Caribou Biosciences, Inc. 10-Q

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations. You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and the related notes included in Part I, Item 1, of this Quarterly Report on Form 10-Q for the fiscal quarter ended September 30, 2025 (“Form 10-Q”) and with the audited consolidated financial statements and the related notes for the fiscal year ended December 31, 2024, included in our Annual Report on Form 10-K (“Form 10-K”) filed with the U.S. Securities and Exchange Commission (“SEC”) on March 10, 2025. Special Note Regarding Forward-Looking Statements This Form 10-Q contains “forward-looking” statements within the meaning of Section 27A of the Securities Act of 1933, as amended (“Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (“Exchange Act”). All statements, other than statements of historical facts, contained in this Form 10-Q are forward-looking statements, including statements regarding our business strategy, plans, and objectives; expectations regarding our clinical development programs, including our expectations about the timing of such programs; the expected timing of disclosure of clinical data from such programs; the safety, efficacy, and potential advantages of our product candidates; future regulatory filings and interactions with regulatory authorities; our results of operations and financial position; and plans and objectives of management for future operations. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. As a result of many factors, including but not limited to the risks described in the section of our Form 10-K titled “Risk Factors,” in the section entitled “Risk Factors” in this Form 10-Q, and in other filings we make with the SEC, the events and circumstances reflected in our forward-looking statements may not be achieved or may not occur, and actual results could differ materially from those described in or implied by the forward-looking statements. As a result of these risks, you should not place undue reliance on these forward-looking statements. We assume no obligation to revise or update any forward-looking statements for any reason, except as required by law. Overview We are a clinical-stage C lustered R egularly I nterspaced S hort P alindromic R epeats (“CRISPR”) genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. Our genome-editing platform, including our novel chRDNA ( C RISPR h ybrid R NA- DNA , pronounced “chardonnay”) technology, enables more precise genome editing of allogeneic cell therapies. Our allogeneic, or off-the-shelf, chimeric antigen receptor (“CAR”)-T (“CAR-T”) cell therapy product candidates are manufactured in advance with cells from healthy donors, with the goal of enabling broad patient access, rapid patient treatment, and increased manufacturing scale. Our allogeneic CAR-T cell therapy product candidates in clinical development are directed at established cell surface targets against which autologous CAR-T cell therapeutics have already demonstrated clinical proof of concept, CD19 and B cell maturation antigen (“BCMA”). We use our chRDNA technologies to armor our allogeneic CAR-T cell therapies through multiple genome-editing strategies, such as checkpoint disruption and immune cloaking, to enhance activity against hematologic malignancies. We are advancing two clinical-stage allogeneic CAR-T cell therapies for the treatment of patients with hematologic malignancies: • Vispacabtagene regedleucel (“vispa-cel,” formerly CB-010): an allogeneic anti-CD19 CAR-T cell therapy that is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (“r/r B-NHL”) in our ANTLER phase 1 clinical trial • CB-011: an allogeneic anti-BCMA CAR-T cell therapy that is being evaluated in patients with relapsed or refractory multiple myeloma (“r/r MM”) in our CaMMouflage phase 1 clinical trial Vispa-cel has received regenerative medicine advanced therapy (“RMAT”) designation for relapsed or refractory large B cell lymphoma (“r/r LBCL”) as well as fast track designation for r/r B-NHL from the U.S. Food and Drug Administration (“FDA”); CB-011 has received fast track designation for r/r MM from the FDA. 20 Tabl e of Contents To our knowledge, vispa-cel is the first clinical-stage allogeneic CAR-T cell therapy with a genome-edited knockout of the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface. On November 3, 2025, we announced that, as of the September 2, 2025, safety data cutoff date, 84 patients have been enrolled in the ANTLER phase 1 clinical trial, including a confirmatory cohort of 22 CD19-naïve second-line (“2L”) large B cell lymphoma (“LBCL”) patients. The confirmatory cohort was designed to prospectively confirm the positive outcomes of partial human leukocyte antigen (“HLA”) matching (i.e., greater than or equal to four matched HLA alleles) observed in earlier retrospective analyses. Patients in the confirmatory cohort received a single dose of vispa-cel at the recommended phase 2 dose (“RP2D”) of 80×10⁶ viable CAR-T cells following lymphodepletion (“LD”) with 60 mg/kg/day for two days of cyclophosphamide and 25 mg/m 2 /day for five days of fludarabine. As of the September 29, 2025, efficacy data cutoff date, data from the 22-patient confirmatory cohort showed that a single dose of partially matched vispa-cel led to an 82% overall response rate (“ORR”), a 64% complete response (“CR”) rate, and 51% progression-free survival (“PFS”) at 12 months. Median follow up for the confirmatory cohort was 6.0 months. By leveraging our allogeneic CAR-T clinical data set of more than 140 patients dosed across multiple clinical trials, we identified key factors that we believe are linked to successful patient outcomes, and we have incorporated these learnings to develop an optimized vispa-cel profile. Two of the key attributes of the optimized vispa-cel profile