A single-shot gene therapy could slash cholesterol levels by 62%, potentially ‘transforming cardiovascular care’. 

Results from the Heart-2 study into VERVE-102, published in The New England Journal of Medicine, showed reductions of up to 62% in a cohort of 35 participants, with durability observed for up to 18 months following treatment. 

Eli Lilly’s VERVE-102 is an investigational in vivo base editing medicine, designed to durably turn off the PCSK9 gene in the liver. A single infusion can subsequently lower blood low-density lipoprotein cholesterol (LDL-C) in adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD). 

VERVE‑102 was well tolerated across all dose levels with no treatment‑related serious adverse events (AEs) and no dose-limiting toxicities reported. 

Worldwide, CAD remains a leading cause of death, affecting more than 300 million people. HeFH also affects approximately one in 200 to 250 people and is characterised by lifelong elevations in LDL-C, leading to premature cardiovascular disease, including CAD.  

The US Food and Drug Administration (FDA) has granted Fast Track designation for VERVE-102 to reduce LDL-C in participants with hyperlipidaemia and high lifetime cardiovascular risk.  

“Twenty years ago, genetics showed us that people born with PCSK9 naturally turned off have low LDL-C for life and are remarkably protected from heart attack, yet today’s chronic therapies struggle to deliver this lifelong lowering,” said Sekar Kathiresan, Lilly Senior Vice President and Co-Founder of Verve Therapeutics.  

“The Heart-2 results provide early clinical evidence that a single dose of VERVE-102 may mimic the LDL-C lowering effects of PCSK9 cardioprotective variants, potentially transforming cardiovascular care from chronic management to a one-time treatment.” 

Lilly now plans to initiate the Phase II clinical study of VERVE-102 by the end of 2026.