A first-in-human Phase 1 trial from Cleveland Clinic showed that a single infusion of an experimental CRISPR-Cas9 therapy, called CTX310, lowered LDL cholesterol and triglycerides by roughly half in patients whose lipid disorders had not responded adequately to existing medications. The reductions persisted for a full year after treatment, according to results presented at the 2026 European Society of Cardiology annual meeting and published simultaneously in the New England Journal of Medicine.
The therapy works by carrying the CRISPR editing system to the liver, where it switches off the ANGPTL3 gene, which helps regulate fats in the blood. In the 15-patient study, those who received the highest dose (0.8 mg/kg) saw a 52.5% reduction in LDL cholesterol and a 47.8% drop in triglycerides from baseline at 12 months. No serious adverse events related to the treatment were reported during the follow-up period.
The findings are encouraging but preliminary, as the trial involved only a small number of participants and CTX310 remains experimental. Researchers plan to continue long-term safety monitoring for another 15 years, in line with FDA recommendations for gene-editing therapies. The study was funded by CRISPR Therapeutics AG, and the first author's institution has received research funding from the company.