CRISPR Therapeutics AG
For the period ended Mar 31, 2026. Filed May 4, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed May 4, 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-Q
Period ended 2025-09-30; filed 2025-11-10.
0001193125-25-273337
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-05-04.
0001193125-26-204188
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 141 previous paragraphs and 124 current paragraphs.
57 unchanged; 31 reworded; 33 added; 51 removed; 2 previous and 3 current paragraphs rearranged.
Tables are not compared: 5 previous and 3 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 (33)
Current report
In Vivo Liver Editing
Current report
Ourin vivo portfolio includes cardiovascular programs, such as CTX310, directed towards angiopoietin-related protein 3, 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
In addition, we have a number of earlier stage investigationalin 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), in development for patients with elevated Lp(a), and CTX460, directed towards SERPINA1 using our proprietary SyNTase editing platform, for the treatment of alpha-1 antitrypsin deficiency. CTX340, CTX321 and CTX460 are currently in IND/CTA-enabling studies. We are also pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells and T cells.
Current report
siRNA-based Programs
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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 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 ongoing Phase 2 clinical trials, including in patients undergoing total knee arthroplasty.
Current report
Zugocabtagene geleucel
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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
In autoimmune disease, it is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the product candidate in adult patients across multiple indications, including an initial clinical trial in systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis; a second clinical trial in immune thrombocytopenia purpura and warm autoimmune hemolytic anemia; and a third clinical trial in autoimmune neurologic diseases, including progressive multiple sclerosis, neuromyelitis optica spectrum disorder, myelin oligodendrocyte glycoprotein antibody-associated Disease (MOGAD), N-methyl-D-aspartate receptor (NMDAR) and leucine-rich glioma-inactivated Protein 1 (LGI1) autoimmune encephalitis, and stiff person syndrome.
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Regenerative Medicine
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Next-generation Editing Modalities
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Research and Development Expenses
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General and Administrative Expenses
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Collaboration Expense, Net
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Research and Development Expenses
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The decrease of approximately $3.9 million was primarily attributable to a decrease in employee-related costs, including stock-based compensation expenses, offset by an increase in external research and development expenses.
Current report
General and Administrative Expenses
Current report
Collaboration Expense, Net
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Debt
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Notes, net
Current report
In March 2026, we issued $600.0 million aggregate principal amount of our Convertible Senior Notes due 2031, or the Notes, pursuant to an indenture dated March 16, 2026, or the Indenture, between us and U.S. Bank Trust Company, National Association, as trustee, in a private offering to qualified institutional buyers, or the 2026 Note Offering. The Notes issued in the 2026 Note Offering include the exercise in full of the initial purchasers’ option to purchase $50.0 million of additional Notes. The investors in the Notes agreed to an effective coupon of 1.125%. Because of anticipated 35% withholding on interest payments on the Notes under Swiss tax law, we agreed to increase the coupon by 0.6058% to 1.7308% to effectively eliminate the impact of such anticipated withholding on any holders who are not eligible to receive a refund.
Current report
The Notes are senior, unsecured obligations of ours and will mature on March 1, 2031, or the maturity date, unless earlier converted, redeemed or repurchased. The Notes will accrue interest payable semiannually in arrears on March 1 and September 1 of each year, beginning on September 1, 2026. Holders may convert all or any portion of their Notes at their option prior to the maturity date, other than during a “conversion freeze period” (as defined in the Indenture). Upon conversion, we will deliver our common shares, nominal value CHF 0.03 per share, or the common shares, at an initial conversion rate of 13.0617 common shares per $1,000 principal amount of Notes (equivalent to an initial conversion price of approximately $76.56 per common share), subject to adjustment in some events in accordance with the terms of the Indenture but will not be adjusted for any accrued and unpaid interest.
Current report
In addition, if certain corporate events occur or are anticipated to occur prior to the maturity date or if we deliver a notice of optional redemption, we will, in certain circumstances, increase the conversion rate for a holder who elects to convert our Notes in connection with such a corporate event or called (or deemed called) for redemption in connection with such notice of optional redemption. Initially, a maximum of 11,363,580 common shares may be delivered upon conversion of the Notes, based on the initial maximum conversion rate of 18.9393 common shares per $1,000 principal amount of Notes, which is subject to customary conversion rate adjustment provisions.
