A one-time gene-editing therapy called VERVE-102 may permanently lower LDL cholesterol by silencing the PCSK9 gene in the liver. In a phase Ib trial involving patients with inherited high cholesterol or early heart disease, a single infusion reduced LDL by up to 62% and PCSK9 levels by up to 88%. Results appeared durable at one year of follow-up. Unlike daily statins or regular injections, this approach aims to be a permanent fix. The therapy uses a lipid nanoparticle delivery system redesigned to improve safety over its predecessor. No dose-limiting toxicities were observed, though mild infusion reactions and temporary liver enzyme elevations occurred. Results were published in the New England Journal of Medicine.
