Intellia Therapeutics, Inc.
For the period ended Jun 30, 2025. Filed Aug 7, 2025.
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Jun 30, 2025 · Filed Aug 7, 2025
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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 clinical-stage gene editing company, focused on revolutionizing medicine with CRISPR-based therapies. CRISPR is a gene editing technology which is also sometimes referred to as CRISPR/Cas or CRISPR/Cas9 when referring to the use of CRISPR technology with the Cas9 enzyme. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. To fully realize the transformative potential of CRISPR-based technologies, the Company is building a full-spectrum gene editing company, by leveraging its modular platform, to advancein vivo andex vivo therapies for diseases with high unmet need by pursuing two primary approaches. Forin vivo applications to address genetic diseases, the Company deploys CRISPR as the therapy. The Company’sin vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. In addition, the Company is advancingex vivo applications to address immuno-oncology and autoimmune diseases, where it uses CRISPR as the tool to create the engineered cell therapy. For itsex vivo programs, CRISPR is used to engineer human cells outside the body.
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, 2024.
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 June 30, 2025, the Company has funded its operations through its initial public offering (“IPO”) and concurrent private placements, follow-on public offerings, at-the-market offerings and the sale of convertible preferred stock, as well as through its collaboration agreements. The Company expects that its cash, cash equivalents and marketable securities as of June 30, 2025 will enable the Company to fund its ongoing operating expenses and capital expenditure requirements for at least the twelve-month period following the issuance of these 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, 2024. There have been no material changes to these policies during the six months ended June 30, 2025.
Recently Adopted Accounting Pronouncements
In December 2023, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. This ASU updates income tax disclosure requirements primarily by requiring specific categories and greater disaggregation within the rate reconciliation and disaggregation of income taxes paid by jurisdiction. This ASU is effective for annual periods beginning after December 15, 2024 and is applicable to the Company’s fiscal year beginning January 1, 2025, with early application permitted. The adoption of this ASU is not expected to have a material impact on the Company’s consolidated financial statements and disclosures.
Recently Issued Accounting Pronouncements Not Yet Effective
In November 2024, the FASB issued 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.
- 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 | $248,233 | $190 | $(106) | $248,317 |
| Financial institution debt securities | 146,226 | 461 | (40) | 146,647 |
| Corporate debt securities | 79,222 | 193 | (45) | 79,370 |
| Total | $473,681 | $844 | $(191) | $474,334 |
| AmortizedCost | Gross UnrealizedGains | Gross UnrealizedLosses | Estimated FairValue | |
| (In thousands) | ||||
| Marketable securities: | ||||
| U.S. Treasury and other government-backed securities | $352,309 | $580 | $(273) | $352,616 |
| Financial institution debt securities | 217,544 | 528 | (245) | 217,827 |
| Corporate debt securities | 94,924 | 163 | (160) | 94,927 |
| Other asset-backed securities | 7,168 | 10 | - | 7,178 |
| Total | $671,945 | $1,281 | $(678) | $672,548 |
The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. There wereno material realized gains or losses in thesix months ended June 30, 2025 or the year ended December 31, 2024. The Company did not reclassify any amounts out of accumulated other comprehensive 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 fromthe balance sheet date and that the Company does not intend to dispose of within the next twelve months. At June 30, 2025 and December 31, 2024, 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.
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 | $55,925 | $55,925 | $ | - | $ | - |
| Marketable securities: | ||||||
| U.S. Treasury and other government-backed securities | 248,317 | 147,562 | 100,755 | - | ||
| Financial institution debt securities | 146,647 | - | 146,647 | - | ||
| Corporate debt securities | 79,370 | - | 79,370 | - | ||
| Total marketable securities | 474,334 | 147,562 | 326,772 | - | ||
| Investment in Kyverna Therapeutics, Inc. | 3,604 | 3,604 | - | - | ||
| Total assets | $533,863 | $207,091 | $326,772 | $ | - | |
| Total · Level 1 · Level 2 · Level 3 | ||||||
| (In thousands) | ||||||
| Assets | ||||||
| Cash equivalents and restricted cash equivalents | $66,898 | $66,898 | $ | - | $ | - |
| Marketable securities: | ||||||
| U.S. Treasury and other government-backed securities | 352,616 | 169,735 | 182,881 | - | ||
| Financial institution debt securities | 217,827 | - | 217,827 | - | ||
| Corporate debt securities | 94,927 | - | 94,927 | - | ||
| Other asset-backed securities | 7,178 | - | 7,178 | - | ||
| Total marketable securities | 672,548 | 169,735 | 502,813 | - | ||
| Investment in Kyverna Therapeutics, Inc. | 4,390 | 4,390 | - | - | ||
| Total assets | $743,836 | $241,023 | $502,813 | $ | - | |
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.
