Jonathan W. Weinsaft, MD, on Working to Bring Novel Therapies to Cardiovascular Disease


The chief of cardiology at Weill Cornell Medical College discussed gene therapy research into Friedreich ataxia and more.

“We're particularly excited at the ability of genomics, proteomics and transcriptomics. To transform our understanding of cardiovascular pathophysiology, and to inform our ability to provide more personalized treatments for cardiovascular conditions we've used, we're actively exploring the power of gene therapy to treat patients with genetically mediated cardiomyopathy or genetically mediated heart failure.”

Gene therapy research is growing in relatively newer fields to the technology, including cardiology. Gene editing therapy recently made a splash in the field when promising data from the phase 1b heart-1 clinical trial (NCT05398029) were recently presented at the American Heart Association’s (AHA) Scientific Sessions 2023 in November.

heart-1 is evaluating Verve Therapeutics’ VERVE-101, an investigational gene-editing therapy intended to treat heterozygous familial hypercholesterolemia (HeFH). The therapy has been well-tolerated, and the trial is continuing to dose patients based off a positive Data Safety Monitoring Board Recommendation. Investigators observed dose-dependent decreases in blood PCSK9 and blood LDL-C percentages from baseline, some of which have lasted for up to 155 days after treatment.

CGTLive spoke with Jonathan W. Weinsaft, MD, chief of cardiology and professor of medicine at Weill Cornell Medical College, to learn more about research that Weill Cornell is engaged in with the goal of bringing more of these novel therapies to patients. He touched on a few different programs, including a gene therapy program for Friedreich Ataxia and cardiomyopathies, and basic research looking deeper into cardiovascular disease. He also noted that the center is trying to leverage AI in powering research as well.

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.
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