ZUG, Switzerland and BOSTON — January 12, 2026 — Leads & Copy — CRISPR Therapeutics (Nasdaq: CRSP) has announced its strategic priorities and anticipated milestones for 2026, highlighting a data- and milestone-rich year across its portfolio.
The biopharmaceutical company, focused on gene-based medicines, is starting 2026 with approximately $2 billion in cash, cash equivalents, and marketable securities. Key priorities include accelerating the launch of CASGEVY®, which has multi-billion-dollar potential, and advancing a broad pipeline supported by gene-editing and siRNA platforms.
Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics, stated that the company is well-positioned with CASGEVY® gaining momentum and multiple programs rapidly advancing through clinical trials. Clinical data has demonstrated the potential of CTX310™ for severe cardiovascular disease, as well as zugo-cel in oncology and autoimmune diseases. The company has also added a siRNA pillar through a partnership with Sirius, advancing CTX611™ targeting Factor XI through the clinic.
CRISPR Therapeutics anticipates several key milestones in 2026, including the continued global commercialization of CASGEVY, with ongoing quarterly updates. Vertex is expected to initiate global regulatory submissions for CASGEVY in patients ages 5 – 11 in the first half of 2026.
Updates from CTX310® are expected in the second half of 2026, along with updates from the Lp(a) program. Top-line Phase 2 clinical data from CTX611 in patients undergoing total knee arthroplasty (TKA) are expected in the second half of 2026. Updates across autoimmune disease and immuno-oncology for zugocabtagene geleucel (zugo-cel; formerly CTX112™) are also expected in the second half of 2026.
The company expects to initiate a clinical trial for CTX460 in alpha-1 antitrypsin deficiency (AATD) in mid-2026, and a clinical trial for CTX340 in refractory hypertension in the first half of 2026.
CASGEVY has now been approved in the U.S., the United Kingdom, the EU, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates, and Kuwait for patients 12 years and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). In 2025, CASGEVY exceeded $100 million in revenue, with more than 60 patients receiving infusions, and patient initiations and first cell collections nearly tripled compared to 2024.
CRISPR Therapeutics continues to advance its in vivo gene editing programs, including CTX310, which demonstrated reductions of triglycerides (TG) and low-density lipoprotein (LDL) in Phase 1 trials. CTX320™, targeting LPA, has shown reductions of up to 73% in clinical trials, and the company is advancing a next-generation LPA program, CTX321™.
The siRNA-based portfolio includes CTX611 (SRSD107), which is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). CRISPR Therapeutics will lead global Phase 3 development of CTX611, excluding Greater China.
Zugocabtagene geleucel (zugo-cel; formerly CTX112™) is advancing in autoimmune disease and hematologic malignancies. Phase 1 clinical trials are ongoing across multiple autoimmune indications, and the first patient with SLE has maintained drug-free DORIS clinical remission through month 9 following CAR-T therapy.
CRISPR Therapeutics continues to advance its regenerative medicine portfolio, including CTX213™ for diabetes, which has demonstrated compelling preclinical efficacy.
CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT. Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide 60/40 with CRISPR Therapeutics.
CRISPR Therapeutics and Sirius Therapeutics entered into a collaboration in 2025 to develop and commercialize novel small interfering RNA (siRNA) therapies. The companies will co-develop CTX611 and share costs and profits equally.
Investor Contact:
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Source: CRISPR Therapeutics
