ZUG, Switzerland and BOSTON — May 4, 2026 — Leads & Copy —
CRISPR Therapeutics (Nasdaq: CRSP) has released its financial results for the first quarter, which ended March 31, 2026.
Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics, stated that the first quarter showed continued execution across the company’s platform. The company expanded zugo-cel into new autoimmune indications and advanced multiple in vivo liver-directed programs toward the clinic, while CASGEVY continued its momentum. Kulkarni believes 2026 will be a defining year for CRISPR Therapeutics, with a strengthened balance sheet and multiple upcoming milestones.
Recent highlights include CASGEVY’s approval in the U.S., Canada, the U.K., the EU, 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). 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.
CASGEVY generated first quarter 2026 revenue of $43 million, and more than 500 people globally have now initiated the CASGEVY treatment journey.
Vertex has completed the U.S. regulatory submission for approval of CASGEVY in children ages 5 to 11 years old with SCD or TDT. The U.S. Food and Drug Administration (FDA) awarded a Commissioner’s National Priority Voucher for this pediatric submission, supporting an accelerated review timeline once accepted.
Access to CASGEVY continued to expand across key markets. Most recently, a pricing agreement was secured for eligible patients with SCD or TDT in Germany, with final implementation underway. As of year-end 2025, approximately 90% of patients in the U.S. had reimbursed access to CASGEVY, which is also reimbursed in the U.K., Italy, Austria, Denmark, Luxembourg, KSA, the Kingdom of Bahrain, the UAE, and Kuwait.
CRISPR Therapeutics continues to advance its in vivo hematopoietic stem cell editing approach using lipid nanoparticle (LNP)-mediated delivery. This approach has the potential to expand the addressable patient populations for SCD and TDT.
The company is also progressing with the development of CTX310, an investigational therapy targeting angiopoietin-related protein 3 (ANGPTL3), in a Phase 1b clinical trial. The company is prioritizing indications in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. CRISPR Therapeutics recently received FDA clearance of its Investigational New Drug (IND) application, supporting expansion of the ongoing trial into the U.S. An update is expected in the second half of 2026.
Additional preclinical in vivo gene editing candidates include CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), which is currently in IND/CTA-enabling studies. A clinical trial for CTX460 is expected to begin in mid-2026. CTX340, targeting angiotensinogen (AGT) for refractory hypertension, is currently in the IND/CTA-enabling phase, with a clinical trial expected to begin in the first half of 2026. CTX321, the company’s next-generation LPA program, is progressing through IND/CTA-enabling studies. An Lp(a) program update is anticipated in 2026.
CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs targeting cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics. CTX611 (SRSD107), a long-acting siRNA therapeutic targeting Factor XI (FXI), is advancing through a Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). An update is expected in the second half of 2026.
Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance across both autoimmune disease and hematologic malignancies. In autoimmune disease, zugo-cel is currently being evaluated in two ongoing Phase 1 basket trials: a rheumatology basket and a hematology basket. The FDA cleared the IND application for a third Phase 1 trial in autoimmune neurologic diseases, which has been initiated. Enrollment across the zugo-cel autoimmune clinical program continues to progress, with over 14 patients dosed to date and over 10 clinical trial sites activated globally. Further updates are expected in the second half of 2026.
In immuno-oncology, the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing, with updates anticipated in the second half of 2026. The company has also initiated a combination study evaluating zugo-cel with pirtobrutinib in aggressive B-cell lymphomas, under the company’s existing collaboration with Lilly.
CRISPR Therapeutics is also advancing a proprietary in vivo CAR-T platform with potential applications across autoimmune disease and oncology.
The company is developing CTX213, a deviceless beta cell replacement candidate for Type 1 diabetes. CTX213 has demonstrated compelling preclinical efficacy and is progressing toward the clinic. Additional updates are expected as development progresses.
First Quarter 2026 Financial Results:
Cash Position: Cash, cash equivalents, and marketable securities were $2,441.8 million as of March 31, 2026, compared to $1,975.8 million as of December 31, 2025. The increase in cash was primarily driven by net proceeds of $585.4 million from the issuance of convertible senior notes in March 2026, offset by operating expenses.
R&D Expenses: R&D expenses were $68.6 million for the first quarter of 2026, compared to $72.5 million for the first quarter of 2025. The decrease in R&D expense was primarily attributable to a decrease in employee-related costs, including stock-based compensation expenses.
G&A Expenses: General and administrative expenses were $17.2 million for the first quarter of 2026, compared to $19.3 million for the first quarter of 2025. The decrease in G&A expense was primarily attributable to a decrease in employee-related costs.
Collaboration Expense: Collaboration expense, net, was $45.9 million for the first quarter of 2026, compared to $57.5 million for the first quarter of 2025. The decrease was primarily attributable to an increase in the Company’s share of CASGEVY revenue.
Net Loss: Net loss was $122.9 million for the first quarter of 2026, compared to a net loss of $136.0 million for the first quarter of 2025.
About 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). 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.
Under an amended agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY, and Vertex and CRISPR Therapeutics share profits and program costs on a 60/40 basis.
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technology.
Zugocabtagene geleucel (zugo-cel) is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications.
CTX611 is a novel double-stranded, long-acting siRNA, designed to target the human coagulation factor XI, or FXI, messenger RNA and inhibit FXI protein expression.
CRISPR Therapeutics and Sirius Therapeutics entered into a strategic collaboration in 2025 to develop and commercialize siRNA therapies for thromboembolic disorders and other serious diseases.
CRISPR Therapeutics is a leading biopharmaceutical company focused on developing transformative gene-based medicines for serious human diseases. The Company is also expanding its gene editing toolkit through SyNTase™ editing, its novel, proprietary platform designed to enable precise, efficient, and scalable gene correction. To accelerate its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California.
CRISPR THERAPEUTICS® standard character mark and design logo, CTX112™, CTX213™, CTX310®, CTX321™, CTX340™, CTX460™, CTX611™ and SyNTase™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners.
Source: CRISPR Therapeutics
