Caribou Biosciences, Inc.
For the period ended Sep 30, 2025. Filed Nov 12, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 12, 2025
CRBU$0.45+8.8%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-12.
0001619856-25-000061
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-12.
0001619856-25-000071
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 144 previous paragraphs and 143 current paragraphs.
99 unchanged; 37 reworded; 7 added; 6 removed; 2 previous and 0 current paragraphs rearranged.
Tables are not compared: 6 previous and 6 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (7)
Current report
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.
Current report
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.
Current report
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.
Current report
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 (150x106 viable CAR-T cells, N=6; 300x106 viable CAR-T cells, N=13; 450x106 viable CAR-T cells, N=13; and 800x106 viable CAR-T cells, N=3). We also announced on November 3, 2025, that we have selected the 450x106 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 450x106 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.
Current report
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.
Current report
Comparison of the Nine Months Ended September 30, 2025, and September 30, 2024
Current report
Comparison of the Nine Months Ended September 30, 2025, and September 30, 2024
Removed paragraphs (6)
Previous report
To our knowledge, CB-010 is the first clinical-stage allogeneic anti-CD19 CAR-T cell therapy with a genome-edited knockout of the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface. At a poster presentation during the 2024 American Society of Clinical Oncology (“ASCO”) Annual Meeting in June 2024, we presented safety, efficacy, and translational data for the first 46 patients evaluated in our ANTLER phase 1 clinical trial after a single dose of CB-010. CB-010 was generally well-tolerated with adverse events as expected for anti-CD19 CAR-T cell therapies. A retrospective analysis of all patient data demonstrated that patients who received a dose of CB-010 manufactured from a donor with at least four matching human leukocyte antigen (“HLA”) alleles (of 12 total alleles) with the patient (referred to as “partial HLA matching”) resulted in improved progression-free survival (“PFS”) compared to patients who received a single dose of CB-010 from a donor with fewer than four matching HLA alleles. To confirm the partial HLA matching strategy, we have completed enrollment of approximately 20 additional 2L LBCL patients in the ongoing ANTLER phase 1 clinical trial. In addition, we are enrolling a proof-of-concept cohort of up to 10 patients in our ANTLER phase 1 clinical trial who have relapsed following any prior CD19-targeted therapy in this population of unmet need.
Previous report
To our knowledge, CB-011 is the first anti-BCMA 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”). This immune cloaking armoring strategy results in no endogenous class I HLA alleles expressed on the CAR-T cell surface, except for expression of the B2M-HLA-E transgene. This reduces the number of potential mismatched HLA alleles to six from 12, resulting in a reduced risk of rapid immunologic clearing of the CAR-T cells by the patient. In the dose escalation portion of our CaMMouflage phase 1 clinical trial, we implemented a deeper lymphodepletion regimen that includes an increased dose of cyclophosphamide (up from the original 300 mg/m 2/day to 500 mg/m 2/day together with the same fludarabine dose of 30 mg/m 2/day for three days). We have completed planned enrollment of additional patients with this lymphodepletion regimen across multiple dose levels (150x106 viable CAR-T cells, 300x106 viable CAR-T cells, 450x106 viable CAR-T cells, and 800x106 viable CAR-T cells) in order to further define safety and efficacy and to determine the recommended dose(s) for expansion. No dose-limiting toxicities have been observed at any dose level.
Previous report
Our common stock is currently listed for trading on the Nasdaq Global Select Market under the symbol “CRBU.” The continued listing of our common stock on Nasdaq is subject to our compliance with a number of listing standards. On May 7, 2025, we received a letter from the Listing Qualifications Staff (“Staff”) of The Nasdaq Stock Market LLC notifying us that, for the prior 30 consecutive business days, the bid price for our shares of common stock had closed below $1.00 per share, which is the minimum bid price required to maintain continued listing on the Nasdaq Global Select Market under Nasdaq Listing Rule 5450(a)(1) (“Minimum Bid Price Rule”). On June 18, 2025, we received a written notice (“Compliance Notice”) from the Staff notifying us that, from June 3, 2025, to June 17, 2025, the closing bid price of our common stock had been $1.00 per share or greater and, accordingly, we had regained compliance with the Minimum Bid Price Rule.
