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Rocket Pharmaceuticals Announces Danon Disease Presentation at the American Heart Association (AHA) Scientific Sessions 2021
—Updated clinical data from the ongoing Phase 1 trial of RP-A501 to be presented— CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ:
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Updated clinical data from the ongoing Phase 1 trial of RP-A501 to be presented—\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021.\n\nDetails for Rocket’s ePoster presentation are as follows:\n\nTitle: Results From First-In-Human Clinical Trial Of RP-A501 (AAV9:LAMP2B) Gene Therapy Treatment For Danon Disease\nPresenter: Barry Greenberg, MD, FHFSA, University of California, San Diego Medical Center, La Jolla, CA\nPoster Number: P2838\n\nThe abstract can be found online at https://professional.heart.org/en/meetings/scientific-sessions. Posters will be available on demand to registrants starting November 13, 2021, at 8 a.m. ET.\n\nAbout RP-A501\n\nRP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B). RP-A501 is currently being evaluated in an ongoing Phase 1 clinical trial, from which preliminary data of the low-dose cohort showed it was generally well tolerated and provided evidence of improved cardiac function in patients.\n\nAbout Danon Disease\n\nDanon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon Disease.\n\nAbout Rocket Pharmaceutical...