Current report
We may not redeem the Notes prior to March 6, 2029. On or after such date, we may redeem for cash all or any portion of the Notes (subject to the partial redemption limitation described in the Indenture), at our option, if the last reported sale price of our common shares has been at least 130% of the conversion price for the Notes then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending on, and including, the trading day immediately preceding the date on which we provide notice of optional redemption, at a redemption price equal to 100% of the principal amount of the Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the optional redemption date. No sinking fund is provided for the Notes.
Current report
If we undergo a fundamental change (as defined in the Indenture), then, subject to certain conditions and except as described in the Indenture, holders may require us to repurchase for cash all or any portion of their Notes at a fundamental change repurchase price equal to 100% of the principal amount of the Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date.
Current report
The Indenture includes customary covenants and sets forth certain events of default after which the Notes may be declared immediately due and payable and sets forth certain types of bankruptcy or insolvency events of default involving us after which the Notes become automatically due and payable.
Current report
We received net proceeds from the 2026 Note Offering of approximately $585.4 million, after deducting the initial purchasers’ discounts and commissions and the estimated offering expenses payable by us. Refer to Note 8 to our condensed consolidated financial statements included in this Form 10-Q for other details related to the Notes.
Current report
At-the-Market Offerings (ATM)
Current report
As of December 31, 2025, we had 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 remained available under the 2021 ATM.
Current report
No common shares were issued and sold under the 2025 ATM for the three months ended March 31, 2026. As of March 31, 2026, 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
Convertible debt
Current report
We account for our convertible debt instruments as a single unit of accounting, a liability, as we concluded that the conversion features do not require bifurcation as a derivative under ASC Topic 815-15, Derivatives and Hedging, as we did not issue our convertible debt instruments at a substantial premium. We record debt issuance costs as contra-liabilities in our condensed consolidated balance sheets at issuance and amortize debt issuance costs over the contractual term of the convertible debt instrument using the effective interest rate. The balance of our Notes presented in our condensed consolidated balance sheets represents the principal balance of the convertible debt instrument less unamortized debt issuance costs. Please refer to Note 8 of the notes to the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
Removed paragraphs (51)
Previous report
CD19 candidates
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CD70 candidates
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CTX131, our next-generation allogeneic CAR T program targeting Cluster of Differentiation 70, was previously in development for both solid tumors and hematologic malignancies. While the Phase 1 data are encouraging, we have strategically redirected resources away from this program to advance other programs with the greatest potential for long-term value creation.
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In Vivo
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Cardiovascular and metabolic diseases
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Our first twoin 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.
Previous report
CTX310 is being investigated in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein and triglyceride levels, in four patient groups: homozygous familial hypercholesterolemia, severe hypertriglyceridemia, heterozygous familial hypercholesterolemia, or mixed dyslipidemias. Both low-density lipoprotein and triglyceride are validated surrogate endpoints accepted by regulatory agencies.
Previous report
CTX320 is being investigated in an ongoing Phase 1 clinical trial targeting LPA, the gene encoding apo(a), a critical component of Lp(a), in patients with elevated Lp(a), a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events.
Previous report
We continue to advance our earlier stagein vivo programs through preclinical studies, including: CTX340, targeting angiotensinogen for the treatment of refractory hypertension, as well as CTX450, targeting 5’-aminolevulinate synthase 1 for the treatment of acute hepatic porphyria; and CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency. CTX340 and CTX450 are currently progressing through IND/CTA-enabling studies. CTX460 is the first investigational candidate we are advancing using our novel SyNTase editing platform. SyNTase enables precise, in vivo gene correction, and represents an important expansion of our toolkit. We are also pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells.
