Lessons learned from the clinical development and market authorization of Glybera
Open accessAbstract
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.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Even when patients adhere to the very-low-fat diet and are monitored by a registered dietitian nutritionist, TG levels may remain dangerously high.
“Even when patients adhere to the very-low-fat diet and are monitored by a registered dietitian nutritionist (RDN), TG levels may remain dangerously high.3,20,21”
From Familial chylomicronemia syndrome: Bringing to life dietary recommendations throughout the life span (2018) · cites this paper as [21]
MCT oil may be used as a nutritional supplement for patients with FCS because MCT is metabolized through a chylomicron-independent pathway.
“MCT oil may be used as a nutritional supplement for patients with FCS because MCT is metabolized through a chylomicron-independent pathway.21”
From Familial chylomicronemia syndrome: Bringing to life dietary recommendations throughout the life span (2018) · cites this paper as [21]
The citation lists haven’t been fetched yet
References 19 · Cited by 189
- Authors
- Bryant, L. M., Christopher, D. M., Giles, A. R., Hinderer, C., Rodriguez, J. L., Smith, J. B., Traxler, E. A., Tycko, J., Wojno, A. P., & Wilson, J. M.
- Affiliations
- University of Pennsylvania (US)
- Corresponding author
- Wilson, James M.
- Venue
- Human Gene Therapy Clinical Development
- Publisher
- SAGE Publications
- Published
- 2013-06
- Volume
- 24
- Issue
- 2
- Pages
- 55-64
- ISSN
- 2324-8637 (ISSN-L)
- 2324-8645
- PMC
- PMC3992977
- OpenAlex
- W2003329601
- Cited by
- 189
- References
- 19
- Citations by year
- 2026: 5 · 2025: 7 · 2024: 7 · 2023: 12 · 2022: 6 · 2021: 18 · 2020: 16 · 2019: 11 · 2018: 16 · 2017: 22 · 2016: 20 · 2015: 21 · 2014: 23 · 2013: 5
- FWCI
- 15.26
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Human Gene Therapy Clinical Development (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Bill and Melinda Gates Foundation (51061)
- U.S. Public Health Service (HHSN268201200041C)
- National Heart, Lung, and Blood Institute (HL059407, P01HL059407)
- National Eye Institute (RC3EY020792, EY020792)
- National Institute of Diabetes and Digestive and Kidney Diseases (P30DK047757, DK047757)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (P01HD057247, HD057247)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Keywords
- gene therapy, clinical development, drug approval, regulatory approval, preclinical research, animal studies
- MeSH terms
- Dependovirus, Animals, Clinical Trials as Topic, Drug Evaluation, Preclinical, Genetic Vectors, Humans, Lipoprotein Lipase, Hyperlipoproteinemia Type I, Genetic Therapy
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
