Intellia Therapeutics, Inc.

Notes to Condensed Consolidated Financial Statements (unaudited)

1. Overview and Basis of Presentation

Intellia Therapeutics, Inc. (“Intellia,” or the “Company”) is a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. The Company’s mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments. With deep scientific, technical and clinical development experience, Intellia aims 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. This includes the development of lonvoguran ziclumeran (“lonvo-z,” previously referred to as NTLA-2002) for the treatment of hereditary angioedema (“HAE”) and nexiguran ziclumeran (“nex-z,” previously referred to as NTLA-2001) for the treatment of transthyretin (“ATTR”) amyloidosis. These lead product candidates are the firstin vivo genome editing product candidates to advance into Phase 3 clinical development. 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. On September 8, 2026, the Company announced that the U.S. Food and Drug Administration (“FDA”) has accepted the Biologics License Application (“BLA”) for lonvo-z and granted the BLA Priority Review with a Prescription Drug User Fee Act (“PDUFA”) target action date of March 10, 2027.

The condensed consolidated financial statements of the Company included herein have been prepared, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these condensed consolidated financial statements should be read in conjunction with the financial statements and notes thereto included in the Company’s Annual Report on Form 10-K (“Annual Report”) for the year ended December 31, 2025.

The unaudited condensed consolidated financial statements include the accounts of Intellia Therapeutics, Inc. and its wholly owned subsidiary, Intellia Securities Corp. All intercompany balances and transactions have been eliminated in consolidation. Comprehensive loss is comprised of net loss and unrealized gain/loss on marketable securities.

The preparation of financial statements in conformity with U.S. 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 condensed consolidated financial statements have been made in connection with the calculation of revenues, research and development expenses and stock-based compensation expense. The Company bases its estimates on historical experience and various other assumptions that management believes to be reasonable under the circumstances at the time such estimates are made. Actual results could differ from those estimates. The Company periodically reviews its estimates in light of changes in circumstances, facts and experience. The effects of material revisions in estimates, if any, would be reflected in the condensed consolidated financial statements prospectively from the date of the change in estimate.

In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period.

Liquidity

Since its inception through September 30, 2026, the Company has funded its operations through its initial public offering (“IPO”) and concurrent private placements, follow-on public offerings, at-the-market offerings, the sale of convertible preferred stock and debt financing, as well as through its collaboration agreements. The Company expects that its cash, cash equivalents and marketable securities as of September 30, 2026 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 condensed consolidated financial statements.

2. Summary of Significant Accounting Policies

The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies,” to the consolidated financial statements included in its Annual Report for the year ended December 31, 2025. Since the date of those financial statements, except as set forth below under “Debt,” there have been no material changes to these policies in the nine months ended September 30, 2026.

Debt

The Company accounts for debt instruments in accordance with the Financial Accounting Standards Board’s (“FASB”) Accounting Standards Codification (“ASC”) No. 470, Debt. Debt is initially recorded at the amount of cash proceeds received, adjusted for debt discounts, premiums, and issuance costs, and is subsequently measured at amortized cost using the effective interest method. Debt is classified as current or noncurrent based on the contractual maturity date and the absence or presence of conditions that would require repayment within twelve months of the balance sheet date.

The Company’s financing arrangements may include non-revolving delayed draw commitments. Fees paid at inception of a delayed draw arrangement are allocated proportionally among the funded and unfunded tranches based on commitment amounts. Amounts allocated to funded tranches are recorded as a direct deduction of the carrying amount of the funded debt and amortized over the term of the loan using the effective interest method. Amounts allocated to unfunded tranches are deferred and included as other noncurrent assets on the Company’s condensed consolidated balance sheet until the related tranche is drawn, at which point a proportionate amount is reclassified as a direct deduction of the carrying amount of the issued debt. If it becomes probable that all or a portion of a loan commitment will not be drawn, the related portion of the loan commitment asset is expensed immediately. Fees payable upon repayment or maturity are included in the determination of the effective interest rate and are accreted into the carrying value of the debt over its term.

Debt arrangements are evaluated for embedded features that may require bifurcation and separate accounting under ASC No. 815, Derivatives and Hedging. Embedded features that meet the definition of a derivative and are not clearly and closely related to the debt host are bifurcated unless a scope exception applies. If bifurcation is required, embedded derivatives are initially and subsequently measured at fair value, with changes in fair value recognized in earnings.

