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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Rocket’s gene therapy could restore cardiac function in Danon disease

Rocket Pharmaceuticals has presented preclinical data demonstrating the efficacy of AAV-based gene therapy in restoring cardiac function in animal models of Danon disease, a fatal heart disease.

Data presented at the American Society of Gene and Cell Therapy 2019 Annual Meeting in Washington has revealed the potential of AAV-based gene therapy in preventing, reducing and reversing cardiac dysfunction in mice and cynomolgus monkeys.

Danon disease is a rare, X-linked neuromuscular and cardiovascular disease characterized by profound cardiomyopathy, skeletal myopathies, and mild cognitive impairment. It is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), a protein that is involved in autophagy, and has an estimated prevalence of 15,000 to 30,000 patients in the US and EU. Currently there are no specific therapies available, although patients may undergo cardiac transplantation.

Rocket’s RP-A501 is an AAV-based gene therapy where the AAV vector carries a gene for the missing LAMP-2 protein. It was developed under a collaboration with the University of California San Diego School of Medicine.

Animal studies have now shown that RP-A501 conferred high vector copy number in heart and LAMP2 protein expression was higher in all the four heart chambers. Protein expression in the heart was higher in treated non-human primates versus untreated ones.

Investigational New Drug application (IND)-enabling toxicology studies were conducted in wild-type mice and non-human primates. No adverse events were observed any of the animals. In addition, the presentation also highlighted the efficacy of RP-A501 in LAMP-2 knockout mice where delivery of AAV-LAMP-2 resulted in dose-dependent improvements of cardiac function in these mice.

With FDA’s IND approval earlier this year, Rocket is now set to start its Phase 1 study later this year to evaluate the safety, tolerability and preliminary efficacy of RP-A501 in humans.

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

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