Eli Lilly reported new Phase 1b data showing that a single dose of its investigational in vivo gene-editing therapy VERVE-102 produced durable reductions in LDL cholesterol and PCSK9 protein levels in patients with high cardiovascular risk, highlighting growing momentum behind one-time genetic medicines for cardiometabolic disease.1
The data, presented from the ongoing Heart-2 trial, demonstrated dose-dependent LDL cholesterol (LDL-C) reductions of up to 62% and PCSK9 reductions of up to 88% following a single infusion of the therapy in adults with heterozygous familial hypercholesterolemia or premature coronary artery disease.2 The findings position VERVE-102 as one of the most advanced in vivo base-editing programs currently being evaluated for cardiovascular disease.
The therapy originated at Verve Therapeutics, which Lilly acquired in 2025 as part of its broader expansion into genetic medicines and cardiovascular disease innovation.3
How does VERVE-102 work to lower LDL cholesterol?
VERVE-102 is an investigational base-editing therapy designed to permanently inactivate the PCSK9 gene in the liver after a single intravenous infusion.2 The therapy uses messenger RNA encoding an adenine base editor alongside a guide RNA directed against PCSK9, both delivered using a lipid nanoparticle platform.
PCSK9 has become a major therapeutic target in cardiovascular medicine because the protein regulates LDL receptor degradation, influencing circulating LDL cholesterol levels. Existing PCSK9 inhibitors have demonstrated significant LDL-lowering efficacy, but they require chronic administration. VERVE-102 aims to provide lifelong LDL reduction through a one-time treatment approach.2
The company is preparing to initiate Phase 2 development following completion of the dose-escalation portion of the Heart-2 trial.
According to Lilly, reductions in LDL-C and PCSK9 observed during the Heart-2 trial remained durable for up to 18 months in some participants.2
Why is in vivo gene editing attracting attention in cardiovascular disease?
The cardiovascular gene-editing field has drawn increasing industry interest because of the potential to address treatment adherence challenges associated with chronic lipid-lowering therapies. Verve Therapeutics previously noted that many patients discontinue LDL-lowering medications within the first year of treatment, potentially limiting long-term cardiovascular risk reduction.4
Unlike ex vivo gene-editing approaches used in oncology, in vivo therapies are administered directly into the body and are designed to edit target genes inside patient tissues. Developers believe the strategy could eventually support broader use across common chronic diseases such as atherosclerotic cardiovascular disease.
Lilly stated that VERVE-102 was generally well tolerated in the ongoing study, with no treatment-related serious adverse events or clinically significant laboratory abnormalities reported in interim analyses.2 The company is preparing to initiate Phase 2 development following completion of the dose-escalation portion of the Heart-2 trial.5
Could one-time cholesterol gene editing reshape long-term cardiovascular treatment?
Although the program remains in early-stage development, the data reflect growing confidence that gene editing could eventually shift portions of cardiovascular disease management away from lifelong maintenance therapies toward durable single-course interventions.
The competitive landscape for cardiovascular genetic medicines has expanded rapidly in recent years as pharmaceutical companies pursue therapies targeting PCSK9, ANGPTL3, and lipoprotein(a) pathways associated with inherited and acquired cardiovascular risk.3
Analysts and researchers have suggested that durable LDL-lowering approaches may become particularly important in patients with familial hypercholesterolemia or persistently elevated cardiovascular risk despite currently available therapies.
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