are donor age (young donors drive enhanced outcomes relative to older donors) and partial HLA matching (matching two or more alleles correlates with enhanced patient outcomes). Of the 84 patients treated in our ANTLER phase 1 clinical trial, 35 CD19-naïve LBCL patients received vispa-cel with an optimized profile. The optimized profile cohort included 20 of the 22 patients in the confirmatory cohort and an additional 15 patients from dose escalation and expansion. The results for the 35-patient optimized profile cohort included an 86% ORR, a 63% CR rate, and 53% PFS at 12 months. Median follow up for the optimized profile cohort was 11.8 months. Based on internal analysis and FDA interactions to date, we anticipate evaluating vispa-cel in a randomized, controlled pivotal phase 3 clinical trial in approximately 250 2L LBCL CD19-naïve patients who are ineligible for transplant and autologous CAR-T cell therapy; patients randomized to the study arm would receive a single dose of vispa-cel at the RP2D following LD and patients randomized to the comparator arm would be treated with the investigator’s choice of standard of care immunochemotherapy regimens. We intend to further refine our pivotal trial design through continued engagement with the FDA prior to initiation of this pivotal trial. Although we believe this approach provides a straightforward regulatory path to full FDA approval of vispa-cel based on FDA interactions to date, we cannot assure you that, in future discussions with the FDA, the FDA will not recommend or request additional requirements for our planned pivotal phase 3 clinical trial design for vispa-cel. To our knowledge, CB-011 is the first allogeneic CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the endogenous beta-2 microglobulin (“B2M”) protein and insertion of a beta-2-microglobulin–human-leukocyte-antigen-E–peptide transgene (“B2M–HLA-E”). On November 3, 2025, we announced results from the dose escalation portion of our ongoing CaMMouflage phase 1 trial evaluating CB-011 in r/r MM patients. Forty-eight fourth-line or later (“4L+”) patients were enrolled in the dose escalation portion of the trial; enrollment in dose escalation is now completed. Patients had been treated with three or more prior lines of therapy including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. Prior treatment with an autologous CAR-T cell therapy was not permitted and no BCMA-targeted therapy was permitted within the prior three months. We evaluated two different LD regimens and multiple CAR-T cell dose levels. Thirty-five patients were treated with an LD regimen of 500 mg/m 2 cyclophosphamide and 30 mg/m 2 fludarabine daily for three days (“the selected LD regimen”). A single dose of CB-011 preceded by the selected LD regimen resulted in responses at all dose levels evaluated (150x10 6 viable CAR-T cells, N=6; 300x10 6 viable CAR-T cells, N=13; 450x10 6 viable CAR-T cells, N=13; and 800x10 6 viable CAR-T cells, N=3). We also announced on November 3, 2025, that we have selected the 450x10 6 viable CAR-T cell dose with the selected LD regimen as our recommended dose for expansion (“RDE”). As of the September 24, 2025, the data cutoff date, data from the 12-patient, BCMA-naïve cohort treated with the selected LD regimen and a single dose of 450x10 6 viable CAR-T cells had the following outcomes: an ORR of 92%, a CR/stringent CR (“sCR”) rate of 75%, and 91% of evaluable patients achieved minimal residual disease (“MRD”) negativity. Seven of the 12 patients remained on study as of the data cutoff date in very good partial response (“VGPR”) or better six months or longer following receipt of a single dose of CB-011. The median follow up for these 12 patients was 8.3 months. On April 24, 2025, we announced a strategic pipeline prioritization with workforce and cost reduction initiatives to focus resources on our vispa-cel and CB-011 product candidates. At that time, we disclosed that we had discontinued our GALLOP phase 1 trial of vispa-cel for the treatment of lupus prior to dosing the first patient; our AMpLify phase 1 clinical trial of CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy, for the treatment of relapsed or refractory acute myeloid leukemia, as additional data would be needed to advance this program; and our preclinical research. Patients treated in the AMpLify phase 1 clinical trial will continue to be followed as part of our long-term follow-up study. Additionally, we 21 Tabl e of Contents announced that our workforce was reduced by 47 employees, or approximately 32% of our workforce. As a result, we recorded cash severance costs, benefits, and transition support services expenses of $1.8 million for the nine months ended September 30, 2025, which we recorded as research and development expenses or general and administrative expenses in our unaudited condensed consolidated statements of operations and comprehensive loss. For the nine months ended September 30, 2025, we recorded wind down costs of $0.4 million, as research and development expenses in our unaudited condensed consolidated statements of operations and comprehensive loss related to the discontinuation of our GALLOP and AMpLify phase 1 clinical trials. In connection with the discontinuation of the AMpLify phase 1 clinical trial for our CB-012 product candidate, we terminated our Exclusive License Agreement, dated November 13, 2020, with Memorial Sloan Kettering Cancer Center (“MSKCC”) (as amended, “MSKCC Agreement”), effective August 11, 2025. In conjunction with the previously announced strategic pipeline prioritization, we have reduced the usage of our leased office and lab space, and we may sublease the unused space. Accordingly, for the second quarter of 2025, we evaluated the related leasehold improvements, right of use assets, and lab equipment and concluded