Adeno-associated virus (AAV) gene therapies are generating much excitement in the rare disease field, particularly for previously untreatable neurological conditions. Efficacy has been claimed for several gene therapy products and the number of trials is rapidly increasing. However, reports of severe treatment-related adverse reactions are emerging, including death. There is still insufficient knowledge about their aetiology, prevention and treatment. We therefore undertook to systematically review publicly available data on AAV gene therapies in order to collate existing information on both safety and efficacy. Here, we review emerging efficacy reports of these novel therapies, many of which show promise. We also collate an increasing number of adverse reactions. Overwhelmingly, these results make a case for unified reporting of adverse events. This is likely to be critical for improving the safety of these promising treatments.
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Contributors LS, FM, CB and RHH conceptualised the study. EH, DS and RHH completed the literature search. TM and RHH performed the data extraction and risk of bias assessment. RHH and LS wrote the paper.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests FM has received honoraria for scientific advisory boards from Novartis Gene Therapies Inc., Biogen, Novartis, PTC, Roche, Pfizer, Dyne Therapeutics and Sarepta. LS has received consulting fees from Sarepta, Pfizer, Roche, Affina, Renegex Bio, Audentes, Evox Therapeutics and Novartis. He has received honoraria from Sarepta, Roche, Audentes and Novartis. He is the secretary of the World Muscle Society. DS, EH, TM, RHH and CGB have no conflicts of interest to declare. TM is a scholar of the Onassis Foundation (Scholarship ID: F ZQ 040-1/2020-2021).
Provenance and peer review Not commissioned; externally peer reviewed.
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