Verve Hits The Clinic With Gene-Edited Cholesterol-Lowering Drug

Verve Therapeutics, Inc. has reached the clinic earlier than anticipated with its base-editing medicine for lowering cholesterol, bringing it into the growing group of PCSK9-targeting therapies looking to succeed where marketed drugs from Amgen, Inc. and Sanofi/Regeneron Pharmaceuticals, Inc. have largely failed.

The company said 12 July that it dosed the first patient in the Phase Ib heart-1 clinical trial of VERVE-101, a gene-editing drug designed to permanently turn off the PCSK9 gene in the liver and thus reduce LDL cholesterol in patients with heterozygous familial hypercholesterolemia (HeFH)