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Rocket Pharmaceuticals Announces Science Translational Medicine Publication of its Program in Danon Disease
– Results Demonstrate AAV9 Gene Therapy of LAMP2B Reverses Clinical Manifestations of Danon Disease in Mouse Study – NEW YORK--(BUSINESS WIRE)-- Rocket
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Results Demonstrate AAV9 Gene Therapy of LAMP2B Reverses Clinical Manifestations of Danon Disease in Mouse Study –\n\n NEW YORK--(BUSINESS WIRE)--\nRocket 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.\n\n\nIn this study, researchers evaluated the efficacy of a single intravenous (IV) infusion of recombinant adeno-associated serotype nine (AAV9) capsid containing the human LAMP2B transgene (AAV9.LAMP2B) in LAMP2-deficient mice, an established model of Danon Disease. Two cohorts of male mice, aged two- or six-months-old, were treated with four separate dose levels of AAV9.LAMP2B to determine the therapeutic potential of this gene therapy to prevent the development of (in the case of the 2 month-old cohort) or reverse an established disease phenotype (in the case of the 6 month-old cohort).\n\n\n“The data published in this manuscript show that a single intravenous injection of AAV9.LAMP2B resulted in dose-dependent human LAMP2B protein expression in heart, liver and skeletal muscle tissues,” said Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health, Professor of Medicine at University of California San Diego School of Medicine and co-author of the manuscript. “Importantly, LAMP2B gene transfer conferred improved cardiac function and higher survival rates in the older cohort of mice receiving high vector doses. These results validate the AAV9.LAMP2B gene therapy approach to reverse the pathological effects of Danon Disease in the tissues most affected by this disorder including the heart, liver and skeletal muscle.”\n\n\...