Gene Editing May Help Treat Dangerously High Cholesterol with Just One Infusion
A new gene-editing therapy, VERVE-102, shows promise in treating familial hypercholesterolaemia with just one infusion. Early clinical trial results indicate that this treatment can significantly lower harmful cholesterol levels for at least a year. Experts believe this approach could revolutionize cholesterol management and reduce reliance on daily medications.
- ▪VERVE-102 is a gene-editing therapy developed by Verve Therapeutics, a subsidiary of Eli Lilly.
- ▪The therapy targets the PCSK9 gene in the liver, which regulates harmful cholesterol levels in the bloodstream.
- ▪In a trial of 35 patients, cholesterol levels dropped significantly, with some maintaining lower levels for over a year.
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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.
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Excerpt limited to ~120 words for fair-use compliance. The full article is at Discover Magazine.