Intellia Therapeutics, Inc.
For the period ended Mar 31, 2026. Filed May 11, 2026.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed May 11, 2026
Extracted filing text, with the paragraph anchors used by the comparison.
See the whole filing, with its images and exhibitsContents
Notes to Condensed Consolidated Financial Statements
Notes to Condensed Consolidated Financial Statements (unaudited)
- 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. In April 2026, the Company reported positive topline data from the global Phase 3 HAELO clinical trial of lonvo-z in HAE, and preparations are underway for the planned commercial launch of lonvo-z in the first half of 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 March 31, 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 and the sale of convertible preferred stock, as well as through its collaboration agreements. The Company expects that its cash, cash equivalents and marketable securities as of March 31, 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.
- 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. There have been no material changes to these policies in the three months ended March 31, 2026.
Recently Issued Accounting Pronouncements Not Yet Effective
In November 2024, the Financial Accounting Standards Board (“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 FASB’s Accounting Standard Codification (“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.
- Marketable Securities
The following table summarizes the Company’s available-for-sale marketable securities:
| AmortizedCost | Gross UnrealizedGains | Gross UnrealizedLosses | Estimated FairValue | |
|---|---|---|---|---|
| (In thousands) | ||||
| Marketable securities: | ||||
| U.S. Treasury and other government-backed securities | $192,071 | $51 | $(281) | $191,841 |
| Financial institution debt securities | 116,598 | 106 | (160) | 116,544 |
| Corporate debt securities | 74,119 | 106 | (60) | 74,165 |
| Total | $382,788 | $263 | $(501) | $382,550 |
| AmortizedCost | Gross UnrealizedGains | Gross UnrealizedLosses | Estimated FairValue | |
| (In thousands) | ||||
| Marketable securities: | ||||
| U.S. Treasury and other government-backed securities | $220,790 | $345 | $(18) | $221,117 |
| Financial institution debt securities | 146,375 | 458 | (18) | 146,815 |
| Corporate debt securities | 81,478 | 266 | (6) | 81,738 |
| Total | $448,643 | $1,069 | $(42) | $449,670 |
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 three months ended March 31, 2026 and 2025. The Company did not reclassify any amounts out of accumulated other comprehensive (loss) income 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 March 31, 2026 and December 31, 2025, the Company didnot 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.
- 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:
| Total · Level 1 · Level 2 · Level 3 | ||||||
|---|---|---|---|---|---|---|
| (In thousands) | ||||||
| Assets | ||||||
| Cash equivalents and restricted cash equivalents | $79,145 | $79,145 | $ | - | $ | - |
| Marketable securities: | ||||||
| U.S. Treasury and other government-backed securities | 191,841 | 129,324 | 62,517 | - | ||
| Financial institution debt securities | 116,544 | - | 116,544 | - | ||
| Corporate debt securities | 74,165 | - | 74,165 | - | ||
| Total marketable securities | 382,550 | 129,324 | 253,226 | - | ||
| Investment in Kyverna Therapeutics, Inc. | 10,130 | 10,130 | - | - | ||
| Total assets | $471,825 | $218,599 | $253,226 | $ | - | |
| Total · Level 1 · Level 2 · Level 3 | ||||||
| (In thousands) | ||||||
| Assets | ||||||
| Cash equivalents and restricted cash equivalents | $92,263 | $92,263 | $ | - | $ | - |
| Marketable securities: | ||||||
| U.S. Treasury and other government-backed securities | 221,117 | 150,852 | 70,265 | - | ||
| Financial institution debt securities | 146,815 | - | 146,815 | - | ||
| Corporate debt securities | 81,738 | - | 81,738 | - | ||
| Total marketable securities | 449,670 | 150,852 | 298,818 | - | ||
| Investment in Kyverna Therapeutics, Inc. | 11,034 | 11,034 | - | - | ||
| Total assets | $552,967 | $254,149 | $298,818 | $ | - | |
Other financial instruments, including accounts receivable, accounts payable and accrued expense, are carried at cost, which approximates fair value due to the short duration and term to maturity.
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.
- Accrued Expenses
Accrued expenses consisted of the following:
| March 31, | December 31, | |
|---|---|---|
| 2026 | 2025 | |
| (In thousands) | ||
| Accrued research and development | $11,790 | $17,366 |
| Employee compensation and benefits | 9,323 | 23,072 |
| Accrued legal and professional expenses | 8,703 | 2,242 |
| Accrued other | 8,104 | 7,182 |
| Total accrued expenses | $37,920 | $49,862 |
- 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.
