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Therapeutic editing for cardiovascular diseases

Award Number: R35HL183558
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 03/20/2026
PERIOD OF PERFORMANCE END DATE: 01/31/2033

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Therapeutic editing for cardiovascular diseases - PROJECT SUMMARY For the past decade, my independent laboratory has pioneered the use of gene-editing tools for therapeutic This work has culminated in clinical trials of PCSK9 and ANGPTL3 base editing, with PCSK9 representing the first base-editing therapeutic administered to patients anywhere in the world; initial results from this clinical trial demonstrated durable reductions in blood low-density lipoprotein cholesterol (LDL-C) levels in patients with familial hypercholesterolemia and atherosclerotic cardiovascular disease (ASCVD). Building on this initial success, going forward, I will focus on three exemplary translational challenges in the cardiovascular field: (1) the use of liver-centered, tunable, multiplex editing to safely prevent ASCVD, the leading cause of death worldwide; (2) the use of therapeutic editing to definitively treat inherited connective tissue disorders, such as pseudoxanthoma elasticum (PXE) and Marfan syndrome; and (3) the use of heart-directed therapeutic editing to treat congenital heart disease and inherited cardiomyopathies, empowered by our development of a non-viral approach to deliver editing tools to the myocardium early in life. applications.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $1,137,435 )
2026	2026	TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, THE	3451 WALNUT ST	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	Cardiovascular Diseases Research	000	1	3/20/2026	NEW	$1,137,435
														Subtotal = $1,137,435

Grand Total All Awards = $1,137,435

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Therapeutic editing for cardiovascular diseases

Award Number: R35HL183558

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 03/20/2026

PERIOD OF PERFORMANCE END DATE: 01/31/2033

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