Federal Grant Award Summary

The National Heart, Lung, and Blood Institute (NHLBI) awarded the University of Pennsylvania a Project Grant of $1,137,435 under the Cardiovascular Diseases Research program (CFDA 93.837) for the period March 20, 2026, through January 31, 2033. The grant supports translational research in therapeutic gene editing for cardiovascular disease prevention and treatment. The research program focuses on three primary research objectives: (1) developing liver-centered, tunable, multiplex gene-editing approaches to prevent atherosclerotic cardiovascular disease; (2) applying therapeutic editing to treat inherited connective tissue disorders including pseudoxanthoma elasticum and Marfan syndrome; and (3) developing heart-directed gene-editing approaches, including non-viral delivery mechanisms, to treat congenital heart disease and inherited cardiomyopathies.

This award builds on the principal investigator's prior clinical successes, including the first base-editing therapeutic for PCSK9 (proprotein convertase subtilisin/kexin type 9) administered to patients with familial hypercholesterolemia and atherosclerotic cardiovascular disease, which demonstrated durable reductions in low-density lipoprotein cholesterol levels. The funded research represents a continuation of advanced translational work converting gene-editing tools into clinical applications addressing the leading causes of cardiovascular mortality worldwide. Research will be performed at the University of Pennsylvania's facilities in Philadelphia, Pennsylvania.