Eli Lilly has reported promising early results for its experimental gene-editing treatment VERVE-102, with the therapy significantly lowering “bad” LDL cholesterol levels in patients at high cardiovascular
risk after just a single dose.
The treatment, originally developed by Verve Therapeutics before its acquisition by Lilly, is designed as a one-time gene-editing therapy that permanently switches off the PCSK9 gene in the liver, a gene closely linked to cholesterol regulation. By disabling PCSK9, researchers hope to create long-lasting reductions in LDL cholesterol, potentially reducing the lifetime risk of heart disease.
Up To 62% Reduction In LDL Cholesterol
The findings come from the ongoing Phase 1b Heart-2 clinical trial involving adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease, patients who remain at elevated cardiovascular risk despite existing cholesterol-lowering treatments.
According to Lilly, a single intravenous infusion of VERVE-102 produced dose-dependent reductions in LDL cholesterol levels, with the highest dose cohort recording an average LDL reduction of 62%. The therapy also reduced circulating PCSK9 protein levels by up to 88%, with the effects sustained for up to 18 months during follow-up.
Researchers noted that no treatment-related serious adverse events or dose-limiting toxicities were observed during the trial. Mild infusion-related reactions and fatigue were among the side effects reported.
Why This Matters
Current cholesterol-lowering therapies such as statins, PCSK9 inhibitors and injectable drugs require long-term adherence, something many patients struggle with over time. Scientists behind VERVE-102 believe a one-time gene-editing treatment could potentially change cardiovascular care from lifelong management to durable prevention.
“Many patients with elevated LDL-C struggle to achieve sustained control despite ongoing efforts with the medicines available today,” cardiologist Riyaz S. Patel said while discussing the study findings.
Heart disease remains one of the leading causes of death globally, and high LDL cholesterol is a major contributing factor.
A Major Bet On Gene Editing
Lilly acquired Verve Therapeutics in a deal reportedly valued at nearly $1 billion, signalling growing confidence in gene-editing approaches beyond rare diseases.
The company plans to begin Phase 2 trials of VERVE-102 later this year. However, experts caution that the research is still at an early stage and longer-term studies will be needed to fully understand both safety and durability.
If future trials continue to show positive outcomes, therapies like VERVE-102 could potentially reshape how high cholesterol and heart disease are treated in the future, moving from daily pills and regular injections towards a single long-lasting intervention.
