The AFM-Telethon and its laboratory Genethon are delighted with the treatment of the first patient included in the European gene therapy trial for limb-girdle muscular dystrophy linked to the FKRP gene (LGMD2I/R9). This is a major step, the result of 30 years of excellence in research led by Isabelle Richard, head of the Progressive Muscular Dystrophies team at Genethon. The trial is being conducted by Atamyo Therapeutics, a biotechnology company created by Genethon to accelerate the development of gene therapy for limb-girdle muscular dystrophies.
The phase 1-2 clinical trial, which is taking place in Denmark, France and the UK, aims to evaluate the safety and efficacy of a gene therapy combining an AAV vector and the FKRP gene and administered intravenously. The first patient was treated by Prof. John Vissing, Director of the Copenhagen Neuromuscular Centre at the National Rigshospital and principal investigator of this study.
Limb Girdle Muscular Dystrophies (LGMD) are a group of diseases characterised by progressive degeneration of the pelvic girdle and shoulder muscles, leading to loss of motor function in the upper and lower limbs. There are more than 30 subtypes of limb-girdle muscular dystrophy associated with genetic mutations. Two gene therapy projects on other limb-girdle myopathies (gamma-sarcoglycanopathy and calpainopathy) were developed in the preclinical phases by Isabelle Richard’s team at Genethon and then licensed to Atamyo Therapeutics for clinical development.
