On February 12, 2026 CRISPR Therapeutics (Nasdaq: CRSP) reported financial results for the fourth quarter and full year ended December 31, 2025.
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"As we close out the fourth quarter, CRISPR Therapeutics continues to make steady progress across a broad and increasingly mature pipeline," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "We made meaningful advances across multiple clinical and preclinical programs, including encouraging data from zugo-cel in autoimmune disease and oncology, continued global uptake of CASGEVY, and important developments across our in vivo liver editing portfolio, and momentum in our siRNA collaboration with Sirius Therapeutics. At the same time, we continue to strengthen our platform capabilities to support long-term value creation. Together, these developments reflect continued execution across the portfolio."
Recent Highlights and Outlook
Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])
CASGEVY is approved in the U.S., EU, Great Britain, Canada, Switzerland, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain, Qatar, the United Arab Emirates (UAE), and Kuwait for patients 12 years and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
CASGEVY generated fourth quarter 2025 revenue of $54 million and full year 2025 revenue of $116 million, reflecting continued growth in treated patients. 64 patients received infusions of CASGEVY during the year, including 30 in the fourth quarter. Globally, 147 people with SCD or TDT initiated the treatment process with their first cell collection during the year.
Patient initiations and first cell collections increased nearly three-fold in 2025 compared to 2024, with continued momentum going into 2026.
Access to CASGEVY continued to expand across key markets. As of year-end, approximately 90% of patients in the U.S. have reimbursed access to CASGEVY. CASGEVY is also reimbursed in the U.K., Italy, Austria, Denmark, Luxembourg, KSA, the Kingdom of Bahrain, the UAE, and Kuwait. Most recently, in January, Vertex secured reimbursed access for eligible patients with SCD in Scotland, consistent with the prior reimbursement agreement reached in 2025 for patients with TDT.
At the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2025, positive pediatric data from the pivotal studies in children ages 5-11 years with SCD or TDT were presented and featured in the "Best of ASH (Free ASH Whitepaper)" program. Global regulatory submissions in this age group are expected to begin in the first half of 2026. The U.S. Food and Drug Administration (FDA) awarded a Commissioner’s National Priority Voucher for this pediatric submission, supporting an expedited review once submissions are complete.
CRISPR Therapeutics continues to advance its in vivo hematopoietic stem cell editing approach utilizing lipid nanoparticle (LNP)-mediated delivery through preclinical studies. This initiative has the potential to expand the addressable patient populations for SCD and TDT.
In Vivo Liver Editing
CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary LNP delivery platform.
The Company continues to advance CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. The Company expects to provide an update in the second half of 2026.
Building on insights from CTX320, the Company is advancing its next-generation LPA program, CTX321. CTX321 incorporates an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected in 2026.
CRISPR Therapeutics continues to advance several preclinical in vivo gene editing candidates, including:
CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate to emerge from the Company’s SyNTase editing platform. The Company expects to initiate a clinical trial for CTX460 in mid-2026.
CTX340, targeting angiotensinogen (AGT) for refractory hypertension, is currently in IND/CTA-enabling studies. The Company expects to initiate a clinical trial for CTX340 in the first half of 2026.
siRNA-based Programs
CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics.
CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.
The program has the potential to target a range of thromboembolic and clotting-related indications and represents a multi-billion-dollar market opportunity, including atrial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), thrombosis in chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD). CRISPR Therapeutics is expected to lead global Phase 3 development of CTX611 with Sirius Therapeutics responsible for development activities in greater China.
CRISPR Therapeutics has the option to nominate up to two siRNA targets for research and development and expects to provide an update in 2026. For each target, CRISPR Therapeutics will fund research and retain opt-in rights to lead clinical development and commercialization. Sirius will be eligible to receive milestone payments and contingent payments, as well as tiered royalties.
Autoimmune Disease and Immuno-Oncology
Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance in both autoimmune disease and hematologic malignancies.
In autoimmune disease, Phase 1 clinical trials of zugo-cel are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA), with continued progress across studies. The Company expects to provide updates in the second half of 2026.
In immuno-oncology, the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing. The Company expects to provide updates in the second half of 2026. Zugo-cel is also being evaluated in combination with pirtobrutinib in aggressive B-cell lymphomas under an existing collaboration with Lilly.
CRISPR Therapeutics continues to leverage its proprietary lipid nanoparticle (LNP) delivery platform, mRNA, and conjugation capabilities to advance its in vivo CAR-T platform with potential applications in autoimmune disease and oncology.
CRISPR Therapeutics’ autoimmune disease and immuno-oncology platforms 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.
Regenerative Medicine
CRISPR Therapeutics continues to advance its regenerative medicine portfolio, including its efforts in diabetes. The Company is advancing CTX213, a deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from edited induced pluripotent stem cells (iPSCs), for the treatment of Type 1 diabetes. CTX213 has demonstrated compelling preclinical efficacy via direct administration and is progressing towards the clinic. The Company expects to provide additional updates as development progresses.
Upcoming Events
The Company will present at the upcoming events:
Citi’s 2026 Virtual Oncology Leadership Summit
Date: Wednesday, February 18, 2026
Time: 10:45 a.m. ET
TD Cowen 46th Annual Health Care Conference
Date: Monday, March 2, 2026
Time: 11:10 a.m. ET
Leerink Partners Global Healthcare Conference
Date: Wednesday, March 11, 2026
Time: 1:00 p.m. ET
Fourth Quarter and Full Year 2025 Financial Results
Cash Position: Cash, cash equivalents, and marketable securities were $1,975.8 million as of December 31, 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.
R&D Expenses: R&D expenses were $83.5 million for the fourth quarter of 2025, compared to $71.7 million for the fourth quarter of 2024. The increase in R&D expense was primarily driven by an increase in licensing fees.
G&A Expenses: General and administrative expenses were $18.4 million for the fourth quarter of 2025, compared to $18.1 million for the fourth quarter of 2024.
Collaboration Expense: Collaboration expense, net, was $53.7 million for the fourth quarter of 2025, compared to $10.4 million for the same period in 2024. In 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 amended collaboration agreement with Vertex. The year-over-year increase in collaboration expense reflects the absence of a comparable deferral in 2025.
Net Loss: Net loss was $130.6 million for the fourth quarter of 2025, compared to a net loss of $37.3 million for the fourth 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 and older by multiple regulatory bodies around the world.
(Press release, CRISPR Therapeutics, FEB 12, 2026, View Source [SID1234662637])