CRSP

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with (i) our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and (ii) our audited consolidated financial statements and related notes and management’s discussion and analysis of financial condition and results of operations included in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the Securities and Exchange Commission, or the SEC, on February 12, 2026. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and impact and potential impacts on our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including, without limitation, those factors set forth in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2025 and the “Risk Factors” section of subsequent Quarterly Reports on Form 10-Q, our actual results or timing of certain events could differ materially from the results or timing described in, or implied by, these forward-looking statements. Overview 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 an ex vivo approach, in which we edit cells outside of the human body before administering them to the patient, or an in vivo editing approach, where we deliver the CRISPR-based therapeutic directly to target cells within the human body. 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. 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. Hemoglobinopathies CASGEVY CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin in red blood cells, which can compensate for the defective adult hemoglobin in patients with sickle cell disease, or SCD, or transfusion-dependent beta thalassemia, or TDT. CASGEVY is the first therapy to emerge from our strategic partnership with Vertex and is being advanced under a joint development and commercialization agreement between us and Vertex and certain of its affiliates. 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, including the Kingdom of Saudi Arabia, for the treatment of eligible patients 12 years and older with SCD or TDT. Additionally, in July 2026, the FDA approved expanded use of CASGEVY in the United States for the treatment of eligible patients ages 2 years and older with either SCD or TDT. CASGEVY is the first approved genetic therapy indicated for children as young as 2 years for both SCD and 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. Additional candidates 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. 20 Table of Contents In Vivo Liver Editing We have established a leading platform for in 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. Our in 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. In addition, we have initiated Phase 1 clinical trials of CTX340, directed towards angiotensinogen, in patients for the treatment of refractory hypertension and CTX460, directed towards SERPINA1 using our proprietary SyNTase editing platform, for the treatment of alpha-1 antitrypsin deficiency. Additional candidates 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 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). CTX321 is 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. siRNA-based Programs 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. 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. CTX611 is in ongoing Phase 2 clinical trials, including in patients undergoing total knee arthroplasty. CAR-T 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. Zugocabtagene geleucel 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. 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, or SLE, 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. 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. Additional candidates 21 Table of Contents 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. Regenerative Medicine 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. Next-generation Editing Modalities 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. Partnerships 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. 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. 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. 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. Financial Overview 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. 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 22 Table of Contents personnel; incur facilities costs; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company. Revenue Recognition 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 six months ended June 30, 2026 was $10.2 million and $11.6 million, respectively, primarily related to an upfront payment on an immaterial license and collaboration agreement entered into during the second quarter of 2026. Revenue for the three and six months ended June 30, 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. Research and Development Expenses Research and development expenses consist primarily of costs incurred for our research activities, including our product discovery efforts and the development of our product candidates, which include: •employee-related expenses, including salaries, benefits and equity-based compensation expense; •costs of services performed by third parties that conduct research and development and preclinical and clinical activities on our behalf; •costs of purchasing lab supplies and non-capital equipment used in our preclinical activities and in manufacturing preclinical study materials, as well as supplies and materials used to manufacture clinical drug material; •consultant fees; •facility costs, including rent, depreciation and maintenance expenses; and •fees and other payments related to acquiring and maintaining licenses under certain of our third-party licensing agreements. Our external research and development expenses support our various preclinical and clinical programs, and, as such, we do not break down external research and development expenses further. Our internal research and development expenses consist of payroll and benefits expenses, facilities expense, and other indirect research and development expenses incurred in support of overall research and development activities and, as such, are not allocated to a specific development stage or therapeutic area. Research and development costs are expensed as incurred. Nonrefundable advance payments for research and development goods or services to be received in the future are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed. At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of any product candidates we may identify and develop. This is due to the numerous risks and uncertainties associated with developing such product candidates, including the uncertainty of: •successful completion of preclinical studies and IND/CTA-enabling studies; •successful enrollment in, and completion of, clinical trials; •receipt of marketing approvals from applicable regulatory authorities; •establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; •obtaining and maintaining patent and trade secret protection and non-patent exclusivity; •launching commercial sales of the product, if and when approved, whether alone or in collaboration with others; •acceptance of the product, if and when approved, by patients, the medical community and third-party payors; •effectively competing with