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.
Other Investments
The Company’s investment in SparingVision SAS (“SparingVision”) was initially recorded at fair value, determined according to Level 3 inputs in the fair value hierarchy described above. The SparingVision investment is included in “Investments and other assets” in the condensed consolidated balance sheets. This investment is accounted for using the measurement alternative at cost minus impairment, adjusted for changes in observable prices. There were no changes in observable prices or impairment of this investment as of June 30, 2025. The carrying value of the SparingVision investment was$14.6 million as of June 30, 2025 and December 31, 2024.
AvenCell Therapeutics, Inc.
The Company’s investment in AvenCell Therapeutics, Inc. (“AvenCell”) was initially recorded at fair value, determined according to Level 3 inputs in the fair value hierarchy described above. The AvenCell investment is included in “Investments and other assets” in the condensed consolidated balance sheets. This investment is accounted for using the measurement alternative at cost minus impairment, adjusted for changes in observable prices. There were no changes in observable prices or impairment of this investment as of June 30, 2025. The Company previously accounted for the AvenCell investment under the equity method; refer to Note 8 for further details including the change in fair value. The carrying value of the AvenCell investment was$7.9 million as of June 30, 2025 and December 31, 2024.
- Accrued Expenses
Accrued expenses consisted of the following:
| June 30, | December 31, | |
|---|---|---|
| 2025 | 2024 | |
| (In thousands) | ||
| Accrued research and development | $22,927 | $26,362 |
| Employee compensation and benefits | 12,405 | 24,075 |
| Accrued legal and professional expenses | 2,773 | 2,845 |
| Accrued other | 3,448 | 2,073 |
| Total accrued expenses | $41,553 | $55,355 |
In January 2025, the Company announced the prioritization of its current and near-term clinical programs and a strategic restructuring to streamline its operations. In connection with this portfolio prioritization and strategic restructuring, the Company reduced its employee headcount by approximately 27%. In the first quarter of 2025, the Company recognized $6.5 million of restructuring charges in the condensed consolidated statement of operations and comprehensive loss related to these actions. These charges included severance and other employee termination-related costs. The workforce reductions were substantially complete as of March 31, 2025. As of June 30, 2025, there was $1.0 million in accrued expenses recorded as “employee compensation and benefits,” related to the restructuring, which the Company expects to be paid in the second half of 2025.
- 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 asserting claims under Sections 10(b) and 20(a) and Rule 10b-5 of the Securities Exchange Act of 1934 (the “Exchange Act”) on behalf of a putative class of stockholders. 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 the defendants made certain false and/or misleading statements between January 4, 2024 and January 8, 2025, relating to the Company’s NTLA-3001 program and the demand for viral-based editing. The amended complaint seeks unspecified damages, interest, reasonable attorneys’ fees and other costs. 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. 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 six months ended June 30, 2025, except as noted above, there have been no material changes to any outstanding 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 June 30, 2025, 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 June 30, 2025, the Company’s accounts receivable and contract liabilities were related to its collaboration agreements.
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 | $8,517 | $20,439 | $(17,603) | $11,353 | |
| Contract liabilities - deferred revenue | $38,917 | $1,079 | $(10,303) | $29,693 | |
| Balance atBeginning ofPeriod · Additions · Deductions · Balance at Endof Period | |||||
| Accounts receivable | $36,456 | $11,809 | $(35,936) | $12,329 | |
| Contract liabilities - deferred revenue | $60,993 | $ | - | $(10,757) | $50,236 |
The Company recognized the following revenues as a result of changes in the contract liability balance (in thousands):
| Six Months Ended June 30, | ||
|---|---|---|
| Revenue recognized in the period from: | 2025 | 2024 |
| Amounts included in the contract liability at the beginning of the period | $10,245 | $10,757 |
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.
Collaboration Agreements
Regeneron Pharmaceuticals, Inc.
