CRISPR Therapeutics AG
For the period ended Dec 31, 2025. Filed Feb 12, 2026.
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A company's yearly report to the SEC, with audited financial statements. It is the fullest account a company gives of its business, its finances and the risks it sees.
FY2025 · Filed Feb 12, 2026
CRSP$50.07+3.1%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-K
Period ended 2024-12-31; filed 2025-02-11.
0000950170-25-017899
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-K
Period ended 2025-12-31; filed 2026-02-12.
0001193125-26-048957
Item 7. 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 151 previous paragraphs and 160 current paragraphs.
91 unchanged; 39 reworded; 27 added; 20 removed; 1 previous and 3 current paragraphs rearranged.
Tables are not compared: 3 previous and 4 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 (27)
Current report
In Vivo Liver Editing
Current report
Ourin vivo portfolio includes cardiovascular programs, such as CTX310, directed towards angiopoietin-related protein 3 or ANGPTL3, which is currently in an ongoing Phase 1b clinical trial in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia.
Current report
siRNA-based Programs
Current report
Our siRNA-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics and certain of its affiliates, or Sirius.
Current report
CTX611 (formerly known as SRSD107) is a novel double-stranded, long-acting siRNA, designed to target the human coagulation factor XI, or FXI, messenger RNA and inhibit FXI protein expression. Through modulation of the intrinsic coagulation pathway, CTX611 is intended to provide anticoagulant and antithrombotic effects. Supported by clinical experience conducted by Sirius in two Phase 1 clinical trials, CTX611 is being developed as a long-acting FXI inhibitor with the potential to support infrequent, including semi-annual, subcutaneous administration.
Current report
CTX611 is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty.
Current report
Zugocabtagene geleucel
Current report
In oncology, the Phase 1/2 clinical trial in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy is ongoing. Eligible disease subtypes include large B-cell lymphoma, or LBCL, follicular lymphoma grade 1-3a, marginal zone lymphoma, and mantle cell lymphoma. Initial positive clinical data generated through December 2025 support the advancement of zugo-cel into the Phase 2 portion of the ongoing Phase 1/2 trial. We have also established a collaboration and clinical supply agreement with Eli Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program’s development in oncology. Zugo-cel has been granted RMAT designation by the U.S. Food and Drug Administration for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.
Current report
Next-generation Editing Modalities
Current report
siRNA. In May 2025, we partnered with Sirius and entered into the Sirius Agreement pursuant to which, among other things, we and Sirius will collaborate on the research, development, manufacture, commercialization and use of the Sirius Collaboration Products, including co-development and co-commercialization of CTX611; and (2) Sirius granted us options to exclusively license Sirius siRNA technology to target up to two licensed targets from a list of seven reserved targets for the research, develop, manufacture and commercialization of siRNA Licensed Products, For the first Sirius Collaboration Product successfully developed, we will be the lead party responsible for commercialization efforts in the United States and Sirius will be the lead party responsible for commercialization efforts in Greater China.
Current report
Acquired In-Process Research and Development Expenses
Current report
Asset acquisition costs related to acquired technology are expensed as acquired in-process research and development at the point that they have no established alternative future use. We classify asset acquisitions of acquired in-process research and development as investing activities on our consolidated statements of cash flows.
Current report
Other Income (Expense), Net
Current report
$10.4 million of increased sublicense and license fees, primarily attributable to costs incurred related to a contingent liability as of December 31, 2025, as described in Note 9 of the notes to the consolidated financial statements included in this Annual Report on Form 10-K.
Current report
Acquired In-Process Research and Development Expenses
Current report
Acquired in-process research and development expenses were $96.3 million for the year ended December 31, 2025. There were no acquired in-process research and development expenses for the year ended December 31, 2024. The $96.3 million acquired in-process research and development expense is attributable to the costs incurred upon entering the Sirius Agreement during the second quarter of 2025, as described in Note 8 of the notes to the consolidated financial statements included in this Annual Report on Form 10-K.
Current report
Collaboration expense, net, was $213.5 million for the year ended December 31, 2025, compared to $120.7 million for the year ended December 31, 2024. In 2024, we exercised our option to defer specified costs under the CASGEVY program in excess of the $110.3 million deferral limit under the A&R Vertex JDCA, as amended. The increase of approximately $92.8 million in collaboration expense, net, was primarily attributable to reaching the deferral limit in 2024, as no such limit was applicable in 2025. Absent the deferral limit, the increase was primarily attributable to an increase in our share of CASGEVY operating expenses related to increased commercial and manufacturing costs for CASGEVY when compared to the prior period, which was partially offset by an increase in our share of CASGEVY revenue.
