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261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June
Laurent Servais1, Rebecca Horton2, Dimah Saade3
1MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK; Division of Child Neurology, Centre de Référence des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège and University of Liège, Avenue de l'Hôpital 1 4000 Liege, Belgium.
Abstract:
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.
