Work

Lessons Learned from the Clinical Development and Market Authorization of Glybera

Year: 2013

Type: article

Abstract: 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.

Source: Human Gene Therapy Clinical Development

DOI: 10.1089/humc.2013.087

Authors: Laura M. Bryant, Devin M. Christopher, April R. Giles, Christian Hinderer, Jesse L. Rodriguez, Jenessa B. Smith, Elizabeth A. Traxler, Josh Tycko, Adam P. Wojno, James M. Wilson

Institution: University of Pennsylvania

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

Funders: Bill and Melinda Gates Foundation, U.S. Public Health Service, National Heart, Lung, and Blood Institute, National Eye Institute, National Institute of Diabetes and Digestive and Kidney Diseases, Eunice Kennedy Shriver National Institute of Child Health and Human Development

Awards: G2798287860, Molecular Therapy for Cystic Fibrosis, Gene Therapy for Urea Cycle Disorders, AAV Gene Therapy for Lipid Disorders, Novel Adeno-Associated Viral Therapy for X-linked Retinitis Pigmentosa, G7965915398

OpenAlex ID: W2003329601

FWCI: 11.64

Cites: 19

Cited by: 191

Related to: 10