Preclinical Data of RP-A501 in Danon Disease Demonstrates Restoration
and Durable Prevention of Cardiac, Muscle and Liver Structure and
Function
NEW YORK–(BUSINESS WIRE)–Rocket
Pharmaceuticals, Inc. (Nasdaq:RCKT) (“Rocket”), a leading U.S.-based
multi-platform clinical-stage gene therapy company, today announces one
oral poster presentation and two poster presentations at the upcoming
American College of Cardiology 2019 Annual Meeting being held March
16-18, 2019 in New Orleans, LA. RP-A501 is the Company’s first
adeno-associated viral vector (AAV)-based gene therapy for the treatment
of Danon disease that is in development under a collaboration with the
University of California San Diego School of Medicine (UCSD). UCSD
Health will be the initial and lead center for the planned Phase 1
clinical trial. Barry Greenberg, M.D., Director of the Advanced Heart
Failure Treatment Program at UC San Diego Health and Professor of
Medicine at University of California San Diego School of Medicine, will
be the principal investigator….