Current report
At-the-Market Offerings (ATM)
Current report
We entered into an Open Market Sale Agreement SM, or the Sales Agreement, with Jefferies LLC under which we, at our sole discretion, are able to offer and sell, from time to time at prevailing market prices, our common shares. The following are in connection with the Sales Agreement.
Current report
2021 ATM
Current report
In January 2021, we filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million, or, together with the subsequent prospectus supplements filed in July 2021 and August 2024 relating to the common shares remaining under the original prospectus supplement, the 2021 ATM. In 2025, we issued and sold an aggregate of 6.1 million common shares under the 2021 ATM at an average price of $59.63 per share for aggregate proceeds of $359.0 million, which were net of equity issuance costs of $4.7 million, excluding stamp taxes of $3.6 million.
Current report
As of December 31, 2025, we issued and sold an aggregate of 8.0 million common shares under the 2021 ATM at an average price of $74.77 per share for aggregate proceeds of $592.2 million, which were net of equity issuance costs of $7.8 million, excluding stamp taxes of $5.9 million. As of December 31, 2025, no common shares remain available under the 2021 ATM.
Current report
2025 ATM
Current report
In October 2025, we filed a new prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million, referred to herein as the 2025 ATM. In 2025, we issued and sold an aggregate of 0.7 million common shares under the 2025 ATM at an average price of $60.81 per share for aggregate proceeds of $42.3 million, which were net of equity issuance costs of $0.5 million, excluding stamp taxes of $0.4 million. Common shares having aggregate gross proceeds up to $557.2 million remain available under the 2025 ATM.
Current report
Share Issuance Agreement with Sirius Therapeutics
Current report
As described in Note 8 of the notes to the consolidated financial statements included in this Annual Report on Form 10-K, we and Sirius entered into a share issuance agreement and we registered and issued 1,842,105 common shares to Sirius, nominal value CHF 0.03 per share, at an issue price of $38.00 per share as partial consideration for entering into the Sirius Agreement.
Current report
Net cash used in investing activities for the year ended December 31, 2025 was $31.8 million, compared to net cash used in investing of $280.5 million for the year ended December 31, 2024. The decrease in net cash used in investing activities was primarily a result of the net purchase position for marketable debt securities for the year ended December 31, 2024, compared to a net maturity position for marketable debt securities for the year ended December 31, 2025.
Removed paragraphs (20)
Previous report
CD19 Candidates
Previous report
CD70 Candidates
Previous report
CTX131 is being developed for both solid tumors and hematologic malignancies. It is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the candidate in adult patients with relapsed or refractory solid tumors, as well as in hematologic malignancies, including, including T cell lymphomas, or TCL. We believe allogeneic CAR T approaches for TCL may have greater potential to meet the unmet need in this patient population given the patients’ own T cells are not suitable for autologous manufacturing.
Previous report
In Vivo
Previous report
Cardiovascular disease
Previous report
Our first two in vivo programs utilizing our proprietary LNP platform, CTX310 and CTX320, aim to address cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3, or ANGPTL3, and lipoprotein (a), or Lp(a), respectively. CTX310 is being investigated in an ongoing clinical trial targeting ANGPTL3 in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia. In addition,
Previous report
Building upon CTX310 and CTX320, we are progressing CTX340, targeting angiotensinogen for refractory hypertension, as well as CTX450, targeting 5’-aminolevulinate synthase 1 for acute hepatic porphyria, through preclinical studies. In addition, we have programs focused on gene correction in the liver, including programs leveraging technologies developed by our CRISPR-X research team. Finally, we are pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells.
Previous report
CRISPR-X
Previous report
Diabetes. Beginning in 2018, we partnered with ViaCyte, Inc., or ViaCyte (now a wholly-owned subsidiary of Vertex), to pursue the discovery, development and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes. In 2023, ViaCyte elected to opt-out of the collaboration with us for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, the on-going collaboration assets will be wholly owned by us, subject to a royalty on future sales owed to ViaCyte. Our product candidate, CTX211, being developed for the potential treatment of T1D, resulted from this collaboration, and which we are continuing to advance in a Phase 1 clinical trial. Additionally, in 2023, we entered into a non-exclusive license agreement with Vertex for Vertex to utilize certain of our gene-editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent/requiring diabetes throughout the world. To date, we have recognized revenue of $205 million in upfront and milestone payments and remain eligible to receive additional research and development milestones and royalties on future products under the license.
Previous report
Research and development expenses
Previous report
Research and Development Expenses
Previous report
$12.9 million of decreased sublicense and license fees, primarily attributable to a decrease in research and development licenses and milestones due to strategic partners;
Previous report
Collaboration expense, net, was $110.3 million for the year ended December 31, 2024, compared to $130.3 million for the year ended December 31, 2023. The decrease of $20.0 million was attributable to our share of certain costs in 2023 arising from a license agreement between Vertex and a third party, which we agreed to pay to Vertex upon the occurrence of an event specified in Amendment No. 1 to the A&R Vertex JDCA. No similar costs were incurred in 2024. Refer to Note 8 of the notes to our consolidated financial statements included in this Annual Report on Form 10-K for a description of revenue recognized related to Vertex.
