Phase 1 CTX310 trial reports sustained lipid reductions at one year

A Cleveland Clinic-led first-in-human phase 1 trial evaluated CTX310, an experimental CRISPR-Cas9 therapy, in 15 patients with lipid disorders inadequately controlled by medications. Participants received a single infusion at doses ranging from 0.1 to 0.8 mg/kg, preceded by corticosteroids and antihistamines. Researchers monitored safety and changes in ANGPTL3, LDL cholesterol and triglycerides over one year.

CTX310 delivers gene-editing machinery to the liver to disable ANGPTL3, a gene involved in regulating circulating lipids. At 12 months, the highest-dose group had reductions from baseline of 52.5% in LDL cholesterol and 47.8% in triglycerides. No serious adverse events related to treatment were reported during follow-up. According to the source, the findings were presented at the 2026 European Society of Cardiology annual meeting and simultaneously published in the New England Journal of Medicine.

The findings suggest a durable lipid-lowering effect, but this small early-phase trial does not establish cardiovascular benefit or long-term safety. The source does not provide dose-group sizes or detailed adverse-event data. CTX310 remains experimental, and an additional 15 years of safety follow-up is planned. CRISPR Therapeutics funded the study; the lead author's institution also received research funding from the company.