that this asset group is impaired. As a result, we recognized impairment charges totaling $10.0 million for the second quarter of 2025, of which $7.4 million and $2.6 million, respectively, were related to the tenant improvements and right of use asset associated with the underlying leased property. We also identified certain laboratory equipment that we no longer intend to use. As a result, this equipment was deemed impaired, resulting in a $2.2 million impairment charge for the second quarter of 2025. Since our founding in 2011, we have devoted substantially all of our resources to organizing and staffing, business planning, raising capital, expanding our genome-editing platform technologies, developing our product candidates and building our pipeline, creating and maintaining our intellectual property portfolio, and establishing arrangements with third parties for the manufacture, testing, and clinical trial evaluations of our product candidates. We do not have any products approved for commercial sale and have not generated any revenue from product sales. We have incurred operating losses since commencement of our operations. To date, we have primarily funded our operations through proceeds from the sales of our capital stock, revenue from our license and collaboration agreements, and proceeds from the sale of shares of Intellia Therapeutics, Inc. (“Intellia”) common stock. Our net losses for the three months ended September 30, 2025, and September 30, 2024, were $27.5 million and $34.7 million, respectively. Our net losses for the nine months ended September 30, 2025, and September 30, 2024, were $121.6 million and $113.6 million, respectively. We had an accumulated deficit of $570.0 million as of September 30, 2025. Our net losses and operating losses may fluctuate from quarter to quarter and year to year depending primarily on the timing of expenses associated with our clinical trials and development of our product candidates. We anticipate that our expenses will increase substantially as we: • advance clinical trials for our CAR-T cell therapy product candidates, including through succeeding clinical phases of development for these product candidates; • seek regulatory and marketing approvals for any of our product candidates that successfully complete clinical trials, if any; • expand manufacturing capabilities and supply chain capacity for our product candidates; • experience any delays, challenges, or other issues associated with any of the above, including the failure of our clinical trials meeting endpoints or clinical trial data subject to differing interpretations, or the occurrence of potential safety issues or other development or regulatory challenges; • hire additional personnel, as needed, to support our clinical development efforts; • acquire or in-license intellectual property or new technologies; • expand, maintain, enforce, and defend our intellectual property portfolio; • make royalty, milestone, or other payments under current, and any future, agreements with third parties; • establish a sales, marketing, and distribution infrastructure to commercialize any product candidates for which we obtain marketing approval; and • continue to operate as a public company, including defending against any future class action securities litigation and shareholder derivative lawsuits. 22 Tabl e of Contents We do not own or operate any manufacturing facilities. We use multiple contract manufacturing organizations (“CMOs”) to individually manufacture, under current good manufacturing processes, our chRDNA guides, Cas9 and Cas12a proteins, plasmids, and adeno-associated virus serotype 6 (“AAV6”) vectors used in the manufacture of our cell therapy product candidates as well as the CAR-T cell therapy product candidates themselves. We expect to continue to rely on our CMOs for manufacturing our clinical trial materials, and most of these CMOs have capabilities for commercial manufacturing. Additionally, we may decide to build our own manufacturing facility in the future to provide greater flexibility and control over our clinical or commercial manufacturing needs. Components of Results of Operations Licensing and Collaboration Revenue We have not generated any revenue from product sales to date and do not expect to generate any revenue from the sale of products in the foreseeable future. We cannot predict if, when, or to what extent we will generate revenue from the commercialization and sale of our product candidates if we succeed in obtaining regulatory approval for these product candidates. To date, all of our revenue consists of licensing and collaboration revenue earned from collaboration and/or licensing agreements entered into with third parties, including related parties. Under these agreements, we license rights to certain intellectual property controlled by us. The terms of these arrangements typically include payments to us of one or more of the following: nonrefundable, upfront license fees or exclusivity fees; annual maintenance fees; regulatory and/or commercial milestone payments; research and development payments; and royalties on the net sales of products and/or services. Each of these payments results in licensing and collaboration revenue. Revenue under such licensing and collaboration agreements was $2.2 million and $2.0 million for the three months ended September 30, 2025, and September 30, 2024, respectively, and $7.2 million and $7.9 million for the nine months ended September 30, 2025, and September 30, 2024, respectively. See Notes 5 and 7 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information. For the foreseeable future, we expect substantially all our revenue will be generated from licensing and collaboration agreements. See Note 2 to the consolidated financial statements included in our Form 10-K for