Previous report
In 2024, 2023 and 2022, we exercised our option to defer specified costs on the CASGEVY program in excess of the deferral limit under A&R Vertex JDCA, as amended, resulting in deferred costs of $102.8 million, $80.9 million and $36.1 million, respectively. Any deferred amounts are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amount per year. These deferred costs on the CASGEVY program will be recognized by us when recoverability of such deferred amounts by Vertex is probable and the amount can be reasonably estimated. As of December 31, 2024, no contingent payments have been accrued to date. Refer to Note 8 of the notes to our consolidated financial statements in this Annual Report on Form 10-K for further discussion on our arrangements with Vertex.
Previous report
Other income, net
Previous report
At-the-Market Offerings
Previous report
In August 2019, we entered into an Open Market Sale Agreement SM with Jefferies under which we were able to offer and sell, from time to time at our sole discretion through Jefferies, as our sales agent, our common shares, par value of CHF 0.03 per share, or the August 2019 Sales Agreement. In connection with the August 2019 Sales Agreement, as amended, we have filed several prospectus supplements with the SEC to offer and sell, from time to time, common shares. We filed our current prospectus supplement for $378.6 million in August 2024.
Previous report
In 2022, the sale of common shares pursuant to the 2019 Sales Agreement and the applicable prospectus supplement was not significant.
Previous report
As of December 31, 2024, we have common shares having aggregate gross proceeds up to $363.7 million remaining under our current prospectus supplement, and we have issued and sold an aggregate of 1.9 million common shares at an average price of $122.73 per share for aggregate proceeds of $233.2 million, which were net of equity issuance costs of $3.1 million, excluding stamp taxes.
Previous report
Net cash used in investing activities for the year ended December 31, 2024 was $280.5 million and consisted primarily of purchases of marketable securities in excess of maturities of marketable securities of $255.4 million. Net cash provided by investing activities for the year ended December 31, 2023 was $374.6 million and consisted primarily of maturities of marketable securities in excess of purchases of marketable securities of $386.6 million.
Reworded paragraphs (39)
Previous wording
Our mission is to create transformative gene-based medicines for serious human diseases. We are a leading gene editing company focused on the development of CRISPR-based therapeutics, including by using CRISPR/Cas9 technology. CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We aim to apply this technology to disrupt, delete, correct and insert genes to treat genetic diseases and to engineer advanced cellular therapies. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex. We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, CAR T, in vivo approaches and type 1 diabetes. Depending on the program, we take either anex vivo approach, in which we edit cells outside of the human body before administering them to the patient, or anin vivo editing approach, where we deliver the CRISPR-based therapeutic directly to target cells within the human body.
Current wording
Our mission is to create transformative gene-based medicines for serious human diseases. We are a leading biopharmaceutical company focused on the development of CRISPR-based therapeutics, including by using CRISPR/Cas9 technology. We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, in vivo approaches, CAR T, and regenerative medicine. Depending on the program, we take either anex vivo approach, in which we edit cells outside of the human body before administering them to the patient, or anin vivo editing approach, where we deliver the CRISPR-based therapeutic directly to target cells within the human body.
Previous wording
We continue to innovate on our platform to develop next-generation technologies that can enable new therapies. Through our efforts, we aim to unlock the full potential of CRISPR-based therapeutics to create medicines that can transform people's lives. We believe that our innovative research, translational expertise, and clinical development experience, position us as a leader in the development of CRISPR-based therapeutics and may enable us to create an entirely new class of highly effective and potentially curative therapies for patients with both rare and common diseases for whom current biopharmaceutical approaches have had limited success.
Current wording
We continue to innovate on our platform to develop next-generation technologies that can enable new therapies. We are developing other technologies, including delivery technologies and other gene editing technologies, like SyNTase. Through our efforts, we aim to unlock the full potential of gene-based therapeutics to create medicines that can transform people’s lives. We believe that our innovative research, translational expertise, and clinical development experience, position us as a leader in the development of CRISPR-based therapeutics and may enable us to create an entirely new class of highly effective and potentially curative therapies for patients with both common and rare diseases for whom current biopharmaceutical approaches have had limited success.
Previous wording
Bahrain and the United Arab Emirates for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including (1) three clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, (2) two clinical trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and (3) long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.
Current wording
In 2023, CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in the world. To date, CASGEVY has been approved in the United States, European Union, Great Britain, Canada, Switzerland and certain countries in the Middle East for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including in clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, two pivotal trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. Overall, CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.
