-CASGEVY® momentum accelerating; nearly 300 patients have been referred to Authorized Treatment Centers (ATCs), approximately 165 patients have completed their first cell collection and 39 have received infusions across all regions; Vertex expects clear line of sight to over $100 million in total CASGEVY revenue this year and significant growth expected in 2026-
-Pediatric development of exa-cel is advancing in SCD and TDT, with enrollment in two global Phase 3 studies completed; initial data will be presented at the upcoming American Society of Hematology (ASH) annual meeting-
-Clinical trials ongoing for CTX112™, targeting CD19, across multiple indications; broad updates for CTX112 in autoimmune disease and oncology expected by year-end-
-Preclinical data for CTX460™, presented at the European Society of Gene and Cell Therapy (ESGCT) annual congress, demonstrated in vivo gene correction of alpha-1 antitrypsin deficiency (AATD) with a potential best-in-class profile; clinical trial initiation planned for mid-2026-
-Phase 2 clinical trial ongoing for SRSD107, targeting Factor XI for the prevention of thromboembolic disorders; first patient dosed and trial expanded to additional sites in Europe-
-Strong balance sheet with approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2025-
ZUG, Switzerland and BOSTON, Nov. 10, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the third quarter ended September 30, 2025.
"This has been another strong quarter of execution and progress across our portfolio," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "CASGEVY® momentum continues to build globally, reflecting growing patient engagement and clinical advancement. Enrollment has been completed in two global Phase 3 pediatric studies, and dosing is on track to complete this quarter. Additionally, positive Phase 1 data for CTX310® presented in a late-breaking presentation at the American Heart Association Scientific Sessions and published in The New England Journal of Medicine, highlight the breadth and potential of our platform to address serious cardiovascular disease. We continue to advance our broader pipeline, including dosing the first patient in the Phase 2 clinical trial of SRSD107 and unveiling our novel SyNTase™ editing platform with CTX460™, highlighting continued innovation and expansion of our therapeutic toolkit. With strong execution and growing momentum across our programs, CRISPR Therapeutics is well positioned to lead the next wave of gene editing innovation and deliver potentially transformative therapies to patients."
Recent Highlights and Outlook
Hemoglobinopathies and CASGEVY® (exagamglogene autotemcel [exa-cel])
CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), designed to eliminate both vaso-occlusive crises (VOCs) and transfusion requirements. CASGEVY is approved in the U.S., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Qatar, Canada, Switzerland and the United Arab Emirates (UAE) for the treatment of both SCD and TDT. Across these markets, there are more than 60,000 eligible patients in these countries, including approximately 37,000 in North America and Europe and more than 23,000 in the Middle East. With a well-established treatment pathway and a growing number of patients progressing through each stage, CASGEVY has strong momentum heading into late 2025 and beyond.
In September, a reimbursement agreement was reached in Italy for patients with SCD and TDT. Italy represents the largest population of individuals with TDT in Europe, with approximately 5,000 people aged 12 years and older with TDT and approximately 2,300 with SCD.
Globally, since launch through September 30th, 2025, approximately 165 patients with SCD or TDT have completed their first cell collection, including 50 people in the third quarter. 39 patients have received infusions of CASGEVY, including 10 infused in the third quarter of 2025. Nearly 300 patients have been referred by their physicians to an authorized treatment center (ATC) to begin the treatment process. This includes 110 cell collections in the first nine months of 2025, double the total for all of 2024.
Enrollment of children 5 to 11 years of age with SCD or TDT in two global Phase 3 studies of CASGEVY has been completed, and dosing is expected to be completed this quarter. Initial data from these studies will be presented at the American Society of Hematology (ASH) annual meeting on December 6th, 2025.
The number of ATCs initiating patients continues to increase in the U.S., Europe, and the Middle East. Through the end of September, 25 ATCs had initiated more than 5 patients, with at least one ATC in each region initiating 20 or more patients.
Momentum continues to build through the final months of 2025. With continued uptake and reimbursement progress across major regions, Vertex expects a clear line of sight to over $100 million in total CASGEVY revenue this year with significant growth expected in 2026.
In Vivo Liver Editing
CRISPR Therapeutics is advancing a pipeline of in vivo gene editing candidates addressing major unmet needs in cardiovascular, metabolic and rare diseases using its proprietary, de-risked lipid nanoparticle (LNP) delivery platform.
CTX310®, targeting ANGPTL3, is in an ongoing Phase 1 clinical trial in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias. Phase 1 data were presented in a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM). Results from the Phase 1 clinical trial highlight the potential of CTX310 to safely and durably lower both triglycerides (TG) and low-density lipoprotein (LDL) following a single-course IV administration. CRISPR Therapeutics is advancing CTX310 into Phase 1b clinical trials, prioritizing development in sHTG and mixed dyslipidemia.
CTX320™ is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with an increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels affect up to 20% of the global population and remain unaddressed by current therapies. The Company plans to provide an update in the first half of 2026.
CRISPR Therapeutics continues to advance its preclinical in vivo programs: CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD); CTX340™, targeting AGT for the treatment of refractory hypertension; and CTX450™, targeting ALAS1 for the treatment of acute hepatic porphyria (AHP).