other therapies and treatment options; •a continued acceptable safety profile following approval; •enforcing and defending intellectual property and proprietary rights and claims; and •achieving desirable medicinal properties for the intended indications. A change in the outcome of any of these variables with respect to the development of any product candidates or the subsequent commercialization of any product candidates we may successfully develop could significantly change the costs, timing and viability associated with the development of that product candidate. Research and development activities are central to our business model. We expect to continue to incur research and development costs consistent with research and development at companies of our size and stage of development, which may increase in the foreseeable future as our current development programs progress, new programs are added and we continue to prepare regulatory filings. These increases will likely include the costs related to the implementation and expansion of clinical trial sites and related patient enrollment, monitoring, program management and manufacturing expenses for current and future clinical trials. 23 Table of Contents Acquired In-Process Research and Development Expenses 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. General and Administrative Expenses General and administrative expenses consist primarily of employee-related expenses, including salaries, benefits and equity-based compensation, for personnel in executive, finance, accounting, business development, human resources and other general and administrative functions. Other significant costs include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting and consulting services. We expect to continue to incur general and administrative expenses consistent with general and administrative functions at 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. In addition, we anticipate ongoing expenses related to the reimbursements of third-party patent related expenses in connection with certain of our in-licensed intellectual property. Collaboration Expense, Net Collaboration expense, net, consists of operating expense under our collaboration with Vertex for the hemoglobinopathies program. Other Income (Expense), Net 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. Results of Operations Comparison of three months ended June 30, 2026 and 2025 (in thousands): Three Months Ended June 30, Period to Period 2026 2025 Change Revenue: Collaboration revenue $ 10,000 $ — $ 10,000 Grant revenue 181 892 (711 ) Total revenue 10,181 892 9,289 Operating expenses: Research and development 67,152 69,894 (2,742 ) Acquired in-process research and development 2,473 96,253 (93,780 ) General and administrative 17,553 18,916 (1,363 ) Collaboration expense, net 40,272 45,153 (4,881 ) Total operating expenses 127,450 230,216 (102,766 ) Loss from operations (117,269 ) (229,324 ) 112,055 Other income, net 27,075 22,067 5,008 Loss before income taxes (90,194 ) (207,257 ) 117,063 Provision for income taxes (960 ) (1,292 ) 332 Net loss $ (91,154 ) $ (208,549 ) $ 117,395 Collaboration Revenue Collaboration revenue was $10.0 million for the three months ended June 30, 2026 related to an upfront payment on an immaterial license and collaboration agreement entered into during the second quarter of 2026. There was no collaboration revenue for the three months ended June 30, 2025. 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 on significant collaborative arrangements. Research and Development Expenses Research and development expenses were $67.2 million for the three months ended June 30, 2026, compared to $69.9 million for the three months ended June 30, 2025. The following table summarizes our research and development expenses for the three months ended June 30, 2026 and 2025, together with the changes in those items in dollars (in thousands): 24 Table of Contents Three Months Ended June 30, Period to Period 2026 2025 Change External research and development expenses $ 20,794 $ 20,831 $ (37 ) Employee-related expenses 11,729 16,968 (5,239 ) Facility expenses 21,708 23,265 (1,557 ) Stock-based compensation expenses 7,840 7,805 35 Sublicense and license fees 5,081 728 4,353 Other expenses — 297 (297 ) Total research and development expenses $ 67,152 $ 69,894 $ (2,742 ) The decrease of approximately $2.7 million was primarily attributable to a decrease in employee-related costs and facility-related expenses, offset by an increase in license fees. Acquired In-Process Research and Development Expenses Acquired in-process research and development expenses were not material for the three months ended June 30, 2026. Acquired in-process research and development costs were $96.3 million for the three months ended June 30, 2025 and were 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. General and Administrative Expenses General and administrative expenses were $17.6 million for the three months ended June 30, 2026, compared to $18.9 million for the three months ended June 30, 2025. The decrease of approximately $1.3 million was primarily associated with a decrease in employee-related costs, including stock-based compensation expenses. Collaboration Expense, Net Collaboration expense, net, was $40.3 million for the three months ended June 30, 2026, compared to $45.2 million for the three months ended June 30, 2025. The decrease was primarily attributable to an increase in our share of CASGEVY revenue. Other Income, Net Other income was $27.1 million for the three months ended June 30, 2026, compared to $22.1 million of income for the three months ended June 30, 2025. The increase of approximately $5.0 million in other income was primarily due to the change in fair value of corporate equity securities during the three months ended June 30, 2026. Comparison of six months ended June 30, 2026 and 2025 (in thousands): Six Months Ended June 30, Period to Period 2026 2025 Change Revenue: Collaboration revenue $ 11,000 $ — $ 11,000 Grant revenue 639 1,757 (1,118 ) Total revenue 11,639 1,757 9,882 Operating expenses: Research and development 135,726 142,378 (6,652 ) Acquired in-process research and development 2,473 96,253 (93,780 ) General and administrative 34,736 38,212 (3,476 ) Collaboration expense, net 86,221 102,662 (16,441 ) Total operating expenses 259,156 379,505 (120,349 ) Loss from operations (247,517 ) (377,748 ) 130,231 Other income, net 35,231 35,604 (373 ) Loss before income taxes (212,286 ) (342,144 ) 129,858 Provision for income taxes (1,799 ) (2,401 ) 602 Net loss $ (214,085 ) $ (344,545 ) $ 130,460 Collaboration Revenue Collaboration revenue was $11.0 million for the six months ended June 30, 2026 and was related to an upfront payment received as part of an immaterial license and collaboration agreement entered into during the second quarter of 2026. There was no collaboration revenue for the six months ended June 30, 2025. Please refer to Note 6 of the notes to our unaudited condensed 25 Table of Contents consolidated financial statements included in this Quarterly Report on Form 10-Q for further information on significant collaboration agreements. Research and Development Expenses Research and development