Previous wording
We continue to advance our internally developed targeted conditioning program, as well as in vivo hematopoietic stem cell editing approaches utilizing lipid nanoparticle-mediated delivery through preclinical studies. Both initiatives could significantly expand the addressable patient populations for SCD and TDT.
Current wording
We continue to advance our internally developed targeted conditioning program, as well asin vivo hematopoietic stem cell editing approaches utilizing lipid nanoparticle-mediated delivery through preclinical studies. Both initiatives could significantly expand the addressable patient populations for SCD and TDT.
Previous wording
Ourin vivo gene editing strategy focuses on gene disruption and whole gene correction – the two technologies required to address the vast majority of the most prevalent severe monogenic diseases as well as many common diseases. We have established a leading platform forin vivo gene editing and are rapidly advancing a broad portfolio ofin vivo programs, supported by an internal lipid nanoparticle, or LNP, team to enable liver-directed and extrahepatic programs with novel lipids, formulations, and targeting moieties. Our firstin vivo programs target the liver, taking advantage of validated LNP delivery technologies, and aim to treat diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association.
Current wording
We have established a leading platform forin vivo gene editing and are rapidly advancing a pipeline of in vivo gene editing candidates that target the liver, taking advantage of validated lipid nanoparticle, or LNP, delivery technologies, and aim to treat diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association. We have established a proprietary LNP delivery platform to enable gene-editing in the liver using both CRISPR/Cas9 and our novel, proprietary SyNTase editing technologies.
Previous wording
CTX320 is being investigated in an ongoing clinical trial targeting LPA, the gene encoding apo(a), a critical component of Lp(a) in patients with elevated Lp(a), which has shown to have an independent association with major adverse cardiovascular events.
Current wording
In addition, we have a number of earlier stage investigational in vivo programs leveraging gene disruption in the liver for both common and rare diseases, including CTX340, directed towards angiotensinogen for the treatment of refractory hypertension; our next-generation LPA program, CTX321 directed towards LPA, the gene encoding apolipoprotein(a), a major component of lipoprotein(a), or Lp(a), and CTX460, directed towards SERPINA1 using our proprietary SyNTase editing platform, for the treatment of alpha-1 antitrypsin deficiency. CTX340 and CTX321 are currently in IND-enabling studies in patients with refractory hypertension and in patients with elevated Lp(a), which has been shown to have an independent association with major adverse cardiovascular events, respectively. We are progressing CTX460 through preclinical studies. We are also pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells and T cells.
Previous wording
We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing several cell therapy programs for oncology and/or autoimmune indications, including two next-generation allogeneic CAR T programs, CTX112 targeting Cluster of Differentiation 19, or CD19, and CTX131 targeting Cluster of Differentiation 70, or CD70. These product candidates incorporate edits designed to enhance CAR T potency, reduce CAR T exhaustion and evade the immune system. An additional edit in CTX131 is designed to prevent CAR T cells from killing other CAR T cells. In addition, these next-generation candidates exhibit increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch. We are producing CTX112 and CTX131 for clinical trials at our internal GMP manufacturing facility in Framingham, Massachusetts.
Current wording
We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing cell therapy programs for autoimmune indications and oncology.
Previous wording
CTX112 is being developed for both hematologic malignancies and autoimmune indications. It is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy, as well as an ongoing clinical trial in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis. Early clinical studies conducted by third parties have shown that CD19-directed autologous CAR T therapy can produce long-lasting remissions in multiple autoimmune indications by deeply depleting B cells. Our first generation allogeneic CD19-directed CAR T program has demonstrated effective depletion of B cells in oncology settings, which supports the potential for CTX112 in autoimmune diseases.
Current wording
In autoimmune disease, it is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with systemic lupus erythematosus, or SLE, systemic sclerosis, and inflammatory myositis, and a second clinical trial in immune thrombocytopenia purpura and warm autoimmune hemolytic anemia.
Previous wording
Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 for the potential treatment of solid tumors.
Current wording
Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR T programs. In addition, we are developing both transient and integrated in vivo CAR T therapies by targeting T cells with LNPs and leveraging our delivery, mRNA, and gene editing expertise.
Previous wording
We are developing gene-edited stem cell-derived therapies for the treatment of T1D. We believe our gene editing capabilities have the potential to enable a beta-cell replacement product candidate that may deliver durable benefit to patients without the need for long-term immunosuppression. We have three parallel efforts to achieve this goal: (1) CTX211, an allogeneic, gene-edited, hypoimmune, stem cell derived product candidate in a device that is implanted into patients and intended to produce insulin in a glucose-dependent manner, and which is in an ongoing clinical trial; (2) CTX213, a research stage deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from edited stem cells; and (3) we have granted a non-exclusive license to certain of our CRISPR/Cas9 intellectual property to Vertex to accelerate Vertex’s development of hypoimmune cell therapies for T1D in exchange for certain milestones and royalties.
