Rocket Pharmaceuticals (Nasdaq: RCKT) 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.

As quoted in the press release:

“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.”

Click here to read the full press release.