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February 8, 2023

FDA grants RMAT designation to Rocket Pharmaceutical’s therapy for Danon Disease

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Rocket Pharmaceuticals’ RP-A501, an investigational adeno-associated virus (AAV)-based gene therapy to treat Danon Disease.

FDA grants RMAT designation to Rocket Pharmaceutical’s therapy for Danon Disease