The gene therapy used in this trial combines an AAV-type viral vector and a shortened version of the dystrophin gene, the microdystrophin, designed by researchers and experts from Genethon – a laboratory funded by AFM-Telethon – in collaboration with the team of Prof. George Dickson (University of London) and the team of Caroline Le Guiner in Nantes (gene therapy laboratory, CHU / Inserm).
On the basis of the positive results observed in preclinical studies in terms of efficacy and safety (Nature Communications, July 2017), Généthon and Sarepta Therapeutics, leaders in precision genetic medicine for rare diseases, have announced a collaboration to finalize the preclinical development then co-develop the clinical program.
As part of this international trial coordinated by Prof. Francesco Muntoni (Great Ormond Street Hospital, London), young boys with Duchenne muscular dystrophy will be included in France, United Kingdom, United States and Israel.
In France, the trial will take place in several research centres. In addition to I-Motion for Paris (Dr Silvana De Lucia), there will be the Hautepierre Hospital in Strasbourg (Prof. Vincent Laugel) and the hospitals in Brest, Bordeaux, Lyon, Marseille and Lille.
