Rocket Pharmaceuticals Provides Update on Anticipated Registration Path for RP-A501 in Danon Disease Following End-of-Phase 1 FDA Meeting

Dec 22, 2022

Rocket+Pharmaceuticals%2C+Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces updates from the Company's end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) regarding RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease. During the meeting, Rocket reviewed the positive Phase 1 dataset with the FDA and proposed a study design and endpoints for ongoing clinical development of the investigational gene therapy.

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