CRISPR Therapeutics AG
For the period ended Jun 30, 2025. Filed Aug 4, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Aug 4, 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 2025-03-31; filed 2025-05-06.
0000950170-25-064468
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent 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 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 112 previous paragraphs and 138 current paragraphs.
76 unchanged; 28 reworded; 30 added; 7 removed; 1 previous and 4 current paragraphs rearranged.
Tables are not compared: 3 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 (30)
Current report
Cardiovascular and metabolic diseases
Current report
CTX310 is being investigated in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein and triglyceride levels, in four patient groups: homozygous familial hypercholesterolemia, severe hypertriglyceridemia, heterozygous familial hypercholesterolemia, or mixed dyslipidemias. Both low-density lipoprotein and triglyceride are validated surrogate endpoints accepted by regulatory agencies. 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 report
SRSD107
Current report
In May 2025, we entered into a strategic collaboration with Sirius Therapeutics and certain of its affiliates, or Sirius, pursuant to which, among other things, we and Sirius will jointly develop and commercialize small interfering RNA, or siRNA therapies, beginning with SRSD107. SRSD107 is a next generation, long-acting siRNA designed to selectively inhibit human coagulation factor
Current report
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 report
Two Phase 1 clinical trials of SRSD107 have been completed by Sirius, and single doses of SRSD107 have been well tolerated. SRSD107 demonstrated robust pharmacodynamic effects, including reductions of over 93% in FXI levels and FXI activity, along with more than a twofold increase in activated partial thromboplastin time relative to baseline. These effects were sustained, with responses maintained for up to six months post-dosing. 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 report
Sirius Therapeutics
Current report
In May 2025, we entered into a collaboration, option and license agreement, or the Sirius Agreement, with Sirius, pursuant to which, among other things, we and Sirius will collaborate on the research, development, manufacture, commercialization and use of the Sirius Collaboration Products; and (2) Sirius granted us options to exclusively license Sirius siRNA technology to target up to two licensed targets from a list of seven reserved targets for the research, develop, manufacture and commercialization of Sirius Licensed Products, in exchange for the potential to receive certain option fees, milestone payments and royalties. In connection with entering into the Sirius Agreement, we made an upfront cash payment to Sirius of $25.0 million and also entered into a share issuance agreement with Sirius, pursuant to which we agreed to issue to Sirius the Sirius Shares.
Current report
With respect to Sirius Collaboration Products, we and Sirius will share equally all development and commercialization costs. For the first collaboration product candidate successfully developed, we will be the lead party responsible for commercialization efforts in the United States and Sirius will be the lead party responsible for commercialization efforts in Greater China. The parties will share equally the net profits and net losses incurred under the Sirius Agreement with respect to all Sirius Collaboration Products,
Current report
except in the event that a party opts out of the joint development and commercialization. We will also pay Sirius future one-time development and regulatory milestones with respect to Sirius Collaboration Products that achieve the applicable milestone events.
Current report
Asset acquisition costs related to acquired technology are expensed as acquired in-process research and development at the point that they have no established alternative future use. We classify asset acquisitions of acquired in-process research and development as investing activities on our condensed consolidated statements of cash flows.
Current report
The decrease of approximately $10.3 million was primarily attributable to the following:
Current report
$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 report
$1.8 million of decreased facility expenses primarily driven by lower laboratory-related costs; offset by
Current report
$2.2 million of increased external research and development costs, primarily associated with an increase in variable external research and manufacturing costs.
Current report
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 report
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 report
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 report
Collaboration Revenue
Current report
The decrease of approximately $14.0 million was primarily attributable to the following:
Current report
$9.9 million of decreased employee-related expenses, including stock-based compensation expenses; and
Current report
$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.
Current report
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.
Current report
Other Income, Net
Current report
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 report
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 report
Share Issuance Agreement with Sirius Therapeutics
Current report
As described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q, we and Sirius entered into a share issuance agreement and we registered and issued 1,842,105 common shares to Sirius, nominal value CHF 0.03 per share, at an issue price of $38.00 per share as partial consideration for entering into the Sirius Agreement.
