CRISPR therapy CTX310 halved LDL cholesterol for a full year

Cleveland Clinic's Phase 1 trial of the CRISPR-Cas9 therapy CTX310 found a single infusion cut LDL cholesterol by 52.5% and triglycerides by 47.8% at 12 months in 15 patients with lipid disorders. No serious adverse events were reported; results were presented at ESC 2026 and published in NEJM.

• Single CTX310 infusion cut LDL cholesterol by 52.5% at 12 months
• Triglycerides fell 47.8% at the highest dose
• Trial enrolled 15 patients at doses of 0.1–0.8 mg/kg
• Safety follow-up will continue for another 15 years