Verve Therapeutics’ New Gene Editing Therapy Shows Promise in Lowering Cholesterol, Overcoming Previous Setback

Verve Therapeutics’ experimental gene editing treatment, Verve-102, has demonstrated promising safety and efficacy in a small clinical trial, signaling a potential rebound after a previous safety-related setback. The company is developing a one-time treatment aimed at permanently reducing heart attack risk by editing the PCSK9 liver gene.

Following earlier concerns about potential liver damage and low platelet counts with its initial therapy, linked to the lipid nanoparticle delivery system, Verve pivoted to Verve-102, utilizing a different, potentially safer nanoparticle.

The latest trial results, involving 14 participants with HeFH or premature coronary artery disease, showed no serious treatment-related side effects after 28 days of follow-up. Notably, patients receiving the highest tested dose experienced a 53% reduction in LDL (“bad”) cholesterol and a 60% decrease in PCSK9 protein levels.

Analysts, including William Blair’s Myles Minter and Jefferies’ Roger Song, viewed the data as a significant positive, suggesting Verve has addressed prior safety concerns and demonstrated “best-in-class potential” compared to existing cholesterol-lowering therapies. Verve aims to compete with established PCSK9 inhibitors like Amgen’s and Regeneron’s antibodies and Novartis’ RNA-based therapy, Leqvio. CEO Sek Kathiresan argued that the high discontinuation rates of these treatments highlight the need for a durable, one-time solution.

The company is currently enrolling patients in a higher-dose cohort and plans to initiate a Phase 2 trial in the U.S. later this year, following FDA clearance. Verve reported sufficient cash reserves to fund operations through mid-2027.Eli Lilly, collaborating with Verve on Verve-102, expressed strong interest in the data and will decide by late 2025 whether to co-develop the therapy.