An experimental gene-editing treatment dramatically lowered cholesterol levels — maybe permanently — after one infusion, a preliminary study found.
The study, published May 25 in The New England Journal of Medicine, gave 35 patients six doses of VERVE-102, an investigational base-editing therapy that deactivates proprotein convertase subtilisin-kexin type 9 in the liver. People with loss-of-function variants of PCSK9 have reduced levels of LDL cholesterol and fewer atherosclerotic cardiovascular disease events.
A single infusion of the highest dose reduced LDL cholesterol levels by up to 62%, and the change has lasted for patients who were treated 18 months ago.
This preliminary study will be followed by a larger study of 200 patients, The New York Times reported May 25.
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