Gene‑Editing Therapy Cuts LDL 62% in Early Trial

An experimental gene-editing therapy has shown the potential to substantially and durably reduce harmful cholesterol with a single treatment, according to interim results from an early-stage clinical trial. The therapy, VERVE-102, is designed to permanently inactivate a gene in the liver that regulates cholesterol levels.

The findings, published in The New England Journal of Medicine, come from an interim analysis of 35 adults with heterozygous familial hypercholesterolemia or premature coronary artery disease. Participants received a single intravenous infusion of VERVE-102 at varying doses. At the highest dose tested, patients experienced a mean reduction of 62% in low-density lipoprotein (LDL) cholesterol, with corresponding reductions of up to 88% in PCSK9 protein levels in the blood. These reductions were sustained for up to 18 months in follow-up data. Lower doses produced smaller, dose-dependent effects.

The treatment was generally well-tolerated, with no treatment-related serious adverse events or dose-limiting toxicities reported. Some participants experienced mild-to-moderate infusion-related reactions and transient elevations in liver enzymes. One serious adverse...