Gonzalez v. Intellia Therapeutics, Inc.
On February 11, 2025, a purported stockholder of the Company filed a lawsuit, captioned Gonzalez v. Intellia Therapeutics, Inc., No. 1:25-cv-01353 (D. Mass.), in the U.S. District Court for the District of Massachusetts against the Company and certain of its officers (the “defendants”) on behalf of a putative class of stockholders who purchased Company shares from July 30, 2024 through January 8, 2025. On May 26, 2025, the court entered an order appointing co-lead plaintiffs and, on July 23, 2025, co-lead plaintiffs filed an amended complaint. 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. 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. 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, allegations of 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 months ended March 31, 2026, 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.
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 March 31, 2026, the satisfaction and timing of the contingent payments is uncertain and not reasonably estimable.
- 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 March 31, 2026 and December 31, 2025, the Company’s accounts receivable and contract liabilities were related to its collaboration with Regeneron.
The following table presents changes in the Company’s accounts receivable and contract liabilities (in thousands):
| Balance atBeginning ofPeriod · Additions · Deductions · Balance at Endof Period | |||||
|---|---|---|---|---|---|
| Accounts receivable | $9,468 | $8,445 | $(8,782) | $9,131 | |
| Contract liabilities - deferred revenue | $7,290 | $ | - | $(6,660) | $630 |
| Balance atBeginning ofPeriod · Additions · Deductions · Balance at Endof Period | |||||
| Accounts receivable | $8,517 | $10,203 | $(8,216) | $10,504 | |
| Contract liabilities - deferred revenue | $38,917 | $ | - | $(5,095) | $33,822 |
The Company recognized the following revenues as a result of changes in the contract liability balance (in thousands):
| Three Months Ended March 31, | ||
|---|---|---|
| Revenue recognized in the period from: | 2026 | 2025 |
| Amounts included in the contract liability at the beginning of the period | $6,660 | $5,095 |
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 notified the Company that it was exercising 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 recognized$5.2 million and $5.1 million of collaboration revenue in thethree months ended March 31, 2026 and 2025, respectively, in the condensed consolidated statements of operations and comprehensive loss related to the 2016
Regeneron Agreement. As of March 31, 2026, there was approximately $0.6 million of the aggregate transaction price remaining to be recognized that will be recognized through April 2026, the remaining period of the collaboration.
In March 2025, Regeneron provided notice of the achievement of a development milestone under the 2016 Regeneron Agreement. As a result of meeting this milestone, the Company recognized $1.8 million of previously constrained variable consideration as collaboration revenue within the condensed consolidated statement of operations and comprehensive loss in thethree months ended March 31, 2025.
In 2018, the Company entered into a co-development and co-promotion (“Co/Co”) agreement with Regeneron for transthyretin (“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 $8.4 million and $7.0 million of collaboration revenue, primarily representing payments due from Regeneron pursuant to the ATTR Co/Co agreement, in thethree months ended March 31, 2026 and 2025, respectively. The Company didnot incur material costs related to the Hemophilia A Co/Co agreement during the three months ended March 31, 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, other than as described above.
As of March 31, 2026 and December 31, 2025, the Company had accounts receivable of$9.1 million and $9.5 million, respectively, and deferred revenue of $0.6 million and $7.3 million, respectively, related to the Regeneron agreements.
Other Agreements
The Company has existing license and collaboration agreements with 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 thethree months ended March 31, 2026 and 2025.
- Investments and Other Assets
Investments and other assets consisted of the following:
| March 31, | December 31, | |
|---|---|---|
| 2026 | 2025 | |
| (In thousands) | ||
| Investment in Kyverna | $10,130 | $11,034 |
| Other investments | 11,424 | 11,424 |
| Restricted cash equivalents, long-term | 15,404 | 12,104 |
| Prepaid expenses and other assets, long-term | 1,052 | 978 |
| Total investments and other assets | $38,010 | $35,540 |
Kyverna Therapeutics, Inc.
