Audentes Therapeutics, a biotechnology company focused on developing and commercializing gene therapy products, today announced it has commenced dosing of patients in a Phase 1 / 2 clinical trial of AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM). This trial is a multicenter, multinational, open-label, ascending dose study to evaluate the safety and preliminary efficacy of AT132 in approximately twelve XLMTM patients less than five years of age. Preliminary data is expected to be available in the fourth quarter of 2017.
Last april, team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease.
This work, published in Molecular Therapy, has been achieved thanks to donations from the French Telethon and the support of the Myotubular Trust.