In a case of an unlikely coupling, French non profit Généthon and the infamous American company Sarepta have partnered up to develop a gene therapy for Duchenne Muscular Dystrophy (DMD).
Duchenne muscular dystrophy is the most common form of Muscular Dystrophy. It is characterized by general muscle weakness and wasting which primarily affects the pelvis, upper arms, and upper legs; eventually involving all voluntary muscles. The symptoms start showing before the third year of a persons’ life and by the age of twelve, the individual would be wheelchair bound. This inherited disorder allows for a very low survival chance.
This partnership appears to be a curious case given the French biotech though has not yet brought a drug out onto the market, is well established as a nonprofit; credit partially belonging to its creator, the French Muscular Dystrophy Association, AFM Téléthon. The company which is already developing a micro-dystrophin gene therapy which has proven itself applicable to the disease and owing to all these factors, is clearly an attractive partner for any company looking to stumble into the field.
Whereas the company, Sarepta neither has an impressive track record, nor an exceptionally good reputation in the industry. During the trial for its FDA-approved drug for
DMD, Exondys 51, which was later sold off, the FDA admitted that patients of the trial did not clearly experience any benefit from the drug in the study. This circumstance later prompted a renowned journalist to sue the company for the documents pertaining to the decision; an attack that sent Sarepta’s stock to a downward spiral in May earlier this year.With an overtly crowded arena, every company battling to successfully launch the next DMD drug in market, this unorthodox joining of forces could just raise above expectations to bring in success.
The financial details of the deal have not been disclosed.
