Rocket Pharmaceuticals Announces Science Translational Medicine Publication of its Program in Danon Disease
Rocket Pharmaceuticals, Inc. (RCKT)
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Source: Business Wire
– Results Demonstrate AAV9 Gene Therapy of LAMP2B Reverses Clinical Manifestations of Danon Disease in Mouse Study – NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the peer-reviewed publication of a study evaluating the efficacy of an adeno-associated virus (AAV)-based gene therapy delivering human LAMP2B in a mouse model of Danon Disease, a rare genetic disorder often resulting in heart failure. The Science Translational Medicine publication “AAV9.LAMP2B Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease” describes data demonstrating that vector-mediated transfer of LAMP2B to deficient mice improved heart function and survival. These findings underscore the promise of Rocket’s AAV-based gene therapy candidate for Danon Disease, RP-A501.In this study, researchers eva
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