Lilly shares data update on base editing gene therapy from Verve buyout

Eli Lilly announced positive phase 1b study results for VERVE-102, a potential first-in-class in vivo gene editing medicine targeting PCSK9, a gene linked to cholesterol levels and cardiovascular health.

In the Heart-2 trial evaluating VERVE-102 in adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD), a single intravenous infusion of VERVE-102 resulted in meaningful lowering of circulating PCSK9 protein and corresponding reductions in LDL-C across all evaluated dose levels. In this interim analysis of 35 participants, a single dose of VERVE‑102 resulted in dose-dependent mean reductions in PCSK9 ranging from 51% to 88%, at the lowest 0.3 mg/kg dose to the highest 1.0 mg/kg dose, respectively. These reductions were sustained over time, with durability observed for up to 18 months following treatment.

These data were presented as a late-breaking oral presentation at the European Atherosclerosis Society Congress and simultaneously published in The New England Journal of Medicine.

Through Lilly’s acquisition of Beam Therapeutics in October 2023, the drugmaker picked up certain product rights to Verve’s cardiovascular in vivo gene editing programs targeting PCSK9 and ANGPTL3, which included its lead program, VERVE-102. Lilly then purchased Verve in a deal worth approximately $1.3 billion in June 2025.

Lilly says it plans to begin enrolling the phase 2 clinical study of VERVE-102 by the end of this year.