Rocket Pharmaceuticals Presents Preclinical Data of RP-A501 for Danon Disease at the American Society of Gene and Cell Therapy 2019 Annual Meeting


– Biodistribution of RP-A501 Demonstrates High Concentration in Heart, the End-Organ Target in Danon Disease –

– Phase 1 Clinical Trial to Begin in Second Quarter of 2019 –

NEW YORK, NY, USA I May 02, 2019 I Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today 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 (AAV)-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.”

“Additional preclinical RP-A501 data continue to augment the evidence regarding this vector’s potential to prevent, reduce, or reverse cardiac dysfunction. RP-A501 conferred high vector copy number (VCN) and LAMP2 protein expression in all four heart chambers, suggesting optimal tropism and uptake of the gene therapy. Specifically, VCNs in the ~10 range were about ten-fold higher in heart chambers versus skeletal muscle and most central nervous system tissues. Importantly, protein expression in all four heart chambers was higher in treated non-human primates versus wild type; this differential was most pronounced in cardiac muscle. Transduction and expression in the heart is critically important for Danon disease patients because heart failure is the overwhelming cause of mortality,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President, Clinical Development of Rocket.

Investigational New Drug application (IND)-enabling toxicology studies were conducted in wild-type mice and non-human primates. 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 (KO) mice which showed dose-dependent improvements and restoration of cardiac function, with responses observed in both older and younger KO mice.

Full results from the ASGCT presentation will be available online at the conclusion of the oral presentation:

About Danon Disease

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 U.S. 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.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket’s lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Rocket’s additional pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for a devastating, pediatric heart failure indication, Danon disease. For more information about Rocket, please visit

This story was republished from Rocket Pharmaceutical’s press release.


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