Gene therapy VERVE-102 cuts bad cholesterol by 62%. A one-time infusion that permanently lowers “bad cholesterol” may be on the horizon. US drugmaker Eli Lilly and its subsidiary Verve Therapeutics reported that their gene therapy VERVE-102 reduced LDL cholesterol by about 62% in an early trial, with effects lasting 18 months.
LDL, or low-density lipoprotein, is called “bad cholesterol” because it sticks to artery walls and forms plaque. That narrows arteries and increases the risk of heart attacks and strokes. Current treatments — statins, ezetimibe, and PCSK9 antibody injections — require daily pills or frequent shots.VERVE-102 works differently. It’s a form of gene editing delivered via a single IV infusion. It targets the PCSK9 gene in liver cells and permanently reduces the liver’s ability to produce PCSK9, a protein that regulates LDL.
In the phase 1 trial, patients saw an average 60% drop in LDL after one dose. The reduction was especially strong in those with familial hypercholesterolemia, a genetic condition causing very high cholesterol.
“This shows that a concept once considered futuristic — one-time genetic treatment for lifelong cholesterol control — has begun to enter human clinical testing,” said Prof David E Newby of the University of Edinburgh.Experts are cautious. The trial was small and follow-up was less than 2 years. It’s unclear if the effect will last decades. There are also questions about long-term safety, since gene editing is irreversible.
Dr Newby noted that while lowering LDL is proven to reduce cardiovascular risk, VERVE-102 itself has not yet been shown to prevent heart attacks. Larger trials are needed.
The therapy could be most useful for patients with very high cardiovascular risk or those who struggle with daily medication. But cost and access will be hurdles. Gene therapies are expensive and require specialized delivery.
If successful, VERVE-102 would mark a shift from managing cholesterol daily to correcting it at the source. For millions at risk of heart disease, that could mean fewer pills — and fewer heart attacks.