Recently Issued Accounting Pronouncements Not Yet Effective

In November 2024, the FASB issued Accounting Standards Update (“ASU”) No. 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses. This ASU requires disclosure of specified information about certain costs and expenses in the footnotes to the financial statements. This ASU is effective for annual periods beginning after December 15, 2026 and is applicable to the Company’s fiscal year beginning January 1, 2027, with early application permitted. The Company is currently evaluating the impact of adopting this ASU on its consolidated financial statements and disclosures.

In December 2025, the FASB issued ASU No. 2025-12, Codification Improvements. This ASU’s purpose is to update the ASC for a broad range of topics in order to clarify, correct errors, or make minor improvements that make the ASC easier to understand and apply. This ASU is effective for interim reporting periods within annual periods beginning after December 15, 2026 and is applicable to the Company’s fiscal year beginning January 1, 2027. The Company is currently evaluating the impact of adopting this ASU on its consolidated financial statements and disclosures.

3. Marketable Securities

The following table summarizes the Company’s available-for-sale marketable securities:

The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. There were no material realized gains or losses in the nine months ended September 30, 2026 or the year ended December 31, 2025. The Company did not reclassify any amounts out of accumulated other comprehensive income (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. As such, the Company has classified these losses as temporary in nature.

The Company’s available-for-sale securities that are classified as short-term marketable securities in the condensed consolidated balance sheets mature within one year or less as of the balance sheet date. Available-for-sale securities that are classified as noncurrent in the condensed consolidated balance sheets are those that mature afterone year but within five years from the balance sheet date and that the Company does not intend to dispose of within the next twelve months. At September 30, 2026 and December 31, 2025, the Companydidnot 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 condensed consolidated balance sheets.

4. Fair Value Measurements

The Company classifies fair value-based measurements using a three-level hierarchy that prioritizes the inputs used to measure fair value. This hierarchy requires entities to maximize the use of observable inputs and minimize the use of unobservable inputs. The three levels of inputs used to measure fair value are as follows: Level 1, quoted market prices (unadjusted) in active markets for identical assets or liabilities; Level 2, observable inputs other than quoted market prices included in Level 1, such as quoted market prices for markets that are not active or other inputs that are observable or can be corroborated by observable market data; and Level 3, unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities, including certain pricing models, discounted cash flow methodologies and similar techniques that use significant unobservable inputs.

Certain of the Company’s financial assets, including cash equivalents, restricted cash equivalents and marketable securities, have been initially valued at the transaction price, and subsequently revalued at the end of each reporting period, utilizing third party pricing services or other observable market data. The pricing services utilize industry standard valuation models and observable market inputs to determine value.

The Company’s financial assets recognized at fair value on a recurring basis consisted of the following:

Other financial instruments, including accounts receivable, accounts payable and accrued expenses, are carried at cost, which approximates fair value due to the short duration and term to maturity.

The Company’s debt is considered a Level 2 financial liability. The fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. As of September 30, 2026, the carrying amount of the debt reasonably approximated the estimated fair value. Refer to Note 10, “Debt,” for further details.

The Company has determined that the estimated fair value of its investment in Kyverna Therapeutics, Inc. (“Kyverna”), a publicly traded company, is reported as Level 1 as it is valued at a quoted market price in an active market. The investment in Kyverna is classified within “Investments and other assets” in the condensed consolidated balance sheets. Refer to Note 8, “Investments and Other Assets,” for further details.

5. Accrued Expenses

Accrued expenses consisted of the following:

6. Commitments and Contingencies

Litigation

From time to time, the Company may be involved in legal and administrative proceedings and claims of various types. In some actions, the claimants seek damages, as well as other relief, which, if granted, would require significant expenditures. The Company records a liability in its consolidated financial statements for these matters when a loss is known or considered probable and the amount can be reasonably estimated. The Company reviews these estimates each accounting period as additional information is known and adjusts the loss provision when appropriate. If a matter is both probable to result in a liability to the Company and the amount of the loss can be reasonably estimated, the Company estimates and discloses the possible loss or range of loss. If the loss is not probable or cannot be reasonably estimated, a liability is not recorded in its consolidated financial statements.

BlueAllele Corp. v. Intellia Therapeutics, Inc.