additional information about our revenue recognition policy related to our licensing and collaboration agreements. Operating Expenses Research and Development Expenses Our research and development expenses consist of internal and external expenses incurred in connection with the development of our product candidates and our platform technologies, and our in-licensing, assignment, and other third-party agreements. External costs include: • costs associated with acquiring technology and intellectual property licenses that have no alternative future uses, sublicensing revenues, and milestones; • costs incurred in connection with the clinical development and manufacturing of our product candidates, including under agreements with CMOs, suppliers, clinical research organizations (“CROs”), and clinical sites; and • other research and development costs, including lab supplies, and consulting services. Internal costs include: • personnel-related costs, including salaries, benefits, and stock-based compensation expense, for our research and development personnel; and • allocated facilities and other overhead expenses, including expenses for rent, facilities maintenance, and depreciation. 23 Tabl e of Contents We expense research and development costs as incurred. Costs of certain activities are recognized based on an evaluation of the progress to completion of specific tasks. However, payments made prior to the receipt of goods or services that will be used or rendered for future research and development activities are deferred and capitalized as prepaid expenses and other current assets in our unaudited condensed consolidated balance sheets. The capitalized amounts are recognized as expenses as the goods are delivered or as related services are performed. We separately track certain external costs on a program-by-program basis; however, we do not track costs that are deployed across our programs. We do not allocate internal costs as several of our departments support our programs and our payroll and other personnel expenses are not tracked on a program-by-program basis. Clinical development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will increase substantially for the foreseeable future as we continue to implement our business strategy; advance our product candidates through clinical trials; conduct translational research to support our product candidates; seek regulatory approvals for our product candidates that successfully complete clinical trials; and hire additional personnel to support our clinical development efforts. The successful development of our CAR-T product candidates is highly uncertain. Accordingly, at this time, we cannot reasonably estimate or know the nature, timing, and costs of the efforts that will be necessary to complete the development of our product candidates. We are also unable to predict when, if ever, we will generate revenue and material net cash inflows from the commercialization and sale of any of our product candidates for which we may obtain marketing approval. We may never succeed in achieving regulatory approval for any of our product candidates. The duration, costs, and timing of clinical trials and development of our product candidates will depend on a variety of factors, including: • sufficiency of our financial and other resources; • acceptance of our CRISPR chRDNA genome-editing technology; • ability to develop differentiating features so that our products have a competitive edge; • establishment, maintenance, enforcement, and defense of our patents and other intellectual property rights; • our ability to not infringe, misappropriate, or otherwise violate third-party intellectual property rights; • successful enrollment in, and completion of, our clinical trials of our product candidates; • data from our clinical trials that support an acceptable risk-benefit profile of our product candidates for the intended patient populations and that demonstrate safety and efficacy; • entry into collaborations to further the development of our product candidates; • successful development of our internal process development and transfer to CMOs; • establishment of agreements with CMOs and suppliers for clinical and commercial supplies and scaling up manufacturing processes and capabilities to support our clinical trials; • receipt of timely responses and marketing approvals from applicable regulatory authorities; • grant of regulatory exclusivity for our product candidates; • establishment of sales, marketing, and distribution capabilities necessary for commercialization of our product candidates if and when approved, whether by us or in collaboration with third parties; • maintenance of a continued acceptable safety profile of our products post-approval; • acceptance of our product candidates, if and when approved by the applicable regulatory authorities, by patients, the medical community, and third-party payors; • ability of our products to compete with other therapies and treatment options; • establishment and maintenance of healthcare coverage and adequate reimbursement; and 24 Tabl e of Contents • expanded indications and patient populations for our products. The following table summarizes our research and development expenses for the periods indicated: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 (in thousands) (in thousands) External costs: Expenses related to licenses, sublicensing revenue, and milestones $ 421 $ 375 $ 1,972 $ 4,429 Services provided by CROs, CMOs, and third parties that conduct preclinical studies and clinical trials on our behalf 9,373 13,060 35,441 37,215 Other research and development expenses 2,491 3,655 11,844 18,065 Total external costs 12,285 17,090 49,257 59,709 Internal costs: Personnel-related expenses 7,475 9,869 27,291 30,344 Facilities and other allocated expenses 2,641 