Previous report
Licensing and collaboration revenue decreased by $0.8 million to $2.7 million for the three months ended June 30, 2025, from $3.5 million for the three months ended June 30, 2024. This decrease primarily relates to a $1.6 million decrease related to the Exclusive License Agreement for Veterinary Therapeutics (as amended, the “Edge chRDNA License Agreement”) with Edge Animal Health (“Edge”) reflecting the issuance of additional shares of convertible preferred stock by Edge to us in the three months ended June 30, 2024, which was partially offset by a $0.8 million increase related to other licensees.
Previous report
Comparison of the Six Months Ended June 30, 2025, and June 30, 2024
Previous report
Comparison of the Six Months Ended June 30, 2025, and June 30, 2024
Reworded paragraphs (37)
Previous wording
We are a clinical-stage Clustered Regularly Interspaced Short Palindromic Repeats (“CRISPR”) genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. Our genome-editing platform, including our novel chRDNA (CRISPRh ybrid RNA-DNA, or “chRDNA,” 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 devastating diseases.
Current wording
We are a clinical-stage Clustered Regularly Interspaced Short Palindromic Repeats (“CRISPR”) genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. Our genome-editing platform, including our novel chRDNA (CRISPRh ybrid RNA-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.
Previous wording
•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
Current wording
•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
Previous wording
CB-010 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.
Current wording
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.
Previous wording
On April 24, 2025, we announced a strategic pipeline prioritization with workforce and cost reduction initiatives to focus resources on our CB-010 and CB-011 product candidates. At that time, we disclosed that we had discontinued our GALLOP phase 1 trial of CB-010 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 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 each of the three- and six-month periods ended June 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 each of the three- and six-month periods ended June 30, 2025, we recorded wind down costs of $0.3 million as research and development expenses in our unaudited condensed consolidated statements of operations and comprehensive loss related to the discontinuation of our GALLOP phase 1 clinical trial and AMpLify phase 1 clinical trial. In connection with the discontinuation of the AMpLify phase 1 clinical trial for our CB-012 product candidate, we provided notice of termination on May 13, 2025, to Memorial Sloan Kettering Cancer Center (“MSKCC”) of our Exclusive License Agreement, dated November 13, 2020, with MSKCC (as amended, “MSKCC Agreement”), which termination was effective on August 11, 2025. Under the MSKCC Agreement, we had exclusively licensed from MSKCC know-how, biological materials, and related patent families to the single-chain variable fragment (“scFv”) used in our CB-012 product candidate.
Current wording
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 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.
Previous wording
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, in connection with the preparation of the financial statements for the three and six months ended June 30, 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 each of the three- and six-month periods ended June 30, 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 each of the three- and six-month periods ended June 30, 2025.
Current wording
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.
Previous wording
Our net losses for the three months ended June 30, 2025, and June 30, 2024, were $54.1 million and $37.7 million, respectively. Our net losses for the six months ended June 30, 2025, and June 30, 2024, were $94.1 million and $78.9 million, respectively. We had an accumulated deficit of $542.5 million as of June 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 nonclinical studies and our other research and development expenses. We anticipate that our expenses will increase substantially as we:
Current wording
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:
Previous wording
•experience any delays, challenges, or other issues associated with any of the above, including the failure of 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;
Current wording
•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;
Previous wording
•continue to operate as a public company, including defending against any class action securities litigation and shareholder derivative lawsuits.
Current wording
•continue to operate as a public company, including defending against any future class action securities litigation and shareholder derivative lawsuits.
Previous wording
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 $2.7 million and $3.5 million for the three months ended June 30, 2025, and June 30, 2024, respectively, and $5.0 million and $5.9 million for the six months ended June 30, 2025, and June 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.
Current wording
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.
Previous wording
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 MSKCC Agreement.
Current wording
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.
