Gene Therapy Clinical Trials 2020 MEDWEBUS00117 Many Diseases

Gene Therapy Clinical Trials 2020 MEDWEB-US-00117

Many Diseases Are Promising Targets for Gene Therapy Indications Addressed by Gene Therapy Clinical Trials Much of the previous research into gene therapy has focused on cancer and monogenic diseases 1 • Improved understanding of the genetics of inherited diseases is increasingly prompting research into gene therapy applications in a broader range of disorders 1 • Clinical trials of gene therapy for infectious diseases, cardiovascular disease, neurologic diseases, and inflammatory diseases are ongoing 2, 3 2, 2% 1, 8% 1, 3% 1, 7% 3 1, 9% Cancer (n=1951) Monogenic diseases (n=338) 6, 2% Infectious diseases (n=184) 6, 3% Cardiovascular diseases (n=183) Neurologic diseases (n=52) Ocular diseases (n=37) Inflammatory diseases (n=15) 11, 5% Other diseases (n=63) Gene marking (n=50) 66, 6% Healthy volunteers (n=57) Figure reproduced from The Journal of Gene Medicine, © 2017 John Wiley and Sons Ltd. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: http: //www. abedia. com/wiley/indications. php. Accessed February 18, 2019. 1. Beitelshees M, et al. Discov Med 2017; 24(134): 313– 322; 2. Ginn SL, et al. J Gene Med 2018; 20(5): e 3015; 3. The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, August 2018. Available at: http: //www. abedia. com/wiley/indications. php. Accessed February 11, 2019. 2

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 worldwide 1 Number of Gene Therapy Clinical Trials Approved Each Year Worldwide, 1989 – 2018* , 2 200 150 100 50 92 19 93 19 94 19 95 19 96 19 97 19 98 19 99 20 00 20 01 20 02 20 03 20 04 20 05 20 06 20 07 20 08 20 09 20 10 20 11 20 12 20 13 20 14 20 15 20 16 20 17 20 18 19 19 8 1 0 9 19 90 Number of clinical trials approved each year 250 *Based on a global analysis of gene therapy clinical activity. The numbers of trials in recent years tend to be under-represented due to the time needed for results to be published, causing a lag in obtaining information on recent trials 3. Figure reproduced from The Journal of Gene Medicine, © 2017 John Wiley and Sons Ltd. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: http: //www. abedia. com/wiley/years. php. Accessed February 18, 2019. 1. The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: http: //www. abedia. com/wiley/phases. php. Accessed February 18, 2019; 2. The Journal of Gene Medicine. Gene Therapy Clinical Trials Worldwide, December 2018. Available at: http: //www. abedia. com/wiley/years. php. Accessed February 18, 2019; 3. Ginn SL, et al. J Gene Med 2018; 20(5): e 3015. 3