Verve Therapeutics Expands Base Editing Trial for Cholesterol Treatment to US

Verve Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to expand its gene editing trial for a novel cholesterol and heart disease treatment into the United States. The company's investigational therapy, VERVE-102, aims to provide a one-time treatment for individuals with genetically elevated cholesterol levels.

VERVE-102: A Groundbreaking Approach to Cholesterol Management

VERVE-102 utilizes base editing technology to alter a single nucleotide in the PCSK9 gene, which plays a crucial role in regulating liver cells' uptake of cholesterol from the blood. This innovative approach differs from existing PCSK9-targeting therapies, such as antibody drugs and RNA medicines, which require regular injections ranging from every other week to every six months.

Sekar Kathiresan, CEO of Verve Therapeutics, emphasized the potential impact of their treatment: "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."

Ongoing Clinical Trials and Future Plans

Verve is currently conducting a Phase 1b trial, dubbed Heart-2, in Australia, New Zealand, Canada, and the United Kingdom. With the FDA's recent clearance, the company can now expand this trial to include U.S. participants. Initial safety and efficacy data from the Heart-2 trial are expected to be announced before the end of June 2025.

The company has ambitious plans for VERVE-102, including:

1. Publishing dose escalation data later in the year
2. Initiating a Phase 2 clinical trial
3. Potentially collaborating with Eli Lilly, which has the option to share development costs and profits for VERVE-102 after reviewing data from the Heart-2 trial

Addressing Previous Setbacks and Market Implications

VERVE-102 is a successor to an earlier treatment, VERVE-101, which faced challenges in clinical trials. Verve paused the VERVE-101 trial in 2024 due to a study participant experiencing concerning liver enzyme increases and thrombocytopenia. The company suggested that the lipid nanoparticle used for delivery in VERVE-101 may have been a contributing factor.

In response, VERVE-102 employs a different lipid nanoparticle for delivery into the body, while maintaining similar mechanics for targeting the PCSK9 gene.

The news of FDA clearance for VERVE-102's U.S. trial expansion has positively impacted Verve Therapeutics' stock, with shares rising by up to 8% following the announcement. This development comes as a welcome boost for the company, which recently experienced a setback when Vertex Pharmaceuticals ended a partnership with Verve in February 2025.

As Verve Therapeutics continues to advance its gene editing therapies, including two other base editing treatments in various stages of development, the pharmaceutical industry will be closely watching the progress of VERVE-102 and its potential to revolutionize cholesterol management for patients with genetic predispositions to high cholesterol levels.