Genethon (GEN) is a non-profit R&D organization created in 1990 and funded by the Association Française contre les Myopathies (AFM-Telethon), a French association that supports patients and their families. The mission of GEN is to develop gene therapies for genetic diseases and to provide patients with access to these treatments. With over 230 scientists, physicians, engineers and regulatory affairs specialists, GEN also operates manufacturing of lentiviral vectors (LV), adeno-associated (AAV) vectors, and genetically modified hematopoietic stem cells. GEN is developing a pipeline of gene therapies at both preclinical and clinical level in the areas of immune and blood deficiencies and neuromuscular, eye, and liver diseases.
GEN has the required full range of skills and tools necessary to convert proofs of concept into treatments validated in patients: (1) production of vectors at small and large scale, including AAV vectors, (2) bioprocess and analytical development for vector production and characterization, (3) pre-clinical development of gene therapy (GT) products for inherited diseases, including muscular disorders such as myotubular myopathy (4) regulatory affairs and clinical development (some of the GT products have reached the clinical trial phase), (5) an intellectual property portfolio and business development unit that ensure national and international partnerships.
Within MYOCURE, Genethon will be responsible of producing and validating a myospecific AAV-MTM1 vector in animal models of myotubular myopathy. In addition, GEN will be in charge of developing a scalable process for the novel myospecific AAV vectors and producing the vectors for preclinical toxicology.