Verve Therapeutics has reported promising early clinical results from its Phase 1b HEART-2 trial of VERVE-102, a groundbreaking in vivo gene-editing therapy designed to permanently lower LDL cholesterol in patients with heterozygous familial hypercholesterolemia (HeFH).
The company’s approach leverages base editing technology to make a single, permanent change in the PCSK9 gene within liver cells, aiming to reduce the body’s production of PCSK9 protein—a key regulator of LDL cholesterol. By switching off this gene, VERVE-102 can significantly lower cholesterol levels with just one infusion.
In this first-in-human trial, Verve tested escalating single doses of VERVE-102 in individuals with HeFH, a genetic condition that causes abnormally high LDL cholesterol and significantly increases the risk of premature cardiovascular disease. The most notable results came from the highest dose group (0.6 mg/kg), which experienced a 55% reduction in LDL cholesterol and an 84% drop in PCSK9 protein levels—both achieved with just a single treatment.
The safety profile of VERVE-102 has also been encouraging. Across all dosing levels, the therapy was well tolerated, with no treatment-related serious adverse events or dose-limiting toxicities reported. Some participants experienced mild, transient side effects, but nothing that required clinical intervention.
“These early results are a major step toward a functional cure for familial hypercholesterolemia,” said executives from Verve. “The potential of a one-time gene editing therapy to permanently lower LDL cholesterol could dramatically change the landscape of cardiovascular care.”
Verve plans to advance VERVE-102 into a Phase 2 trial in 2025, with larger cohorts and longer follow-up periods to further evaluate durability and long-term safety. Full data from the HEART-2 trial are expected by 2027.
This news follows a growing trend in the biotech space toward in vivo gene editing therapies aimed at addressing common yet serious chronic conditions like cardiovascular disease. If successful, VERVE-102 could become the first one-time gene editing solution for high cholesterol—replacing lifelong regimens of statins, injectables, or other lipid-lowering treatments.
Source – Biopharma
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