CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310 Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and Lipid Lowering

-Data presented in a late-breaking presentation at the American Heart Association (AHA) Scientific Sessions 2025-

-Phase 1 clinical data for CTX310 demonstrate robust, dose-dependent reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%), and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose-

-Among participants with elevated baseline TG (>150 mg/dL), a mean reduction of 60% in TG were observed at therapeutic doses-

-CTX310 was well tolerated with no treatment-related serious adverse events and no ≥Grade 3 changes in liver transaminases-

-Findings simultaneously published in The New England Journal of Medicine entitled “First-in-Human Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3”-

ZUG, Switzerland and BOSTON, Nov. 08, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose. These data demonstrate the potential of CTX310 to deliver meaningful and sustained lipid lowering following a single-course intravenous (IV) infusion.

These data were presented today during a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM) in a peer-reviewed article entitled “Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3.”

"The publication and presentation of these Phase 1 results mark an important milestone for CRISPR Therapeutics and for the field of in vivo gene editing,” said Naimish Patel, M.D., Chief Medical Officer of CRISPR Therapeutics. “For the first time, we’ve shown that a single-course in vivo CRISPR treatment can safely and durably lower ANGPTL3, leading to clinically meaningful reductions in triglycerides and LDL. These data provide strong support for continued advancement of CTX310 and our broader cardiovascular gene-editing portfolio.”