Current report
Acquired In-process Research and Development Expenses
Current report
In-process research and development that is acquired in a transaction that does not qualify as a business combination under U.S. GAAP and that does not have an alternative future use is recorded as “Acquired in-process research and development” in our condensed consolidated statements of operations and comprehensive loss.
Removed paragraphs (7)
Previous report
Cardiovascular disease
Previous report
Research and Development Expenses
Previous report
General and Administrative Expenses
Previous report
Collaboration Expense, Net
Previous report
Research and Development Expenses
Previous report
General and Administrative Expenses
Previous report
Collaboration Expense, Net
Reworded paragraphs (28)
Previous wording
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 SCD and 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.
Current wording
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.
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, Kingdom of Saudi Arabia, Kingdom of Bahrain and the United Arab Emirates for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including (1) three clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, (2) two clinical trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and (3) long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.
Current wording
In 2023, CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in the world. To date, CASGEVY has been approved in the United States, European Union, Great Britain, Kingdom of Saudi Arabia, Kingdom of Bahrain, Qatar, Canada, Switzerland, and the United Arab Emirates for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including in (1) three clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, (2) two clinical trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and (3) long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.
Previous wording
We continue to advance our internally developed targeted conditioning program, as well asin vivo hematopoietic stem cell, or HSC, editing approaches utilizing lipid nanoparticle-mediated delivery through preclinical studies. Both initiatives could significantly expand the addressable patient populations for SCD and TDT.
Current wording
We continue to advance our internally developed targeted conditioning program, as well asin vivo hematopoietic stem cell editing approaches utilizing lipid nanoparticle-mediated delivery through preclinical studies. Both initiatives could significantly expand the addressable patient populations for SCD and TDT.
Previous wording
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 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.
Previous wording
Ourin vivo gene editing strategy focuses on gene disruption and whole gene correction – the two technologies required to address the vast majority of the most prevalent severe monogenic diseases as well as many common diseases. We have established a leading platform forin vivo gene editing and are rapidly advancing a broad portfolio ofin vivo programs, supported by an internal lipid nanoparticle, or LNP, team to enable liver-directed and extrahepatic programs with novel lipids, formulations and targeting moieties. Our firstin vivo programs target the liver, taking advantage of validated LNP delivery technologies, and aim to treat diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association.
Current wording
Ourin vivo gene editing strategy focuses on gene disruption and whole gene correction – the two technologies required to address the vast majority of the most prevalent severe monogenic diseases as well as many common diseases. We have established a leading platform forin vivo gene editing and are rapidly advancing a pipeline ofin vivo gene editing candidates targeting major unmet needs in cardiovascular and metabolic diseases using our proprietary lipid nanoparticle, or LNP, delivery platform. Our firstin vivo programs target the liver, taking advantage of validated LNP delivery technologies, and aim to treat diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association.
Previous wording
Our first twoin vivo programs utilizing our proprietary LNP platform, CTX310 and CTX320, aim to address cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3, or ANGPTL3, and lipoprotein (a), or Lp(a), respectively. CTX310 is being investigated in an ongoing clinical trial targeting ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein and triglyceride levels, in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia. In addition, CTX320 is being investigated in an ongoing clinical trial targeting LPA, the gene encoding apo(a), a critical component of Lp(a) in patients with elevated Lp(a), which has shown to have an independent association with major adverse cardiovascular events.
Current wording
Our first twoin vivo programs utilizing our proprietary LNP platform, CTX310 and CTX320, aim to address cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3, or ANGPTL3, and lipoprotein (a), or Lp(a), respectively.
Previous 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. 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
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.
Previous wording
Beginning in 2018, we partnered with ViaCyte, Inc., or ViaCyte (now a wholly-owned subsidiary of Vertex), to pursue the discovery, development and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes. In 2023, ViaCyte elected to opt-out of the collaboration with us for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, the on-going collaboration assets will be wholly owned by us, subject to a royalty on future sales owed to ViaCyte. Our product candidate, CTX211, being developed for the potential treatment of T1D, resulted from this collaboration, and which we are continuing to advance in a Phase 1 clinical trial. Additionally, in 2023, we entered into a non-exclusive license agreement with Vertex for Vertex to utilize certain of our gene-editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of T1D, diabetes type 2 or insulin dependent/requiring diabetes throughout the world. To date, we have recognized revenue of $205.0 million in upfront and milestone payments and remain eligible to receive additional research and development milestones and royalties on future products under the license.
