaboration Revenue Collaboration revenue increased by $9.8 million to $67.7 million during the year ended December 31, 2025, as compared to $57.9 million during the year ended December 31, 2024.
The increase in collaboration revenue during the year ended December 31, 2024 is primarily due to the recognition of $21.0 million of previously eliminated intra-entity profit under our license and collaboration agreement with AvenCell Therapeutics, Inc. (the “AvenCell LCA”) and a $12.8 million increase in revenue related to Regeneron Pharmaceuticals, Inc.
The increase in collaboration revenue during the year ended December 31, 2025 is due to a $23.7 million increase, primarily in cost reimbursements related to our collaboration with Regeneron Pharmaceuticals, Inc.
The decrease in the year ended December 31, 2023 is primarily due to $17.4 million in marketable securities purchased (net of maturities) and $14.0 million in cash used for the purchase of property and equipment.
The increase in the year ended December 31, 2025 is primarily due to $229.2 million in marketable securities that matured (net of purchases), offset in part by $1.1 million in cash used for the purchase of property and equipment. During the year ended December 31, 2024 we added $125.6 million of net cash through investing activities.
These contracts are generally cancelable at any time by us upon prior written notice. We do not include any potential future pass-through milestone payments or royalty payments we may be required to make under our existing license agreements or the merger agreement related to our acquisition of Rewrite Therapeutics, Inc.
These contracts are generally cancelable at any time by us upon prior written notice. 84 We are also party to license and other agreements, which may include contingent payments. As of December 31, 2025, the satisfaction and timing of the contingent payments is uncertain and not reasonably estimable.
(“we,” “us,” “our,” “Intellia,” or the “Company”) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. CRISPR is a gene editing technology which is also sometimes referred to as CRISPR/Cas or CRISPR/Cas9 when referring to the use of CRISPR technology with the Cas9 enzyme.
(“we,” “us,” “our,” “Intellia,” or the “Company”) is a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. Our mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments.
General and Administrative General and administrative expenses increased by $9.3 million to $125.8 million during the year ended December 31, 2024, compared to $116.5 million during the year ended December 31, 2023. This increase was primarily related to an increase in stock-based compensation of $8.2 million.
Research and Development Research and development expenses decreased by $77.5 million to $388.9 million during the year ended December 31, 2025, as compared to $466.3 million during the year ended December 31, 2024.
Removed
Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia’s deep scientific, technical and clinical development experience, along with its people, are helping set the standard for a new class of medicine.
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With deep scientific, technical and clinical development experience, we aim to reset the standard for medicine by durably treating the root causes of disease. For over a decade, Intellia has applied its proprietary technologies and expertise, including CRISPR-based gene editing technologies, oligonucleotides, and lipid nanoparticles (“LNPs”), to develop novel, first-in-class product candidates.
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To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies.
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This includes the development of lonvoguran ziclumeran (“lonvo-z,” also referred to as NTLA-2002) for the treatment of hereditary angioedema (“HAE”) and nexiguran ziclumeran (“nex-z,” also referred to as NTLA-2001) for the treatment of transthyretin (“ATTR”) amyloidosis. These lead product candidates are the first in vivo genome editing product candidates into Phase 3 development.
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To fully realize the transformative potential of CRISPR/Cas9-based technologies, we are building a full-spectrum gene editing company, by leveraging our modular platform, to advance in vivo and ex vivo therapies for diseases with high unmet need by pursuing two primary approaches. For in vivo applications to address genetic diseases, we deploy CRISPR/Cas9 as the therapy.
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These systemically administered CRISPR-based candidates are designed to address diseases with high unmet need with a single intravenous (“IV”) infusion that is administered in an outpatient setting. Lonvo-z and nex-z are currently in Phase 3 clinical development, and we are preparing for the planned commercial launch of lonvo-z in the first half of 2027.
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Our in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. In addition, we are advancing ex vivo applications to address immuno-oncology and autoimmune diseases, where we use CRISPR/Cas9 as the tool to create the engineered cell therapy. For our ex vivo programs, CRISPR/Cas9 is used to engineer human cells outside the body.
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(“Regeneron”) and $9.0 million in revenue that was recognized as a result of the termination of our License, Collaboration and Option Agreement with SparingVision SAS (the “SparingVision LCA”), offset in part by the recognition of $21.0 million of previously eliminated intra-entity profit under our License and Collaboration Agreement with AvenCell Therapeutics, Inc.
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Our deep scientific, technical and clinical development experience, along with our robust intellectual property (“IP”) portfolio, have enabled us to unlock broad therapeutic applications of CRISPR/Cas9 and related technologies to create new classes of genetic medicine.
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Prior year external development expenses related to NTLA-3001 have been reclassified to “Research materials and contracted services” in the table above to conform with the current year’s presentation. General and Administrative General and administrative expenses decreased by $6.0 million to $119.8 million during the year ended December 31, 2025, compared to $125.8 million during the year ended December 31, 2024.
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During the year ended December 31, 2023, other income (expense), net consisted of interest income earned on our cash, cash equivalents, restricted cash equivalents and marketable securities, loss from our equity method investment and change in fair value of contingent consideration.
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This decrease was primarily related to a decrease in stock-based compensation offset in part by increased expenses related to the ongoing buildout of our commercial infrastructure and reductions to right-of-use assets for accelerated amortization and impairment.
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Other Income (Expense), Net The decrease in other income (expense), net of $18.9 million is primarily related to $32.6 million in expense due to the change in fair value of our investments in Kyverna Therapeutics, Inc.
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Other Income, Net The increase in other income, net of $13.1 million is primarily related to a $31.7 million change in unrealized losses of our investments in equity securities in the year ended December 31, 2025 as compared to the prior year, which included an unrealized loss of $27.0 million as a result of a reduction in the carrying value of our investment in AvenCell.
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(“Kyverna”) and AvenCell and a $2.0 million decrease in interest income, offset in part by a $15.6 million change related to our equity method loss recorded in the year ended December 31, 2023.
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The increase was offset in part by an $18.6 million decrease in interest income due to lower average cash and marketable securities balances in the current year.
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As of December 31, 2024, $249.1 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended.
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We expect the decrease in research and development expenses to be mostly offset by increased expenses in 2026 related to our ongoing buildout of a commercial infrastructure.
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Additionally, we are eligible to earn milestone payments and royalties, in each case, on a per-product basis under our collaborations with SparingVision SAS (“SparingVision”), ONK Therapeutics, Ltd. (“ONK”) and ReCode Therapeutics, Inc.
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Cash Flows The following is a summary of cash flows: Year Ended December 31, 2025 2024 (In thousands) Net cash used in operating activities $ (394,736 ) $ (348,880 ) Net cash provided by investing activities 228,031 125,567 Net cash provided by financing activities 131,486 185,747 83 Net cash used in operating activities Net cash used in operating activities of $394.7 million during the year ended December 31, 2025 primarily consists of a net loss of $412.7 million, further reduced by accretion of investment discounts and premiums of $5.9 million and net changes in operating assets and liabilities of $73.8 million.
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(“ReCode”), on a per-target basis under our collaboration with Regeneron, and upon achievement of certain events with Kyverna, subject to the provisions of our agreements with each of them. Except for these sources of funding, we will not have any committed external source of liquidity.
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Included in the net cash used in operating activities is approximately $65.0 million of non-recurring cash payments associated with our previously announced portfolio prioritization, workforce reduction, and real estate consolidation.
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Net cash used in operating activities of $394.1 million during the year ended December 31, 2023 primarily consists of a net loss of $481.2 million, further reduced by changes in operating assets and liabilities of $52.5 million, including the receipt of $18.7 million in payments from our collaboration partners during that period and offset in part by non-cash charges of stock-based compensation of $134.1 million, loss on equity method investment of $22.3 million and depreciation of $9.0 million.
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These decreases are offset in part by stock-based compensation of $80.2 million, $6.2 million for accelerated amortization and impairment of right-of-use assets, $0.9 million in net adjustments to the fair value of our investments in Kyverna and SparingVision, and depreciation of $9.8 million.
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(“Rewrite”) due to the uncertainty of the occurrence of the events requiring payment under those agreements. These payments are not reflected in the disclosures above. In January 2023, a research milestone related to Rewrite was achieved and settled.
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Contractual Obligations We have entered into arrangements that contractually obligate us to make payments that will affect our liquidity and cash flows in future periods.
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For additional information on our leases and timing of future payments refer to Note 11, “Leases”, of the consolidated financial statements included in this Annual Report on Form 10-K. Property Leases - Not Yet Commenced In February 2025, we entered into a lease agreement for office and laboratory space at 400 Technology Square in Cambridge, Massachusetts.
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In connection therewith, we have committed to making at least $195.1 million in rental payments over a lease term of 147 months estimated to begin in the second half of 2026.
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We only apply the five-step model to contracts when we determine that collection of substantially all consideration for goods and services that are transferred is probable based on the customer’s intent and ability to pay the promised consideration.