expenses were $135.7 million for the six months ended June 30, 2026, compared to $142.4 million for the six months ended June 30, 2025. The following table summarizes our research and development expenses for the six months ended June 30, 2026 and 2025, together with the changes in those items in dollars (in thousands): Six Months Ended June 30, Period to Period 2026 2025 Change External research and development expenses $ 42,290 $ 38,686 $ 3,604 Employee-related expenses 26,720 36,700 (9,980 ) Facility expenses 43,942 47,224 (3,282 ) Stock-based compensation expenses 15,694 17,537 (1,843 ) Sublicense and license fees 6,741 1,526 5,215 Other expenses 339 705 (366 ) Total research and development expenses $ 135,726 $ 142,378 $ (6,652 ) The decrease of approximately $6.7 million was primarily attributable to a decrease in employee-related costs, including stock-based compensation expenses, as well as a decrease in facility costs. The changes noted were offset by an increase in external research and development expenses and license fees. Acquired In-Process Research and Development Expenses Acquired in-process research and development expenses were not material for the six months ended June 30, 2026. Acquired in-process research and development expenses were $96.3 million for the six months ended June 30, 2025 and was 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. General and Administrative Expenses General and administrative expenses were $34.7 million for the six months ended June 30, 2026, compared to $38.2 million for the six months ended June 30, 2025. The decrease of approximately $3.5 million was primarily associated with a decrease in employee-related costs, including stock-based compensation expenses, as well as a decrease in consulting costs. Collaboration Expense, Net Collaboration expense, net, was $86.2 million for the six months ended June 30, 2026, compared to $102.7 million for the six months ended June 30, 2025. The decrease was primarily attributable to an increase in our share of CASGEVY revenue. Other Income, Net Other income was $35.2 million for the six months ended June 30, 2026, compared to $35.6 million of income for the six months ended June 30, 2025. Liquidity and Capital Resources We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of June 30, 2026, we had $2,364.4 million in cash, cash equivalents and marketable securities, of which approximately $308.1 million was held outside of the United States, and an accumulated deficit of $2,161.6 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. Debt Notes, net 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 26 Table of Contents 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. 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. 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. 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. 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. 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. 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. At-the-Market Offerings (ATM) We have entered into an Open Market Sale AgreementSM, 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. 2021 ATM 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 three and six months ended June 30, 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. 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. 2025 ATM 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. No common shares were issued and sold under the 2025 ATM for the three and six months ended June 30, 2026. As of June 30, 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. 27 Table of Contents Share Issuance Agreement with Sirius Therapeutics 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. Funding Requirements 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. 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. 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. Outlook Based on our research and development plans and our timing expectations related to the progress of our programs, we expect our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditures for at least the next 24 months without giving effect to any additional proceeds we may receive under our license agreements and collaborations, including with Vertex, and any other capital raising transactions we may complete. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. Given our need for additional financing to support the long-term clinical development and future commercialization of our programs, as applicable, we intend to consider additional financing opportunities when market terms are favorable to us. Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our gene editing technology platform, as well as our delivery and other technologies; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance of our product candidates, either directly or with a collaborator or distributor, if approved, including for CASGEVY; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, defending, protecting and expanding our estate of intellectual property rights, including patents, trade secrets and know-how; and attracting, hiring and retaining qualified personnel. 28 Table of Contents Cash Flows The following table provides information regarding our cash flows for each of the periods below (in thousands): Six Months Ended June 30, Period to Period 2026 2025 Change Net cash used in operating activities $ (192,395 ) $ (167,827 ) $ (24,568 ) Net cash (used in) provided by investing activities (466,402 ) 50,222 (516,624 ) Net cash provided by financing activities 599,088 12,846 586,242 Effect of exchange rate changes on cash (26 ) 121 (147 ) Net decrease in cash $ (59,735 ) $ (104,638 ) $ 44,903 Operating Activities Net cash used in operating activities was $192.4 million for the six months ended June 30, 2026, compared to $167.8 million for the six months ended June 30, 2025. The $24.6 million increase in net cash used in operating activities was primarily driven by a $57.4 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 $130.5 million. Investing Activities Net cash used in investing activities for the six months ended June 30, 2026 was $466.4 million, compared to net cash provided by investing activities of $50.2 million for the six months ended June 30, 2025. The increase in net cash used in investing activities was primarily driven by a net increase in purchases of our marketable securities. Financing Activities Net cash provided by financing activities for the six months ended June 30, 2026 was $599.1 million, compared with $12.8 million for the six months ended June 30, 2025. Net cash provided by financing activities for the six months ended June 30, 2026 consisted primarily of $585.4 million in net proceeds from the issuance of the Notes in March 2026. Critical Accounting Policies and Estimates 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 six months ended June 30, 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. Convertible debt 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. Recent Accounting Pronouncements Refer to Note 1 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for a discussion of recent accounting pronouncements. 29 Table of Contents