In April 2016, the Company entered into a license and collaboration agreement with Regeneron Pharmaceuticals, Inc. (“Regeneron”) (as amended from time to time, the “2016 Regeneron Agreement”). In October 2023, Regeneron notified the Company that it was exercising its one-time option to extend the technology collaboration term for an additionaltwo years, until April 2026, in exchange for a nonrefundable payment of $30.0 million that was paid in April 2024. The Company recognized $5.0 million and $10.1 million of collaboration revenue in thethree and six months ended June 30, 2025, respectively, and $5.2 million and $10.3 million during thethree and six months ended June 30, 2024, respectively, in the condensed consolidated statements of operations and comprehensive loss related to the 2016 Regeneron Agreement.
In March 2025, Regeneron provided notice of the achievement of a development milestone for the hemophilia B program 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 thesix months ended June 30, 2025.
As of June 30, 2025, there was approximately $16.3 million of the aggregate transaction price remaining to be recognized that will be recognized through April 2026, the remaining term of the existing collaboration agreement.
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 Co/Co”) agreements. In March 2024, the Company notified Regeneron that it was opting out of its hemophilia B Co/Co agreement. The Company continued to have obligations under the hemophilia B Co/Co agreement until the agreement ended in September 2024. The Company continues to support Regeneron with the development of gene editing products directed to hemophilia B, as applicable, under the 2016 Regeneron Agreement. That agreement will control the parties’ obligations to develop and commercialize gene editing products directed to hemophilia B.
The Company recognized $7.9 million and $14.9 million of collaboration revenue in thethree and six months ended June 30, 2025, respectively, and $5.1 million and $9.7 million in thethree and six months ended June 30, 2024, respectively, primarily representing payments due from Regeneron pursuant to the ATTR Co/Co agreement.
The Company recognized contra-revenue related to the Hemophilia Co/Co agreements amounting to approximately $0.1 million in each of thethree and six months ended June 30, 2025, and $4.4 million and $7.6 million in thethree and six months ended June 30, 2024, respectively. The Company recognized $0.1 million and $1.6 million related to the hemophilia B program in thethree and six months ended June 30, 2025, respectively, representing cost reimbursements under the 2016 Regeneron Agreement.
In September 2023, Regeneron and Intellia further expanded the research collaboration (the “2023 Regeneron Amendment”) to develop additionalin vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. The Company didnot recognize material collaboration revenue in thethree and six months ended June 30, 2025, and recognized $0.6 million and $1.1 million of collaboration revenue in thethree and six months ended June 30, 2024, respectively, in the condensed consolidated statements of operations and comprehensive loss related to the 2023 Regeneron Amendment.
Since December 31, 2024, there have been no material changes to the key terms of the 2016 Regeneron Agreement, ATTR Co/Co, Hemophilia Co/Co, or 2023 Regeneron Amendment (the “Regeneron Agreements”), other than as described above.
As of June 30, 2025 and December 31, 2024, the Company had accounts receivable of $10.2 million and $7.2 million, respectively, and deferred revenue of $17.2 million and $26.4 million, respectively, related to the Regeneron Agreements.
In October 2021, the Company and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, entered into a license and collaboration agreement (the “SparingVision LCA”) to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.
Since December 31, 2024, there have been no material changes to the key terms of the SparingVision LCA.
The Company recognizedno revenue related to the SparingVision LCA for thethree and six months ended June 30, 2025 andapproximately $0.5 million and $1.1 million, respectively, related to the SparingVision LCA for the three and six months ended June 30, 2024. The Company didnot have accounts receivable related to the SparingVision LCA as of June 30, 2025. At December 31, 2024, the Company had $0.6 million in accounts receivable related to the SparingVision LCA. The Company had deferred revenue of $12.5 million as of June 30, 2025 and December 31, 2024, related to the SparingVision LCA, which is expected to be recognized on a cost incurred basis over a six to nine year period from the signing of the agreement.
ReCode Therapeutics, Inc. (“ReCode”)
On February 14, 2024, the Company entered into a license, collaboration and option agreement with ReCode Therapeutics, Inc. (the “ReCode LCA”), a clinical-stage genetic medicines company, to develop novel genomic medicines for the treatment of cystic fibrosis.
Since December 31, 2024, there have been no material changes to the key terms of the ReCode LCA.
The Company is entitled to cost reimbursements for certain research activities, which will be recorded as revenue. The Company did not recognize material revenue from the ReCode LCA during the three and six months ended June 30, 2025 and 2024.