On July 8, 2024, BlueAllele Corp. (“BlueAllele”) filed a complaint alleging infringement by the Company of various patents in the U.S. District Court for the District of Delaware. 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. On September 12, 2024, the Company filed a motion to dismiss the complaint, and on December 9, 2024, the court denied the Company’s motion to dismiss and discovery began. On January 6, 2025, the Company filed its answer and counterclaims, and BlueAllele filed a motion to dismiss the Company’s counterclaims on January 27, 2025. 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.

Aiello v. Bhanji et al.

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. 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, allegedly excessive compensation to the Company’s non-employee directors. 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. 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 three and nine months ended September 30, 2026, there have been no material changes to any outstanding litigation, nor is the Company a party to any material new litigation.

License and Other Agreements

The Company is party to license and other agreements, which may include contingent payments. These payments could include up to $130.0 million related to Rewrite Therapeutics, Inc. (“Rewrite”), including $100.0 million upon achievement of a regulatory approval milestone and $30.0 million upon achievement of pre-specified research milestones. As of September 30, 2026, the satisfaction and timing of the contingent payments is uncertain and not probable.

7. Collaborations and Other Arrangements

To accelerate the development and commercialization of gene editing products in multiple therapeutic areas, the Company has formed, and intends to seek other opportunities to form, strategic alliances with collaborators who can augment its leadership in CRISPR/Cas9 therapeutic development. As of September 30, 2026 and December 31, 2025, the Company’s accounts receivable and contract liabilities were related to its collaboration with Regeneron Pharmaceuticals, Inc. (“Regeneron”).

The following table presents changes in the Company’s accounts receivable and contract liabilities (in thousands):

The Company recognized the following revenues as a result of changes in the contract liability balance (in thousands):

The Company has not incurred significant expenses to obtain collaboration agreements and costs to fulfill those contracts do not generate or enhance resources of the Company. As such, no costs to obtain or fulfill a contract have been capitalized in any period.

Regeneron Pharmaceuticals, Inc.

In April 2016, the Company entered into a license and collaboration agreement with Regeneron (as amended from time to time, the “2016 Regeneron Agreement”), which consisted of a product development component and a technology collaboration component. In October 2023, Regeneron exercised its one-time option to extend the term of the technology collaboration until April 2026, in exchange for a nonrefundable payment of $30.0 million that was paid in April 2024. The Company did not recognize any collaboration revenue in thethree months ended September 30, 2026 related to the 2016 Regeneron Agreement. The Company recognized $5.8 million of collaboration revenue in the nine months ended September 30, 2026, and$5.3 million and $15.6 million during thethree and nine months ended September 30, 2025, respectively, in the condensed consolidated statements of operations and comprehensive loss related to the 2016 Regeneron Agreement.

In 2018, the Company entered into a co-development and co-promotion (“Co/Co”) agreement with Regeneron for ATTR amyloidosis (the “ATTR Co/Co”). In May 2020, the Company entered into co-development and co-funding agreements for the treatment of hemophilia A and hemophilia B (the “Hemophilia A Co/Co” and the “Hemophilia B Co/Co” agreements). The Hemophilia B Co/Co ended in September 2024. The Company recognized $6.8 million and $21.8 million of collaboration revenue in thethree and nine months ended September 30, 2026, respectively, and $8.4 million and $23.3 million of collaboration revenue in thethree and nine months ended September 30, 2025, respectively, representing payments due from Regeneron pursuant to the ATTR Co/Co agreement. The Company didnot incur material costs related to the Hemophilia A Co/Co agreement during thethree and nine months ended September 30, 2026 and 2025. Since December 31, 2025, there have been no material changes to the key terms of the 2016 Regeneron Agreement, ATTR Co/Co or Hemophilia A Co/Co.

As of September 30, 2026 and December 31, 2025, the Company had accounts receivable of $7.5 million and $9.5 million, respectively, related to the Regeneron agreements. The Company hadno deferred revenue as of September 30, 2026, and deferred revenue of $7.3 million as of December 31, 2025, related to the Regeneron agreements.

Other Agreements

The Company has existing license and collaboration agreements with AvenCell Therapeutics, Inc. (“AvenCell”), Kyverna, and ONK Therapeutics, Ltd. (“ONK”). Since December 31, 2025, there have been no material changes to the key terms of the AvenCell, Kyverna and ONK license and collaboration agreements. The Company hadno material revenue from these collaborations during the three and nine months ended September 30, 2026 and 2025.