3,462 9,076 9,636 Total internal costs 10,116 13,331 36,367 39,980 Total research and development expenses $ 22,401 $ 30,421 $ 85,624 $ 99,689 General and Administrative Expenses Our general and administrative expenses consist primarily of personnel-related costs, intellectual property costs, consulting costs, and allocated overhead, including rent, equipment depreciation, and utilities. Personnel-related costs consist of salaries, benefits, and stock-based compensation for our general and administrative personnel. Intellectual property costs include expenses for filing, prosecuting, and maintaining patents and patent applications, including certain patents and patent applications that we license from third parties. We are entitled to receive reimbursement from third parties of a portion of the costs for filing, prosecuting, and maintaining certain patents and patent applications. We accrue for these reimbursements as the respective expenses are incurred and classify such reimbursements as a reduction of general and administrative expenses. We expect that our general and administrative expenses will increase in the future if our clinical trials are successful and if we prepare for potential commercialization of our product candidates, as expenses would increase to support the growth and operations of a public company with late-stage clinical programs and potential commercial products. Impairment Charges Impairment charges consist of charges related to the strategic pipeline prioritization with workforce and cost reduction initiatives announced on April 24, 2025, and include impairment of our leasehold improvements, right of use assets, and lab equipment. See Note 14 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information. Other Income (Expense) Other income (expense) consists primarily of impairment of an equity investment, interest income earned on cash and marketable securities and, the change in fair value of the MSKCC success payments liability under our now-terminated MSKCC Agreement. 25 Tabl e of Contents Results of Operations Comparison of the Three Months Ended September 30, 2025, and September 30, 2024 The following table summarizes our results of operations for the periods indicated: Three Months Ended September 30, 2025 2024 Change (in thousands) Licensing and collaboration revenue $ 2,198 $ 2,024 $ 174 Operating expenses: Research and development 22,401 30,421 (8,020) General and administrative 9,197 9,841 (644) Total operating expenses 31,598 40,262 (8,664) Loss from operations (29,400) (38,238) 8,838 Other income (expense): Change in fair value of the MSKCC success payments liability — (164) 164 Other income, net 1,852 3,718 (1,866) Total other income (expense) 1,852 3,554 (1,702) Net loss $ (27,548) $ (34,684) $ 7,136 Licensing and Collaboration Revenue Licensing and collaboration revenue increased by $0.2 million to $2.2 million for the three months ended September 30, 2025, from $2.0 million for the three months ended September 30, 2024. This increase relates to a $0.2 million increase related to other licensees. The following table summarizes our revenue by licensee for the periods indicated: Three Months Ended September 30, 2025 2024 Change (in thousands) Pfizer, related party 622 622 — Other licensees 1,576 1,402 174 Total licensing and collaboration revenue $ 2,198 $ 2,024 $ 174 Research and Development Expenses Research and development expenses decreased by $8.0 million to $22.4 million for the three months ended September 30, 2025, from $30.4 million for the three months ended September 30, 2024. This decrease was primarily related to decreases of (i) $3.7 million in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates, driven by decreases of (a) $3.0 million due to timing of CMO activities and (b) $0.7 million in CRO activities for our clinical trials, (ii) $2.4 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization, (iii) $1.1 million in other research and development expenses primarily related to the reduction in workforce and strategic pipeline prioritization, and (iv) $0.8 million in other facilities and allocated expenses. General and Administrative Expenses General and administrative expenses decreased by $0.6 million to $9.2 million for the three months ended September 30, 2025, from $9.8 million for the three months ended September 30, 2024. This decrease was primarily related to a decrease of $1.0 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization; partially offset by an increase of $0.4 million in legal and other service-related expenses. 26 Tabl e of Contents Total Other Income (Expense) Total other income (expense) decreased by $1.7 million for the three months ended September 30, 2025, as compared to the three months ended September 30, 2024. During the second quarter of 2025, we re-measured the fair value of the MSKCC success payments liability and estimated the fair value to be zero; thus, no subsequent changes in fair value were recognized through the August 11, 2025 termination date. We recognized a loss related to the change in the fair value of the MSKCC success payments liability in the amount of $0.2 million for the three months ended September 30, 2024. Other income, net decreased by $1.9 million for the three months ended September 30, 2025, compared to September 30, 2024. This decrease was primarily related to a $1.9 million decrease in interest income earned from marketable securities. Comparison of the Nine Months Ended September 30, 2025, and September 30, 2024 The following table summarizes our results of operations for the periods indicated: Nine Months Ended September 30, 2025 2024 Change (in thousands) Licensing and collaboration revenue $ 7,218 $ 7,917 $ (699) Operating expenses: Research and development 85,624 99,689 (14,065) General and administrative 29,335 