Lilly posts new Heart-2 data for one-time gene-editing therapy

Eli Lilly reported new Phase 1b Heart-2 data showing that a single intravenous dose of VERVE-102, an investigational in vivo base-editing therapy, lowered PCSK9 by up to 88% and LDL cholesterol by up to 62% in adults with heterozygous familial hypercholesterolemia or premature coronary artery disease. Lilly said the effect was dose-dependent and remained durable through follow-up of up to 18 months, with results presented at the 2026 European Atherosclerosis Society Congress and published in The New England Journal of Medicine. VERVE-102 was originally developed by Verve Therapeutics, which Lilly acquired in 2025. (investor.lilly.com)

Why it matters: While this is a human cardiovascular story, it’s notable for veterinary professionals because it reflects how one-time gene-editing approaches are moving from concept toward clinically meaningful biomarker changes in chronic disease. For veterinarians following translational medicine, the Heart-2 data add to evidence that lipid nanoparticle-delivered base editing can produce durable effects in vivo, a platform concept that could eventually influence how the industry thinks about inherited and long-term metabolic disease across species, even though VERVE-102 itself is not a veterinary product. (nejm.org)

What to watch: Lilly said it plans to start a Phase 2 trial of VERVE-102 by the end of 2026. (biopharmadive.com)