8. Investments and Other Assets

Investments and other assets consisted of the following:

Kyverna Therapeutics, Inc.

As of September 30, 2026 and December 31, 2025, the Company’s investment in Kyverna is valued at$8.0 million and $11.0 million, respectively. The Company recognized an unrealized loss of$2.5 million and$3.1 million, respectively, recorded within “Change in fair value of investments, net” in the condensed consolidated statements of operations and comprehensive loss during the three and nine months ended September 30, 2026, associated with changes in the fair value of Kyverna’s common stock. The Company recognized an unrealized gain of $3.4 million and $2.7 million, respectively, recorded within “Change in fair value of investments, net”in the condensed consolidated statements of operations and comprehensive loss during the three and nine months ended September 30, 2025, associated with changes in the fair value of Kyverna’s common stock.

Other Investments

The Company’s other investments consist of investments in private companies including SparingVision SAS (“SparingVision”) and AvenCell, both of which are accounted for under ASC No. 321, Investments in Equity Securities, using the measurement alternative at cost minus impairment, adjusted for changes in observable prices. The carrying value of the Company’s investment in SparingVision included within “Other investments” in the table above was$3.5 million at September 30, 2026 and December 31, 2025. In the third quarter of 2026, AvenCell completed a Series C financing, which represented an observable price change in the investment in AvenCell. The Company recognized an unrealized loss of $7.9 million, recorded within “Change in fair value of investments, net”in the condensed consolidated statement of operations and comprehensive loss during the three and nine months ended September 30, 2026.

9. Leases

In February 2025, as part of its efforts to streamline its real estate portfolio, the Company entered into a Second Amendment (the “Winter Street Amendment”) which will terminate the lease entered into by the Company in 2022 for space at 840 Winter Street, Waltham, Massachusetts (the “840 Winter Lease”) on or before June 30, 2028. In connection with the Winter Street Amendment, the Company paid the landlord lease modification payments totaling $78.0 million in three installments, with $34.0 million paid in February 2025, $30.0 million paid in April 2025 and $14.0 million paid in January 2026. In conjunction with the Winter Street Amendment, also in February 2025, the Company entered into a lease agreement (the “Tech Square Lease”) with the same landlord as the 840 Winter Lease, for office and laboratory space located at 400 Technology Square, Cambridge, Massachusetts (“400 Tech Square”).

Under the terms of the Tech Square Lease and subsequent amendments, the Company will lease approximately 125,000 square feet of office and laboratory space at 400 Tech Square. In September 2026, the Company determined, in accordance with ASC No. 842, Leases (Topic 842) that the criteria for commencement of the Tech Square Lease had been met as the lessor had made the space available for the Company’s use. The Company recorded a right of use asset of $136.9 million and a lease liability of $82.1 million related to the Tech Square Lease. The right-of-use asset includes $54.8 million of prepaid rent recognized prior to the commencement date. The initial term of the Tech Square Lease istwelve years andthree months, ending in November 2038. The Company has an option to extend the Tech Square Lease for an additional term of five years, which was not reasonably certain of exercise as of the commencement date and therefore not included in the measurement of the lease liability. The Tech Square Lease is subject to 3% fixed rate rent escalations and requires the Company to make monthly payments for operating costs such as real estate taxes, maintenance costs, and utilities. These costs are variable in nature and have therefore been excluded from the measurement of the lease liability and will be recorded as lease costs when they are incurred.

In August 2026, the Company entered into a Third Amendment to the Tech Square Lease which added approximately 23,000 square feet on the 5th floor of the 400 Tech Square building. The Company identified a separate lease component for this additional space (the “5th Floor Component”). The 5th Floor Component has not commenced as of September 30, 2026.

In April 2026 the Company amended its lease for 130 Brookline Street in Cambridge, Massachusetts to shorten the term from mid-2031 to December 31, 2026, in exchange for a $3.9 million fee payable in two equal installments, due in July 2026 and January 2027, which resulted in a reduction of the right-of-use asset from the remeasurement of the lease liability of approximately $2.1 million.

The table below reconciles the undiscounted cash flows for each of the next five years and total of the remaining years to the operating lease liabilities recorded in the condensed consolidated balance sheet as of September 30, 2026:

Excluded from the Future Operating Lease Payments table above is $33.9 million related to the 5th Floor Component of the Tech Square Lease, which had not yet commenced as of September 30, 2026.

