The Food and Drug Administration has authorized Verve Therapeutics to expand its clinical trial for a novel gene editing treatment targeting high cholesterol into the United States, the biotechnology company announced Monday.
The experimental therapy, VERVE-102, employs base editing technology to modify a single nucleotide in the PCSK9 gene, which plays a crucial role in how liver cells process blood cholesterol. This approach aims to provide a one-time treatment that could permanently lower cholesterol levels in patients with heterozygous familial
hypercholesterolemia or premature coronary artery disease.
Currently approved PCSK9-targeting treatments, including Amgen’s Repatha which generated over $2.2 billion in 2024 sales, require regular administration ranging from biweekly to twice yearly injections. VERVE-102 represents a potential paradigm shift by offering lasting cholesterol reduction through a single course of treatment.
“Current cholesterol medications can effectively lower LDL-C at individual time points, but patient adherence remains problematic,” explained Sekar Kathiresan, Verve’s CEO. “Our therapeutic approach aims to deliver sustained cholesterol reduction through a one-time treatment, potentially leading to more meaningful clinical outcomes.”
The company has been conducting its Phase 1b Heart-2 trial in multiple countries including Australia, New Zealand, Canada, and the United Kingdom. Initial safety and efficacy results are expected before the end of June, with dose escalation data and Phase 2 trial initiation planned for later this year.
VERVE-102 succeeds the company’s earlier candidate, VERVE-101, which faced setbacks when a trial participant experienced concerning liver enzyme elevations and reduced blood platelet counts. While both treatments utilize similar gene-editing mechanisms, VERVE-102 employs a different lipid nanoparticle delivery system. The company had previously suggested that the delivery vehicle used in VERVE-101 may have contributed to the adverse events.
The development program has attracted significant pharmaceutical industry interest, with Eli Lilly maintaining an option to share development costs and profits for VERVE-102 following Heart-2 trial data review. However, the company recently experienced a setback when Vertex Pharmaceuticals terminated their research collaboration in February.
The FDA clearance news triggered a positive market response, with Verve’s shares rising up to 8% Monday morning, helping to offset some losses following the Vertex partnership dissolution announcement.
Verve’s pipeline includes two additional base editing therapies, with one already advancing into Phase 1 clinical testing. The company’s approach represents part of a broader movement in genetic medicine, where researchers are exploring ways to make precise DNA modifications to treat various diseases.
The expansion of the VERVE-102 trial into the U.S. market marks a significant milestone for the company and the field of genetic medicine, potentially offering a new therapeutic option for patients with inherited high cholesterol conditions. The company’s focus on cardiovascular disease through genetic intervention could provide an alternative to traditional treatment approaches that require ongoing medication adherence.
This development comes at a time when gene editing technologies are increasingly being recognized as potential solutions for chronic diseases, with several companies advancing similar therapeutic strategies through clinical development. Success in this trial could help validate base editing as a viable approach for treating common cardiovascular conditions affecting millions of patients worldwide.