Current wording
We continue to advance our regenerative medicine portfolio, including in diabetes. We are advancing CTX213, a deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from induced pluripotent stem cells for the treatment of T1D. To date, CTX213 has demonstrated preclinical efficacy data via direct administration. In addition, we have granted a non-exclusive license to certain of our CRISPR/Cas9 intellectual property to Vertex to accelerate Vertex’s development of hypoimmune cell therapies for T1D in exchange for certain milestones and royalties.
Previous wording
While we have made significant progress with our current portfolio of programs, we recognize that we need to continue to innovate to unlock the full power of gene editing and bring potentially transformative therapies to even more patients. We have a dedicated early-stage research team called CRISPR-X that focuses on innovating next-generation editing modalities. CRISPR-X is developing technologies to enable whole gene correction and insertion via non-viral DNA delivery and all-RNA systems, without requiring homology-directed repair or viral delivery of DNA.
Current wording
While we have made significant progress with our current portfolio of programs, we recognize that we may be able to bring transformative therapies to even more patients by continuing to innovate to unlock the full potential of gene editing. We are focused on innovating next-generation editing modalities. For example, we have developed a proprietary, next-generation, site-specific gene correction platform called SyNTase editing. In addition, we are also developing technologies to enable whole gene correction and insertion via non-viral DNA delivery and all-RNA systems.
Previous wording
Other Partnerships. We have entered into a number of additional collaborations and license agreements in other therapeutic areas, including an additional agreement with Vertex for the treatment of Duchenne muscular dystrophy and myotonic dystrophy type 1, and others to support and complement our hematopoietic stem cell, CAR T, in vivo and T1D programs and platform, including agreements with: Nkarta, Inc. to develop and commercialize products leveraging donor-derived, gene-edited CAR-NK cells; Capsida Biotherapeutics, Inc. to developin vivo gene editing therapies delivered with engineered adeno-associated virus vectors; Roswell Park Comprehensive Cancer Center to advance a gene-edited autologous CAR T program against a new target; MaxCyte, Inc. onex vivo delivery for our hemoglobinopathy and CAR T programs; CureVac AG on optimized mRNA constructs and manufacturing for certainin vivo programs; and KSQ Therapeutics, Inc. on intellectual property for our allogeneic immuno-oncology programs.
Current wording
Other Partnerships. We have entered into a number of additional collaborations, research and license agreements in other therapeutic areas, including additional agreements with Vertex including for the treatment of Duchenne muscular dystrophy and myotonic dystrophy type 1, as well as diabetes, and others, including to support and complement our hematopoietic stem cell, CAR T, in vivo and diabetes programs and platform.
Previous wording
We have a history of recurring losses and expect to continue to incur losses for the foreseeable future; however, we have been in a net income position in certain previous years due to certain payments associated with our collaboration and license agreements with Vertex. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel growth; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company.
Current wording
We have a history of recurring losses and expect to continue to incur losses for the foreseeable future; however, we have been in a net income position in certain previous years due to certain payments associated with our collaboration and license agreements with Vertex. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; pursue business development activities; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel growth; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company.
Previous wording
We have not generated any revenue to date from sales of any wholly owned product. During the years ended December 31, 2024, 2023 and 2022, we recognized $35.0 million, $370.0 million and $0.4 million, respectively, of collaboration revenue, which is primarily related to our collaboration and license agreements with Vertex.
Current wording
We have not generated any revenue to date from sales of any wholly-owned product. No collaboration revenue was recognized for the year ended December 31, 2025. During the years ended December 31, 2024 and 2023, we recognized $35.0 million and $370.0 million, respectively, of collaboration revenue, which is primarily related to our collaboration and license agreements with Vertex.
Previous wording
For the years ended December 31, 2024, 2023 and 2022, we generated $2.3 million, $1.2 million and $0.8 million, respectively, of grant revenue related to certain contracts with not-for-profit entities.
Current wording
For the years ended December 31, 2025, 2024 and 2023, we generated $3.5 million, $2.3 million and $1.2 million, respectively, of grant revenue related to certain contracts with not-for-profit entities.
Previous wording
For additional information about our revenue recognition policy, see Note 2 and Note 8 of the notes to our audited consolidated financial statements included in this Annual Report on Form 10-K.
Current wording
For additional information about our revenue recognition policy, see Note 2 and Note 8 of the notes to the consolidated financial statements included in this Annual Report on Form 10-K.
Previous wording
costs of services performed by third parties that conduct research and development and preclinical activities on our behalf;
Current wording
costs of services performed by third parties that conduct research and development and preclinical and clinical activities on our behalf;
Previous wording
fees and other payments related to acquiring and maintaining licenses under our third-party licensing agreements.
