CRISPR Therapeutics AG
For the period ended Sep 30, 2025. Filed Nov 10, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 10, 2025
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 2025-06-30; filed 2025-08-04.
0000950170-25-102052
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-10.
0001193125-25-273337
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 138 previous paragraphs and 141 current paragraphs.
97 unchanged; 32 reworded; 9 added; 6 removed; 3 previous and 3 current paragraphs rearranged.
Tables are not compared: 5 previous and 5 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 (9)
Current report
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.
Current report
The decrease of approximately $23.3 million was primarily attributable to the following:
Current report
The decrease of approximately $37.2 million was primarily attributable to the following:
Current report
$4.6 million of decreased facility-related expenses, primarily driven by lower lab consumable costs; and
Current 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.
Current report
We have entered into an Open Market Sale Agreement SM, or the Sales Agreement, with Jefferies LLC under which we, at our sole discretion, are able to offer and sell, from time to time at prevailing market prices, our common shares. The following are in connection with the Sales Agreement.
Current report
2021 ATM
Current report
In January 2021, we filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million, or, together with the subsequent prospectus supplements filed in July 2021 and August 2024 relating to 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.
Removed paragraphs (6)
Previous report
CTX131 is being developed for both solid tumors and hematologic malignancies. It is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the candidate in adult patients with relapsed or refractory solid tumors and hematologic malignancies, including T cell lymphomas, or TCL. We believe allogeneic CAR T approaches for TCL may have greater potential to meet the unmet need in this patient population given the patients’ own T cells are not suitable for autologous manufacturing.
Previous report
The decrease of approximately $10.3 million was primarily attributable to the following:
Previous report
The decrease of approximately $14.0 million was primarily attributable to the following:
Previous report
Collaboration expense, net, was $102.7 million for the six months ended June 30, 2025, compared to $99.1 million for the six months ended June 30, 2024. The increase of approximately $3.6 million in collaboration expense, net, was primarily attributable to an increase in our share of CASGEVY revenue, offset by an increase in costs associated with the commercial launch of CASGEVY.
Previous report
In August 2019, we entered into an Open Market Sale Agreement SM with Jefferies under which we were able to offer and sell, from time to time at our sole discretion through Jefferies, as our sales agent, our common shares, par value of CHF 0.03 per share, or the August 2019 Sales Agreement. In January 2021, in connection with the August 2019 Sales Agreement, 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. In August 2024, we filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplements filed in January 2021 and July 2021, respectively, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement and July 2021 prospectus supplement having aggregate gross proceeds of up to $378.6 million, or, together with the January 2021 prospectus supplement and July 2021 prospectus supplement, the 2021 ATM.
Previous report
For the 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. Activity under the 2021 ATM was not material for the six months ended June 30, 2024. Equity issuance costs related to the 2021 ATM, excluding stamp taxes, were not material for the three and six months ended June 30, 2025 and 2024.
Reworded paragraphs (32)
Previous wording
We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing several cell therapy programs for oncology and/or autoimmune indications, including two next-generation allogeneic CAR T programs, CTX112 targeting Cluster of Differentiation 19, or CD19, and CTX131 targeting Cluster of Differentiation 70, or CD70. These product candidates incorporate edits designed to enhance CAR T potency, reduce CAR T exhaustion and evade the immune system. An additional edit in CTX131 is designed to prevent the CAR T cells from killing other CAR T cells. In addition, these next-generation candidates exhibit increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch. We are producing CTX112 and CTX131 for clinical trials at our internal GMP manufacturing facility in Framingham, Massachusetts.
Current wording
We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing 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.
Previous wording
CTX112 is being developed for both hematologic malignancies and autoimmune indications. It is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy, as well as an ongoing clinical trial in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis. CTX112 has been granted RMAT designation by the FDA for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.
Current 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.
Previous wording
Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 for the potential treatment of solid tumors.
Current wording
Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR T programs.
Previous wording
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. In June 2025, we reported additional data for CTX310, demonstrating dose-dependent reductions in ANGPTL3, triglyceride and low-density lipoprotein following a single administration of CTX310. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in triglyceride and low-density lipoprotein reductions of up to 86% at dose level 4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings.
Current wording
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 wording
Building upon CTX310 and CTX320, we are progressing CTX340, targeting angiotensinogen for refractory hypertension, as well as CTX450, targeting 5’-aminolevulinate synthase 1 for acute hepatic porphyria, through preclinical studies. CTX340 and CTX450 are currently progressing through IND/CTA-enabling studies. In addition, we have programs focused on gene correction in the liver, including programs leveraging technologies developed by our CRISPR-X research team. Finally, we are pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells.