Previous report
SRSD107
Previous report
In May 2025, we entered into a strategic collaboration with Sirius Therapeutics and certain of its affiliates, or Sirius, pursuant to which, among other things, we and Sirius will jointly develop and commercialize small interfering RNA, or siRNA therapies, beginning with SRSD107. SRSD107 is a next generation, long-acting siRNA designed to selectively inhibit human coagulation factor XI, or FXI, a key driver of pathological thrombosis with minimal impact on normal hemostasis. By targeting FXI, SRSD107 aims to reduce thrombotic events while minimizing the risk of bleeding, representing a differentiated approach compared to Factor Xa inhibitors. In addition, SRSD107 may offer the potential for reversibility not observed with other anti-Factor XI modalities. The addressable population for SRSD107 includes patients with atrial fibrillation, venous thromboembolism, or VTE, cancer-associated thrombosis, chronic Coronary Artery Disease, chronic Peripheral Vascular Disease, end-stage renal disease requiring hemodialysis, and patients undergoing major orthopedic surgery, where bleeding risk limits existing therapies.
Previous report
Two Phase 1 clinical trials of SRSD107 have been completed by Sirius, and single doses of SRSD107 have been well tolerated. SRSD107 demonstrated robust pharmacodynamic effects, including reductions of over 93% in FXI levels and FXI activity, along with more than a twofold increase in activated partial thromboplastin time relative to baseline. These effects were sustained, with responses maintained for up to six months post-dosing. SRSD107 is being investigated in an ongoing Phase 2 clinical trial evaluating the safety and efficacy of the candidate in preventing VTE following total knee arthroplasty.
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Type 1 Diabetes
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CRISPR-X
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Hemoglobinopathies
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Diabetes
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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 ongoing 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 T1D, diabetes type 2 or insulin dependent/requiring diabetes throughout the world. To date, we have recognized revenue of $205.0 million in upfront and milestone payments and remain eligible to receive additional research and development milestones and royalties on future products under the license.
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Sirius Therapeutics
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With respect to Sirius Collaboration Products, we and Sirius will share equally all development and commercialization costs. For the first collaboration product candidate 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. The parties will share equally the net profits and net losses incurred under the Sirius Agreement with respect to all Sirius Collaboration Products,
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except in the event that a party opts out of the joint development and commercialization. We will also pay Sirius future one-time development and regulatory milestones with respect to Sirius Collaboration Products that achieve the applicable milestone events.
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Other Partnerships
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Acquired In-Process Research and Development Expenses
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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 condensed consolidated statements of cash flows.
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There was no collaboration revenue for the three months ended September 30, 2025 and 2024. Please refer to Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
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The decrease of approximately $23.3 million was primarily attributable to the following:
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$10.5 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs;
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$8.9 million of decreased employee-related expenses, including stock-based compensation expenses, primarily driven by decreased headcount; and
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$4.0 million of decreased facility expenses primarily driven by lower laboratory-related costs.
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Collaboration Revenue
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There was no collaboration revenue for the nine months ended September 30, 2025 and 2024. Please refer to Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
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The decrease of approximately $37.2 million was primarily attributable to the following:
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$18.9 million of decreased employee-related expenses, including stock-based compensation expenses, primarily driven by decreased headcount;
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$9.5 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs;
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$4.6 million of decreased facility-related expenses, primarily driven by lower lab consumable costs; and
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$3.5 million of decreased sublicense and license fees, primarily attributable to a decrease in research and development licenses and milestones payable under certain strategic partnerships and other research and development agreements.
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Acquired In-Process Research and Development Expenses
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Acquired in-process research and development expenses were $96.3 million for the nine months ended September 30, 2025. There was no acquired in-process research and development expenses for the nine months ended September 30, 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 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
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General and administrative expenses were $55.1 million for the nine months ended September 30, 2025, compared to general and administrative expenses of $54.9 million for the nine months ended September 30, 2024. The increase of approximately $0.3 million was primarily associated with an increase in stock-based compensation expense, offset by a decrease in intellectual property costs.
Previous report
Collaboration expense, net, was $159.8 million for the nine months ended September 30, 2025, compared to $110.3 million for the nine months ended September 30, 2024. In the third quarter of 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 $49.5 million in collaboration expense, net, was primarily attributable to the timing of when we reached 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 offset by an increase in our share of CASGEVY revenue.