Current wording
fees and other payments related to acquiring and maintaining licenses under certain of our third-party licensing agreements.
Previous wording
Collaboration expense, net, consists of operating expenses related to the CASGEVY program under our collaboration with Vertex. Under the A&R Vertex JDCA, as amended, we have an option to defer our portion of specified costs on the CASGEVY program in excess of $110.3 million for the years ended December 31, 2022, 2023 and 2024. The $110.3 million for 2023 does not include amounts attributable to our share of certain costs arising from a license agreement between Vertex and a third party, which we agreed to pay to Vertex upon the occurrence of an event specified in Amendment No. 1 to the A&R Vertex JDCA and further described in Note 8 of the notes to our consolidated financial statements included in this Annual Report on Form 10-K. In 2024, 2023 and 2022, we exercised our option to defer our portion of specified costs incurred for the CASGEVY program in excess of the deferral limit. Any deferred amounts are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amount per year.
Current wording
Collaboration expense, net, consists of operating expenses under our collaboration with Vertex for the hemoglobinopathies program.
Previous wording
Other income, net consists primarily of interest income earned on investments.
Current wording
Other income, net consists primarily of interest income earned on investments, as well as the change in fair value of corporate equity securities.
Previous wording
Refer to Note 2 of the notes to our consolidated financial statements included in this Annual Report on Form 10-K for a discussion of recent accounting pronouncements.
Current wording
Refer to Note 2 of the notes to the consolidated financial statements included in this Annual Report on Form 10-K for a discussion of recent accounting pronouncements.
Previous wording
Collaboration revenue was $35.0 million for the year ended December 31, 2024, compared to $370.0 million for the year ended December 31, 2023. Collaboration revenue for the year ended December 31, 2024 was related to Vertex's achievement of a $10.0 million research milestone and $25.0 million research milestone under the Non-Ex License Agreement with Vertex in the fourth quarter of 2024. Collaboration revenue for the year ended December 31, 2023 was related to (i) the achievement of a $200.0 million milestone in connection with the approval of CASGEVY under our collaboration with Vertex and (ii) an upfront payment of $100.0 million and a milestone achieved of $70.0 million under the Non-Ex License Agreement with Vertex. Refer to Note 8 of the notes to our consolidated financial statements included in this Annual Report on Form 10-K for a description of revenue recognized related to Vertex.
Current wording
No collaboration revenue was recognized for the year ended December 31, 2025. Collaboration revenue was $35.0 million for the year ended December 31, 2024 and was related to Vertex’s achievement of a $10.0 million research milestone and $25.0 million research milestone under the Non-Ex License Agreement with Vertex in 2024. Refer to Note 8 of the notes to the consolidated financial statements included in this Annual Report on Form 10-K for a description of revenue recognized related to Vertex.
Previous wording
Grant revenue was $2.3 million and $1.2 million, respectively, for the years ended December 31, 2024 and 2023.
Current wording
Grant revenue was $3.5 million and $2.3 million, respectively, for the years ended December 31, 2025 and 2024.
Previous wording
Research and development expenses were $320.7 million for the year ended December 31, 2024, compared to $387.3 million for the year ended December 31, 2023. The following table summarizes our research and development expenses for the years ended December 31, 2024 and 2023, together with the changes in those items in dollars (in thousands):
Current wording
Research and development expenses were $284.8 million for the year ended December 31, 2025, compared to $310.2 million for the year ended December 31, 2024. The following table summarizes our research and development expenses for the years ended December 31, 2025 and 2024, together with the changes in those items in dollars (in thousands):
Previous wording
The decrease of approximately $66.7 million was primarily attributable to the following:
Current wording
The decrease of approximately $25.4 million was primarily attributable to the following:
Previous wording
$7.3 million of decreased employee-related expenses.
Current wording
$25.8 million of decreased employee-related expenses, including stock-based compensation expenses, primarily driven by decreased headcount;
Previous wording
$7.9 million of decreased facility expenses primarily driven by lower laboratory-related costs; and
Current wording
$5.6 million of decreased facility expenses primarily driven by lower laboratory-related costs;
Previous wording
$39.5 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs;
Current wording
$3.6 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs; offset by
Previous wording
General and administrative expenses were $73.0 million for the year ended December 31, 2024, compared to $76.2 million for the year ended December 31, 2023. The decrease of $3.2 million was primarily attributable to decreased employee-related expenses and consulting and professional services-related expenses.
Current wording
General and administrative expenses were $73.5 million for the year ended December 31, 2025, compared to $73.0 million for the year ended December 31, 2024.
Previous wording
Other income, net, was $103.9 million for the year ended December 31, 2024, compared to $71.8 million for the year ended December 31, 2023. The increase in other income, net, was primarily due to an increase in interest income earned on cash, cash equivalents and marketable securities for the year ended December 31, 2024.