10. Debt

On September 4, 2026 (the “Closing Date”), the Company entered into a Credit Agreement (the “Credit Agreement”) with OrbiMed Royalty & Credit Opportunities IV, LP and OrbiMed Royalty & Credit Opportunities V, LP, as the initial lenders (“Lenders”), and OrbiMed Royalty & Credit Opportunities V, LP, as the administrative agent. The Credit Agreement provides the Company with a five-year senior secured credit facility of up to $400 million (the “OrbiMed Credit Facility”), consisting of (i) an initial draw of $75 million on the Closing Date (the “Initial Term Loan”), (ii) several potential future loans (the “Delayed Draw Term Loans”) including a potential additional $75 million loan at the Company’s option upon the approval by the FDA of the Company’s BLA for lonvo-z prior to a certain date and three additional potential $40 million loans at the Company’s option upon achieving certain revenue targets for lonvo-z prior to certain dates, (iii) a potential additional $30 million loan at the Company’s option upon achieving an equity fundraising target prior to a certain date, and (iv) an uncommitted potential additional incremental loan up to $100 million subject to mutual agreement among the Company and the Lenders. The proceeds of the OrbiMed Credit Facility will be used for the working capital needs and general corporate purposes of the Company.

The OrbiMed Credit Facility matures on September 4, 2031 (the “Maturity Date”), and the entire then-outstanding principal amount of the loans will be due on the Maturity Date. Loans outstanding under the OrbiMed Credit Facility bear interest, payable monthly, at a rate per annum equal to (1) the greater of (a)3.00% or (b) the one-month Secured Overnight Financing Rate (“SOFR”) applicable to such period plus (2) an applicable margin of 6.15%. The Delayed Draw Term Loans are also subject to a fee on the undrawn amounts of 0.50% per annum. These fees will end when the relevant delayed-draw commitments are funded or terminated, which will be no later than March 31, 2029. Each Delayed Draw Term Loan commitment will expire if the applicable milestone condition is not satisfied by a specified date. In addition, the Company is required to pay certain customary commitment, administrative and facility fees in connection with the OrbiMed Credit Facility.

In connection with the issuance of the OrbiMed Credit Facility, the Company paid upfront and other fees to the lenders of $1.2 million and incurred debt issuance costs of $1.8 million. An upfront fee of $0.7 million paid to the lender was treated as a deduction to the carrying value of the Initial Term Loan. Debt discounts and issuance costs amounting to$2.3 million were then allocated to each of the components of the OrbiMed Credit Facility proportionally based on commitment amounts, resulting in $0.6 million being allocated to the Initial Term Loan and $1.7 million being allocated to the Delayed Draw Term Loans. The debt discounts and issuance costs allocated to the Initial Term Loan were recorded as a direct deduction of the carrying amount of the Initial Term Loan and are being amortized over the term of the loan using the effective interest rate. As of September 30, 2026, the effective interest rate was 11.1%. The debt discounts and issuance costs allocated to the Delayed Draw Term Loans were recorded to other noncurrent assets. When the Delayed Draw Term Loans are issued, a proportionate amount of the capitalized cost will be reclassified as a direct deduction of the carrying amount of the issued Delayed Draw Term Loan(s).

The Company may elect to prepay all or any portion of the outstanding loans prior to the Maturity Date, subject to payment of accrued and unpaid interest on the principal amount repaid or prepaid, an applicable repayment premium ranging from 0% to 3%, and/or an exit fee ranging from 3.75% to 4.75% of the principal amount repaid or prepaid, in each case based on the timing of repayment. Repayments or prepayments before the 13-month anniversary of the applicable loan closing date are also subject to a make-whole amount equal to specified remaining scheduled interest payments through that anniversary. The OrbiMed Credit Facility is also subject to customary mandatory prepayments from certain asset sales and casualty events.

