GlobeNewswire·Nov 9, 2026, 9:16 AM ET

CRISPR Therapeutics Presents Phase 1b Data for CTX310® Demonstrating Reductions in ANGPTL3 and Triglycerides in Patients with Severe Hypertriglyceridemia at the American Heart Association (AHA) Scientific Sessions 2026

CRSP

CRISPR Therapeutics presented Phase 1b clinical data for CTX310, an investigational in vivo CRISPR/Cas9 gene editing therapy, at the AHA Scientific Sessions 2026.
A single-course treatment with CTX310 resulted in a mean 65% reduction in circulating ANGPTL3 and a 63% reduction in triglycerides (TG) at Day 30 in patients with severe hypertriglyceridemia (sHTG).
Participants in the sHTG cohort had a median baseline TG level exceeding 1,000 mg/dL, with all subjects having diabetes, 30% having a history of pancreatitis, and 20% having clinical atherosclerotic cardiovascular disease.
The therapy demonstrated additional mean reductions of 51% in non-HDL-C, 23% in apolipoprotein B, and 16% in LDL-C from a low baseline.
CTX310 was well tolerated with no treatment-related serious adverse events or clinically relevant elevations in liver transaminases (AST or ALT).
One participant experienced a resolved Grade 2 infusion-related reaction, while no Grade 3 or higher adverse events related to the treatment were reported.
The Phase 1b trial is an open-label, multicenter study involving 10 participants receiving a single intravenous infusion of CTX310 at a fixed dose of 40 mg or 50 mg based on body weight.
The U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designations to CTX310 for the treatment of sHTG and for patients with elevated LDL-C.
Reductions in ANGPTL3 and TG levels were maintained in participants followed for at least 90 days, with no cases of acute pancreatitis reported during the study period.
The trial is currently enrolling at sites in New Zealand, Australia, the United Kingdom, and the United States.

Company: CRISPR Therapeutics AG
Keywords: Nasdaq:CRSP, CRISPR Therapeutics AG