Gene Editing May Help Treat Dangerously High Cholesterol with Just One Infusion

Gene editing may help people born with dangerously high cholesterol lower their levels with just one infusion.

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.

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