One Infusion Switched off a Cholesterol-Raising Gene in Monkeys for Three Years

A single infusion of a CRISPR gene editor switched off the gene ANGPTL3 in the livers of monkeys and cut the protein that gene makes in their blood by more than 90% on average. Verve Therapeutics, the company developing the treatment, reports in Nature that the reduction was confirmed out to three years after that one dose.

ANGPTL3 drives up LDL cholesterol and triglycerides, two leading risk factors for the artery disease behind most heart attacks. The authors write that most patients never reach their target lipid levels on the drugs approved today, which are taken continuously. They describe their own approach as a potential single-course treatment.

The paper reports that in mouse and nonhuman primate models of high blood lipids, switching off the gene was associated with reductions of about 40 to 50% in LDL cholesterol and 50 to 70% in triglycerides. In human liver cells, the editor made the intended single-letter change in the DNA, with no off-target editing detected at any of about 3,000 candidate sites. Verve's researchers also report that the gene inactivation was well tolerated in the monkeys.

The infusion carried the guide RNA plus mRNA coding for the editor, packed inside a lipid nanoparticle tagged with a sugar that steers it to liver cells. Taiji Mizoguchi, Amit V. Khera and colleagues at Verve Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and Company, ran the work. Co-authors are at the Icahn School of Medicine at Mount Sinai and the University of Pennsylvania. The authors tested an editor their own company is developing.

Nature released this peer-reviewed, accepted version on Nov. 6, 2026, ahead of the final edited one. The authors write that these results were the scientific foundation for starting a first-in-human trial in patients whose high blood lipids resist existing treatment. That trial is under way, and the paper carries no results from it.