Indianapolis, Indiana, USA, May 25, 2026
Eli Lilly and Company has announced positive Phase 1b clinical results for VERVE-102, an investigational in vivo base-editing therapy designed to permanently turn off the PCSK9 gene in the liver and significantly reduce low-density lipoprotein cholesterol (LDL-C) levels with a single treatment. The findings from the ongoing Heart-2 trial demonstrated that a one-time intravenous infusion of VERVE-102 produced dose-dependent reductions in both circulating PCSK9 protein and LDL cholesterol levels, with durable effects sustained for up to 18 months following treatment.
The data were presented as a late-breaking oral presentation during the European Atherosclerosis Society (EAS) Congress and simultaneously published in The New England Journal of Medicine, highlighting growing scientific interest in next-generation gene-editing approaches for cardiovascular disease prevention. Researchers believe the therapy may represent a major shift in the future treatment of hypercholesterolemia and coronary artery disease by potentially replacing chronic lipid-lowering therapies with a one-time genetic intervention.
VERVE-102 Demonstrates Strong Cholesterol Reduction
The Heart-2 Phase 1b study is evaluating VERVE-102 in adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease who require additional LDL cholesterol lowering despite maximally tolerated lipid-lowering therapies. According to Lilly, the interim analysis included 35 participants treated across six dose cohorts ranging from 0.3 mg/kg to 1.0 mg/kg.
The results showed meaningful dose-dependent reductions in PCSK9 protein levels ranging from 51% to 88%, while corresponding LDL-C reductions ranged from 9% to 62% depending on dose level. The highest dose cohort achieved a mean LDL-C reduction of 62%, with reductions remaining durable throughout the follow-up period. Approximately 15 participants have now been followed for at least one year, with durability observed for up to 18 months after treatment.
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Researchers stated that the therapy was generally well tolerated across all evaluated dose levels, with no treatment-related serious adverse events or dose-limiting toxicities reported. The most commonly observed treatment-related adverse events included mild infusion-related reactions and fatigue. All participants completed the full planned infusion dose without study withdrawal.
VERVE-102 was specifically designed to mimic naturally occurring loss-of-function variants in the PCSK9 gene, which are associated with lifelong low LDL cholesterol levels and significantly reduced cardiovascular disease risk. Scientists believe the therapy may provide a durable and potentially transformative treatment option for patients struggling to achieve long-term cholesterol control using currently available therapies.
Gene Editing Advances Cardiovascular Treatment Innovation
VERVE-102 is an investigational in vivo base-editing medicine consisting of messenger RNA encoding an adenine base editor along with a guide RNA targeting the PCSK9 gene. Both components are delivered through a proprietary lipid nanoparticle platform designed to selectively reach liver cells following a single intravenous infusion.
Lilly stated that the therapy utilizes Verve Therapeutics’ proprietary GalNAc-LNP delivery technology, enabling the treatment to access liver cells through both LDL receptors and asialoglycoprotein receptors. By permanently modifying the PCSK9 gene inside liver cells, researchers aim to create long-term suppression of LDL cholesterol production without requiring continuous medication administration.
The U.S. Food and Drug Administration has already granted Fast Track designation to VERVE-102 for reducing LDL cholesterol in patients with hyperlipidemia and elevated lifetime cardiovascular risk. Familial hypercholesterolemia affects approximately 1 in every 200 to 250 individuals globally, while coronary artery disease remains one of the leading causes of death worldwide.
Lilly Expands Long-Term Genetic Medicine Strategy
Lilly confirmed plans to initiate enrollment for a Phase 2 clinical study of VERVE-102 later this year as part of its broader cardiovascular genetic medicine strategy. Industry analysts believe the program could represent one of the most important developments in gene-editing-based cardiovascular therapy, particularly as healthcare systems seek longer-lasting treatment solutions for chronic diseases.
The company also continues evaluating additional gene-editing programs including VERVE-201, which targets the ANGPTL3 gene for lipid lowering and cardiovascular risk reduction. Experts believe the success of VERVE-102 could accelerate wider adoption of in vivo base-editing technologies across cardiovascular medicine and other chronic disease areas.
As biotechnology companies continue advancing precision genetic therapies, Lilly’s latest clinical milestone highlights the growing potential of one-time gene-editing treatments capable of fundamentally changing the long-term management of cardiovascular disease worldwide.
Source: Eli Lilly press release