As of March 31, 2026 and December 31, 2025, the Company’s investment in Kyverna is valued at$10.1 million and $11.0 million, respectively. The Company recognized unrealized losses of$0.9 million and $2.1 million, respectively, recorded within “change in fair value of investments, net”in the condensed consolidated statements of operations and comprehensive loss during the three months ended March 31, 2026 and 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 and AvenCell, both of which are accounted for under ASC 321, Investments in Equity Securities(“ASC 321”), using the measurement alternative at cost minus impairment, adjusted for changes in observable prices. The carrying value of the Company’s investments in SparingVision and AvenCell included within “Other investments” in the table above were$3.5 million and$7.9 million, respectively as of March 31, 2026. There wasno change in the carrying value of these investments from December 31, 2025.
- Leases
In February 2025, 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 addition, the Tech Square Lease will expand to include an additional 23,000 square feetof office and laboratory space at 400 Tech Square representing the 5 th floor (the “Additional Tech Square Premises”) when such space becomes available.
The initial term of the Tech Square Lease istwelve years andthree months beginning in September 2026 (the “Rent Commencement Date”). The Company’s obligation to pay rent will start in December 2026. The Company has an option to extend the Tech Square Lease for an additional term of five years. As of the Rent Commencement Date, the base rent under the Tech Square Lease will be $108.00 per square foot per year, plus certain operating expenses, and taxes. The base rent is subject to scheduled annual increases of 3% in July of each rent year. In addition, the Company is entitled to receive up to $410.00 per square foot of tenant improvement allowances. The Company has recorded $52.5 million of prepaid rent related to the Tech Square Lease within “Prepaid expenses and other current assets” in the condensed consolidated balance sheet as of March 31, 2026.
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 March 31, 2026:
| Future Operating Lease Payments | |
|---|---|
| (in thousands) | |
| Remainder of 2026 | $17,818 |
| 2027 | 24,125 |
| 2028 | 15,637 |
| 2029 | 12,666 |
| 2030 | 13,043 |
| Thereafter | 13,550 |
| Total lease payments | $96,839 |
| Less: imputed interest | (16,349) |
| Total operating lease liabilities at March 31, 2026 | $80,490 |
Excluded from the Future Operating Lease Payments table above is $189.1 million related to the Tech Square Lease, which has not yet commenced as of March 31, 2026.
- Stock-Based Compensation
Stock-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows:
| Three Months Ended March 31, | ||
|---|---|---|
| 2026 | 2025 | |
| (In thousands) | ||
| Research and development | $7,582 | $12,626 |
| General and administrative | 5,897 | 9,214 |
| Total | $13,479 | $21,840 |
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 March 31, 2026, there were 9,724,526 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 March 31, 2026,1,553,938 shares were available for issuance under the Inducement Plan.
Restricted Stock Units
The following table summarizes the Company’s RSU activity for thethree months ended March 31, 2026:
| Number ofShares | WeightedAverage GrantDate Fair Valueper Share | |
|---|---|---|
| Unvested restricted stock units as of December 31, 2025 | 5,196,121 | $22.80 |
| Granted | 2,818,849 | 14.70 |
| Vested | (1,518,117) | 25.39 |
| Cancelled | (206,940) | 50.13 |
| Unvested restricted stock units as of March 31, 2026 | 6,289,913 | $17.65 |
The weighted-average grant date fair value of all RSUs granted during the three months ended March 31, 2026 and 2025 was $14.70 and $10.67, respectively. The total fair value of RSUs vested (measured on the date of vesting) for the three months ended March 31, 2026 and 2025 was $13.9 million and $17.5 million, respectively.
As of March 31, 2026, there was $72.2 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 1.87 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 three months ended March 31, 2026, the Company granted 2,563,521 RSUs with a service condition to new and existing employees, which have the potential to vest over a period of approximately three years. The weighted average grant date fair value of these RSUs was $13.73.
Unvested restricted stock units as of March 31, 2026 in the table above includes 5,203,446 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 three months ended March 31, 2026 and 2025 was 255,328 and 223,600, respectively. The grant date fair value for the market-based RSUs, calculated using a Monte Carlo valuation model, was $24.43 and $15.76, respectively. The following assumptions were used to determine the grant date fair value for the three months ended March 31, 2026 and 2025, respectively: risk free interest rate: 3.40% and 3.99%; expected volatility:80.9% and 67.6%. The expected term for all grants was approximately 3.0 years; the expected dividend yield was 0.0%.