Other Agreements
The Company has existing license and collaboration agreements with AvenCell, Kyverna, and ONK Therapeutics, Ltd. (“ONK”). Since December 31, 2024, there have been no material changes to the key terms of the AvenCell, Kyverna and ONK license and collaboration agreements.
During the three and six months ended June 30, 2025 and 2024, the Company didnot recognize material revenue under its agreements with Kyverna or ONK.
During the six months ended June 30, 2024, the Company recognized $21.0 million of previously eliminated intra-entity profit related to itslicense and collaboration agreement with AvenCell (the “AvenCell LCA”) in the condensed consolidated statement of operations and comprehensive loss. See Note 8 for further details. The Company recognized $1.0 million in revenue related to material shipments under the AvenCell LCA during thesix months ended June 30, 2025. The Company had $1.0 million and $0.7 million in accounts receivable from AvenCell as of June 30, 2025 and December 31, 2024, respectively. The Company had $1.0 million in accrued expenses as of June 30, 2025 and December 31, 2024 related to the AvenCell LCA.
- Investments and Other Assets
Investments and other assets consisted of the following:
| June 30, | December 31, | |
|---|---|---|
| 2025 | 2024 | |
| (In thousands) | ||
| Investment in Kyverna | $3,604 | $4,390 |
| Prepaid rent Tech Square Lease, long-term | 36,829 | - |
| Other investments | 22,481 | 22,481 |
| Restricted cash equivalents, long-term | 13,776 | 13,605 |
| Prepaid expenses and other assets, long-term | 3,553 | 3,788 |
| Total investments and other assets | $80,243 | $44,264 |
Other Investments
As of June 30, 2025 and December 31, 2024, the carrying value of the Company’s investment in SparingVision was$14.6 million.
AvenCell Therapeutics, Inc.
As of December 31, 2023, the Company held a 33.33% equity interest in AvenCell and accounted for its investment using the equity method of accounting, as the Company had significant influence, but not control, over AvenCell, and the investment was valued at $11.8 million. During the first quarter of 2024, in conjunction with the completion of a debt financing, AvenCell increased the size of their board, with a single investor having control over AvenCell’s operational and financial decisions. From that point forward, the Company no longer had the ability to exercise significant influence over AvenCell, and therefore the Company’s investment in AvenCell has been accounted for in accordance with Accounting Standard Codification (“ASC”) 321, Investments in Equity Securities (“ASC 321”) and AvenCell is no longer considered to be a related party.
The transition from equity method accounting to ASC 321 required the Company to reclassify $2.1 million from accumulated other comprehensive income (loss) and recognize $21.0 million of previously eliminated intra-entity profit, both of which resulted in an increase in the carrying value of the investment in AvenCell. In the fourth quarter of 2024, AvenCell completed a Series B financing, which represented an observable price change in the investment in AvenCell. As of June 30, 2025 and December 31, 2024, the carrying value of the Company’s investment in AvenCell was$7.9 million.
- Leases
In February 2022 and subsequently amended in June 2023, the Company entered into an agreement to lease approximately 140,000 square feet of office, general laboratory and manufacturing space at 840 Winter Street in Waltham, Massachusetts (the “840 Winter Lease”).
In conjunction with the Company’s January 2025 strategic restructuring to streamline its operations, in February 2025, the Company entered into a Second Amendment (the “Winter Street Amendment”) which will terminate the 840 Winter Lease on or before June 30, 2028. In connection with the Winter Street Amendment, the Company will pay to the landlord lease modification payments totaling $78.0 million in three installments, with the first $34.0 million paid in February 2025, a second $30.0 million paid in April 2025 and the remaining $14.0 million to be paid in January 2026. The Company is not subject to paying any base rent, operating expenses or other costs pursuant to the 840 Winter Lease after January 2025. 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, the Company would initially lease approximately 101,000 square feet at 400 Tech Square (the “Initial Tech Square Premises”). In addition, the Tech Square Lease would expand, in two tranches, to include approximately 46,000 square feet of additional office and laboratory space at 400 Tech Square (the “Additional Tech Square Premises”) when each such space becomes available. In July 2025, the Company entered into a First Amendment to the Tech Square Lease (the “Tech Square Lease Amendment”) which added 23,000 square feet to the Initial Tech Square Premises, such that the Initial Tech Square Premises now includes approximately 124,000 square feet and the Additional Tech Square Premises now includes approximately 23,000 square feet. In July 2025, the landlord delivered the Initial Tech Square Premises to the Company for the Company’s construction of tenant improvements (the “Delivery Date”). 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 anoption to extend the Tech Square Lease for an additional term offive 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% on the anniversary of the Delivery Date. In addition, the Company is entitled to receive up to $51.0 million of tenant improvement allowances for the Initial Tech Square Premises, and additional tenant improvement allowances for the Additional Tech Square Premises. The construction of tenant improvements for the Initial Tech Square Premises will be primarily funded by the tenant improvement allowance and is expected to be completed in the second half of 2026.