Current wording
Beginning in 2018, we partnered with ViaCyte, Inc., or ViaCyte (now a wholly-owned subsidiary of Vertex), to pursue the discovery, development and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes. In 2023, ViaCyte elected to opt-out of the collaboration with us for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, the ongoing collaboration assets will be wholly owned by us, subject to a royalty on future sales owed to ViaCyte. Our product candidate, CTX211, being developed for the potential treatment of T1D, resulted from this collaboration, and which we are continuing to advance in a Phase 1 clinical trial. Additionally, in 2023, we entered into a non-exclusive license agreement with Vertex for Vertex to utilize certain of our gene-editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of T1D, diabetes type 2 or insulin dependent/requiring diabetes throughout the world. To date, we have recognized revenue of $205.0 million in upfront and milestone payments and remain eligible to receive additional research and development milestones and royalties on future products under the license.
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., or Nkarta, 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, 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.
Previous wording
Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans and payments related to certain of our license and collaboration agreements with strategic partners.
Current wording
Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business development activities, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans and payments related to certain of our license and collaboration agreements with strategic partners.
Previous wording
We have a history of recurring losses and expect to continue to incur losses for the foreseeable future; however, we have been in a net income position in certain previous years due to certain payments associated with our collaboration and license agreements with Vertex. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel growth; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company.
Current wording
We have a history of recurring losses and expect to continue to incur losses for the foreseeable future; however, we have been in a net income position in certain previous years due to certain payments associated with our collaboration and license agreements with Vertex. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; pursue business development activities; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel growth; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company.
Previous wording
We have not generated any revenue to date from sales of any wholly-owned product and do not expect to do so in the near future. Revenue recognized for the three months ended March 31, 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 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.
Previous wording
fees and other payments related to acquiring and maintaining licenses under our third-party licensing agreements.
Current wording
fees and other payments related to acquiring and maintaining licenses under certain of our third-party licensing agreements.
Previous wording
Research and development expenses were $72.5 million for the three months ended March 31, 2025, compared to $76.2 million for the three months ended March 31, 2024. The following table summarizes our research and development expenses for the three months ended March 31, 2025 and 2024, together with the changes in those items in dollars (in thousands):
Current 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):
Previous wording
The decrease of approximately $3.7 million was primarily attributable to a decrease in employee-related expenses, including stock-based compensation expenses.
Current wording
$7.6 million of decreased employee-related expenses, including stock-based compensation expenses;
Previous wording
Other income was $13.5 million for the three months ended March 31, 2025, compared to $24.7 million of income for the three months ended March 31, 2024. The decrease of approximately $11.2 million was primarily due to the change in fair value of corporate equity securities in the first quarter of 2025, as well as a decrease in interest income earned on cash, cash equivalents and marketable securities for the three months ended March 31, 2025.
Current 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.
Previous wording
General and administrative expenses were $19.3 million for the three months ended March 31, 2025, compared to general and administrative expenses of $18.0 million for the three months ended March 31, 2024. The increase of approximately $1.3 million was primarily associated with an increase in stock-based compensation expense, offset by a reduction in intellectual property costs and employee-related expenses.
Current 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.
Previous wording
Collaboration expense, net, was $57.5 million for the three months ended March 31, 2025, compared to $47.0 million for the three months ended March 31, 2024. The increase of approximately $10.5 million in collaboration expense, net, was primarily attributable to an increase in cost of goods sold and other commercial costs related to CASGEVY and collaboration expenses related toin vivo HSC editing, offset by our share of product sales.
