For people born with familial hypercholesterolaemia, an inherited condition that causes dangerously high cholesterol from birth, managing it isn’t a lifestyle choice. It’s a daily obligation: pills every morning, injections every few weeks, a regimen that begins young and, for most, never ends. A new experimental therapy aims to change that with one infusion.
Results from an early clinical trial, published in the New England Journal of Medicine, show that one dose of VERVE-102, a gene-editing therapy developed by Verve Therapeutics, a subsidiary of Eli Lilly, can lower harmful cholesterol in patients with the inherited condition or early-onset heart disease. In the small group of patients tracked longest, those lower levels stayed down for at least a year.