All obligations under the Credit Agreement are secured on a first-priority basis, subject to certain exceptions, by security interests in substantially all assets of the Company, including its intellectual property. In addition, the Credit Agreement contains customary covenants, including, without limitation, (i) financial covenants to (1) maintain liquidity of at least $50 million in controlled accounts until the FDA approves the BLA for lonvo-z and (2) either achieve certain revenue targets, maintain certain market capitalization thresholds or maintain the outstanding loan principal in cash equivalents in controlled accounts, and (ii) negative covenants that, subject to certain exceptions, restrict the Company’s ability to incur additional indebtedness, grant liens, make investments (including acquisitions), effectuate mergers or consolidations, engage in asset sales and licensing transactions, pay dividends, terminate or modify certain material agreements, pay subordinated indebtedness, and undertake other matters customarily restricted in such agreements. The exceptions to incurring additional indebtedness and granting liens include an exception allowing the Company to enter synthetic royalty transactions, subject to certain restrictions in the Credit Agreement.

The Credit Agreement also contains certain events of default after which loans under the OrbiMed Credit Facility may be due and payable immediately, including payment defaults, material inaccuracy of representations and warranties, covenant defaults, bankruptcy and insolvency proceedings, cross-defaults to certain other agreements, judgments against the Company and its subsidiaries, and change of control.

The OrbiMed Credit Facility contains certain features that are not clearly and closely related to the host instrument and represent derivatives that are required to be remeasured at fair value each reporting period. The Company determined that the estimated fair value of the derivatives at issuance and through September 30, 2026 were not material.

As of September 30, 2026, the carrying amount of the Initial Term Loan, net of unamortized debt discount, issuance costs and exit fee, was approximately $73.7 million. The Initial Term Loan is carried at historical cost, adjusted for the debt discount, debt issuance costs and exit fee, and is not required to be carried at fair value. The principal balance of $75.0 million at September 30, 2026, is due on the Maturity Date, and the Initial Term Loan is classified as a noncurrent liability on the condensed consolidated balance sheet.

The following table summarizes the Company’s outstanding debt liability (in thousands):

The following table summarizes components of the Company’s debt related interest expense (in thousands):

11. Stock-Based Compensation

Stock-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows:

Stock Option and Incentive Plans

In April 2016, the Company adopted the Amended and Restated 2015 Stock Option and Incentive Plan (the “2015 Plan”). The 2015 Plan provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, restricted stock awards (“RSAs”), restricted stock units (“RSUs”) and other stock-based awards. Recipients of incentive stock options and non-qualified stock options are eligible to purchase shares of the Company’s common stock at an exercise price equal to the fair value of such stock on the grant date.

In June 2025, the Company adopted the 2025 Equity Incentive Plan (the “2025 Plan”). The 2025 Plan provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, RSAs, RSUs, unrestricted stock awards, cash-based awards and dividend equivalent rights. Recipients of incentive stock options and non-qualified stock options are eligible to purchase shares of the Company’s common stock at an exercise price equal to the fair value of such stock on the grant date. The Company no longer grants any awards under the 2015 Plan. The shares of common stock underlying any awards that are forfeited, cancelled, or otherwise terminated, other than by exercise, under the 2025 Plan and the 2015 Plan will be added back to the shares of common stock available for issuance under the 2025 Plan. As of September 30, 2026, there were 9,816,122 shares available for future issuance under the 2025 Plan.

In June 2024, the Company adopted the 2024 Inducement Plan, which was amended in December 2025 (as amended, the “Inducement Plan”). The Inducement Plan provides for the grant of non-qualified stock options, stock appreciation rights, RSAs, RSUs, unrestricted stock awards and dividend equivalent rights to individuals who are not employed by the Company. Recipients of non-qualified stock options are eligible to purchase shares of the Company’s common stock at an exercise price equal to the fair value of such stock on the grant date. At September 30, 2026,1,031,090 shares were available for issuance under the Inducement Plan.

Restricted Stock Units

The following table summarizes the Company’s RSU activity for thenine months ended September 30, 2026:

The weighted-average grant date fair value of all RSUs granted during the three and nine months ended September 30, 2026 was $13.31 and $14.43, respectively. The total fair value of RSUs vested (measured on the date of vesting) for thethree and nine months ended September 30, 2026 was $3.2 million and $18.6 million, respectively. The weighted-average grant date fair value of all RSUs granted during thethree and nine months ended September 30, 2025 was $10.22 and $10.48, respectively. The total fair value of RSUs vested (measured on the date of vesting) for thethree and nine months ended September 30, 2025 was $1.2 million and $20.2 million, respectively.

As of September 30, 2026, there was $50.6 million of unrecognized stock-based compensation expense related to all RSUs that are expected to vest. These costs are expected to be recognized over a weighted average remaining vesting period of 1.75 years.