Current wording
Other income, net, was $86.6 million for the year ended December 31, 2025, compared to $103.9 million for the year ended December 31, 2024. The decrease in other income, net, was primarily due to a decrease in interest income earned on cash, cash equivalents and marketable securities for the year ended December 31, 2025.
Previous wording
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of December 31, 2024, we had $1,903.8 million in cash, cash equivalents and marketable securities, of which approximately $53.8 million was held outside of the United States, and an accumulated deficit of $1,366.0 million. We anticipate that we will continue to incur losses for at least the next several years. We expect to continue to incur research and development costs and general and administrative expenses, consistent with costs associated with research and development at companies of our size and stage of development, and, as a result, we will need additional capital to fund our operations, which we may raise through public or private equity or debt financings, strategic collaborations, or other sources.
Current wording
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of December 31, 2025, we had $1,975.8 million in cash, cash equivalents and marketable securities, of which approximately $103.4 million was held outside of the United States, and an accumulated deficit of $1,947.6 million. We anticipate that we will continue to incur losses for at least the next several years. We expect to continue to incur research and development costs and general and administrative expenses, consistent with costs associated with research and development at companies of our size and stage of development, and, as a result, we will need additional capital to fund our operations, which we may raise through public or private equity or debt financings, strategic collaborations, or other sources.
Previous wording
In 2024, we issued and sold 0.4 million common shares at an average price of $55.81 per share for aggregate proceeds of $21.7 million, which were net of equity issuance costs of $0.3 million. An additional $0.2 million of stamp taxes related to this amount was paid in 2024.
Current wording
In 2024, we issued and sold 0.4 million common shares under the 2021 ATM at an average price of $55.81 per share for aggregate proceeds of $21.7 million, which were net of equity issuance costs of $0.3 million, excluding stamp taxes of $0.2 million.
Previous wording
In 2023, we issued and sold 0.5 million common shares at an average price of $72.32 per share for aggregate proceeds of $32.7 million, which were net of equity issuance costs of $0.4 million. An additional $0.3 million of stamp taxes related to this amount was paid in 2023.
Current wording
In 2023, we issued and sold 0.5 million common shares under the 2021 ATM at an average price of $72.32 per share for aggregate proceeds of $32.7 million, which were net of equity issuance costs of $0.4 million, excluding stamp taxes of $0.3 million.
Previous wording
Net cash used in operating activities was $142.8 million for the year ended December 31, 2024, compared to net cash used in operating activities of $260.4 million for the year ended December 31, 2023. The decrease in cash used in operating activities was primarily driven by cash received in 2024 from Vertex in connection with the $200.0 million milestone achieved upon regulatory approval of CASGEVY in 2023, offset by increased net losses.
Current wording
Net cash used in operating activities was $345.0 million for the year ended December 31, 2025, compared to net cash used in operating activities of $142.8 million for the year ended December 31, 2024. The $202.2 million increase in cash used in operating activities was primarily driven by the timing of receipt of milestone payments from Vertex which were recorded in accounts receivable as of the respective year-end periods and paid in the subsequent year ($200.0 million paid in the first quarter of 2024 compared to $25.0 million paid in the first quarter of 2025).
Previous wording
Net cash provided by financing activities for the year ended December 31, 2024 was $332.0 million and primarily consisted of net proceeds of approximately $279.0 million from the issuance of common shares in connection with our registered direct offering, net proceeds of approximately $31.2 million from stock option exercises, and net proceeds of approximately $21.7 million from the issuance of common shares in connection with our 2021 ATM. Net cash provided by financing activities for the year ended December 31, 2023 was $62.7 million and consisted of net proceeds of $32.7 million from the issuance of common shares and net proceeds of approximately $29.9 million from stock option exercises.
Current wording
Net cash provided by financing activities for the year ended December 31, 2025 was $426.0 million, compared to net cash provided by financing of $332.0 million for the year ended December 31, 2024. Net cash provided by financing activities for the year ended December 31, 2025 consisted primarily of net proceeds of approximately $397.3 million from the sale of common shares issued in connection with our 2021 ATM and 2025 ATM, in the aggregate, as well as net proceeds of approximately $27.7 million from stock option exercise proceeds. Net cash provided by financing activities for the year ended December 31, 2024 primarily consisted of net proceeds of approximately $279.0 million from the issuance of common shares in connection with our registered direct offering, net proceeds of approximately $31.2 million from stock option exercises, and net proceeds of approximately $21.7 million from the issuance of common shares in connection with our 2021 ATM.
Previous wording
Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution filing, defense and intellectual property maintenance costs, and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development at companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.
Current wording
Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution filing, defense and intellectual property maintenance costs, business development activities and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development at companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.
