Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing Therapies


The cofounder and chief executive officer of Verve Therapeutics discussed the company’s pipeline.

“We have a second investigational medicine called VERVE-102 that also targets PCSK9 but differs in the delivery vehicle, in the lipid nanoparticle formulation. In this second product VERVE-102, we're using a ligand called GalNAc that targets this lipid nanoparticle even more specifically to the liver. And so that's the main difference.”

Verve Therapeutics has dosed the first patient with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) in the phase 1b in Heart-2 clinical trial (NCT06164730) with VERVE-102, an investigational in vivo base editing therapy.1 Heart-2 is an open-label, single-ascending dose study that is recruiting adult patients in 4 cohorts who need additional lowering of low-density lipoprotein C (LDL-C) and will assess outcomes by measuring pharmacokinetics and changes in levels of the PCSK9 protein and LDL-C in the blood.

VERVE-102 uses a different lipid nanoparticle (LNP) delivery system than the company’s former lead candidate VERVE-101 for the same indication. Verve announced in April 2024 that it was pausing enrollment in its phase 1b heart-1 clinical trial (NCT05398029) of VERVE-101 in consultation with the study’s independent data and safety monitoring board (DSMB) after the sixth participant treated experienced asymptomatic grade 3 adverse events.2

CGTLive® spoke with Sekar Kethiresan, MD, cofounder and chief executive officer of Verve Therapeutics, to learn more about VERVE-102 and the company’s gene editing strategy to help address unmet needs in the field of cardiovascular disease. He also discussed VERVE-101 as well as a next-generation candidate VERVE-201 which the company is aiming to begin a clinical trial for by the second half of 2024.

1. Verve Therapeutics Announces Dosing of First Patient in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102. News release. Verve Therapeutics. May 7, 2024. Accessed May 14, 2024.
2. Verve therapeutics announces updates on its PCSK9 program. Verve Therapeutics. April 2, 2024. Accessed May 14, 2024.
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