Restricted Stock Units - Service Awards

The Company awards RSUs with a service condition to new employees upon hire, non-employee directors upon appointment, and to existing employees and non-employee directors as part of their annual grant. 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. RSUs granted to non-employee directors with a service condition as part of their annual grant generally vest on the first anniversary of the original vesting date.

In the nine months ended September 30, 2026, the Company granted 3,235,991 RSUs with a service condition to new and existing employees and non-employee directors, most of which have the potential to vest over a period of approximately three years. The weighted average grant date fair value of these RSUs was$13.64.

Unvested restricted stock units as of September 30, 2026 in the table above includes 5,223,689 RSUs that are service-based.

Restricted Stock Units - Market Awards

The Company awards market-based RSUs to senior executives which have the potential to vest after a period ofthree years, with a vesting start date of January 1 in the year of grant, and the number of shares to be delivered will depend on the Company’s Total Shareholder Return (“TSR”), a market condition, over that period relative to a defined group of biotechnology companies. The number of market-based RSUs granted in the nine months ended September 30, 2026 was 255,328 and their grant date fair value, calculated using a Monte Carlo valuation model, was $24.43. The following assumptions were used to determine the grant date fair value: risk free interest rate: 3.4%; expected volatility:80.9%. The expected term for these grants was approximately 3.0 years; the expected dividend yield was 0.0%.

Unvested restricted stock units as of September 30, 2026 in the table above includes 713,805 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. The number of PSUs with a relative TSR modifier granted in the year ended December 31, 2024 was 486,617. These PSUs, to the extent earned, shall vest on January 1, 2027, and the number of shares to be delivered will be determined based upon the achievement of certain performance goals, which can range from 0% to 200%. Following the determination of the achievement of performance criteria, the amount of shares awarded will be subject to adjustment based on the application of a TSR modifier, which can range from 75% to 125%. The grant date fair value for these PSUs, calculated using a Monte Carlo valuation model, was $36.16. The following assumptions were used to determine the grant date fair value: risk free interest rate:4.28%; expected volatility:77.2%; expected term: approximately 3.0 years; expected dividend yield:0.0%. The Company recognizes compensation expense ratably over the required service period based on its estimate of the number of shares that will vest based upon the probability of achieving the performance goals.

There wereno PSUs with a relative TSR modifier granted in thenine months ended September 30, 2026. Unvested restricted stock units as of September 30, 2026 in the table above includes 352,662 PSUs with a TSR multiplier.

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 $9.90 per option for those options granted during thenine months ended September 30, 2026. There wereno options granted during thethree months ended September 30, 2026. The weighted average grant date fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $6.88 and $6.80 per option for those options granted during thethree and nine months ended September 30, 2025, respectively. Weighted average assumptions used to apply this pricing model were as follows:

Risk-free Interest Rate. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant with maturities approximately equal to the option’s expected term.

Expected Term. The expected term represents the period that stock option awards are expected to be outstanding. For option grants that are considered to be “plain vanilla,” the Company determines the expected term using the simplified method. The simplified method deems the term to be the average of the time-to-vesting and the contractual life of the options. The Company uses the simplified method because it concluded that its stock option exercise history does not provide a reasonable basis upon which to estimate 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.

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. The maximum term of stock options granted under the 2015 Plan, the 2025 Plan and the Inducement Plan is ten years.

The following is a summary of stock option activity for thenine months ended September 30, 2026:

The total intrinsic value (the amount by which the fair market value exceeded the exercise price) of stock options exercised during the three and nine months ended September 30, 2026 and 2025 was not material.

As of September 30, 2026, there was $6.7 million of unrecognized compensation cost related to stock options that have not yet vested, which are expected to be recognized over a weighted average remaining vesting period of 1.2 years.

Employee Stock Purchase Plan

In May 2016, the Company adopted the 2016 Employee Stock Purchase Plan (the “2016 Plan”). The 2016 Plan allows eligible employees to purchase shares of the Company’s common stock on the last day of each predetermined six-month offering period at 85% of the lower of the fair market value per share at the beginning or end of the applicable offering period. The 2016 Plan provides for six-month offering periods beginning in January and July of each year.

As of September 30, 2026, there were 1,330,666 shares available for future issuance under the 2016 Plan. The number of shares reserved for issuance under the 2016 Plan was increased on January 1, 2026 by 500,000 shares of common stock.