The Company accounted for the Winter Street Amendment and the Tech Square Lease as one combined contract under ASC 842, Leases (Topic 842). The Company identifiedtwo lease components, 840 Winter and 400 Tech Square. The total consideration in the combined contract is $244.4 million, which includes the undiscounted remaining lease payments of the 840 Winter and 400 Tech Square components and the lease prepayments. The Company did not include the lease payments associated with the five-year extension option related to the Tech Square Lease as it is not reasonably certain of exercise. The Company allocated $52.1 million of the consideration to the 840 Winter component and $192.3 million to the 400 Tech Square component based on relative standalone selling prices. Upon allocation, the Company remeasured the 840 Winter lease liability as of the modification date based on the remaining lease payments and using an updated incremental borrowing rate of 6.6%, which reduced the lease liability and right of use asset by $61.9 million. After accounting for the modification in February 2025, the lease liability and right of use asset for the 840 Winter component were $46.5 million and $50.0 million, respectively. As of June 30, 2025, the lease liability and right-of-use asset were $16.4 million and $44.7 million, respectively.
As of June 30, 2025, the Tech Square component had not commenced as the Company had not taken control over the premises and therefore the Company did not record a right-of-use asset or lease liability. The Company has recorded $36.8 million of prepaid rent related to the Tech Square component within “Investments and other assets” in the condensed consolidated balance sheet as of June 30, 2025.
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 June 30, 2025 for all of the Company’s leases.
| Future Operating Lease Payments | |
|---|---|
| Year Ending December 31, | (in thousands) |
| Remainder of 2025 | $12,892 |
| 2026 | 32,219 |
| 2027 | 24,125 |
| 2028 | 15,637 |
| 2029 | 12,666 |
| Thereafter | 26,593 |
| Total lease payments | $124,132 |
| Less: imputed interest | (21,541) |
| Total operating lease liabilities at June 30, 2025 | $102,591 |
Excluded from the table above are $156.8 million and $36.9 million of undiscounted future operating lease payments related to the Tech Square Lease and Tech Square Lease Amendment.
- Stock-Based Compensation
Stock-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| (In thousands) | ||||
| Research and development | $14,059 | $25,439 | $26,685 | $45,600 |
| General and administrative | 7,977 | 15,422 | 17,191 | 29,437 |
| Total | $22,036 | $40,861 | $43,876 | $75,037 |
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.
On April 28, 2025 the Company’s board of directors adopted, and on June 11, 2025 its stockholders approved, 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 2025 Plan became effective on June 11, 2025, and the Company no longer grants any awards under the 2015 Plan. The number of shares initially reserved for issuance under the 2025 Plan is 12,831,965, which includes 7,666,787 shares that remained available for grant under the 2015 Plan upon adoption of the 2025 Plan and an additional 5,165,178 shares authorized for issuance under the 2025 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 June 30, 2025, there were 12,759,619 shares available for future issuance under the 2025 Plan.
In June 2024, the Company adopted the 2024 Inducement Plan (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. In accordance with the Inducement Plan,850,000 shares of common stock were reserved for future issuance; there were 215,638 shares available for future issuance under the Inducement Plan as of June 30, 2025.
