Rocket Pharmaceuticals presents preclinical data of RP-A501 at the American Society of Gene and Cell Therapy 2019 Annual Meeting in Washington, D.C. RP-A501 is the Company’s adeno-associated viral vector-based gene therapy for the treatment of Danon disease. The data is included in an oral presentation by Annahita Keravala, Ph.D., Associate Vice President, AAV Platform, entitled, “Systemic Delivery of AAV9.LAMP2B for the Treatment of Danon Disease: Toxicology Studies in Mice and Cynomolgus Monkeys.” Investigational New Drug application-enabling toxicology studies were conducted in wild-type mice and non-human primates. Three dose levels were tested in mice, including 3X10 13 vg/kg, 1X10 14 vg/kg, and 3X10 14 vg/kg. The highest dose level from the murine study, 3X10 14 vg/kg, was tested in non-human primates. No dose-related adverse events were observed at all tested doses in both mice and non-human primates. Vector genomes, mRNA and protein expression were widely distributed across key tissues with high levels of transduction, transcription and translation detected in the heart, skeletal muscle, diaphragm and liver. The ASGCT presentation also highlights previously reported preclinical efficacy data of RP-A501 in LAMP-2 knockout mice which showed dose-dependent improvements and restoration of cardiac function, with responses observed in both older and younger KO mice.
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