Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial

Image: Illustration of HDL, LDL, and atherosclerosis in blood vessels. Image credit: Brgfx via Magnific. Article by Beth Mole. Ars Technica – May 28, 2026. Research article: The New England Journal of Medicine.

An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial. Researchers running a Phase I safety trial for the drug, dubbed VERVE-102, published interim results from just 35 patients this week in the New England Journal of Medicine. Though the numbers are small and the analysis is preliminary, VERVE-102 appeared safe, with no serious adverse events reported from the treatment, even at the largest doses. The most significant finding was a temporary, mild increase in a liver enzyme that suggested minor injury in the liver, where the drug works. […]

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