Eli Lilly VERVE-102 gene therapy reduces LDL

Eli Lilly and Company announced promising early results from its Phase 1b Heart-2 clinical trial for Verve PCSK9-inhibitor gene therapy, an experimental gene therapy designed to treat hypercholesterolemia. The therapy, which works by permanently turning off the PCSK9 gene in the liver, demonstrated dose-dependent reductions in LDL cholesterol of up to 62% after a single intravenous infusion, with effects lasting up to 18 months. The trial involved 35 adults with heterozygous familial hypercholesterolemia or premature coronary artery disease. Experts like Riyaz S Patel and Ambuj Roy lauded the findings as a significant medical breakthrough, potentially transforming cardiovascular care from chronic management to a one-time treatment. Verve PCSK9-inhibitor gene therapy was well-tolerated with no serious adverse events reported. The United States — Food and Drug Administration has granted Verve PCSK9-inhibitor gene therapy Fast Track designation, and Eli Lilly and Company plans to begin Phase 2 clinical studies by the end of 2026.