The United States’ Regulatory Environment Is Evolving to Accommodate a Coming Boom in Gene Therapy Research
Open accessAbstract
Introduction: A dramatic increase in the number of clinical trials involving gene-modified cell therapy and gene therapy is taking place. The field is on the verge of a boom, and the regulatory environment is evolving to accommodate the growth. Discussion: This commentary summarizes the current state of the field, including an overview of the growth. The United States (US) regulatory structure for gene therapy will be summarized, and the evolution of the oversight structure will be explained. Conclusion: The gene therapy field has recently produced its first FDA-approved therapeutics and has a pipeline of other investigational products in the final stages of clinical trials before they can be evaluated by the FDA as safe and effective therapeutics. As research continues to evolve, so must the oversight structure. Biosafety professionals and IBCs have always played key roles in contributing to the safe, evidence-based advancement of gene therapy research. With the recent regulatory changes and current surge in gene therapy research, the importance of those roles has increased dramatically.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
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?
The majority of gene therapy trials, almost 70%, are based on viral vectors.
“The majority of these gene therapy trials, almost 70%, are based on viral vectors.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [15]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
The Next Phase of Human Gene-Therapy Oversight
Collins, F. S., & Gottlieb, S. L. (2018) · New England Journal of Medicine · doi:10.1056/nejmp1810628 · Cited by 97 · see citing works
Principles of and strategies for germline gene therapy
Wolf, D. P., Mitalipov, P. A., & Mitalipov, S. M. (2019) · Nature Medicine · doi:10.1038/s41591-019-0473-8 · Cited by 73 · see citing works
Points to consider in the design and submission of human somatic cell gene therapy protocols.
(1985) · PubMed · Cited by 19 · see citing works
Flagged first, then by citations
Research works printed but not linked: 4
- [1]1. The Journal of Gene Medicine. Gene therapy clinical trials worldwide. https://www.wiley.com/legacy/wileychi/genmed/clinical/. Accessed March 21, 2019.
- [13]13. Wolinetz C. Changing oversight of emerging technology—challenges for researchers, institutional biosafety committees (IBCs) and the community. Lecture presented at: University of Minnesota; March 6, 2019; Minneapolis, MN. [Google Scholar]
- [17]17. Kaiser J. Update: House spending panel restores U.S. ban on gene-edited babies. Science. https://www.sciencemag.org/news/2019/06/update-house-spending-panel-restores-us-ban-gene-edited-babies. Updated June 4, 2019. Accessed June 4, 2019.
- [21]21. US Food & Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., director of the center for biologics evaluation and research on new policies to advance development of safe and effective cell and gene therapies. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629493.htm. Updated January 15, 2019. Accessed March 21, 2019. Articles from Applied Biosafety: Journal of the American Biological Safety Association are provided here courtesy of ABSA International and Mary Ann Liebert, Inc.
Cited by
Sorted by citations
Ghosh, S., Brown, A. M., Jenkins, C., & Campbell, K. (2020) · Applied Biosafety · doi:10.1177/1535676019899502 · Cited by 218 · see citing works
- Authors
- Eisenman, D.
- Affiliations
- Advarra, Research Triangle Park, NC, USA
- Corresponding author
- Eisenman, Daniel
- ORCID
- Eisenman, Daniel
- Venue
- Applied Biosafety
- Publisher
- SAGE Publications
- Published
- 2019-06-18
- Volume
- 24
- Issue
- 3
- Pages
- 147-152
- ISSN
- 1091-3505 (ISSN-L)
- 1535-6760
- 2470-1246
- PMC
- PMC9134463
- OpenAlex
- W2951787126
- Cited by
- 17
- References
- 4
- Citations by year
- 2026: 2 · 2025: 1 · 2024: 4 · 2023: 3 · 2022: 4 · 2021: 1 · 2020: 2
- FWCI
- 1.20
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Applied Biosafety (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- University of Minnesota
- National Institutes of Health
- Topics
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Keywords
- gene therapy regulation, FDA oversight, clinical trials, investigational products, biosafety, institutional biosafety committees, regulatory change
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
