CRISPR Therapy Cuts LDL Cholesterol by 52.5% for a Year

A single infusion of experimental CRISPR-Cas9 therapy CTX310 reduced LDL cholesterol by 52.5% and triglycerides by 47.8% at 12 months in a Phase 1 trial at Cleveland Clinic with 15 patients. No serious side effects were reported.

• LDL cholesterol fell 52.5% one year after a single infusion
• Triglycerides dropped 47.8% in patients at the highest dose
• Phase 1 trial enrolled 15 patients with doses of 0.1–0.8 mg/kg
• Therapy disables the ANGPTL3 gene in the liver