Created in 1990 by AFM-TELETHON, Genethon is fully dedicated to the design and development of gene therapy treatments for rare diseases.
With over 130 scientists, physicians, engineers and regulatory affairs specialists, Genethon also operates manufacturing of lentiviral vectors (LV), adeno-associated (AAV) vectors, and genetically modified hematopoietic stem cells. Genethon is developing a pipeline of gene therapies at both preclinical and clinical level in the areas of neuromuscular diseases, immune and blood deficiencies, liver as well as ocular disorders.
Genethon 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, (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 ensures national and international partnerships.
Genethon is the coordinator of the CureCN project. Besides the scientific coordination of the project, Genethon will oversee the manufacturing and release of clinical lots of AAV vectors, coordinate clinical monitoring and will be responsible for immune-monitoring of the patients enrolled in the clinical trial. Genethon will also be responsible for the preclinical studies in juvenile animals and vector integration studies, as well as for the nonclinical work relative to vector re-administration. Genethon will also contribute to the work on the development plasmapheresis columns specific to anti-AAV antibodies.
Staff members involved