Previous wording
Although we and our partner, Vertex, have received marketing approvals for CASGEVY in certain jurisdictions, we cannot guarantee we and Vertex will receive additional marketing approvals for CASGEVY or we will receive marketing approvals for our other product candidates in the future. Most of our programs are still in early stages of development and the outcome of our efforts is uncertain, and we cannot estimate the actual amounts necessary to successfully complete the development, manufacture and commercialization of any current or future product candidates, if approved, or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to finance our cash needs through a combination of equity financings, debt financings and payments received in connection with our collaboration and license agreements. We intend to consider opportunities to raise additional funds through the sale of equity or debt securities when market conditions are favorable to us to do so. However, the trading prices for our common shares and other biopharmaceutical companies have been highly volatile. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event could materially and adversely affect our business and the value of our common shares. To the extent that we raise additional capital through the future sale of equity or debt securities, the ownership interests of our shareholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing shareholders. If we raise additional funds through license or collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Current wording
Until such time as we can generate substantial product revenues, if ever, we expect to finance our cash needs through a combination of equity financings, debt financings and payments received in connection with our collaboration and license agreements. We intend to consider opportunities to raise additional funds through the sale of equity or debt securities when market conditions are favorable to us to do so. However, the trading prices for our common shares and other biopharmaceutical companies have been highly volatile. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event, including resulting from the uncertain geopolitical environment, global trade policy and global economic conditions, could materially and adversely affect our business and the value of our common shares. To the extent that we raise additional capital through the future sale of equity or debt securities, the ownership interests of our shareholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing shareholders. If we raise additional funds through license or collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Previous wording
Our operating lease obligations primarily consist of lease payments on our office and laboratory facility in Boston, Massachusetts, as well as lease payments on our cell manufacturing facility in Framingham, Massachusetts, which are described in further detail in Note 7 of our consolidated financial statements included in this Annual Report on Form 10-K. Future contractual payments on operating lease and sublease obligations due within one year of December 31, 2024 are $29.4 million, and future contractual payments on operating lease and sublease obligations due greater than one year from December 31, 2024 are $265.2 million.
Current wording
Our operating lease obligations primarily consist of lease payments on our office and laboratory facility in Boston, Massachusetts, as well as lease payments on our cell manufacturing facility in Framingham, Massachusetts, which are described in further detail in Note 7 of our consolidated financial statements included in this Annual Report on Form 10-K. Future contractual payments on operating lease and sublease obligations due within one year of December 31, 2025 are $29.7 million, and future contractual payments on operating lease and sublease obligations due greater than one year from December 31, 2025 are $235.9 million.
Previous wording
Under the A&R Vertex JDCA, as amended, we are allowed to defer a portion of our share of costs under the arrangement if spending on the CASGEVY program exceeds specified amounts. Any deferred amounts are only payable to Vertex as an offset against future profitability of the CAGEVY program and the amounts payable are capped at a specified maximum amount per year. Deferred costs associated with the CASGEVY program have not been recognized as of December 31, 2024 because a reasonable estimate of future payments against future profitability cannot be made.
Current wording
Under the A&R Vertex JDCA, as amended, for 2022, 2023 and 2024, the Company had an option to defer a portion of its share of costs if spending on the CASGEVY program exceeded specified amounts, which the Company exercised in each such year, resulting in deferred costs of $221.8 million, in the aggregate. Any deferred amounts are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amount per year. Deferred costs associated with the CASGEVY program have not been recognized as of December 31, 2025 because a reasonable estimate of future payments against future profitability cannot be made.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex.
Current report — wording carried from elsewhere
Our lead next-generation product candidate, zugocabtagene geleucel (zugo-cel; formerly CTX112), incorporates edits designed to enhance CAR T potency, reduce CAR T exhaustion and evade the immune system. As a result of the next-generation edits, zugo-cel exhibits increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch. We are producing zugo-cel for clinical trials at our internal GMP manufacturing facility in Framingham, Massachusetts. Zugo-cel continues to advance in both autoimmune disease and hematologic malignancies.
Current report — wording carried from elsewhere
Although we and our partner, Vertex, have received marketing approvals for CASGEVY in certain jurisdictions, we cannot guarantee we and Vertex will receive additional marketing approvals for CASGEVY or we will receive marketing approvals for our other product candidates in the future. Most of our programs are still in early stages of research and development and the outcome of our efforts is uncertain, and we cannot estimate the actual amounts necessary to successfully complete the development, manufacture and commercialization of any current or future product candidates, if approved, or whether, or when, we may achieve profitability.
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
In 2023, CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in the world. To date, CASGEVY has been approved in the United States, European Union, Great Britain, Canada, Switzerland, Kingdom of Saudi Arabia, Kingdom of