Background
Dedicated to the fight against rare diseases, Genethon has developed a unique expertise in the design and development of gene therapy products, from lab research to clinical trials. Its work work has led to several treatments on the market, in clinical trials, or at the R&D stage that are changing the lives of patients and their families.
With 200 scientists and professionals, Genethon pursues its mission: to develop treatments that change the lives of patients suffering from rare genetic diseases and to eradicate these diseases.
Genethon is a non-profit organization, created in 1990 by the French Association against Myopathies (AFM-Téléthon), thanks to donations from the first Telethons.
Description of the Products / Services / Technology
Genethon’s capabilities encompass all the expertise and skills involved in the discovery, preclinical, clinical and technological development, and biomanufacturing of gene therapy vectors:
Therapeutic Research departments:
design gene therapy approaches for several diseases.
Preclinical development, an in vitro and in vivo drug testing platform, which includes:
- a vivarium hosting up to 4,000 rodents;
- a functional investigation facility;
- an imaging and cytometry platform, for molecular and physio-pathological investigation, from the single cell to the whole animal level;
- a histology service.
Technological developments for gene therapy products:
- bioprocess R&D;
- identification and development of biological markers and theranostics
- for monitoring neuromuscular diseases (Duchenne muscular dystrophy,in particular).
GMP biomanufacturing department:
- GMP Production, Quality control and Assurance of clinical batches of drug candidates for gene therapy trials.
- Clinical Development and Regulatory Affairs department: design, implementation and running of clinical trials.
DNA and cell bank:
national platform for the preparation and conservation of human biological samples.
