Rocket Pharmaceuticals, Inc.(Nasdaq: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, today announces the clearance of the Company's Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-A501. 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. UC San Diego Health will be the initial and lead center for the planned Phase 1 clinical trial under the direction of Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California San Diego School of Medicine.

"This acceptance marks the first Rocket-sponsored IND cleared for our AAV platform and the third for our pipeline in only three months," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "RP-A501 represents the first investigational gene therapy for a monogenic heart failure syndrome and has the largest market opportunity for Rocket with an estimated 15,000 - 30,000 patients in the U.S. and E.U. We expect to initiate a Phase 1, first-in-human study in the U.S. in the second quarter of 2019 to assess the safety, tolerability and preliminary efficacy of RP-A501."

"Danon disease is a devastating heart failure syndrome that develops during childhood and adolescence, and is followed by a rapid progression toward end-stage heart failure and death," said Dr. Adler. "We look forward to advancing this potentially curative treatment option for the patients and families affected by this life-threatening disease."

In parallel with the Phase 1 clinical trial, the Company plans to publish a comprehensive literature review of Danon cases and conduct a retrospective chart review. In addition, the Company will initiate a prospective natural history study with enrollment planned to commence in the first quarter of 2019.

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