CRISPR Therapeutics Presents Positive Phase 1b Clinical Data for CTX310

CRISPR Therapeutics reported Phase 1b results for CTX310, an investigational in vivo CRISPR/Cas9 therapy targeting ANGPTL3 in severe hypertriglyceridemia. At Day 30, a single treatment produced mean reductions from baseline of 65% in circulating ANGPTL3 and 63% in triglycerides among participants whose median baseline triglyceride level exceeded 1,000 mg/dL. The data were presented at the 2026 American Heart Association Scientific Sessions, and the FDA has granted CTX310 RMAT designations for severe hypertriglyceridemia and elevated LDL-C.