35,969 (6,634) Impairment charges 12,150 — 12,150 Total operating expenses 127,109 135,658 (8,549) Loss from operations (119,891) (127,741) 7,850 Other income (expense): Impairment of equity investment (9,158) — (9,158) Change in fair value of the MSKCC success payments liability 785 1,934 (1,149) Other income, net 6,627 12,192 (5,565) Total other income (expense) (1,746) 14,126 (15,872) Net loss $ (121,637) $ (113,615) $ (8,022) Licensing and Collaboration Revenue Licensing and collaboration revenue decreased by $0.7 million to $7.2 million for the nine months ended September 30, 2025, from $7.9 million for the nine months ended September 30, 2024. This decrease is primarily due to a $1.6 million decrease in revenue related to the issuance of additional shares of convertible preferred stock received as consideration to us under the Edge chRDNA License Agreement in the nine months ended September 30, 2024, which was partially offset by a $0.9 million increase related to other licensees. The following table summarizes our revenue by licensee for the periods indicated: Nine Months Ended September 30, 2025 2024 Change (in thousands) Edge Animal Health, related party $ — $ 1,623 $ (1,623) Pfizer, related party 1,865 1,865 — Other licensees 5,353 4,429 924 Total licensing and collaboration revenue $ 7,218 $ 7,917 $ (699) 27 Tabl e of Contents Research and Development Expenses Research and development expenses decreased by $14.1 million to $85.6 million for the nine months ended September 30, 2025, from $99.7 million for the nine months ended September 30, 2024. This decrease was primarily related to (i) a decrease of $6.2 million in other research and development expenses primarily related to the reduction in workforce and strategic pipeline prioritization, (ii) a decrease of $3.1 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization, (iii) a decrease of $2.4 million in expenses related to licenses, sublicensing revenue, and milestones, (iv) a net decrease of $1.8 million in external CMO and CRO activities, driven by a decrease of (a) $2.5 million due to timing of CMO activities, and an increase of (b) $0.7 million in CRO activities for our clinical trials, and (v) a decrease of $0.6 million in other facilities and allocated expenses. General and Administrative Expenses General and administrative expenses decreased by $6.6 million to $29.3 million for the nine months ended September 30, 2025, from $36.0 million for the nine months ended September 30, 2024. This decrease was primarily related to a decrease of $4.3 million in legal expenses, including $3.9 million related to the accrual of a securities class action litigation settlement expense in 2024 and a decrease of $2.3 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization. Impairment Charges In connection with the previously announced strategic pipeline prioritization, impairment charges were $12.2 million for the nine months ended September 30, 2025, compared to zero for the nine months ended September 30, 2024. These charges include $7.4 million for tenant improvements, $2.6 million for the right-of-use asset, and $2.2 million for lab equipment. Total Other Income (Expense) Total other income decreased by $15.9 million for the nine months ended September 30, 2025, as compared to the nine months ended September 30, 2024. Impairment of equity investment was $9.2 million related to our equity investment in Edge for the nine months ended September 30, 2025, compared to zero for the nine months ended September 30, 2024. See Note 3 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information. In connection with our now-terminated MSKCC Agreement, we recognized a gain of $0.8 million related to the change in the fair value of the MSKCC success payments liability for the nine months ended September 30, 2025. We recognized a gain of $1.9 million related to the change in the fair value of the MSKCC success payments liability for the nine months ended September 30, 2024. Other income, net decreased by $5.6 million for the nine months ended September 30, 2025, compared to September 30, 2024. This decrease was primarily related to a $5.7 million decrease in interest income earned from marketable securities. Liquidity, Capital Resources, and Capital Requirements Sources of Liquidity Since our inception through September 30, 2025, we have raised an aggregate net proceeds of $841.3 million to fund our operations through our initial public offering (“IPO”); sales of convertible preferred stock; a follow-on public offering; proceeds from our licensing, licensing and collaboration, service, and patent assignment agreements, including sales of Intellia stock; private placements; at-the-market equity offerings; and government grants. As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $159.2 million. Shelf Registration Statements On August 9, 2022, we filed a universal shelf registration statement on Form S-3 (“2022 Shelf Registration Statement”) with the SEC, which allowed us to sell, from time to time, up to $400.0 million of common stock, preferred stock, debt securities, warrants, rights, or units comprised of any combination thereof (including the $100.0 million of 28 Tabl e of Contents common stock reserved under the 2022 Shelf Registration Statement for our at-the-market equity offering program described below). On May 8, 2025, in anticipation of the expiration of the 2022 Shelf Registration Statement on August 16, 2025, we filed a new shelf registration statement on Form S-3 (“2025 Shelf Registration Statement”), which was declared effective by the SEC on May 14, 2025. Upon the effectiveness of the 2025 Shelf Registration Statement, the offering of securities under the 2022 Shelf Registration Statement was deemed terminated. Pursuant to the 2025 Shelf Registration Statement, we may, from time