CRISPR Therapeutics Reports 65% ANGPTL3 And 63% Triglyceride Reductions With CTX310 In Phase 1b Trial For Severe Hypertriglyceridemia; Well Tolerated, With No Treatment-Related Serious Adverse Events And No Clinically Relevant Elevations In Liver Transaminases

-Data presented in a late-breaking oral presentation at AHA-

-A single-course treatment with CTX310 produced a mean reduction from baseline of 65% in circulating ANGPTL3 and 63% in triglycerides (TG) at Day 30 in patients with severe hypertriglyceridemia (sHTG), who had a median baseline TG level above 1,000 mg/dL-

-CTX310 was well tolerated, with no treatment-related serious adverse events and no clinically relevant elevations in liver transaminases-

ZUG, Switzerland and BOSTON, Nov. 09, 2026 (GLOBE NEWSWIRE) --  CRISPR Therapeutics (NASDAQ:CRSP) today presented clinical data from its Phase 1b clinical trial evaluating CTX310®, an investigational, in vivo CRISPR/Cas9 gene editing therapy targeting angiopoietin-like protein 3 (ANGPTL3), in patients with severe hypertriglyceridemia (sHTG). A single-course treatment with CTX310 produced mean reductions from baseline at Day 30 of 65% in circulating ANGPTL3 and 63% in triglycerides (TG) in participants with a median baseline TG level above 1,000 mg/dL. These results show that a single-course treatment with CTX310 can deeply reduce ANGPTL3 and triglycerides in patients with severe hypertriglyceridemia, reinforcing its potential as a one-time gene editing treatment for this high-risk population.

These data were presented today during a late-breaking session at the AHA Scientific Sessions 2026 and build on the Phase 1a results, including one-year durability data presented at the European Society of Cardiology (ESC) Congress 2026 and published in The New England Journal of Medicine. The U.S. Food and Drug Administration (FDA) recently granted Regenerative Medicine Advanced Therapy (RMAT) designations to CTX310 for the treatment of sHTG and for the treatment of patients with elevated low-density lipoprotein cholesterol (LDL-C). CRISPR Therapeutics continues to advance CTX310 in the Phase 1b clinical trial, with U.S. and ex-U.S. trials ongoing.