Unvested restricted stock units as of March 31, 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 thethree months ended March 31, 2026 and 2025. Unvested restricted stock units as of March 31, 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 $10.01 and $6.92 per option for those options granted during the three months ended March 31, 2026 and 2025, respectively. Weighted average assumptions used to apply this pricing model were as follows:
| Three Months Ended March 31, | ||
|---|---|---|
| 2026 | 2025 | |
| Risk-free interest rate | 3.6% | 4.1% |
| Expected term | 6.0 years | 6.0 years |
| Expected volatility of underlying stock | 83.7% | 75.4% |
| Expected dividend yield | 0.0% | 0.0% |
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 thethree months ended March 31, 2026:
| Number ofOptions | WeightedAverageExercisePrice perShare | WeightedAverageRemainingContractualTerm | AggregateIntrinsicValue | |
|---|---|---|---|---|
| (In years) · (In thousands) | ||||
| Outstanding at December 31, 2025 | 5,173,766 | $39.98 | 5.97 | $50 |
| Granted | 731,481 | 13.78 | ||
| Forfeited | (271,503) | 32.54 | ||
| Outstanding at March 31, 2026 | 5,633,744 | $36.93 | 6.30 | $2,457 |
| Exercisable at March 31, 2026 | 4,140,960 | $44.91 | 5.22 | $846 |
There wereno options exercised during the three months ended March 31, 2026 or 2025.
As of March 31, 2026, there was $10.5 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 1.5 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 March 31, 2026, there were 1,524,879 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 three months ended March 31, 2026 and 2025 the Company didnot issue any shares of common stock under the 2016 Plan.
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:
| Three Months Ended March 31, | ||
|---|---|---|
| 2026 | 2025 | |
| Risk-free interest rate | 3.59% | 4.24% |
| Expected term | 0.5 years | 0.5 years |
| Expected volatility of underlying stock | 121.1% | 66.2% |
| Expected dividend yield | 0.0% | 0.0% |
- 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:
| Three Months Ended March 31, | ||
|---|---|---|
| 2026 | 2025 | |
| (In thousands) | ||
| Net loss | $(96,231) | $(114,329) |
| Weighted average shares outstanding, basic and diluted | 118,490 | 103,500 |
| Net loss per share, basic and diluted | $(0.81) | $(1.10) |
The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive:
| Three Months Ended March 31, | ||
|---|---|---|
| 2026 | 2025 | |
| (In thousands) | ||
| Unvested restricted stock units | 6,290 | 5,525 |
| Stock options | 5,634 | 5,903 |
| 11,924 | 11,428 | |
- Stockholders’ Equity
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 March 31, 2026, the Company issued 28,925,501 shares of its common stock under the 2022 Sale Agreement, as amended. During the three months ended March 31, 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. The Company didnot issue any shares of its common stock under the 2022 Sale Agreement, as amended, during the three months ended March 31, 2025. As of March 31, 2026,$368.5 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended.
- 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:
| Three Months Ended March 31, | ||
|---|---|---|
| 2026 | 2025 | |
| Collaboration revenue | $15,048 | $16,627 |
| Less: | ||
| Research and development: | ||
| External development expenses - nex-z | 20,244 | 23,130 |
| External development expenses - lonvo-z | 8,142 | 10,668 |
| Other research and development (1) | 52,351 | 74,629 |
| Total research and development | 80,737 | 108,427 |
| General and administrative (2) | 34,843 | 29,007 |
| Interest income | (5,205) | (8,603) |
| Other segment information (3) | 904 | 2,125 |
| Segment and consolidated net loss | $(96,231) | $(114,329) |
(1)
Includes unallocated research and development expenses including stock-based compensation of $7.6 million and $12.6 million for the three months ended March 31, 2026 and 2025, respectively, as disclosed within Note 10, “Stock-Based Compensation.”
(2)
Includes stock-based compensation of $5.9 million and $9.2 million for the three months ended March 31, 2026 and 2025, respectively, as disclosed within Note 10, “Stock-Based Compensation.”
(3)
Includes change in fair value of investments as disclosed in the Company’s condensed consolidated statements of operations and comprehensive loss.
Depreciation and amortization expense totaled $2.2 million and $2.5 million for the three months ended March 31, 2026 and 2025, respectively.
- Subsequent Event
On April 28, 2026, the Company entered into an underwriting agreement (the “Underwriting Agreement”) related to a 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.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.