CRISPR Therapeutics AG

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, 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.

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 multiple jurisdictions, including the United States, European Union, 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, CASGEVY has been approved for use in younger children in select jurisdictions, including in the United States, United Kingdom and certain countries in the Middle East. We and Vertex continue to investigate CASGEVY in clinical trials, including in 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 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.

Current wording

CTX340, directed towards angiotensinogen, and CTX460, directed towards SERPINA1 using our proprietary SyNTase editing platform, are in Phase 1 clinical trials in patients for the treatment of refractory hypertension and in patients for the treatment of alpha-1 antitrypsin deficiency, respectively.

Previous wording

CTX611 is in ongoing Phase 2 clinical trials, including in patients undergoing total knee arthroplasty.

Current wording

CTX611 is in ongoing Phase 2 clinical trials, including in patients undergoing total knee arthroplasty, which recently completed enrollment.

Previous wording

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.

Current wording

In autoimmune disease, zugo-cel is being investigated in three ongoing Phase 1 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.

Previous 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.

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. 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. Zugo-cel has been granted regenerative medicine advanced therapy designation by the FDA for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.

Previous wording

Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR-T programs. 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.

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 integratedin vivo CAR-T therapies by targeting T cells with LNPs and leveraging our delivery, mRNA, and gene editing expertise.

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 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.

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 and nine months ended September 30, 2026 was $12.7 million and $24.3 million, respectively, and primarily related to $12.3 million of previously deferred collaboration revenue under an amendment to the 2015 Collaboration Agreement with Vertex recognized in the third quarter of 2026, as well as an upfront payment on an immaterial license and collaboration agreement entered into during the second quarter of 2026. Revenue for the three and nine months ended September 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.

Previous wording

Comparison of three months ended June 30, 2026 and 2025 (in thousands):

Current wording

Comparison of the three months ended September 30, 2026 and 2025 (in thousands):

Previous wording

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.

Current wording

Collaboration revenue was $12.3 million for the three months ended September 30, 2026 and was primarily related to $12.3 million in previously deferred collaboration revenue recognized under an amendment to the 2015 Collaboration Agreement with Vertex during the third quarter of 2026. There was no collaboration revenue for the three months ended September 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.

Previous wording

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):

Current wording

Research and development expenses were $71.9 million for the three months ended September 30, 2026, compared to $58.9 million for the three months ended September 30, 2025. The following table summarizes our research and development expenses for the three months ended September 30, 2026 and 2025, together with the changes in those items in dollars (in thousands):

Previous wording

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.

Current wording

Collaboration expense, net, was $41.3 million for the three months ended September 30, 2026, compared to $57.1 million for the three months ended September 30, 2025. The decrease was primarily attributable to an increase in our share of CASGEVY revenue.

Previous wording

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.

Current wording

Other income was $18.7 million for the three months ended September 30, 2026, compared to other income of $26.2 million for the three months ended September 30, 2025. The decrease of approximately $7.5 million in other income was primarily due to interest expense on the Notes, as well as the change in fair value of corporate equity securities during the three months ended September 30, 2026.

Previous wording

Comparison of six months ended June 30, 2026 and 2025 (in thousands):

Current wording

Comparison of the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

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 consolidated financial statements included in this Quarterly Report on Form 10-Q for further information on significant collaboration agreements.

Current wording

Collaboration revenue was $23.3 million for the nine months ended September 30, 2026 and was primarily related to $12.3 million in previously deferred collaboration revenue recognized under an amendment to the 2015 Collaboration Agreement with Vertex during the third quarter of 2026, as well as 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 nine months ended September 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 collaboration agreements.

Previous wording

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):

Current wording

Research and development expenses were $207.6 million for the nine months ended September 30, 2026, compared to $201.3 million for the nine months ended September 30, 2025. The following table summarizes our research and development expenses for the nine months ended September 30, 2026 and 2025, together with the changes in those items in dollars (in thousands):

Previous wording

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.

Current wording

The increase of approximately $6.3 million was primarily attributable to an increase in external research and development expenses and sublicense and license fees, offset by a decrease in employee-related costs, including stock-based compensation expenses.

Previous wording

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.

Current wording

Acquired in-process research and development expenses were $14.1 million for the nine months ended September 30, 2026, compared to $96.3 million for the nine months ended September 30, 2025. Acquired in-process research and development expenses for the nine months ended September 30, 2026 primarily consisted of a development milestone under the Sirius Agreement that was recognized in the third quarter of 2026. Acquired in-process research and development expenses for the nine months ended September 30, 2025 were attributable to the costs incurred upon entering the Sirius Agreement during the second quarter of 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 the Sirius Agreement.

Previous wording

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.

Current wording

General and administrative expenses were $52.6 million for the nine months ended September 30, 2026, compared to $55.1 million for the nine months ended September 30, 2025. The decrease of approximately $2.5 million was primarily associated with a decrease in employee-related costs, including stock-based compensation expenses, as well as a decrease in consulting costs.

Previous wording

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.

Current wording

Collaboration expense, net, was $127.5 million for the nine months ended September 30, 2026, compared to $159.8 million for the nine months ended September 30, 2025. The decrease was primarily attributable to an increase in our share of CASGEVY revenue.

Previous wording

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.

Current wording

We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of September 30, 2026, we had $2,276.6 million in cash, cash equivalents and marketable securities, of which approximately $308.6 million was held outside of the United States, and an accumulated deficit of $2,273.1 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

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.

Current wording

No common shares were issued and sold under the 2025 ATM for the three and nine months ended September 30, 2026. As of September 30, 2026, we have 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 is 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.

Previous wording

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.

Current wording

Net cash used in operating activities was $282.1 million for the nine months ended September 30, 2026, compared to $252.5 million for the nine months ended September 30, 2025. The $29.6 million increase in net cash used in operating activities was primarily driven by acquired in-process research and development related to the Sirius Agreement in 2025 and 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 $125.5 million.

Previous wording

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.

Current wording

Net cash used in investing activities for the nine months ended September 30, 2026 was $480.5 million, compared to net cash used in investing activities of $69.0 million for the nine months ended September 30, 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 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.

Current wording

Net cash provided by financing activities for the nine months ended September 30, 2026 was $603.5 million, compared with $309.6 million for the nine months ended September 30, 2025. Net cash provided by financing activities for the nine months ended September 30, 2026 consisted primarily of $585.4 million in net proceeds from the issuance of the Notes in March 2026 and was offset by a decrease of $287.3 million in proceeds from the issuance of common shares.

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 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.

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 and nine months ended September 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.