Abstract
Somatic gene therapies may be authorised for marketing in the EU under the advanced therapy medicinal product regulation. These therapeutic compounds are sufficiently novel and complex in their potential effects to require specialist evaluation. However, the current definition of gene therapy medicinal products (‘GTMP’) risks excluding molecules which are not manufactured through techniques involving recombination. We consider the way, in which the ‘recombinant nucleic acid’ aspect of the GTMP definition is challenged by developments in gene-editing technology, and why a broader scope of GTMP regulation may be desirable.
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Acknowledgements
The authors are grateful for the review and comments from Alex Faulkner, Andrew Webster, Ben Davies, Dianne Nicol, Edison Bicudo, Jane Kaye, Lisa Eckstein, and Phoebe Li. Any errors are the authors’ own.
Funding
The authors acknowledge support from the Leverhulme Trust project grant RPG-2017-330, Economic and Social Research Council grant number ES/P002943/1 (Biomodifying technologies and experimental space: Organisational & Regulatory Implications for the Translation & Valuation of Health Research).
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Mourby, M., Morrison, M. Gene therapy regulation: could in-body editing fall through the net?. Eur J Hum Genet 28, 979–981 (2020). https://doi.org/10.1038/s41431-020-0607-y
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DOI: https://doi.org/10.1038/s41431-020-0607-y
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