CRISPR Gene Therapy Cuts Cholesterol by 50% in Single Treatment

In a groundbreaking medical breakthrough, researchers have successfully tested CTX310™, an investigational CRISPR-Cas9 gene-editing therapy that achieved unprecedented reductions in cholesterol and triglyceride levels through a single treatment. The Phase 1 first-in-human trial, presented at the American Heart Association's Scientific Sessions 2025, demonstrated that a one-time infusion of CTX310 reduced LDL cholesterol by nearly 50% and triglycerides by approximately 55% in patients with difficult-to-treat lipid disorders. The therapy works by using tiny fat-based particles to deliver CRISPR editing technology to the liver, where it permanently switches off the ANGPTL3 gene—a genetic approach inspired by people born with natural mutations that provide lifelong protection against high cholesterol and cardiovascular disease.

The trial involved 15 adult participants across six sites in Australia, New Zealand, and the United Kingdom, all of whom had elevated lipid levels despite maximum tolerated conventional therapies. Remarkably, cholesterol and triglyceride reductions began within two weeks of treatment and remained sustained for at least 60 days of follow-up, with the highest dose achieving up to 60% reductions in both measures. Lead study author Dr. Luke J. Laffin from the Cleveland Clinic described the results as "unprecedented," noting that this marks the first therapy to simultaneously achieve substantial reductions in both LDL cholesterol and triglycerides—a critical advancement for patients with mixed lipid disorders who typically struggle with elevations in both blood fats.

Safety monitoring revealed only minor infusion-related reactions in three participants, such as back pain and nausea that resolved with medication, and one temporary liver enzyme elevation that normalized without intervention. While these early results are promising, researchers emphasize the need for larger Phase 2 studies with more diverse populations to confirm the findings. The American Heart Association's ongoing Lower Your LDL Cholesterol Now™ Initiative highlights the critical importance of cholesterol management, particularly given that high cholesterol affects approximately 86.4 million U.S. adults and remains a leading risk factor for heart disease and stroke worldwide. Future studies are planned to begin in late 2025 or early 2026, with participants undergoing long-term safety monitoring for up to 15 years as recommended by the FDA for all CRISPR-based therapies.