Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A501 Gene Therapy for Danon Disease

Rocket Pharmaceuticals, Inc. announced that the FDA has granted RMAT designation to RP-A501, the Company’s investigational adeno-associated virus-based gene therapy for the treatment of Danon Disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available.