One shot to lower bad cholesterol? An Expert Explains how new gene-editing therapy works

A new experimental therapy from Eli Lilly and Verve Therapeutics may revolutionize cholesterol control with a single shot infusion. This therapy permanently switches off a gene called PCSK9 in the liver, drastically lowering LDL, or "bad cholesterol," by an average of 62%. The treatment is unique as it administers genetic instructions directly to the patient's body via an intravenous infusion, targeting DNA alterations to permanently reduce liver's PCSK9 production.

Professor David E Newby, a co-author of the VERVE-102 phase 1 trial, explained that traditional medications require ongoing administration to maintain the LDL-lowering effect, while VERVE-102 aims for a "one-and-done" approach. The study showed a roughly 60% reduction in LDL cholesterol, with significant reductions in PCSK9 levels and LDL cholesterol.

However, the long-term benefits and risks of this gene-editing therapy remain uncertain, as it is still early in clinical testing. For now, the therapy could potentially be an option for high-risk patients, but further research is needed to confirm its lasting benefits and safety.