Previous wording
Research and development expenses decreased by $7.8 million to $27.7 million for the three months ended June 30, 2025, from $35.5 million for the three months ended June 30, 2024. This decrease was primarily related to decreases of (i) $4.6 million in other research and development expenses primarily related to the reduction in workforce and strategic pipeline prioritization, (ii) $2.0 million in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates, driven by decreases of (a) $1.4 million due to timing of CMO activities and (b) $0.6 million in CRO activities for clinical trials, (iii) $1.0 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization, and (iv) $0.3 million in other facilities and allocated expenses.
Current wording
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.
Previous wording
General and administrative expenses decreased by $1.1 million to $10.4 million for the three months ended June 30, 2025, from $11.5 million for the three months ended June 30, 2024. This decrease was primarily related to (i) a decrease of $0.9 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization, (ii) a decrease of $0.3 million in patent prosecution and maintenance costs, (iii) a decrease of $0.3 million in legal, and other service related expenses; partially offset by an increase of $0.4 million in other facilities and allocated expenses.
Current wording
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.
Previous wording
Total other income (expense) decreased by $12.3 million for the three months ended June 30, 2025, as compared to the three months ended June 30, 2024.
Current wording
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.
Previous wording
In connection with our termination of the MSKCC Agreement, we recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $0.5 million for the three months ended June 30, 2025. We recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $1.8 million for the three months ended June 30, 2024.
Current wording
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.
Previous wording
Other income, net decreased by $1.8 million for the three months ended June 30, 2025, compared to June 30, 2024. This decrease was primarily related to a $1.9 million decrease in interest income earned from marketable securities.
Current wording
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.
Previous wording
Licensing and collaboration revenue decreased by $0.9 million to $5.0 million for the six months ended June 30, 2025, from $5.9 million for the six months ended June 30, 2024. This decrease primarily relates to a $1.6 million decrease related to the Edge chRDNA License Agreement reflecting the issuance of additional shares of convertible preferred stock by Edge to us in the six months ended June 30, 2024, which was partially offset by a $0.8 million increase related to other licensees.
Current wording
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.
Previous wording
Research and development expenses decreased by $6.0 million to $63.2 million for the six months ended June 30, 2025, from $69.3 million for the six months ended June 30, 2024. This decrease was primarily related to (i) a decrease of $5.1 million in other research and development expenses primarily related to the reduction in workforce and strategic pipeline prioritization, (ii) a decrease of $2.5 million in expenses related to licenses, sublicensing revenue, and milestones, (iii) a decrease of $0.6 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization; partially offset by an increase of $1.9 million in external CMO and CRO activities, driven by increases of (a) $1.4 million in CRO activities for clinical trials, and (b) $0.5 million due to timing of CMO activities, and an increase of $0.3 million in other facilities and allocated expenses.
Current wording
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.
Previous wording
General and administrative expenses decreased by $6.0 million to $20.1 million for the six months ended June 30, 2025, from $26.1 million for the six months ended June 30, 2024. This decrease was primarily related to (i) a decrease of $4.7 million in legal expenses, including $3.9 million related to the accrual of a securities class action litigation settlement expense in 2024, (ii) a decrease of $1.3 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization, (iii) a decrease of $0.3 million in patent prosecution and maintenance costs; partially offset by an increase of $0.3 million in facilities and allocated expenses.
Current wording
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.
Previous wording
In conjunction with the previously announced strategic pipeline prioritization, impairment charges were $12.2 million for the three months ended June 30, 2025, compared to zero for the three months ended June 30, 2024. These charges include $7.4 million related to tenant improvements, $2.6 million for the right-of-use asset, and $2.2 million for lab equipment.
Current wording
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.
Previous wording
Total other income decreased by $5.0 million for the six months ended June 30, 2025, as compared to the six months ended June 30, 2024.
Current wording
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.
Previous wording
Impairment of equity investment was $9.2 million related to our equity investment in Edge for the three months ended June 30, 2025, compared to zero for the three months ended June 30, 2024. See Note 3 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.
Current wording
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.
