Eli Lilly Tests One-Time Gene-Editing Infusion for High Cholesterol

Drugmaker Eli Lilly is testing an experimental gene-editing therapy called VERVE-102 for high cholesterol, according to a company news release issued this week. The therapy is designed to switch off the PCSK9 gene in the liver, which produces a protein that regulates cholesterol levels.

Researchers said the treatment could be given as a one-time infusion lasting about four hours rather than daily pills or repeated injections.

In an initial study of 35 participants, VERVE-102 lowered LDL cholesterol by as much as 62 percent after one infusion. More than one-third of those patients maintained lower cholesterol levels for up to 18 months. The study did not assess whether the treatment reduces heart attacks or strokes. The Food and Drug Administration previously granted the study fast-track status.

Lilly stated it plans to begin a larger Phase 2 study by the end of 2026. Nearly 25 million Americans have high cholesterol, the Centers for Disease Control and Prevention reported, and the condition is a major risk factor for heart disease, the leading cause of death in the United States.