Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Jul;28(7):979-981.
doi: 10.1038/s41431-020-0607-y. Epub 2020 Mar 25.

Gene therapy regulation: could in-body editing fall through the net?

Miranda Mourby  1 , Michael Morrison  2
Affiliations

Gene therapy regulation: could in-body editing fall through the net?

Miranda Mourby et al. Eur J Hum Genet. 2020 Jul.

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.

PubMed Disclaimer

Conflict of interest statement

The authors declare that they have no conflict of interest.

References

    1. Council Regulation (EU) 536/2014 of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC [2014] OJ L 158/1, Article 90. This reflects the prohibition in Article 13 Oviedo Convention, to which some EU Member States are party.
    1. Council Regulation (EC) 1394/2007 of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 [2007] OJ L324/121.
    1. Cornel MC, Howard HC, Lim D, Bonham VL, Wartiovaara K. Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic? Eur J Hum Genet. 2019;27:484–7. doi: 10.1038/s41431-018-0309-x. - DOI - PMC - PubMed
    1. Bender C. Regulating the gene-therapy revolution. Nature. 2018;564:S20–S22. doi: 10.1038/d41586-018-07641-1. - DOI - PubMed
    1. European Medicines Agency, ʻScientific recommendation on classification of advanced therapy medicinal products EMA/814824/2018ʼ, 21 November 2018. https://www.ema.europa.eu/en/documents/report/scientific-recommendation-.... Accessed 17 March 2020.

Publication types

MeSH terms