CRISPR Therapeutics’ CTX310 Shows Strong Phase 1 Results in Gene‑Editing Lipid Therapy

CRISPR Therapeutics has revealed positive Phase 1 clinical trial data for its experimental in‑vivo gene‑editing drug CTX310, targeting the gene ANGPTL3, showing both deep and durable reductions in key lipids. The data, presented at the American Heart Association Scientific Sessions 2025 and published in the The New England Journal of Medicine, show a mean ANGPTL3 reduction of 73 % (up to 89 %), triglyceride (TG) reductions averaging 55 % (up to 84 %), and LDL cholesterol reductions averaging 49 % (up to 87 %). Patients with elevated baseline TG levels saw a mean 60 % TG drop at therapeutic doses. Importantly, CTX310 was well tolerated: no treatment‑related serious adverse events and no Grade 3 or higher liver enzyme elevations. CTX310 works via a single intravenous dose of lipid‑nanoparticle delivered CRISPR/Cas9 editing of ANGPTL3 in hepatocytes. With more than 40 million US patients estimated to have elevated TG and/or LDL despite standard care, the therapy could be a game‑changer for severe or refractory dyslipidemia. CRISPR Therapeutics is now moving CTX310 into Phase 1b trials, initially focused on severe hypertriglyceridemia (sHTG) and mixed dyslipidemia. #geneediting #lipidtherapy #CRISPR #cardiovascularinnovation #SlimScan #GrowthStocks #CANSLIM