Genethon is making strategic investments into technological innovation for the benefit of its therapeutic research projects, in order to ensure that the chosen drug candidates have the best possible chance of success.
The Bioprocess Department develops innovative large-scale production and purification methods that are suitable for the therapy vectors being researched in each of its treatment programs. Work is conducted in order to facilitate bioproduction of these treatments in accordance with Good Manufacturing Practices.
Genethon aims at identifying biomarkers that can be used to assess patients' response to gene therapy. It is also leading an international network aimed at identifying biomarkers specific for Duchenne muscular dystrophy. Eventually, these biomarkers will be used to perfect new diagnostic and assessment technics that can benefit future therapies.
The Applied Vectorology group relies on an in-depth understanding of the structure and method of action of AAV (adeno-associated virus) and lentiviral vectors, in order to improve their efficacy and suitability for large-scale production.
Assessing and monitoring immune response is an essential part of ensuring the successful development of many gene therapy products. Genethon is developing experimental models and numerous immune tests in order to assess innate, cell-mediated and humoral immune response to administration of a vector and a transgene, first using in vitro and in vivo preclinical studies and then in patients enrolled in clinical trials.