Gene-Editing Therapy Cuts LDL by 62% in 35-Patient Heart Disease Study
Updated
Updated · The New York Times · May 25
Gene-Editing Therapy Cuts LDL by 62% in 35-Patient Heart Disease Study
4 articles · Updated · The New York Times · May 25
A single infusion of an experimental gene-editing treatment cut LDL cholesterol by as much as 62% in 35 patients with genetically high cholesterol or heart disease, according to an interim trial analysis.
The effect appears durable: in a subgroup treated 18 months ago, the LDL reduction has been sustained, raising the prospect of a one-time preventive therapy rather than repeated lifelong treatment.
The study, published in The New England Journal of Medicine, is unusually preliminary for the journal; editor in chief Dr. Eric Rubin said it appears to work well and targets the leading U.S. cause of death.
Researchers plan to expand the trial to as many as 85 participants and then move to a larger 200-patient study, testing whether the approach can help prevent cardiovascular disease, which kills nearly 800,000 Americans a year.
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Recent clinical trials are transforming cardiovascular care with innovative gene-editing therapies like VERVE-102. After receiving FDA clearance in March 2025, VERVE-102 advanced based on promising interim data from the Heart-2 trial, which tested different doses to evaluate changes in LDL cholesterol and PCSK9 protein levels. The highest doses of therapies targeting ANGPTL3, the same protein VERVE-102 aims to inhibit, led to strong reductions in ANGPTL3 protein and significant improvements in blood lipids within 60 days. Notably, LDL cholesterol dropped by nearly 49% and triglycerides by over 55%, showing the powerful potential of these new treatments.