You have obtained the status of a pharmaceutical establishment, what does that mean?
That Généthon Bioprod, the laboratory of the French Association against Myopathies (AFM), is authorized to produce drugs. In this case, it is genetical therapy, since the rare diseases that AFM deals with are caused by genetic abnormalities. The evaluation of the first drug candidates begins. It is therefore necessary to manufacture them on a large scale, which Généthon Bioprod allows. Ultimately, the idea is to rely on this laboratory to manufacture these drugs in series.
Why is this an important decision for you?
This is the first time that a non-profit association, the AFM therefore, has obtained this authorization. This follows on from high-level research carried out in the first laboratory set up by the AFM: the Généthon. This is where we were able to better understand the mechanisms of these diseases and design treatments to correct them. Laboratories around the world (in the United States, in Japan, etc.) are now asking to work with us. But the operating budget of this laboratory depends on the Telethon …
What are the first expected drug genes?
A trial is underway in children with immunodeficiency (absence of immune defenses), a disease similar to that of bubble babies. It takes place in France, Great Britain and the United States. Another will start for rare retinal damage (Leber’s disease). In 2014, trials should start on Fanconi (rare anemia) and Duchenne (myopathy) diseases.
