Genethon, one of the principal international centers for research and preclinical and clinical development of gene therapy for rare diseases. Its mission is to develop innovative treatments for patients.
In 2012, Genethon received the prix Galien France for pharmaceutical research into “Medicines destined for rare diseases-cell therapy and gene therapy”.
In 2013, Genethon has opened Genethon BioProd, a 5000 m² facility fully dedicated to GMP-production and quality control of gene therapy products.
EFS-Atlantic BIO GMP (ABG) is a platform for the production of innovative medications, which is under the operational direction of the French Blood Agency.
The CHU (University Hospital Center) hosts the Gene Therapy Laboratory led by Professor Philippe Moullier which includes:
- A research team that develops protocols for gene transfer in vivo, particularly as applied to retinal diseases and neuromuscular diseases (Duchenne).
- An R & D team that develops new tools and processes of production and purification which are consistent with production of clinical grade vectors as well as the development of quality control tests for clinical application.
- A production platform of preclinical viral vectors.
GenoSafe is a full Contract Research and Consulting Organization which specializes in evaluating the Quality, Safety and Efficacy of innovative biological products. We meet our clients’ specific needs by performing custom studies in strict compliance with regulatory requirements.
Winner of the Investissements d’Avenir call for proposals in the field of health and biotechnology pre-industrial demonstrators in April 2011, the PGT project has received funding from the ANR.