Verve Therapeutics Expands Gene Editing Trial for Cholesterol Treatment to the U.S.

Verve Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to expand its ongoing clinical trial of VERVE-102, an experimental gene editing therapy aimed at lowering cholesterol and reducing the risk of heart disease, the company announced on Monday.

VERVE-102 targets the PCSK9 gene, which is responsible for producing a protein that regulates the liver’s ability to remove cholesterol from the bloodstream. The therapy works by making a precise change to a single nucleotide, or base, within this gene, with the goal of permanently reducing PCSK9 activity.

Currently, there are approved antibody drugs and an RNA-based medicine in the U.S. that target PCSK9 to lower cholesterol levels. These medications, such as Amgen’s blockbuster drug Repatha, which generated over $2.2 billion in sales in 2024, require regular injections. In contrast, VERVE-102 offers the potential for a lifelong reduction in cholesterol levels following a single course of treatment by effectively “turning off” the PCSK9 gene in the liver. Verve is currently evaluating the therapy in individuals with heterozygous familial hypercholesterolemia, an inherited condition characterized by persistently high cholesterol, or premature coronary artery disease.

Sekar Kathiresan, CEO of Verve Therapeutics, emphasized the need for a more durable solution, stating, “There are multiple cholesterol lowering medicines currently available that can lower LDL-C at a single time point; however, time on treatment for these medicines remains low. To address this unmet need, Verve’s medicines are designed to deliver lifelong cholesterol lowering after a single course of treatment and, consequently, drive more meaningful efficacy.”

Verve has been conducting a Phase 1b trial of VERVE-102, known as Heart-2, in Australia, New Zealand, Canada, and the U.K. The company anticipates announcing initial safety and efficacy data from this international trial before the end of June. Later in 2025, Verve plans to release dose escalation data and initiate a Phase 2 clinical trial for VERVE-102.

VERVE-102 is the successor to an earlier Verve therapy, VERVE-101. The trial for VERVE-101 was paused last year after a participant experienced an increase in liver enzymes and a decrease in platelet counts. While both therapies target the PCSK9 gene through similar mechanisms, VERVE-102 utilizes a different lipid nanoparticle for delivering the gene editing components into the body. At the time of the VERVE-101 pause, Verve suggested that the lipid nanoparticle used in that earlier treatment may have been a contributing factor to the observed issues. Verve also has other base editing therapies in its pipeline, with one currently in Phase 1 testing. The company has a partnership with Eli Lilly, which retains the option to participate in the development and profit-sharing for VERVE-102 following a review of the data from the Heart-2 trial.

Following the FDA clearance announcement, shares in Verve Therapeutics experienced an increase of as much as 8% on Monday morning, partially recovering from a recent decline that occurred after Vertex Pharmaceuticals ended its partnership with the company in February.

https://www.biopharmadive.com/news/verve-us-base-editing-study-ind-fda-heart-2/743338/