Current wording
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 wording
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, 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.
Current wording
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 wording
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. In July 2025, the European Medicines Agency authorized the initiation of a Phase 2 clinical trial of SRSD107 for thromboembolic disorders. The study is designed to evaluate the safety and efficacy of SRSD107 in preventing venous thromboembolism in patients undergoing total knee arthroplasty and aims to confirm its anticoagulant potential.
Current wording
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.
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, or DMD, and myotonic dystrophy type 1, or DM1, and others to support and complement our hematopoietic stem cell, CAR T, in vivo and T1D programs and platform, including agreements with: Nkarta, Inc. to develop and commercialize products leveraging donor-derived, gene-edited CAR-NK cells; Capsida Biotherapeutics, Inc. to developin vivo gene editing therapies delivered with engineered adeno-associated virus, or AAV, vectors; Roswell Park Comprehensive Cancer Center to advance a gene-edited autologous CAR T program against a new target; MaxCyte, Inc. onex vivo delivery for our hemoglobinopathy and CAR T programs; CureVac AG on optimized mRNA constructs and manufacturing for certainin vivo programs; and KSQ Therapeutics, Inc. on intellectual property for our allogeneic immuno-oncology programs.
Current wording
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.
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, 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 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.
Previous wording
Research and development expenses were $69.9 million for the three months ended June 30, 2025, compared to $80.2 million for the three months ended June 30, 2024. The following table summarizes our research and development expenses for the three months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands):
Current 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):
Previous wording
$2.2 million of increased external research and development costs, primarily associated with an increase in variable external research and manufacturing costs.
Current wording
$10.5 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs;
Previous wording
$9.9 million of decreased employee-related expenses, including stock-based compensation expenses; and
Current wording
$8.9 million of decreased employee-related expenses, including stock-based compensation expenses, primarily driven by decreased headcount; and
Previous wording
$1.8 million of decreased facility expenses primarily driven by lower laboratory-related costs; offset by
Current wording
$4.0 million of decreased facility expenses primarily driven by lower laboratory-related costs.
Previous wording
General and administrative expenses were $18.9 million for the three months ended June 30, 2025, compared to general and administrative expenses of $19.5 million for the three months ended June 30, 2024. The decrease of approximately $0.6 million was primarily associated with a decrease in intellectual property costs, offset by an increase in facilities-related costs.
Current 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.
Previous wording
Collaboration expense, net, was $45.2 million for the three months ended June 30, 2025, compared to $52.1 million for the three months ended June 30, 2024. The decrease of approximately $6.9 million in collaboration expense, net, was primarily attributable to an increase in our share of CASGEVY revenue, as well as a decrease in operating expenses for the program.
Current 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.
Previous wording
Other income was $22.1 million for the three months ended June 30, 2025, compared to $26.1 million of income for the three months ended June 30, 2024. The decrease of approximately $4.0 million was primarily due to the change in fair value of corporate equity securities during the three months ended June 30, 2025, as well as a decrease in interest income earned on cash, cash equivalents and marketable securities for the three months ended June 30, 2025.
Current 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.
Previous wording
Comparison of six months ended June 30, 2025 and 2024 (in thousands):
Current wording
Comparison of nine months ended September 30, 2025 and 2024 (in thousands):
Previous wording
There was no collaboration revenue for the six months ended June 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.
Current wording
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.
Previous wording
Research and development expenses were $142.4 million for the six months ended June 30, 2025, compared to $156.3 million for the six months ended June 30, 2024. The following table summarizes our research and development expenses for the six months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands):
Current wording
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):
Previous wording
$7.6 million of decreased employee-related expenses, including stock-based compensation expenses;
Current wording
$18.9 million of decreased employee-related expenses, including stock-based compensation expenses, primarily driven by decreased headcount;
Previous wording
$2.8 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;
Current wording
$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.
Previous wording
Acquired in-process research and development expenses were $96.3 million for the three months ended June 30, 2025. There was no acquired in-process research and development expenses for the three months ended June 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.
Current wording
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.
Previous wording
General and administrative expenses were $38.2 million for the six months ended June 30, 2025, compared to general and administrative expenses of $37.4 million for the six months ended June 30, 2024. The increase of approximately $0.8 million was primarily associated with an increase in stock-based compensation expense, offset by a decrease in intellectual property costs.
Current wording
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 wording
Other income was $35.6 million for the six months ended June 30, 2025, compared to $50.9 million of income for the six months ended June 30, 2024. The decrease of approximately $15.3 million was primarily due to the change in fair value of corporate equity securities during the six months ended June 30, 2025, as well as a decrease in interest income earned on cash, cash equivalents and marketable securities for the six months ended June 30, 2025.
