6 May 2019 - - US-based multi-platform clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has presented preclinical data of RP-A501 at the American Society of Gene and Cell Therapy 2019 annual meeting in Washington, the company said.

DC 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."

Three dose levels were tested in mice, including 3×1013 vg/kg, 1×1014 vg/kg, and 3×1014 vg/kg. The highest dose level from the murine study, 3×1014 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.

Danon disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. It is estimated to have a prevalence of 15,000 to 30,000 patients in the US and the European Union. The disease is often fatal in male patients in the second or third decade of life due to rapidly progressive heart failure.

Available therapies for Danon disease include 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.

Rocket Pharmaceuticals is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases.