World Duchenne Muscular Dystrophy Day provides an opportunity to note the acceleration in the development of innovative therapies for this neuromuscular disease, which is the most common in children. In particular, gene therapy represents a very promising avenue. Généthon, the AFM-Téléthon laboratory, is currently developing a drug candidate which has demonstrated its efficacy in the first patients to be treated.
A drug candidate showing efficacy in the first patients treated.
The gene therapy drug candidate developed by Genethon combines a microdystrophin and an AAV8 vector. Administered by a single intravenous injection, it is currently being evaluated as part of a trial being conducted in France and the United Kingdom. 5 children aged between 6 and 9 have been treated as part of this trial.
Presented at the Myology2024 scientific congress last April, the results from the first patients treated show good tolerance of this drug candidate, as well as efficacy data, both in terms of microdystrophin expression and functional improvement. Children treated with the therapeutic dose improved their ability to walk, even run, climb stairs and stand up unaided, with or without support. These advances mark a major step forward in the development of the treatment, as Genethon prepares, in collaboration with the European Medicines Agency (EMA), the final phase of evaluation of the drug candidate with the dose that demonstrated its efficacy in the first phase of the trial. This dose is lower than those used in all the other gene therapy trials currently being conducted for Duchenne muscular dystrophy, which represents a major advantage both in terms of patient safety (limitation of side effects) and ease of production, and therefore the cost of treatment.
The gene therapy drug candidate developed by Genethon combines a microdystrophin and an AAV8 vector. Administered by a single intravenous injection, it is currently being evaluated as part of a trial being conducted in France and the United Kingdom. 5 children aged between 6 and 9 have been treated as part of this trial.
Presented at the Myology2024 scientific congress last April, the results from the first patients treated show good tolerance of this drug candidate, as well as efficacy data, both in terms of microdystrophin expression and functional improvement. Children treated with the therapeutic dose improved their ability to walk, even run, climb stairs and stand up unaided, with or without support. These advances mark a major step forward in the development of the treatment, as Genethon prepares, in collaboration with the European Medicines Agency (EMA), the final phase of evaluation of the drug candidate with the dose that demonstrated its efficacy in the first phase of the trial. This dose is lower than those used in all the other gene therapy trials currently being conducted for Duchenne muscular dystrophy, which represents a major advantage both in terms of patient safety (limitation of side effects) and ease of production, and therefore the cost of treatment.
Duchenne muscular dystrophy research at the Institute
Within the Center of Research in Myology, France Pietri-Rouxel heads the Muscle mass and function maintenance & optimization of AAV-based gene therapies Group (MOOVE) group, which aims to optimise the therapeutic approach for treating Duchenne muscular dystrophy, to characterise the phenotypic and genomic characteristics of Becker patients carrying the deletion of exons 45 to 55, and to decipher the mechanisms governing skeletal muscle plasticity, an essential step in understanding its physiopathological processes.
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