Item 7A. Quantitative and Qualitative Disclosures About Market Risk

Market Risk — interest-rate, FX, commodity exposure

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Biggest changeAs of December 31, 2024, we had cash equivalents, restricted cash equivalents and marketable securities of $739.4 million consisting of interest-bearing money market accounts, corporate and financial institution debt securities, U.S. Treasury and other government securities and asset-backed securities.
Biggest changeAs of December 31, 2025, we had cash equivalents, restricted cash equivalents and marketable securities of $541.9 million consisting of interest-bearing money market accounts, corporate and financial institution debt securities and U.S. Treasury and other government securities.
Due to the short-term duration of our investment portfolios and the low risk profile of our investments, we do not believe an immediate change of 100 basis points, or one percentage point, would have a material effect on the fair market value of our investment portfolio. Declines in interest rates, however, would reduce future interest income.
Due to the short-term duration of our investment portfolios and the low risk profile of our investments, we 86 do not believe an immediate change of 100 basis points, or one percentage point, would have a material effect on the fair market value of our investment portfolio. Declines in interest rates, however, would reduce future interest income.
We do not have any foreign currency or derivative financial instruments. Inflation generally affects us by increasing our cost of labor, preclinical and clinical trial costs. We do not believe that inflation had a material effect on our results of operations during the year ended December 31, 2024. Item 8.
We do not have any foreign currency or derivative financial instruments. Inflation generally affects us by increasing our cost of labor and program costs. We do not believe that inflation had a material effect on our results of operations during the year ended December 31, 2025. Item 8.

Other NTLA 10-K year-over-year comparisons