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

Commentary
Video

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.

REFERENCES
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. https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-dosing-first-patient-heart-2-phase
2. Verve therapeutics announces updates on its PCSK9 program. Verve Therapeutics. April 2, 2024. Accessed May 14, 2024. https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-updates-its-pcsk9-program.

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Recent Videos
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira, and Kilian Guse, PhD, the vice president of genetic medicine platforms at Pacira
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira
Jeffrey Chamberlain, PhD
Tami John, MD
Tami John, MD
Tami John, MD
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.