Cleveland Clinic cardiologist Luke Laffin led a first-in-human Phase 1 trial testing CTX310, an experimental CRISPR-Cas9 gene-editing therapy from CRISPR Therapeutics, in patients with lipid disorders that had not responded to standard medication.
A single one-time infusion of CTX310 switches off the ANGPTL3 gene in the liver. Among the 15 participants, those who received the highest dose showed a 52.5% reduction in LDL cholesterol and a 47.8% drop in triglycerides after 12 months, with no serious adverse events reported during follow-up.
The results, presented at the European Society of Cardiology's 2026 congress and published in the New England Journal of Medicine, suggest a single treatment could replace a lifetime of daily cholesterol-lowering pills for people with hard-to-treat lipid disorders linked to heart attacks and stroke. Researchers plan to monitor participants for 15 years to confirm the therapy's long-term safety.