Rocket Pharmaceuticals (NASDAQ:RCKT) has commenced patient dosing in Phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease.
The trial is expected to enroll 12-24 pediatric and young adult male patients. The study is designed to assess the safety and tolerability of a single infusion of RP-A501.
Additional outcome measures include cardiomyocyte and skeletal muscle transduction by gene expression, histologic correction via endomyocardial biopsy, and clinical stabilization via cardiopulmonary testing.
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