Current wording
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 wording
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of March 31, 2025, we had $1,855.3 million in cash, cash equivalents and marketable securities, of which approximately $108.7 million was held outside of the United States, and an accumulated deficit of $1,501.9 million. We anticipate that we will continue to incur losses for at least the next several years. We expect to continue to incur research and development costs and general and administrative expenses consistent with costs associated with research and development at companies of our size and stage of development, and, as a result, we will need additional capital to fund our operations, which we may raise through public or private equity or debt financings, strategic collaborations, or other sources.
Current wording
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of 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.
Previous wording
For the three months ended March 31, 2025, we issued and sold an aggregate of 0.2 million common shares under the 2021 ATM at an average price of $54.33 per share for aggregate proceeds of $8.7 million. Activity under the 2021 ATM was not material for the three months ended March 31, 2024. Equity issuance costs, excluding stamp taxes, were not material for the three months ended March 31, 2025 and 2024.
Current wording
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.
Previous wording
As of March 31, 2025, we have common shares having aggregate gross proceeds up to $354.8 million remaining under our current prospectus supplement, and we have issued and sold an aggregate of 2.1 million common shares 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.
Current 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.
Previous wording
Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution, filing, defense and intellectual property maintenance costs, and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development at companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.
Current wording
Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution, filing, defense and intellectual property maintenance costs, business development activities, and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.
Previous wording
Although we and our partner, Vertex, have received marketing approvals for CASGEVY in certain jurisdictions, we cannot guarantee we and Vertex will receive additional marketing approvals for CASGEVY or we will receive marketing approvals for our other product candidates in the future. Most of our programs are still in early stages of development and the outcome of our efforts is uncertain, and we cannot estimate the actual amounts necessary to successfully complete the development, manufacture and commercialization of any current or future product candidates, if approved, or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to finance our cash needs through a combination of equity financings, debt financings and payments received in connection with our collaboration and license agreements. We intend to consider opportunities to raise additional funds through the sale of equity or debt securities when market conditions are favorable to us to do so. However, the trading prices for our common shares and other biopharmaceutical companies have been highly volatile. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event, 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.
Current wording
Although we and our partner, Vertex, have received marketing approvals for CASGEVY in certain jurisdictions, we cannot guarantee we and Vertex will receive additional marketing approvals for CASGEVY or we will receive marketing approvals for our other product candidates in the future. Most of our programs are still in early stages of 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.
Previous wording
Net cash used in operating activities was $53.9 million for the three months ended March 31, 2025, compared to cash provided by operating activities of $109.8 million for the three months ended March 31, 2024. The $163.7 million increase in net cash used in operating activities was primarily driven by a decrease in net changes of operating assets and liabilities of $155.6 million, primarily due to the impact of receivables from Vertex which were paid during the quarter ($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 $11.3 million, primarily driven by the change in fair value of corporate equity securities, and our net loss position increased by $19.4 million, from a net loss of $116.6 million for the first quarter of 2024 compared to a net loss of $136.0 million for the first quarter of 2025.
Current 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.
Previous wording
Net cash used in investing activities for the three months ended March 31, 2025 was $19.8 million, compared to net cash used in investing activities of $97.8 million for the three months ended March 31, 2024. The decrease in net cash used in investing activities was primarily driven by a net decrease in purchases of our marketable securities.
Current wording
Net cash 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.
Previous wording
Net cash provided by financing activities for the three months ended March 31, 2025 was $10.6 million, compared with $305.9 million for the three months ended March 31, 2024. Net cash provided by financing activities for the three months ended March 31, 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 three months ended March 31, 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 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.
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 months ended March 31, 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 changes to the below policy.
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 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.
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 to these condensed consolidated financial statements for further discussion on the Vertex Hemoglobinopathy Agreements.
Current 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.
Rearranged current paragraphs (4)
Current report — wording carried from elsewhere
CTX320 is being investigated in an ongoing Phase 1 clinical trial targeting LPA, the gene encoding apo(a), a critical component of Lp(a), in patients with elevated Lp(a), a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events.
Current report — wording carried from elsewhere
Comparison of six months ended June 30, 2025 and 2024 (in thousands):
Current report — wording carried from elsewhere
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 report — wording carried from elsewhere
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):
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
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