Restricted Stock Units
The following table summarizes the Company’s RSU activity for thesix months ended June 30, 2025:
| Number ofShares | WeightedAverage GrantDate Fair Valueper Share | |
|---|---|---|
| Unvested restricted stock units as of December 31, 2024 | 5,547,530 | $39.13 |
| Granted | 2,407,807 | 10.49 |
| Vested | (1,686,670) | 42.66 |
| Cancelled | (987,049) | 39.70 |
| Unvested restricted stock units as of June 30, 2025 | 5,281,618 | $24.84 |
The weighted-average grant date fair value of all RSUs granted during the three and six months ended June 30, 2025 was $8.10 and $10.49, respectively. The total fair value of RSUs vested (measured on the date of vesting) for thethree and six months ended June 30, 2025 was $1.4 million and $18.9 million, respectively. The weighted-average grant date fair value of all RSUs granted during the three and six months ended June 30, 2024 was $25.16 and $34.20, respectively. The total fair value of RSUs vested (measured on the date of vesting) for the three and six months ended June 30, 2024 was $4.6 million and $35.5 million, respectively.
As of June 30, 2025 there was $69.4 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 1.55 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 six months ended June 30, 2025, the Company granted 2,184,207 RSUs with a service condition to new and existing employees and non-employee directors, which have the potential to vest over a period of approximately three years. The weighted average grant date fair value of these RSUs was $9.96.
Unvested restricted stock units as of June 30, 2025 in the table above includes 4,274,020 RSUs that are service-based.
Restricted Stock Units - Market Awards
Since 2022, market-based RSUs have been granted to senior executives. These RSUs 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 six months ended June 30, 2025 was 223,600 and their grant date fair value, calculated using a Monte Carlo valuation model, was $15.76. The following assumptions were used to determine the grant date fair value: risk free interest rate:3.99%; expected volatility:67.6%. The expected term for these grants was approximately 3.0 years; the expected dividend yield was 0.0%.
Unvested restricted stock units as of June 30, 2025 in the table above includes 596,138 RSUs that are market-based.
Restricted Stock Units - Performance-Based Awards with TSR Multiplier
In 2024, performance-based RSUs (“PSUs”) with a relative TSR modifier were also 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 thesix months ended June 30, 2025. Unvested restricted stock units as of June 30, 2025 in the table above includes 389,251 PSUs with a TSR multiplier.
Restricted Stock Units - Performance-Based Awards
In 2022, the Company granted 66,296 performance-based RSUs to certain non-executive employees that would vest upon obtaining certain scientific milestones. There weretwo separate tranches, each attached to a different set of milestones. The milestone related to the first tranche, made up of 21,878 RSUs, was achieved in the first quarter of 2023 and these RSUs vested. The remaining performance milestones were considered not probable of achievement as of June 30, 2025 and, therefore, no related stock-based compensation expense was recorded during the period then ending.
Unvested restricted stock units as of June 30, 2025 in the table above includes 22,209 performance-based RSUs.
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 $5.74 and $6.79 per option for those options granted during thethree and six months ended June 30, 2025, respectively, and $17.50 and $22.01 per option for those options granted during thethree and six months ended June 30, 2024, respectively. Weighted average assumptions used to apply this pricing model were as follows:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Risk-free interest rate | 4.1% | 4.3% | 4.1% | 4.2% |
| Expected term | 5.5 years | 5.6 years | 5.9 years | 6.0 years |
| Expected volatility of underlying stock | 79.4% | 76.3% | 75.8% | 76.4% |
| Expected dividend yield | 0.0% | 0.0% | 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 does not have sufficient historical option exercise data to provide a reasonable basis upon which to estimate the expected term.
Expected Volatility. Expected volatility is estimated based on actual movements in the Company’s stock price over the most recent historical periods, over the expected term of their stock option grants.
Expected Dividend Yield. The Company has not paid cash dividends and has no intention to pay cash dividends in the future.
Stock options generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining two years, unless they contain specific vesting provisions. 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 thesix months ended June 30, 2025:
| Number ofOptions | WeightedAverageExercisePrice perShare | WeightedAverageRemainingContractualTerm | AggregateIntrinsicValue | |
|---|---|---|---|---|
| (In years) · (In thousands) | ||||
| Outstanding at December 31, 2024 | 5,412,786 | $47.27 | 6.18 | $350 |
| Granted | 720,901 | 9.90 | ||
| Forfeited | (692,170) | 68.50 | ||
| Outstanding at June 30, 2025 | 5,441,517 | $39.62 | 6.08 | $255 |
| Exercisable at June 30, 2025 | 4,176,532 | $45.36 | 5.14 | $174 |
The total intrinsic value (the amount by which the fair market value exceeded the exercise price) of stock options exercised during the three and six months ended June 30, 2024 was $0.8 million and $2.0 million, respectively. There wereno options exercised during thethree and six months ended June 30, 2025.
