Gene therapy involves modification of genetic material present within a living cell with a special focus on mutated genes that lead to a number of disorders. ¹ It is a promising front being explored, particularly during recent years, with the potential to revolutionize the way we think about medicine. ² It is directed towards addressing the unmet medical needs concentrating on diseases with no cure but rather a lifetime of medication regimen such as diabetes, cystic fibrosis, AIDS, cardiovascular conditions, and cancer among the others. ³

Several gene and cell therapy projects are currently in the pipeline, and according to a statement made by then FDA commissioner Scott Gottlieb in January 2019, the agency would be approving between 10 and 20 gene and cell therapies per year by the year 2025, we can, therefore, expect the market to grow significantly in the years to come. ⁴ The potential of these therapies to cure the underlying pathology rather than just treat the symptoms has made them a subject of great interest leading to investments worth billions in order to run the clinical trials. ⁵

The future prospects of this market are not only promising for the developers and investors but also the patients living with life-long conditions relying on pharmaceutical management. ⁶ One major setback in the past has been the safety of vectors, which has been significantly improved now. ⁷ Currently, oncologic and non-oncologic rare diseases remain to be the focus areas,^8,9,10 and a number of aspects are being explored, including the vectors, in vivo and ex vivo approaches, regulation, and cost. ¹¹

Clearly, gene and cell therapies seem to be the future of the pharmaceutical industry with a goal to fulfill the unmet medical needs of patients for the foreseeable future to come.

References:

1 NIH. What is gene therapy? Available from https://ghr.nlm.nih.gov/primer/therapy/genetherapy (Last Accessed May 27, 2020)

2 Hood, L., Balling, R., & Auffray, C. (2012). Revolutionizing medicine in the 21st century through systems approaches. Biotechnology journal, 7(8), 992-1001.

3 Rocholl, B. Gene Therapy: Revolutionizing Medicine.

4 U.S Food and 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. Available from https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics (Last Accessed May 27, 2020)

5 Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018). Gene therapy clinical trials worldwide to 2017: An update. The journal of gene medicine, 20(5), e3015.

6 Hanna, E., Rémuzat, C., Auquier, P., & Toumi, M. (2017). Gene therapies development: slow progress and promising prospect. Journal of market access & health policy, 5(1), 1265293.

7 Picanço-Castro, V., Pereira, C. G., Covas, D. T., Porto, G. S., Athanassiadou, A., & Figueiredo, M. L. (2020).

8 Lang, F. F., Bruner, J. M., Fuller, G. N., Aldape, K., Prados, M. D., Chang, S., … & Chandler, W. (2003). Phase I trial of adenovirus-mediated p53 gene therapy for recurrent glioma: biological and clinical results. Journal of Clinical Oncology, 21(13), 2508-2518.

9 Touchefeu, Y., Harrington, K. J., Galmiche, J. P., & Vassaux, G. (2010). gene therapy, recent developments and future prospects in gastrointestinal oncology. Alimentary pharmacology & therapeutics, 32(8), 953-968.

10 O’Reilly, M., Kohn, D. B., Bartlett, J., Benson, J., Brooks, P. J., Byrne, B. J., … & Gargiulo, L. (2013). Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Human gene therapy, 24(4), 355-362.

11 Cavazzana-Calvo, M., Thrasher, A., & Mavilio, F. (2004). The future of gene therapy. Nature, 427(6977), 779-781.