Lessons learned from the clinical development and market authorization of Glybera

Hum Gene Ther Clin Dev. 2013 Jun;24(2):55-64. doi: 10.1089/humc.2013.087. Epub 2013 Jun 29.


Bryant and colleagues follow the development of Glybera (alipogene tiparvovec), the first gene therapy product approved in the European Union, from early preclinical studies through the approval process. They review key data from human and animal studies with an emphasis on issues that will be critical to other gene therapy products. The article concludes with an analysis of the complex review process that eventually led to Glybera's approval.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Dependovirus / genetics
  • Drug Evaluation, Preclinical
  • Genetic Therapy
  • Genetic Vectors / genetics
  • Genetic Vectors / metabolism*
  • Humans
  • Hyperlipoproteinemia Type I / metabolism
  • Hyperlipoproteinemia Type I / pathology
  • Hyperlipoproteinemia Type I / therapy
  • Lipoprotein Lipase / deficiency
  • Lipoprotein Lipase / genetics
  • Lipoprotein Lipase / metabolism*


  • Lipoprotein Lipase