Deepak L. Bhatt, MD, MPH, MBA, on Encouraging Data With Gene Editing for Hypercholesteremia


The director of the Mount Sinai Fuster Heart Hospital shared his optimism on data seen so far with VERVE-101.

“Within the context of heart-1, it appeared to be a safe approach. The patients enrolled in heart-1 were, I would say, rather high risk from cardiovascular perspective, they all had severe advanced coronary artery disease… within that context, that therapy seemed to be well tolerated, and seemed to be safe. Again, of course, we need more patients, we need longer term follow. But these first signs and signals from heart-1 in terms of the effective LDL lowering, I think, are promising and open a new door for research in cardiovascular medicine and one that could result in therapeutics that are extremely useful.”

The first of its kind approval of Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) CRISPR gene-edited cell therapy marked a major milestone in the field of gene editing. One therapy that may be poised to follow in its footsteps is Verve Therapeutics’ VERVE-101, an investigational gene-editing therapy intended to treat heterozygous familial hypercholesterolemia (HeFH).

Recent data from the phase 1b heart-1 trial (NCT05398029) presented at the American Society of Hematology meeting in November 2023 showed that VERVE-101 showed dose-dependent decreases in blood PCSK9 and LDL-C percentage. While durability data are still premature, significant decreases have been sustained for 180 days after treatment.

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 VERVE-101 and its unique advantages for cardiovascular disease. He shared his excitement over the encouraging data seen so far in the heart-1 trial.

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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