Rocket Pharmaceuticals Announces Presentations at the American College of Cardiology 2019 Annual Meeting

 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.

"Danon is a disease of deficient autophagy caused by mutations in the gene for LAMP-2 that leads to death in adolescence and young male adulthood. Insights from literature including UCSD's recently published natural history retrospective demonstrate that male patients have severe disease progression, leading to death at a median age of 19-21 years. Heart transplant and other interventions are not curative. Women have a delayed onset of cardiac symptoms, but may present with equally severe cardiomyopathy and suffer life-threating complications. Global natural history studies are underway to expand upon these findings and inform trial design," said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President, Clinical Development.

Dr. Schwartz continued, "Preclinical efficacy studies of RP-A501 have been conducted in LAMP-2 knockout mice at 2 different ages, one representing a young adolescent male before the onset of significant cardiac disease and the other representing an older male with established symptoms of Danon disease. These studies demonstrate both reversal and durable prevention of cardiac, muscle and liver dysfunction."

"RP-A501 is the first investigational gene therapy for Danon disease and has the potential to revolutionize the treatment of this devastating cardiomyopathy. We are excited to move RP-A501 into the clinic in the second quarter of 2019," concluded Dr. Schwartz.

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