Current wording
Other income was $61.8 million for the nine months ended September 30, 2025, compared to $75.9 million of income for the nine months ended September 30, 2024. The decrease of approximately $14.1 million was primarily due to a decrease in interest income earned on cash, cash equivalents and marketable securities for the nine months ended September 30, 2025.
Previous wording
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of June 30, 2025, we had $1,721.2 million in cash, cash equivalents and marketable securities, of which approximately $83.5 million was held outside of the United States, and an accumulated deficit of $1,710.5 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.
Current 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.
Previous wording
From June 30, 2025 through the date of issuance of this Quarterly Report on Form 10-Q, we have issued and sold an aggregate of 2.3 million common shares under the 2021 ATM at an average price of $58.18 per share for aggregate proceeds of $130.7 million, which were net of equity issuance costs of $1.6 million. An additional $1.3 million of stamp taxes will be owed on this amount.
Current wording
From September 30, 2025 through the date of issuance of this Quarterly Report on Form 10-Q, we have issued and sold an aggregate of 1.0 million common shares under the 2021 ATM at an average price of $70.53 per share for aggregate proceeds of $72.2 million, which were net of equity issuance costs of $1.0 million, excluding stamp taxes of $0.7 million.
Previous wording
As of June 30, 2025, the Company has issued and sold an aggregate of 2.1 million common shares under the 2021 ATM at an average price of $117.41 per share for aggregate proceeds of $241.9 million, which were net of equity issuance costs of $3.3 million, excluding stamp taxes. As of June 30, 2025, common shares having aggregate gross proceeds up to $354.8 million remain available under the 2021 ATM.
Current 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.
Previous wording
Net cash used in operating activities was $167.8 million for the six months ended June 30, 2025, compared to cash provided by operating activities of $14.2 million for the six months ended June 30, 2024. The $182.0 million increase in net cash used in operating activities was primarily driven by a decrease in net changes of operating assets and liabilities of $182.6 million, primarily due to the impact of receivables from Vertex which were paid in the period ($200.0 million paid in the first quarter of 2024 compared to $25.0 million paid in the first quarter of 2025). Additionally, non-cash expense increased by $102.2 million, primarily driven by expense related to the upfront payment as part of the Sirius Agreement described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q. Further, our net loss position increased by $101.5 million, from a net loss of $243.0 million for the six months ended June 30, 2024 compared to a net loss of $344.5 million for the six months ended June 30, 2025.
Current 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).
Previous wording
Net cash provided by investing activities for the six months ended June 30, 2025 was $50.2 million, compared to net cash used in investing activities of $224.7 million for the six months ended June 30, 2024. The decrease in net cash used in investing activities was primarily driven by a net increase in maturities of our marketable securities.
Current 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.
Previous wording
Net cash provided by financing activities for the six months ended June 30, 2025 was $12.8 million, compared with $305.5 million for the six months ended June 30, 2024. Net cash provided by financing activities for the six months ended June 30, 2025 consisted primarily of $8.7 million in net proceeds from the sale of common shares issued in connection with our 2021 ATM, as well as option exercise proceeds, net of issuance costs. Net cash provided by financing activities for the six months ended June 30, 2024 consisted of 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. We received net proceeds of $279.0 million, excluding stamp taxes due of $2.8 million.
Current 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.
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 six months ended June 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, with the exception of the new policy for acquired in-process research and development expenses, as well as changes to our policy around collaboration arrangements which was disclosed in our Quarterly Report on Form 10-Q filed with the SEC on May 6, 2025.
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, 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.
Previous wording
Collaboration costs specific to the Vertex Hemoglobinopathy Agreements accounted for under ASC 808 are presented within “collaboration expense, net” in the condensed consolidated statements of operations and comprehensive loss. 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 discussion on the Vertex Hemoglobinopathy Agreements.
Current wording
Collaboration costs specific to the Vertex Hemoglobinopathy Agreements (as defined in Note 6) accounted for under ASC 808 are presented within “collaboration expense, net” in the condensed consolidated statements of operations and comprehensive loss. 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 discussion on the Vertex Hemoglobinopathy Agreements.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
$9.5 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs;
Current report — wording carried from elsewhere
Acquired In-Process Research and Development Expenses
Current report — wording carried from elsewhere
Acquired In-process Research and Development Expenses
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
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
Previous report — wording carried elsewhere
$4.2 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.
Previous report — wording carried elsewhere
Acquired in-process research and development expenses were $96.3 million for the six months ended June 30, 2025. There was no acquired in-process research and development expenses for the six months ended June 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.