In the After-hours session last night, Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) surged 33.16% to $42.65 after a report of phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy candidate expressing LAMP2B for the treatment of Danon Disease, today announces early results from its open-label, Phase 1 clinical trial of RP-A501.
Danon Disease is a rare inherited X-linked condition caused by genetic defects in the LAMP2 gene contributing to autophagosomal and glycogen accumulation, especially in the cardiac muscle and other tissues, leading eventually to severe and often fatal cardiomyopathy. Preliminary results from low dose RP-A501 cohort patients indicated that gene therapy was relatively well-received, and early evidence of clinical benefit was given.