to time, sell up to $300.0 million of common stock, preferred stock, debt securities, warrants, rights, or units comprised of any combination thereof (including the $100.0 million of common stock reserved under the 2025 Shelf Registration Statement for our at-the-market equity offering program described below). As of September 30, 2025, we had the full amount available for sale under the 2025 Shelf Registration Statement. At-the-Market Equity Offering Program On August 9, 2022, we entered into an Open Market Sale Agreement SM (“ATM Sales Agreement”) with Jefferies LLC (“Jefferies”), pursuant to which, on the terms and subject to the conditions and limitations set forth in the ATM Sales Agreement, from time to time, we could have issued and sold, through Jefferies, acting as sales agent, up to $100.0 million of our shares of common stock under the 2022 Shelf Registration Statement. Through May 14, 2025, we issued and sold an aggregate of 3,588,696 shares of our common stock under the ATM Sales Agreement and the 2022 Shelf Registration Statement at an average price per share of $4.71 for aggregate gross proceeds of $16.9 million ($16.2 million net of offering expenses). During each of the three- and nine-month periods ended September 30, 2025, we did not sell any shares of our common stock under the 2022 ATM Sales Agreement. With the effectiveness of the 2025 Shelf Registration Statement, we refreshed our at-the-market program under the ATM Sales Agreement. We may, from time to time, sell and issue shares of our common stock, through Jefferies as sales agent under the ATM Sales Agreement, having an aggregate offering price of up to $100.0 million in gross proceeds under the 2025 Shelf Registration Statement, by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act. Jefferies has agreed to use commercially reasonable efforts consistent with its normal sales and trading practices to sell shares from time to time, based on our instructions (including any price or size limits or other customary parameters or conditions we may impose). During each of the three- and nine-month periods ended September 30, 2025, we did not sell any shares of our common stock pursuant to the ATM Sales Agreement under either the 2022 Shelf Registration Statement or the 2025 Shelf Registration Statement. As of September 30, 2025, $100.0 million of shares of our common stock remained available for sale under the 2025 Shelf Registration Statement pursuant to the ATM Sales Agreement. Funding Requirements We expect that our existing cash, cash equivalents, and marketable securities will be sufficient to fund our operations for at least the next 12 months from the date this Form 10-Q is filed. We have based these estimates on our current assumptions, which may require future adjustments based on our ongoing business decisions. We will continue to be dependent on equity financing, debt financing, collaboration and licensing arrangements, and/or other forms of capital raises to fund operating expenses, including to fully fund our planned vispa-cel pivotal trial, at least until we are able to generate significant positive cash flows from our operations. We have no current ongoing material financing commitments, such as lines of credit or guarantees, that are expected to affect our liquidity over the next five years, except for our lease commitments and payments under certain of our license agreements as described in Notes 4 and 8 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q. Our primary use of cash is to fund operating expenses and research and development expenses, which primarily consists of expenditures related to clinical trials. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable, accrued expenses, and prepaid expenses. Our future funding requirements will depend on many factors, including the following: • the initiation, progress, timing, costs, and results of clinical trials for our product candidates; • the clinical development plans we establish for these product candidates; 29 Tabl e of Contents • the outcome, timing, and cost of meeting regulatory requirements established by the FDA and other comparable foreign regulatory authorities; • the potential impact of reductions in government spending and personnel under the new Administration; • whether we enter into any collaboration agreements and the terms of any such agreements; • the cost of filing and prosecuting our patent applications, and maintaining and enforcing our patents and other intellectual property rights; • the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against our products after we receive regulatory approval; • the effect of competing technological and market developments; • the cost and timing of completion of commercial-scale outsourced manufacturing activities or the cost and timing of completion of clinical-scale and commercial-scale internal manufacturing activities; • the cost of establishing sales, marketing, and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products; • the amount of revenue, if any, received from commercial sales of our product candidates, should any of our product candidates receive marketing approval; • the achievement of milestones or occurrence of other developments that trigger payments by or to third parties; • our implementation of various computerized informational systems and efforts to enhance operational systems; • the impact of public health crises or geopolitical events on our clinical development or operations; • the impact of inflationary pressures and potential tariffs on the cost of our operations; and • the costs of operating as a public company, including defending against any future class action securities litigation and shareholder derivative lawsuits. Furthermore, our operating plans may change, and we expect to need additional