France-based Genethon, a not-for-profit organization which develops biotherapies for orphan genetic diseases, has received authorization from the French National Agency for Drug Safety (ANSM) to become a pharmaceutical manufacturer.

Genethon BioProd, is authorized to produce drugs in a first for a laboratory created by the French Muscular Dystrophy Association (AFM), which supports patients with neuromuscular diseases and their parents, and financed in part by donations from France's annual AFM-Telethon which raised 94.1 million euros ($121million) in 2011.

Genethon'’s mission is to design gene therapy products for rare diseases, to ensure their pre-clinical and clinical development as well as their production in order to provide patients with access to these innovative treatments.

Neuromuscular conditions, one of the key research targets at Genethon, encompass approximately 200 disorders which affect 30,000 to 40,000 people in France. Genethon has sponsored a Phase I clinical trial for gamma-sarcoglycanopathy (LGMD2C). The trial, which began in November 2006, was based on the intramuscular administration of an AAV (adeno-associated virus) vector carrying a normal copy of the gene. So far, nine patients have been enrolled in the trial and results are being analyzed.

Gamma-sarcoglycanopathy, or LGMD2C, is a rare form of limb girdle muscular dystrophy caused by mutations in the gamma-sarcoglycan gene (gSG). It is characterized by proximal and progressive muscular weakness which begins before the age of 10 on average, with a loss of ambulation occurring at puberty. Cardiomyopathy and respiratory failure may occur as the disease progresses, which can worsen the prognosis of the disease and may cause premature death.