Gene Therapies in Research: Overview
Below is a review of the gene therapy development landscape and exploration of marketed agents.
Investigational New Drug Applications For Gene Therapies Are Increasing in the US
- Prior to clinical testing in human subjects, an IND application is submitted to the FDA for authorization to administer an investigational drug to humans1
- The number of IND applications for gene therapy products submitted to the FDA has been steadily increasing since 2011, with an all-time high achieved in 20181
Increasing Numbers of Gene Therapy Clinical Trials Are Being Approved Each Year
As of December 2018, just over 2900 gene therapy clinical trials have been completed or are ongoing worldwide5
Number of Gene Therapy Clinical Trials Approved Each Year Worldwide, 1989–20186

Figure adapted from The Journal of Gene Medicine, © 2017 John Wiley and Sons Ltd. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: https://www.abedia.com/wiley/years.php. Accessed September 17, 2019.
Gene Therapy Clinical Trials Span Across All Development Phases
Phases of Gene Therapy Clinical Trials5
Figure reproduced from The Journal of Gene Medicine, © 2017 John Wiley and Sons Ltd. Gene Therapy Clinical Trials Worldwide, December 2018.
Available at: https://www.abedia.com/wiley/phases.php. Accessed September 17, 2019.
Many Diseases Are Promising Targets for Gene Therapy
Much of the previous research into gene therapy has focused on cancer and monogenic diseases8
- Improved understanding of the genetics of inherited diseases is increasingly prompting research into gene therapy applications in a broader range of disorders8
- Clinical trials of gene therapy for infectious diseases, cardiovascular disease, neurological diseases, and inflammatory diseases are ongoing7,9
Indications Addressed by Gene Therapy Clinical Trials9
Figure reproduced from The Journal of Gene Medicine, © 2017 John Wiley and Sons Ltd. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: https://www.abedia.com/wiley/indications.php. Accessed September 17, 2019.
Summary/Module Recap
The FDA attributes this surge in gene therapies to the development of safer and effective vectors3
Consequently, approvals of gene therapies are expected to increase over the coming years3
Cancer and monogenic diseases have been the primary targets of gene therapy, but its use is now being explored in a diverse range of diseases7
References
- Eisenman D. Applied Biosafety: J ABSA International 2019;24(3):147–152.
- Marks P. Presented at CASSS Cell & Gene Therapy Symposium 2018, Rockville, MD, USA.
- U.S. FDA. Press announcement. January 15, 2019. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629493.htm. Accessed September 17, 2019.
- ClinicalTrials.gov. Search term results. Available at: https://bit.ly/2UVsFXM. Accessed September 17, 2019.
- The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: https://www.abedia.com/wiley/phases.php. Accessed September 17, 2019.
- The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: https://www.abedia.com/wiley/years.php. Accessed September 17, 2019.
- Ginn SL, et al. J Gene Med 2018;20(5):e3015.
- Beitelshees M, et al. Discov Med 2017;24(134):313–322.
- The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, August 2018. Available at: https://www.abedia.com/wiley/indications.php. Accessed September 17, 2019.
- European Commission. Zynteglo. Available at: https://ec.europa.eu/health/documents/community-register/html/h1367.htm. Accessed September 17, 2019.
- European Commission. Strimvelis. Available at: https://ec.europa.eu/health/documents/community-register/html/h1097.htm. Accessed September 17, 2019.
- Yescarta® [package insert]. 2017. Available at: https://www.yescarta.com/files/yescarta-pi.pdf. Accessed September 17, 2019.
- U.S. FDA. News release. May 24, 2019. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease. Accessed September 17, 2019.
- Kymriah® [package insert]. 2018. Available at: https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymriah.pdf. Accessed September 17, 2019.
- Luxturna™ [package insert]. 2017. Available at: https://sparktx.com/LUXTURNA_US_Prescribing_Information.pdf. Accessed September 17, 2019.
- Eisenman D. Applied Biosafety: J ABSA International 2019;24(3):147–152.
- Marks P. Presented at CASSS Cell & Gene Therapy Symposium 2018, Rockville, MD, USA.
- U.S. FDA. Press announcement. January 15, 2019. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629493.htm. Accessed September 17, 2019.
- ClinicalTrials.gov. Search term results. Available at: https://bit.ly/2UVsFXM. Accessed September 17, 2019.
- The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: https://www.abedia.com/wiley/phases.php. Accessed September 17, 2019.
- The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: https://www.abedia.com/wiley/years.php. Accessed September 17, 2019.
- Ginn SL, et al. J Gene Med 2018;20(5):e3015.
- Beitelshees M, et al. Discov Med 2017;24(134):313–322.
- The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, August 2018. Available at: https://www.abedia.com/wiley/indications.php. Accessed September 17, 2019.
- European Commission. Zynteglo. Available at: https://ec.europa.eu/health/documents/community-register/html/h1367.htm. Accessed September 17, 2019.
- European Commission. Strimvelis. Available at: https://ec.europa.eu/health/documents/community-register/html/h1097.htm. Accessed September 17, 2019.
- Yescarta® [package insert]. 2017. Available at: https://www.yescarta.com/files/yescarta-pi.pdf. Accessed September 17, 2019.
- U.S. FDA. News release. May 24, 2019. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease. Accessed September 17, 2019.
- Kymriah® [package insert]. 2018. Available at: https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymriah.pdf. Accessed September 17, 2019.
- Luxturna™ [package insert]. 2017. Available at: https://sparktx.com/LUXTURNA_US_Prescribing_Information.pdf. Accessed September 17, 2019.