Genethon present its “DMD” project in Milan

The 18th annual congress of the European Society of Gene and Cell Therapy (ESGCT) was held in Milan at the end of October. Genethon sponsors this event and Philippe Moullier, Scientific Director, presented one of Genethon’s major projects concerning Duchenne muscular dystrophy (DMD): a unique network of laboratories has been established to bring together the necessary skills (Institute Myology in Paris and Nantes, research units INSERM UMR 649 and INRA UMR 703). The goal of this project is to develop a biotherapy with curative aim for Duchenne muscular dystrophy.

Today, it is necessary to establish the effective dose of therapeutic product to be used as well as its biosafety in a “large animal” model: the myopathic GRMD (Golden Retriever Muscular Dystrophy) dog, in which the myopathy is identical to that of Duchenne patients. “The existence of a natural canine model of this disease is a unique opportunity and allows us to accumulate a set of clinically relevant data on the effectiveness of our product, while using a locoregional injection mode suitable for application in humans,” said Philippe Moullier.

This project is supported by the AFM and ADNA program (Advanced Diagnostics for New therapeutic Approaches), coordinated by the Mérieux Institute and supported for research and innovation by the French agency, OSEO.