Eli Lilly reported early clinical data for its PCSK9 base-editing program VERVE-102, showing the one-time gene-editing therapy reduced LDL cholesterol by up to 62% in a Phase 1 open-label, single-ascending dose study. Lilly said there were no treatment-related serious adverse events in the trial. The results extend Lilly’s strategy after its 2025 acquisition of Verve Therapeutics for $1 billion. VERVE-102 is designed as a base editor that changes DNA letters in the PCSK9 gene, aiming for durable LDL lowering without repeated dosing. Lilly framed the program as a potential route to broader heart disease prevention for patients who struggle with existing therapies, including statins and PCSK9-targeting antibodies. The company’s next steps depend on upcoming development milestones, including additional clinical validation beyond Phase 1. Overall, the data keep attention on gene editing as a near-term modality in cardiovascular disease—where durability, tolerability, and safety signals will be decisive for progression into later-stage studies.
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