Can a Single Gene Edit Stop Heart Disease?

Lilly’s Verve deal propels research into lasting genetic fixes for cholesterol

7 Nov 2025

Eli Lilly is making a billion-dollar bet on the future of genetic medicine. The drugmaker’s planned $1.3 billion acquisition of Verve Therapeutics signals how quickly gene editing is moving from lab experiments to mainstream cardiovascular research. The agreement includes about $1 billion upfront, with another $300 million tied to future milestones.

At the center of this deal is VERVE-102, Verve’s experimental base-editing therapy now in Phase 1b trials. The one-time infusion is designed to permanently dial down LDL, or “bad,” cholesterol by altering a single letter of DNA in the PCSK9 gene inside liver cells. The edit switches off a key driver of high cholesterol, potentially replacing the need for lifelong medication.

Early trial data, based on results through March and shared in April, showed LDL drops of more than half for some participants, with a few nearing 70. Scientists caution that the findings are still preliminary and that long-term safety remains unproven, but the results hint at what might be possible with precise, single-dose gene editing.

For Lilly, the Verve acquisition expands its reach into in-vivo gene editing, a fast-evolving area of biotechnology. Known for its work in diabetes and cancer, the company is now betting on genetics to tackle heart disease, still the world’s leading cause of death. Analysts see the move as both bold and strategic, positioning Lilly to compete in one of science’s most promising new arenas.

Verve’s approach stands out for its use of base editing, which rewrites DNA rather than cutting it. That could make the process safer and more controlled than traditional CRISPR methods. But scaling up production and ensuring lasting safety will be complex and costly challenges.

Even so, the Lilly-Verve deal marks a pivotal moment. If the science holds up, the partnership could usher in a new chapter of cardiovascular research, one where preventing heart disease starts not with a pill, but with a precise tweak to the genome itself.