7 – GNT

Association Genethon (France) – www.genethon.fr

 SCIDNET Principal Investigator: Fulvio Mavilio (fmavilio@genethon.fr)

Genethon is a non-profit, translational R&D organization created in 1990 and funded by the Association Française contre les Myopathies.The mission of GNT is to develop gene therapies for genetic diseases. With over 230 scientists, physicians, engineers and regulatory affairs specialists, GNT develops gene therapy products based on lentiviral vectors (LV), adeno-associated (AAV) vectors and genetically modified hematopoietic stem cells from vector design to pre-clinical proof of concept, efficacy and safety, including process and analytical development. GNT is developing a pipeline of gene therapies at both preclinical and clinical level in the areas of hematopoietic, neuromuscular, eye and liver diseases. GNT currently hosts 12 R&D units, 4 technology platforms/services, a department of medical/regulatory affairs, and a business/intellectual property unit. GNT hosts a unit of the INSERM, the French National Research Institute, which provides academic affiliation to scientists and access to training, technological platforms, and resources.