As of June 30, 2025, there was $11.0 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 1.23 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 June 30, 2025, there were 1,201,725 shares available for future issuance under the 2016 Plan. The number of shares reserved for issuance under the 2016 Plan are subject to cumulative increases on each January 1 st by the lesser of a) one percent of the number of shares of common stock issued and outstanding on the immediately preceding December 31 st, b)500,000 shares of common stock, or c) such lesser number of shares of common stock as determined by the board of directors.
During the six months ended June 30, 2025 and 2024 the Company issued 155,172 and 87,751 shares of common stock under the 2016 Plan, respectively. The weighted-average purchase prices of shares issued under the 2016 Plan were $7.97 and $19.02 per sharefor the six-month periods ended June 30, 2025 and 2024, 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:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Risk-free interest rate | 4.24% | 5.24% | 4.24% | 5.24% |
| Expected term | 0.5 years | 0.5 years | 0.5 years | 0.5 years |
| Expected volatility of underlying stock | 66.2% | 56.7% | 66.2% | 56.7% |
| Expected dividend yield | 0.0% | 0.0% | 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 June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| (In thousands) | ||||
| Net loss | $(101,255) | $(146,975) | $(215,584) | $(254,411) |
| Weighted average shares outstanding, basic and diluted | 103,732 | 96,975 | 103,617 | 96,238 |
| Net loss per share, basic and diluted | $(0.98) | $(1.52) | $(2.08) | $(2.64) |
The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive:
| Three and Six Months Ended June 30, | ||
|---|---|---|
| 2025 | 2024 | |
| (In thousands) | ||
| Unvested restricted stock units | 5,282 | 5,371 |
| Stock options | 5,442 | 5,709 |
| 10,724 | 11,080 | |
- 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 (the “2022 Sale Agreement, as amended”) to increase the size of the at-the-market offering program from $400.0 million to $750.0 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 June 30, 2025, the Company issued 15,981,790 shares of its common stock under the 2022 Sale Agreement, as amended. During the three and six months ended June 30, 2025, the Company issued 1,459,257 shares of its common stock, in a series of sales, at an average price of$9.64 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of$13.7 million, after payment of cash commissions. As of June 30, 2025, $0.3 million of these proceeds are included in “Prepaid expenses and other current assets” on the Company’s condensed consolidated balance sheet, representing offerings with trade dates in June 2025 that were settled in July 2025. During the six months ended June 30, 2024, the Company issued 3,802,761 shares of its common stock, in a series of sales, at an average price of $25.58 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $94.4 million, after payment of cash commissions and approximately $0.2 million related to legal, accounting and other fees in connection with the sales.
As of June 30, 2025,$235.1 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 the chief 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, 2024. 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 June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Collaboration revenue | $14,245 | $6,957 | $30,872 | $35,892 |
| Less: | ||||
| Research and development: | ||||
| External development expenses - nex-z | 23,958 | 14,706 | 47,088 | 28,364 |
| External development expenses - lonvo-z | 12,053 | 10,676 | 22,721 | 19,469 |
| Other research and development (1) | 61,024 | 88,825 | 135,653 | 178,221 |
| Total research and development | 97,035 | 114,207 | 205,462 | 226,054 |
| General and administrative (2) | 27,206 | 31,793 | 56,213 | 62,884 |
| Interest income | (7,402) | (12,422) | (16,005) | (25,054) |
| Other segment information (3) | (1,339) | 20,354 | 786 | 26,419 |
| Segment and consolidated net loss | $(101,255) | $(146,975) | $(215,584) | $(254,411) |
(1)
Includes unallocated research and development expenses, including stock-based compensation of $14.1 million and $26.7 million for thethree and six months ended June 30, 2025, respectively, and $25.4 million and $45.6 million for thethree and six months ended June 30, 2024, respectively, as disclosed within Note 10, “Stock-Based Compensation.”
(2)
Includes stock-based compensation of $8.0 million and $17.2 million for thethree and six months ended June 30, 2025, respectively, and $15.4 million and $29.4 million for thethree and six months ended June 30, 2024, 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 $2.5 million and $5.0 million for thethree and six months ended June 30, 2025, respectively, and $2.6 million and $5.1 million for thethree and six months ended June 30, 2024, respectively.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.