Rocket Pharma Says EMA Grants PRIME Designation For RP-A501 Gene Therapy For Danon Disease

(RTTNews) - Biotechnology company Rocket Pharmaceuticals, Inc. (RCKT) announced Wednesday that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501.

RP-A501 is the Company's investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease, a devastating and fatal inherited cardiomyopathy for which there are no curative therapies.

PRIME designation was granted based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A501 in patients with Danon Disease and the potential of RP-A501 to meet the high unmet medical need in this population.

Results from the Phase 1 trial represent one of the most comprehensive investigational gene therapy datasets for any cardiac condition.

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