CRISPR Therapeutics Eyes Strong Growth in 2026

The company highlights momentum in CASGEVY rollout, progress in in vivo programs, and strong cash reserves.

November 11, 2025

CRISPR Therapeutics Reports Robust Q3 2025 Amid Broad Pipeline Advancements

CRISPR Therapeutics is pushing forward on all fronts. In its latest quarterly update, the company laid out steady progress across multiple clinical programs—some moving through global late-stage trials, others entering the clinic for the first time. What connects them is a clear focus: expand access, scale delivery, and meet critical treatment gaps with gene-based medicine.

Leading the charge is CASGEVY, a therapy developed with Vertex for patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The treatment is now approved in more than eight regions, including the U.S., Europe, and the Middle East. By the end of Q3, nearly 300 patients had been referred to treatment centers. About 165 had completed their first cell collection, and 39 had received infusions. These numbers are up sharply from earlier in the year, with 110 cell collections recorded in the first nine months—twice as many as in all of 2024.

Momentum is also coming from new reimbursement agreements. Italy, which has Europe’s largest TDT population, signed on in September. Pediatric development is advancing as well, with enrollment now complete in two global Phase 3 studies for children aged 5 to 11. Dosing is expected to wrap up by year-end, and the first data will be presented in December. Vertex sees a clear path to surpassing $100 million in CASGEVY revenue this year and expects meaningful growth in 2026.

Progress Across In Vivo, Oncology, and Autoimmune Programs

Beyond hemoglobinopathies, CRISPR Therapeutics is scaling its in vivo pipeline. CTX310, its candidate targeting ANGPTL3 for cardiovascular risk reduction, delivered encouraging Phase 1 results and is headed into Phase 1b trials. Another program, CTX460, is being developed with the company’s new SyNTase editing platform. In preclinical models for alpha-1 antitrypsin deficiency, it showed strong potential to correct the disease at the gene level. A clinical trial for CTX460 is expected to begin in mid-2026.

The company is also moving forward on CTX320, aimed at reducing lipoprotein(a) levels—a known but largely untreated cardiovascular risk factor. CRISPR plans to provide an update on this trial in the first half of next year.

In autoimmune disease and oncology, the lead asset is CTX112, a CD19-targeting CAR T therapy being tested in both B-cell malignancies and systemic autoimmune disorders. A broader update is expected by the end of 2025. Meanwhile, CTX131 has been stepped back to allow more resources to flow into programs with greater potential impact.

RNA-Based Programs and Regenerative Medicine

CRISPR’s RNA-based platform, developed through its collaboration with Sirius Therapeutics, is also gaining traction. The lead siRNA candidate, SRSD107, entered a Phase 2 trial in Europe this fall. The therapy is designed to prevent clotting complications in high-risk populations with long-acting, subcutaneous dosing. Additional trial sites are being added, and future expansion into multiple thromboembolic and clotting-related conditions is on the roadmap.

Work in regenerative medicine continues as well. CRISPR is building out next-generation therapies for type 1 diabetes using immune-evasive, gene-edited beta cell precursors derived from stem cells. These programs are designed to support insulin independence without chronic immunosuppression. An update is expected later this year.

Financial Update

Financially, the company remains well-capitalized. As of September 30, CRISPR held $1.94 billion in cash, cash equivalents, and marketable securities—up slightly from year-end 2024. R&D costs fell to $58.9 million for the quarter, reflecting reduced manufacturing and personnel expenses. Net loss widened to $106.4 million, in part due to increased collaboration costs as the CASGEVY program matures and deferral limits no longer apply.

Across the board, CRISPR Therapeutics is delivering on its pipeline strategy while investing in new capabilities. With a steady cadence of clinical data, commercial traction in key markets, and a deep bench of candidates entering or preparing for trials, the company is positioning itself for another active year ahead.