CRISPR Therapeutics outlines 2026 milestones with $2 billion cash

CRISPR Therapeutics (NASDAQ: CRSP) announced its strategic priorities and anticipated milestones for 2026, highlighting continued commercialization of its approved gene therapy CASGEVY and advancement of multiple clinical programs across cardiovascular, autoimmune, oncology and rare diseases.

The company reported CASGEVY exceeded $100 million in revenue in 2025, with more than 60 patients receiving infusions. Patient initiations and first cell collections increased nearly three-fold in 2025 compared to 2024. CASGEVY is currently approved in multiple countries for patients 12 years and older with sickle cell disease or transfusion-dependent beta thalassemia.

Regulatory submissions for CASGEVY in patients aged 5-11 years are expected to be initiated by partner Vertex in the first half of 2026. The therapy received a Commissioner's National Priority Voucher for pediatric submissions, enabling accelerated regulatory review.

For its in vivo liver editing programs, CRISPR expects updates from CTX310 in the second half of 2026. The company plans to initiate clinical trials for CTX460 in alpha-1 antitrypsin deficiency in mid-2026 and for CTX340 in refractory hypertension in the first half of 2026.

The company anticipates top-line Phase 2 clinical data from CTX611, a siRNA therapy targeting Factor XI, in patients undergoing total knee arthroplasty in the second half of 2026. Updates from its Lp(a) program are also expected in 2026.

For its autoimmune and immuno-oncology program, zugocabtagene geleucel (zugo-cel), updates are expected in the second half of 2026 across multiple indications including systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis.

CRISPR Therapeutics reported entering 2026 with approximately $2 billion in cash, cash equivalents, and marketable securities. The company develops CASGEVY in collaboration with Vertex, which leads global commercialization under a 60/40 profit-sharing arrangement.

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