CRISPR Therapeutics Q1 2026: CTX310 Expands to US, Casgevy Pediatric sBLA Expected H1 2026

CRISPR Therapeutics reports Q1 2026 financial results and a comprehensive pipeline update. Key clinical milestones: (1) CTX310, targeting ANGPTL3 for severe hypertriglyceridemia and refractory hypercholesterolemia, is advancing in Phase 1b with FDA IND clearance now secured for US site expansion — international sites already enrolling. (2) CTX460, targeting an undisclosed liver-expressed gene for a lipid disorder, is on track for IND submission and first patient dosing in mid-2026. (3) CTX340, another in vivo liver program, is targeting IND submission in H1 2026. (4) Supplemental Biologics License Application (sBLA) for Casgevy (exagamglogene autotemcel) in pediatric patients aged 5–11 years with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) is expected to be submitted to the FDA in H1 2026, which would significantly expand the addressable market. (5) Casgevy commercial momentum: approximately 90% of eligible US patients now have reimbursement access secured; over 45 authorized treatment centers (ATCs) globally are active; more than 40 patients have completed first cell collection. Q1 2026 financial results: cash and equivalents of approximately $1.8 billion providing runway into 2029; royalty revenue from Casgevy partnership with Vertex of approximately $1.46 million for Q1. CRISPR Therapeutics CEO Samarth Kulkarni stated that 2026 will be defined by the Casgevy pediatric expansion and the advancement of the CTX in vivo platform toward additional liver targets.