During the nine months ended September 30, 2026 and 2025 the Company issued 194,213 and 155,172 shares of common stock, respectively, under the 2016 Plan. The weighted-average purchase prices of shares issued under the 2016 Plan were $7.83 and $7.97 per sharefor the nine-month periods ended September 30, 2026 and 2025, respectively.

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:

12. Loss Per Share

The Company calculates basic loss per share by dividing net loss for each respective period by the weighted average number of common shares outstanding for each respective period. The Company computes diluted loss per share after giving consideration to the dilutive effect of stock options and unvested restricted stock units that are outstanding during the period, except where such securities would be anti-dilutive.

Basic and diluted loss per share was calculated as follows:

The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive:

13. Stockholders’ Equity

Follow-on Public Offering

On April 28, 2026, the Company entered into an underwriting agreement related to a follow-on public offering of 16,744,187 shares of its common stock at a public offering price of $10.75 per share. Pursuant to the underwriting agreement, the Company granted a 30-day option to purchase up to an additional 2,511,628 shares of its common stock at the public offering price, less underwriting discounts and commissions. The option was exercised in full on April 29, 2026. The offering closed on April 30, 2026, for aggregate net proceeds of $194.6 million after deducting underwriting discounts and commissions.

At-the-Market Offering Programs

2022 Sale Agreement

In 2022, the Company entered into an Open Market Sale Agreement (the “2022 Sale Agreement”) with Jefferies LLC (“Jefferies”), under which Jefferies was able to offer and sell, from time to time in “at-the-market” offerings, shares of the Company’s common stock having aggregate gross proceeds of up to $400.0 million. In February 2024, the Company entered into an amendment to the 2022 Sale Agreement to increase the size of the at-the-market offering program from $400.0 million to $750.0 million. In March 2026, the Company entered into Amendment No. 2 to the 2022 Sale Agreement (the “2022 Sale Agreement, as amended”) to increase the size of the at-the-market offering program from $750.0 million to $1,035.3 million. The Company agreed to pay cash commissions of up to 3.0% of the gross proceeds of sales of common stock under the 2022 Sale Agreement, as amended. Through September 30, 2026, the Company issued 28,925,501 shares of its common stock under the 2022 Sale Agreement, as amended. During the nine months ended September 30, 2026, the Company issued 2,612,344 shares of its common stock, in a series of sales, at an average price of$13.21 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of$33.6 million, after commissions and offering costs. During the nine months ended September 30, 2025, the Company issued 11,790,624 shares of its 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 payment of cash commissions. As of September 30, 2026,$368.5 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended.

14. Segment Information

The Company has identifiedone operating and reportable segment: the development of gene editing-based therapies. All of the Company’s material assets are held in the U.S. and all of the Company’s collaboration revenue has been generated in the U.S. The operating segment’s revenue is primarily generated through collaboration arrangements with third parties. The Company does not have any intra-entity sales or transfers. The Company manages all business activities on a consolidated basis. The Company’s chief operating decision maker (“CODM”) is thechief executive officer.

The accounting policies for the segment are the same as those described in Note 2, “Summary of Significant Accounting Policies,” in the Company’s Annual Report for the year ended December 31, 2025. 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. The CODM uses net loss to monitor budget versus actual results and to analyze cash flows in assessing performance of the segment and allocating resources. The measure of the operating segment assets is reported on the consolidated balance sheets as total assets.

The following table summarizes the reportable segment’s financial information:

(1)

Includes unallocated research and development expenses, including stock-based compensation of $7.4 million and $24.4 million for the three and nine months ended September 30, 2026, respectively, and $12.2 million and $38.9 million for the three and nine months ended September 30, 2025, respectively, as disclosed within Note 11, “Stock-Based Compensation.”

(2)

Includes stock-based compensation of $7.0 million and $21.6 million for the three and nine months ended September 30, 2026, respectively, and $7.4 million and $24.6 million for the three and nine months ended September 30, 2025, respectively, as disclosed within Note 11, “Stock-Based Compensation.”

(3)

Includes thechange in fair value of investments as disclosed in the Company’s condensed consolidated statements of operations and comprehensive loss.

Depreciation and amortization expense totaled $1.9 million and $6.2 million for the three and nine months ended September 30, 2026, respectively, and $2.4 million and $7.4 million for the three and nine months ended September 30, 2025, respectively.