CTX460 is the first investigational candidate using the Company's novel SyNTase editing platform, unveiled in October. SyNTase is designed to enable precise, in vivo gene correction, and represents an important expansion of CRISPR Therapeutics' toolkit. Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress demonstrated >90% mRNA correction, a 5-fold increase in total AAT levels, and >99% serum M-AAT:Z-AAT ratio in AATD disease models. These findings provide preclinical proof-of-concept for precise, single-dose in vivo gene correction using the SyNTase editing platform and support the potential best-in-class profile of CTX460. CRISPR Therapeutics expects to initiate a clinical trial of CTX460 in mid-2026.
Preclinical data from CTX340 were presented in a late-breaking poster presentation at the American Heart Association (AHA) Scientific Sessions. In a spontaneous hypertensive rat model, CTX340 showed >70% liver editing and mean arterial pressure reduction of 53 mmHg compared to vehicle that was durable throughout the study (~8.5 months). Furthermore, in non-human primates, CTX340 showed greater than 90% AGT reduction with a two-dose regimen showing the additive effects of repeat dosing and enabling dose titration. CTX340 was well tolerated with no hypotension or hypokalemia observed. CTX340 is currently in IND/CTA-enabling studies.
Autoimmune Disease and Immuno-Oncology
CTX112™, targeting CD19, is being developed for autoimmune disease and hematologic malignancies and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma. A Phase 1 clinical trial in autoimmune disease is underway in systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis. In parallel, a Phase 1 clinical trial of CTX112 in relapsed or refractory B-cell malignancies is ongoing. The Company plans to provide a broad update on CTX112 by year-end.
CTX131™, targeting CD70, was previously in development for both solid tumors and hematologic malignancies. While the Phase 1 data are encouraging, the Company has strategically redirected resources away from this program to advance other programs with the greatest potential for long-term value creation.
CRISPR Therapeutics is leveraging its expertise and proprietary lipid nanoparticle (LNP) delivery platform, mRNA, and conjugation capabilities to advance an in vivo CAR T platform with the ability to address autoimmune disease and oncology.
CRISPR Therapeutics' autoimmune disease and immuno-oncology programs are supported by a wholly-owned, GMP manufacturing facility located in Framingham, Massachusetts, which provides end-to-end production capabilities for its cell therapy portfolio and supports both clinical and future commercial supply.
siRNA
In September, CRISPR Therapeutics and its partner Sirius Therapeutics announced that the first patient was dosed in Europe in the Phase 2 clinical trial of SRSD107, a long-acting Factor XI (FXI) small interfering RNA (siRNA) for thromboembolic disorders. The trial is evaluating the safety and efficacy of SRSD107 in preventing venous thromboembolism (VTE) following total knee arthroplasty (TKA) and will inform dose selection for future pivotal trials. SRSD107 has the potential to be a best-in-class FXI inhibitor, showing deep reductions in FXI via semi-annual subcutaneous injection. SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a broader strategic collaboration to advance RNA-based medicines.
CRISPR Therapeutics and Sirius Therapeutics have expanded the Phase 2 clinical trial with additional centers in Europe. SRSD107 is being developed for a range of thromboembolic and clotting-related indications, including arterial fibrillation (AF), cancer-associated thrombosis (CAT), chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD), ischemic stroke and VTE.
Regenerative Medicine
CRISPR Therapeutics continues to advance its regenerative medicine efforts for Type 1 diabetes (T1D). Beyond CTX211™, the Company is developing next-generation programs that leverage induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors. These approaches aim to achieve insulin independence in T1D patients without requiring chronic immunosuppression. The Company expects to provide an update this year.
Upcoming Events
The Company will participate in the following events in November:
Guggenheim 2nd Annual Healthcare Innovation ConferenceDate: Wednesday, November 12, 2025Time: 11:30 a.m. ET
Jefferies Global Healthcare ConferenceDate: Wednesday, November 19, 2025Time: 1:00 p.m. GMT
Third Quarter 2025 Financial Results
Cash Position: Cash, cash equivalents, and marketable securities were $1,944.1 million as of September 30, 2025, compared to $1,903.8 million as of December 31, 2024. The increase in cash was primarily driven by proceeds from the issuance of common shares, option exercise activity and interest income, offset by operating expenses, as well as the $25.0 million upfront cash payment made as part of the Sirius Agreement.
R&D Expenses: R&D expenses were $58.9 million for the third quarter of 2025, compared to $82.2 million for the third quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in variable external research and manufacturing costs, as well as a decrease in employee-related expenses, including stock-based compensation expenses.
G&A Expenses: General and administrative expenses were $16.9 million for the third quarter of 2025, compared to $17.4 million for the third quarter of 2024.
Collaboration Expense: Collaboration expense, net, was $57.1 million for the third quarter of 2025, compared to $11.2 million for the third quarter of 2024. In the third quarter of 2024, we exercised our option to defer specified costs under the CASGEVY program in excess of the deferral limit of $110.3 million under the A&R Vertex JDCA, as amended. The increase 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.
Net Loss: Net loss was $106.4 million for the third quarter of 2025, compared to a net loss of $85.9 million for the third quarter of 2024.
About CASGEVY® (exagamglogene autotemcel [exa-cel])CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient's own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years ...