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Other Income, Net
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Reworded paragraphs (31)
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, or T1D. 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
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, Kingdom of Saudi Arabia, Kingdom of Bahrain, Qatar, Canada, Switzerland, 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 in (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
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 pipeline ofin vivo gene editing candidates targeting major unmet needs in cardiovascular and metabolic diseases using our proprietary lipid nanoparticle, or LNP, delivery platform. 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
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 autoimmune and/or oncology indications. These product candidates incorporate edits designed to enhance CAR T potency, reduce CAR T exhaustion and evade the immune system. In addition, these next-generation candidates exhibit increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch. Our autoimmune disease and immuno-oncology programs are supported by our wholly-owned, GMP manufacturing facility located 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 several cell therapy programs in both autoimmune and immuno-oncology indications.
Previous wording
CTX112, our next-generation allogeneic CAR T program targeting Cluster of Differentiation 19, or CD19, 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 systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis, as well as an ongoing clinical trial in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy. CTX112 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 wording
In immuno-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 regenerative medicine advanced therapy designation by the U.S. Food and Drug Administration for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.
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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.
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 invivo 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 type 1 diabetes, or 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
Given the numerous potential therapeutic applications for CRISPR/Cas9, we have partnered strategically to broaden the indications we can pursue and accelerate development of programs by accessing specific technologies and/or disease-area expertise. We maintain broad partnerships to develop gene editing-based therapeutics in specific disease areas.
Current wording
Given the numerous potential therapeutic applications for CRISPR/Cas9, we have partnered strategically to broaden the indications we can pursue and accelerate development of programs by accessing specific technologies and/or disease-area expertise. We maintain broad partnerships to develop gene-based therapeutics in specific disease areas.
Previous wording
In 2015, we partnered with Vertex and entered into a strategic collaboration, option and license agreement, which focused on the discovery and development of gene-based treatments for hemoglobinopathies and cystic fibrosis using CRISPR/Cas9 gene-editing technology. In 2017, Vertex exercised its option to co-develop and co-commercialize the hemoglobinopathies program and we entered into a joint development and commercialization agreement with Vertex, which we amended and restated in 2021, pursuant to which, among other things, we are co-developing and co-commercializing CASGEVY for TDT and SCD.
Current wording
Hemoglobinopathies. In 2015, we partnered with Vertex and entered into a strategic collaboration, option and license agreement, which focused on the discovery and development of gene-based treatments for hemoglobinopathies and cystic fibrosis using CRISPR/Cas9 gene-editing technology. In 2017, Vertex exercised its option to co-develop and co-commercialize the hemoglobinopathies program and we entered into a joint development and commercialization agreement with Vertex, which we amended and restated in 2021, pursuant to which, among other things, we are co-developing and co-commercializing CASGEVY for TDT and SCD.
Previous wording
In May 2025, we entered into a collaboration, option and license agreement, or the Sirius Agreement, with Sirius, pursuant to which, among other things, we and Sirius will collaborate on the research, development, manufacture, commercialization and use of the Sirius Collaboration Products; 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 Sirius Licensed Products, in exchange for the potential to receive certain option fees, milestone payments and royalties. In connection with entering into the Sirius Agreement, we made an upfront cash payment to Sirius of $25.0 million and also entered into a share issuance agreement with Sirius, pursuant to which we agreed to issue to Sirius the Sirius Shares.
Current wording
siRNA. In May 2025, we partnered with Sirius and entered into the Sirius Agreement pursuant to which, among other things, (1) 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, development, manufacture and commercialization of siRNA Licensed Products. For the first Sirius Collaboration Product successfully developed, we will be the lead party responsible for Phase 3 global development and commercialization efforts in the United States and Sirius will be the lead party responsible for Phase 3 development (subject to the global development plan) and commercialization efforts in Greater China.
Previous wording
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.
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
Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business development activities, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans and payments related to certain of our license and collaboration agreements with strategic partners.
Current wording
Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business development activities, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans, issuance of convertible notes and payments related to certain of our license and collaboration agreements with strategic partners.
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; 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.
