One-Time CRISPR Gene-Editing Therapy Safely Lowers Cholesterol and Triglycerides for One Year in Phase 1 Trial

A Phase 1, first-in-human clinical trial led by Cleveland Clinic demonstrated that a single infusion of the investigational CRISPR-Cas9 gene-editing therapy, CTX310, safely and continuously reduced LDL ("bad") cholesterol by 52.5% and triglycerides by 47.8% after one year in patients with medication-resistant lipid disorders. The findings were presented at the European Society of Cardiology annual meeting and simultaneously published in The New England Journal of Medicine.