Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Correspondence
  • Published:

Glybera and the future of gene therapy in the European Union

Nature Reviews Drug Discovery volume 11, page 419 (2012)Cite this article

Subjects

A recent news article discussed the progress in gene therapy, a field that has had a number of major setbacks (Gene therapies advance towards finish line. Nature Rev. Drug Discov. 10, 719–720 (2011))1. The recent experience of Amsterdam Molecular Therapeutics (AMT) with its investigational gene therapy alipogene tiparvovec (also known as Glybera) provides an illustration of the challenges faced by those working in the field.

Founded in 1998, this small Dutch company developed probably the first viable manufacturing platform using adeno-associated virus (AAV)-derived vectors as the gene delivery vehicle. An important advantage of this platform is that the delivered genes are not integrated into the host chromosome, thereby avoiding potential oncogenic disruptions that have been observed with integrative vectors used to treat patients with X-linked severe combined immunodeficiency. Such a platform could potentially be applied in many monogenic disorders, and perhaps also in more common diseases.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on SpringerLink
  • Instant access to the full article PDF.

USD 39.95

Prices may be subject to local taxes which are calculated during checkout

References

  1. Mullard, A. Gene therapies advance towards finish line. Nature Rev. Drug Discov. 10, 719–720 (2011).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Norman Miller is at Magdalen College, University of Oxford, Oxford OX1 4AU, UK.,

    Norman Miller

Authors
  1. Norman Miller
    View author publications

    Search author on:PubMed Google Scholar

Ethics declarations

Competing interests

The author declares no competing financial interests.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Miller, N. Glybera and the future of gene therapy in the European Union. Nat Rev Drug Discov 11, 419 (2012). https://doi.org/10.1038/nrd3572-c1

Download citation

  • Published:

  • Issue date:

  • DOI: https://doi.org/10.1038/nrd3572-c1

This article is cited by

Search

Advanced search

Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research