Jonathan W. Weinsaft, MD, Future Research With Gene Therapy for Cardiovascular Disease
The chief of cardiology at Weill Cornell Medical College shared his outlook on the future of gene therapy research in the field.
“As we get better at genetic characterization, including whole exome sequencing, and understanding the contributions of polygenic alterations,the interaction between genotype and phenotype, by that I mean people with a given genetic predisposition, who were exposed to an inciting factor environmentally. Soall of these things are going to feed our understanding of how disease progresses and develops and will allow us to target given underlying mechanisms.”
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.
Since then, Verve has paused enrollment in heart-1 in consultation with the study’s independent data and safety monitoring board (DSMB) after the sixth participant treated experienced asymptomatic grade 3 adverse events and is prioritizing its second generation preclinical therapy, VERVE-102, for the same indication.
CGTLive® spoke with Jonathan W. Weinsaft, MD, chief of cardiology and professor of medicine at Weill Cornell Medical College, to learn more about the potential of gene therapy for cardiovascular disease and his outlook on future research.
