Gene therapy clinical trials worldwide to 2017: An update
Abstract
To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our November 2017 update, we have entries on 2597 trials undertaken in 38 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and the genes that have been transferred. Details of the analyses presented, and our searchable database are available via The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: http://www.wiley.co.uk/genmed/clinical. We also provide an overview of the progress being made in gene therapy clinical trials around the world, and discuss key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies, which have the potential to transform the field moving forward.
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About 18.5% of clinical trials use adenoviral vectors for gene therapy.
“About 18.5% of clinical trials use this vector for gene therapy [57].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [57]
Some viral vectors, such as adenovirus vectors and herpesviral vectors, are toxic and can be immunogenic.
“Furthermore, some viral vectors (eg, Adenovirus vector, Herpesviral vector) are toxic and can be immunogenic.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [22]
The AV vector now represents 1 of the 3 major viral vector categories in the gene therapy toolbox, along with AAV and lenti/retrovirus vector systems.
“The AV vector now represents 1 of the 3 major viral vector categories in the gene therapy toolbox, along with AAV and lenti/retrovirus vector systems.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [22]
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- Authors
- Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R.
- Affiliations
- The University of Sydney (AU)
- Chatham House (GB)
- Uppsala University Hospital (SE)
- Corresponding author
- Ginn, Samantha L.
- Venue
- The Journal of Gene Medicine
- Publisher
- Wiley
- Published
- 2018-04-19
- Volume
- 20
- Issue
- 5
- Pages
- e3015-e3015
- ISSN
- 1099-498X (ISSN-L)
- 1521-2254
- OpenAlex
- W2983048398
- Cited by
- 903
- References
- 146
- Citations by year
- 2026: 31 · 2025: 68 · 2024: 86 · 2023: 77 · 2022: 124 · 2021: 141 · 2020: 151 · 2019: 148 · 2018: 36 · 2017: 12 · 2016: 6 · 2015: 6 · 2014: 3 · 2013: 2 · 2012: 5
- FWCI
- 16.66
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- The Journal of Gene Medicine (publishedVersion)
- PubMed (publishedVersion)
- 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 › Oncology › CAR-T cell therapy research
- Keywords
- gene therapy, clinical trials, clinical trial database, gene therapy vectors, disease indications, chimeric antigen receptor T cells, cancer immunotherapy, genome editing, clinical trial trends
- MeSH terms
- Clinical Trials as Topic, Humans, Neoplasms, Global Health, Genetic Therapy, Immunotherapy, Adoptive, Outcome Assessment, Health Care
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