One-off gene-editing therapy could permanently lower cholesterol

One-off gene-editing therapy could permanently lower cholesterol

In an early-stage trial, a single dose of a CRISPR treatment lowered cholesterol levels, possibly permanently

Verve’s One-Time Gene Editor Cuts LDL by Nearly 70% in Early Trial

A phase 1b showed promising results in lowering LDL in patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease.

An innovative therapy based on CRISPR genetic publishing technology has enabled, in a preliminary clinical trial, to reduce cholesterol levels by more than half in one infusion. By specifically targeting the PCSK9 gene — a key actor in hypercholesterolemia — this approach consists of removing a single nucleotide base within liver cells. It could allow prolonged cholesterol control, especially in individuals genetically predisposed to cardiovascu…