Previous wording
In connection with our termination of the MSKCC Agreement, we recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $0.8 million for the six months ended June 30, 2025. We recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $2.1 million for the six months ended June 30, 2024.
Current wording
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.
Previous wording
Other income, net decreased by $3.7 million for the six months ended June 30, 2025, compared to June 30, 2024. This decrease was primarily related to a $3.8 million decrease in interest income earned from marketable securities.
Current wording
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.
Previous wording
Since our inception through June 30, 2025, we have raised an aggregate net proceeds of $840.0 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.
Current wording
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.
Previous wording
As of June 30, 2025, we had cash, cash equivalents, and marketable securities of $183.9 million.
Current wording
As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $159.2 million.
Previous wording
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 common stock reserved under the 2022 Shelf Registration Statement for our at-the-market equity offering program described below). The 2022 Shelf Registration Statement was declared effective by the SEC on August 16, 2022, and was scheduled to expire after three years from the date of effectiveness.
Current wording
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 common stock reserved under the 2022 Shelf Registration Statement for our at-the-market equity offering program described below).
Previous wording
On May 8, 2025, in anticipation of the expiration of the 2022 Shelf Registration Statement, we filed a new shelf registration statement on Form S-3 (“2025 Shelf Registration Statement”). 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). The 2025 Shelf Registration Statement was declared effective by the SEC on May 14, 2025, and will expire after three years from the date of effectiveness. Upon effectiveness of the 2025 Shelf Registration Statement, the offering of securities under the 2022 Shelf Registration Statement was deemed terminated.
Current wording
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.
Previous wording
On August 9, 2022, we entered into an Open Market Sale Agreement SM (“ATM Sales Agreement”) with Jefferies LLC (“Jefferies”), pursuant to which, upon 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 six-month periods ended June 30, 2025, we did not sell any shares of our common stock under the 2022 ATM Sales Agreement.
Current wording
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.
Previous wording
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 upon our instructions (including any price or size limits or other customary parameters or conditions we may impose).
Current wording
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).
Previous wording
During each of the three- and six-month periods ended June 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.
Current wording
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.
Previous wording
We will continue to be dependent upon equity financing, debt financing, collaboration and licensing arrangements, and/or other forms of capital raises 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.
Current wording
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.
Previous wording
Our primary use of cash is to fund operating expenses and research and development expenditures. 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.
Current wording
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.
Previous wording
•the costs of operating as a public company, including defending against class action securities litigation and shareholder derivative lawsuits.
Current wording
•the costs of operating as a public company, including defending against any future class action securities litigation and shareholder derivative lawsuits.
Previous wording
Furthermore, our operating plans may change, and we expect to need additional funds to meet operational needs and capital requirements for clinical trials and other research and development expenditures.
Current wording
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.
Previous wording
Net cash used in operating activities was $65.0 million for the six months ended June 30, 2025, compared to $70.1 million for the six months ended June 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 six months ended June 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.
Current wording
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.
Previous wording
Net cash provided by investing activities was $73.4 million for the six months ended June 30, 2025, compared to $44.2 million for the six months ended June 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.
Current wording
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.
Previous wording
Net cash provided by financing activities was $0.5 million for the six months ended June 30, 2025, compared to $12.6 million for the six months ended June 30, 2024. The decrease was primarily driven by proceeds from the issuance of common stock under the ATM Sales Agreement, net of offering expenses, for the six months ended June 30, 2024. We did not sell any common stock pursuant to the ATM Sales Agreement during the six months ended June 30, 2025, under either the 2022 Shelf Registration Statement or the 2025 Shelf Registration Statement.
Current wording
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.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
In conjunction with the previously announced strategic pipeline prioritization, impairment charges were $12.2 million for the six months ended June 30, 2025, compared to zero for the six months ended June 30, 2024. These charges include $7.4 million related to tenant improvements, $2.6 million for the right-of-use asset, and $2.2 million for lab equipment.
Previous report — wording carried elsewhere
Impairment of equity investment was $9.2 million related to our equity investment in Edge for the six months ended June 30, 2025, compared to zero for the six months ended June 30, 2024. See Note 3 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.