Cleveland Clinic Reports One-Year Safety and Efficacy of CRISPR Gene-Editing Therapy for Lowering Cholesterol and Triglycerides

A Phase 1, first-in-human clinical trial led by Cleveland Clinic has demonstrated that a single infusion of an investigational CRISPR-Cas9 gene-editing therapy, CTX310, safely and continuously reduced LDL ("bad") cholesterol and triglycerides in patients with medication-resistant lipid disorders over a one-year follow-up period. Patients receiving the highest dose experienced a 52.5% reduction in LDL cholesterol and a 47.8% reduction in triglycerides from baseline, with no serious adverse events related to the therapy. The findings were presented at the 2026 European Society of Cardiology annual meeting and simultaneously published in the New England Journal of Medicine.