Verve Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing in vivo gene-editing therapies for cardiovascular disease. Its lead candidate, VERVE-102, is an adenine base editor delivered via lipid nanoparticle that targets the PCSK9 gene in the liver to achieve durable reductions in PCSK9 protein and LDL cholesterol following a single intravenous infusion.
The company is evaluating VERVE-102 in the Phase 1b Heart-2 trial in patients with heterozygous familial hypercholesterolemia or premature coronary artery disease who require additional LDL-C lowering. Interim data showed dose-dependent PCSK9 reductions of 51–88% and LDL-C reductions up to 62%, with effects persisting for up to 18 months and a favorable safety profile consisting primarily of mild infusion-related reactions.
Verve Therapeutics has received FDA Fast Track designation for VERVE-102 and plans to advance into Phase 2. A second program, VERVE-201 targeting ANGPTL3, is also in early clinical development.
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Phase 1b Heart-2: single IV base-editing dose gave dose-dependent, durable PCSK9/LDL-C reductions up to 18 months in HeFH/premature CAD; well tolerated; FDA Fast Track; Phase 2 planned.
