261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands

•Safety issues in AAV-based gene therapies may result from different mechanisms specific to the serotype- the transgene or the underlying condition.•Specific risk management plan should be put in place to prevent and to manage serious adverse events.•Safety events observed in humans are not reproduc...

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Published in:	Neuromuscular disorders : NMD Vol. 33; no. 11; pp. 884 - 896
Main Authors:	Servais, Laurent, Horton, Rebecca, Saade, Dimah, Bonnemann, Carsten, Muntoni, Francesco, Adjali, Dr Oumeya, Beggs, Dr Alan, Bharucha, Dr Diana, Bönnemann, Dr Carsten, Braun, Dr Serge, Byrne, Dr Barry, Corti, Dr Manuela, Buj-Bello, Dr Ana, Chamberlain, Dr Jeff, Ferreiro, Dr Ana, Flanigan, Dr Kevin, Germanenko, Mrs Olga, Goemans, Dr Nathalie, Grant, Dr Daniel, Hopkins, Dr Sam, Horton, Dr Rebecca, Kollb-Sielecka, Dr Marta, Le Guiner, Dr Caroline, Levy, Dr. Dan, Lek, Dr Angela, Miller, Dr Weston, Morris, Dr Carl, Dreghici, Dr Roxana, Muntoni, Dr Francesco, Saade, Dr Dimah, Servais, Dr Laurent, Singh, Dr Teji, Vroom, Drs Elisabeth, Wagner, Dr Kathryn, Van Ieperen, Mr Frank
Format:	Journal Article
Language:	English
Published:	England Elsevier B.V 01-11-2023
Subjects:	Dependovirus - genetics Duchenne Gene therapy Genetic Therapy - adverse effects Humans Immunity Muscular Dystrophy, Duchenne - genetics Netherlands Neuromuscular Diseases - therapy Safety Serious adverse event Spinal miuscular atrophy X linked myotubular myopathy Netherlands X linked myotubular myopathy Spinal miuscular atrophy Duchenne Serious adverse event Safety Gene therapy Immunity
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Summary:	•Safety issues in AAV-based gene therapies may result from different mechanisms specific to the serotype- the transgene or the underlying condition.•Specific risk management plan should be put in place to prevent and to manage serious adverse events.•Safety events observed in humans are not reproduced in animal models and therefore require a strong collaboration between sponsors, investigators, regulators and patients community to be better understood and managed. 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.
Bibliography:	ObjectType-Conference-1 content type line 23 SourceType-Scholarly Journals-1
ISSN:	0960-8966 1873-2364 1873-2364
DOI:	10.1016/j.nmd.2023.09.008