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 and other technologies; 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 and do not expect to do so in the near future. Revenue recognized for the three and nine months ended September 30, 2025 and 2024 was not material. For additional information about our revenue recognition policy, see Note 2, “Summary of Significant Accounting Policies,” in our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on February 11, 2025, as well as Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
Current wording
We have not generated any revenue to date from sales of any wholly-owned product and do not expect to do so in the near future. Revenue recognized for the three months ended March 31, 2026 and 2025 was not material. For additional information about our revenue recognition policy, see Note 2, “Summary of Significant Accounting Policies,” in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 12, 2026, as well as Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
Previous wording
successful completion of preclinical studies and IND-enabling studies;
Current wording
successful completion of preclinical studies and IND/CTA-enabling studies;
Previous wording
Other income (expense), net consists primarily of interest income earned on investments, as well as the change in fair value of corporate equity securities.
Current wording
Other income (expense), net consists primarily of interest expense on debt, interest income earned on investments, as well as the change in fair value of corporate equity securities.
Previous wording
Research and development expenses were $58.9 million for the three months ended September 30, 2025, compared to $82.2 million for the three months ended September 30, 2024. The following table summarizes our research and development expenses for the three months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands):
Current wording
Research and development expenses were $68.6 million for the three months ended March 31, 2026, compared to $72.5 million for the three months ended March 31, 2025. The following table summarizes our research and development expenses for the three months ended March 31, 2026 and 2025, together with the changes in those items in dollars (in thousands):
Previous wording
General and administrative expenses were $16.9 million for the three months ended September 30, 2025, compared to general and administrative expenses of $17.4 million for the three months ended September 30, 2024. The decrease of approximately $0.5 million was primarily associated with a decrease in employee-related costs.
Current wording
General and administrative expenses were $17.2 million for the three months ended March 31, 2026, compared to $19.3 million for the three months ended March 31, 2025. The decrease of approximately $2.1 million was primarily associated with a decrease in employee-related costs.
Previous wording
Collaboration expense, net, was $57.1 million for the three months ended September 30, 2025, compared to $11.2 million for the three months ended September 30, 2024. In the third quarter of 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 $46.0 million in collaboration expense, net, was primarily attributable to the timing of when we reached 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 offset by an increase in our share of CASGEVY revenue.
Current wording
Collaboration expense, net, was $45.9 million for the three months ended March 31, 2026, compared to $57.5 million for the three months ended March 31, 2025. The decrease was primarily attributable to an increase in our share of CASGEVY revenue.
Previous wording
Other income was $26.2 million for the three months ended September 30, 2025, compared to $25.1 million of income for the three months ended September 30, 2024. The increase of approximately $1.2 million was primarily due to the change in fair value of corporate equity securities during the three months ended September 30, 2025, offset by a decrease in interest income earned on cash, cash equivalents and marketable securities for the three months ended September 30, 2025.
Current wording
Other income was $8.2 million for the three months ended March 31, 2026, compared to $13.5 million of income for the three months ended March 31, 2025. The decrease of approximately $5.3 million in other income was primarily due to the change in fair value of corporate equity securities during the three months ended March 31, 2026.
Previous wording
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of September 30, 2025, we had $1,944.1 million in cash, cash equivalents and marketable securities, of which approximately $184.7 million was held outside of the United States, and an accumulated deficit of $1,816.9 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 March 31, 2026, we had $2,441.8 million in cash, cash equivalents and marketable securities, of which approximately $305.2 million was held outside of the United States, and an accumulated deficit of $2,070.5 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 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 our common shares remaining under our original prospectus supplement, the 2021 ATM. For the nine months ended September 30, 2025, we issued and sold an aggregate of 5.1 million common shares under the 2021 ATM at an average price of $57.39 per share for aggregate proceeds of $286.8 million with equity issuance costs of $3.7 million, excluding stamp taxes of $2.9 million. As of September 30, 2025, we have issued and sold an aggregate of 7.0 million common shares under the 2021 ATM at an average price of $75.40 per share for aggregate proceeds of $520.0 million, which were net of equity issuance costs of $6.8 million, excluding stamp taxes of $5.2 million. As of September 30, 2025, common shares having aggregate gross proceeds up to $73.2 million remain available under the 2021 ATM.
Current wording
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 our common shares remaining under our original prospectus supplement, the 2021 ATM.