funds to meet operational needs and capital requirements for our clinical trials and development of our product candidates. Because of the numerous risks and uncertainties associated with therapeutic product development, we may never achieve profitability and, unless and until we are able to develop and commercialize our product candidates, we will need to continue to raise additional capital. Until we can generate significant revenue from product sales, if ever, we expect to finance our operations through equity offerings (including our at-the-market equity offering program), debt financings, collaborations and strategic alliances, licensing arrangements, and/or other sources. There are no assurances that we will be successful in obtaining an adequate level of financing to support our business plans as needed on acceptable terms, or at all. If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our intellectual property, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. Disruptions and volatility in the global and domestic capital markets resulting from heightened inflation, potential tariffs, capital market volatility, interest rate and currency rate fluctuations, artificial intelligence, government agency changes under the new Administration, any potential economic slowdown or recession, including trade wars or civil or political unrest (such as the ongoing war between Ukraine and Russia, conflict in the Middle East, and tension between China and Taiwan) may increase the cost of capital and limit our ability to access capital. If we are unable to raise capital as and when needed or on attractive terms, we may have to significantly delay, reduce, or discontinue the development and commercialization of our product candidates or scale back or terminate our pursuit of new in-licenses and acquisitions. 30 Tabl e of Contents Cash Flows Comparison of the Nine Months Ended September 30, 2025, and September 30, 2024 The following table summarizes our cash flows for the periods indicated: Nine Months Ended September 30, 2025 2024 Change (in thousands) Cash used in operating activities $ (90,168) $ (102,730) $ 12,562 Cash provided by investing activities 84,268 70,677 13,591 Cash provided by financing activities 751 12,861 (12,110) Net decrease in cash, and cash equivalents, and restricted cash $ (5,149) $ (19,192) $ 14,043 Cash Used in Operating Activities Net cash used in operating activities was $90.2 million for the nine months ended September 30, 2025, compared to $102.7 million for the nine months ended September 30, 2024. This decrease was due to (i) changes in the components of net loss primarily related to (a) an increase in non-cash charges primarily for impairment charges and impairment of equity investment incurred for the nine months ended September 30, 2025, and (b) decreases in research and development expenses and general and administrative expenses; and (ii) a decrease in net changes in our operating assets and liabilities primarily related to a decrease in net changes of accrued expenses and other current liabilities; partially offset by an increase in net changes in other assets. Cash Provided by Investing Activities Net cash provided by investing activities was $84.3 million for the nine months ended September 30, 2025, compared to $70.7 million for the nine months ended September 30, 2024. The increase in net cash provided by investing activities was primarily driven by lower cash utilized for purchases of marketable securities; partially offset by a decrease in proceeds from maturities of marketable securities. Cash Provided by Financing Activities Net cash provided by financing activities was $0.8 million for the nine months ended September 30, 2025, compared to $12.9 million for the nine months ended September 30, 2024. The decrease was primarily driven by proceeds from the issuance of common stock under the ATM Sales Agreement, net of offering expenses, during the nine months ended September 30, 2024. We did not sell any common stock pursuant to the ATM Sales Agreement during the nine months ended September 30, 2025, under either the 2022 Shelf Registration Statement or the 2025 Shelf Registration Statement. Critical Accounting Policies and Significant Judgments and Estimates Our critical accounting policies are disclosed in our audited consolidated financial statements for the year ended December 31, 2024, and the related notes included in our Form 10-K. Since the date of such financial statements, there have been no material changes to our significant accounting policies. Other than the estimates noted below, there have been no material changes to our critical accounting estimates as compared to those disclosed in our Form 10-K. Impairment of Long-Lived Assets We review long-lived assets for impairment whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. In order to determine if assets have been impaired, assets are grouped and tested at the lowest level for which identifiable independent cash flows are available. An impairment loss is recognized when the sum of projected undiscounted cash flows is less than the carrying value of the asset group. The measurement of the impairment loss to be recognized is based on the difference between the fair value and the carrying value of the asset group. Fair value may be determined using a market approach or income approach. 31 Tabl e of Contents Recently Issued Accounting Pronouncements See Note 2 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information. Item 3. Quantitative and Qualitative Disclosures about Market Risk. There have been no material changes to our market risk during the nine months ended September 30, 2025. For a discussion of our exposure to market risk, refer to the section titled “Quantitative and Qualitative Disclosures About Market Risk” in our Form 10-K.