An experimental gene-editing therapy called VERVE-102 has shown promising results in an early clinical trial, cutting bad cholesterol by up to 62 percent after a single infusion. The interim Phase I data, published in the New England Journal of Medicine, involved just 35 patients but indicated the drug is safe, with no serious adverse events reported.
The therapy, developed by Verve Therapeutics and acquired by Eli Lilly last year for $1.3 billion, uses mRNA-based base editing to permanently disrupt the PCSK9 gene in liver cells. PCSK9 normally hinders the liver's ability to clear LDL cholesterol. By breaking that gene, the drug aims to provide lifelong reduction in heart disease risk.
The highest dose group saw mean LDL drop from high-risk levels to 78 mg/dL-a 62 percent reduction. Researchers estimate that, if sustained for two decades, this could cut cardiovascular risk by half. Follow-up data so far spans up to 18 months, showing sustained effects. The FDA has granted VERVE-102 Fast Track designation.