Previous wording
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, or the 2025 ATM. Through the date of issuance of this Quarterly Report on Form 10-Q, we have issued and sold an aggregate of 0.2 million common shares under the 2025 ATM at an average price of $73.53 per share for aggregate proceeds of $11.7 million, which were net of equity issuance costs of $0.1 million, excluding stamp taxes of $0.1 million. Common shares having aggregate gross proceeds up to $588.3 million remain available under the 2025 ATM.
Current wording
For the three months ended March 31, 2025, we issued and sold an aggregate of 0.2 million common shares under the 2021 ATM at an average price of $54.33 per share for aggregate proceeds of $8.7 million, which were net of equity issuance costs of $0.1 million, excluding stamp taxes of $0.1 million.
Previous wording
As described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q, 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 wording
As described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q, we and Sirius entered into a share issuance agreement in 2025 and we registered and issued 1,842,105 common shares to Sirius at an issue price of $38.00 per share as partial consideration for entering into the Sirius Agreement.
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, 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 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 employee-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
Net cash used in operating activities was $252.5 million for the nine months ended September 30, 2025, compared to $92.7 million for the nine months ended September 30, 2024. The $159.7 million increase in net 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).
Current wording
Net cash used in operating activities was $108.8 million for the three months ended March 31, 2026, compared to $53.9 million for the three months ended March 31, 2025. The $54.9 million increase in net cash used in operating activities was primarily driven by a $70.3 million overall decrease in net changes of operating assets and liabilities, primarily driven by the timing of receipt of a $25.0 million milestone payment from Vertex which was paid in the first quarter of 2025, offset by a decrease in net loss of approximately $13.1 million.
Previous wording
Net cash used in investing activities for the nine months ended September 30, 2025 was $69.0 million, compared to net cash used in investing activities of $386.3 million for the nine months ended September 30, 2024. The decrease in net cash used in investing activities was primarily driven by a net decrease in purchases of our marketable securities.
Current wording
Net cash used in investing activities for the three months ended March 31, 2026 was $410.7 million, compared to net cash used in investing activities of $19.8 million for the three months ended March 31, 2025. The increase in net cash used in investing activities was primarily driven by a net increase in purchases of our marketable securities.
Previous wording
Net cash provided by financing activities for the nine months ended September 30, 2025 was $309.6 million, compared with $315.1 million for the nine months ended September 30, 2024. Net cash provided by financing activities for the nine months ended September 30, 2025 consisted primarily of $286.6 million in net proceeds from the sale of common shares issued in connection with our 2021 ATM, as well as option exercise proceeds of $23.0 million, net of issuance costs. Net cash provided by financing activities for the nine months ended September 30, 2024 consisted of net proceeds from the sale of approximately $280.0 million of our common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50, as well as option exercise proceeds of $29.4 million, net of issuance costs.
Current wording
Net cash provided by financing activities for the three months ended March 31, 2026 was $591.9 million, compared with $10.6 million for the three months ended March 31, 2025. Net cash provided by financing activities for the three months ended March 31, 2026 consisted primarily of $585.4 million in net proceeds from the issuance of the Notes in March 2026.
Previous wording
There have been no material changes in our critical accounting policies and estimates in the preparation of our condensed consolidated financial statements during the three and nine months ended September 30, 2025 compared to those discussed in our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on February 11, 2025 and discussed in Note 1 in the condensed consolidated financial statements in the Company’s Quarterly Report on Form 10-Q filed with the SEC on August 4, 2025 with respect to acquired in-process research and development expenses and changes to the Company’s policy around collaboration arrangements.
Current wording
There have been no material changes in our critical accounting policies and estimates in the preparation of our condensed consolidated financial statements during the three months ended March 31, 2026 compared to those discussed in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 12, 2026, with the exception of the new policy with respect to convertible debt described below.
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
Collaboration revenue was not material for the three months ended March 31, 2026 and 2025.
Current report — wording carried from elsewhere
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, or the 2025 ATM.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
Comparison of nine months ended September 30, 2025 and 2024 (in thousands):
Previous report — wording carried elsewhere
Research and development expenses were $201.3 million for the nine months ended September 30, 2025, compared to $238.5 million for the nine months ended September 30, 2024. The following table summarizes our research and development expenses for the nine months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands):