Deepak L. Bhatt, MD, MPH, MBA, on Adopting Novel Therapies into Cardiovascular Medicine

Commentary
Video

The director of the Mount Sinai Fuster Heart Hospital discussed trends in research and challenges with adopting new therapies.

“I think the advances in gene editing in other fields other than cardiovascular medicine, per se, will help pave the road for gene editing for cardiovascular indication. So I think it actually helps the cause that there are therapies that are getting approved and other disease states… in this case, time is an ally, with respect to the developments going on for gene editing, in cardiovascular medicine.”

Gene therapy research is expanding to fields such as cardiology, in which gene editing therapy recently made a splash at the American Heart Association’s (AHA) Scientific Sessions 2023 in November. The meeting featured promising data from the phase 1b heart-1 clinical trial (NCT05398029) evaluating Verve Therapeutics’ VERVE-101, an investigational gene-editing therapy intended to treat heterozygous familial hypercholesterolemia (HeFH). Investigators observed dose-dependent decreases in blood PCSK9 and blood LDL-C percentages from baseline with a manageable safety profile and continued dosing at that time.1

Verve has since paused enrollment in heart-1 after the sixth participant treated experienced asymptomatic grade 3 adverse events, in consultation with the study’s independent data and safety monitoring board (DSMB). The company is now prioritizing VERVE-102. its second generation preclinical therapy, for the same indication.2

CGTLive® spoke with Deepak L. Bhatt, MD, MPH, MBA, Director, Mount Sinai Fuster Heart Hospital and Dr. Valentin Fuster Professor of Cardiovascular Medicine, Icahn School of Medicine at Mount Sinai, to learn more about research for novel therapies and gene editing therapies in cardiovascular medicine.

REFERENCES
1. Vafai SB, Gladding PA, Scott R, et al. Safety and pharmacodynamic effects of VERVE-101 an investigational DNA Base editing medicine designed to durably inactivate the PCSK9 gene and lower LDL cholesterol – interim results of the phase 1b heart-1 trial. Presented at: AHA Scientific Sessions 2023; November 10-13; Philadelphia, Pennsylvania.
2. Verve therapeutics announces updates on its PCSK9 program. Verve Therapeutics. April 2, 2024. Accessed April 2, 2024. https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-updates-its-pcsk9-program.

Related Videos
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
Salvador Rico, MD, PhD, on Informing Clinical Trials With Natural History Studies in Dravet
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
Janice Chen, PhD, the cofounder and chief technology officer of Mammoth Biosciences
Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing Therapies
Maria Escolar, MD, the chief medical officer of Forge Biologics
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies
Erika Fullwood Augustine, MD, MS, the associate chief science officer of the Kennedy Krieger Institute
Maria Escolar, MD, the chief medical officer of Forge Biologics
© 2024 MJH Life Sciences

All rights reserved.