CTX310 Shows Durable One-Year ANGPTL3, LDL, and Triglyceride Reductions After Single Infusion

A follow-up letter published August 28, 2026 in the New England Journal of Medicine, and presented simultaneously by Cleveland Clinic cardiologist Luke Laffin at the European Society of Cardiology Congress 2026, reports one-year durability data for CTX310, CRISPR Therapeutics' lipid-nanoparticle-delivered in vivo CRISPR-Cas9 therapy targeting hepatic ANGPTL3. Among the 15-patient Phase 1a cohort with refractory dyslipidemia, participants at the highest dose (0.8 mg/kg) sustained mean reductions from baseline of 78.6% in ANGPTL3, 52.5% in LDL cholesterol, and 47.8% in triglycerides through 12 months after a single infusion. No new treatment-related serious adverse events or dose-limiting toxicities emerged since the initial November 2025 publication, and no ongoing liver-function abnormalities were observed. CRISPR Therapeutics is advancing the program into Phase 1b with US and ex-US sites, marking the longest-duration follow-up yet reported for an in vivo CRISPR cardiovascular therapy.