A Cleveland Clinic Phase 1 clinical trial has shown that a one-time infusion of CRISPR-Cas9 gene-editing therapy CTX310 was effective and safe in reducing LDL cholesterol and triglycerides in people with medication-resistant lipid disorders through 12 months of follow-up. The trial included 15 patients, and those who received the highest dose saw LDL cholesterol reduced by 52.5% from baseline and triglycerides reduced by 47.8% -- effects that persisted for a full year after a single treatment. There were no serious adverse events related to the therapy. The results were presented at the 2026 European Society of Cardiology annual meeting and simultaneously published in the New England Journal of Medicine. CTX310 works by targeting ANGPTL3, a gene in the liver that regulates lipid metabolism. By switching off this gene, the therapy reduces both LDL cholesterol and triglycerides -- both major risk factors for cardiovascular disease. Cardiologist Luke Laffin, the study's first author, called the durability of the effect 'impressive' and noted that additional long-term safety follow-up is planned for the next 15 years as recommended by the FDA.
One-Time CRISPR Injection Slashes Cholesterol by 52% for a Full Year in First-In-Human Trial
Cleveland Clinic reported that a single infusion of CRISPR-Cas9 gene-editing therapy CTX310 reduced LDL cholesterol by 52.5% and triglycerides by 47.8% in 15 patients after 12 months, with no serious adverse events. Results were presented at the European Society of Cardiology meeting.
Key Takeaways
- Cleveland Clinic reported that a single infusion of CRISPR-Cas9 gene-editing therapy CTX310 reduced LDL cholesterol by 52.5% and triglycerides by 47.8% in 15 patients after 12 months, with no serious adverse events.
- Results were presented at the European Society of Cardiology meeting.
AI synthesis4 sources3
