[en] Adeno-associated virus (AAV) gene therapies are demonstrating much promise in the area of neuromuscular disorders. There are now therapies in clinical trials or real-world use for several disorders including spinal muscular atrophy and Duchenne muscular dystrophy. However, there have been several concerning reports of serious adverse events, including deaths. Reporting and monitoring of these is not consistent between trials. Therefore, a group of clinicians, investigators, industry and patient representatives met the weekend of 17th-19th June 2022 to discuss safety issues arising from the use of these therapies. The group shared information on safety events across a spectrum of AAV gene therapy products, both in clinical trials and commercial use. Patterns of serious adverse events were identified and the group discussed methods of identification and management of these as well as new ways of improving information sharing across industry in order to improve the safety of these promising treatments.
Disciplines :
Pediatrics
Author, co-author :
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques
Horton, Rebecca; MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK
Saade, Dimah; Department of Neurology, University of Iowa Carver College of Medicine, Iowa City, IA, USA
Bonnemann, Carsten; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA
Muntoni, Francesco; UCL Great Ormond Street Institute of Child Health, The Dubowitz Neuromuscular Centre, London, UK, National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK
261st ENMC workshop study group
Language :
English
Title :
261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands.
The workshops and next generation programme are made possible thanks to the financial support of the European Neuromuscular Centre (ENMC) and its Full Partners: Association Française contre les Myopathies (France), Deutsche Gesellschaft für Muskelkranke (Germany), Muscular Dystrophy Campaign (UK), Muskelsvindfonden (Denmark), Prinses Beatrix Spierfonds (The Netherlands), Schweizerische Stiftung für die Erforschung der Muskelkrankheiten (Switzerland), Spierziekten Nederland (The Netherlands), Telethon Foundation (Italy). In addition, we would like to thank the Associated Partners: Finnish Neuromuscular Association (Finland), Österreichische Muskelforschung (Austria), SMA Europe and World Duchenne Organisation, and the members of the ENMC Company Forum: Amicus Therapeutics, Astellas, Biogen, Ionis Pharmaceuticals, Lupin Neuroscience, Novartis, PerkinElmer, Roche, Sanofi, and Sarepta. A special thanks to MDA USA for their support to cover travel costs from USA participants. The figure was generated using biorender.
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