1. Bak A, Friis KP, Wu Y. Translating cell and gene biopharmaceutical products for health and market impact. Product scaling from clinical to marketplace: lessons learned and future outlook. J Pharm Sci. 2019;108(10):3169–75. https://doi.org/10.1016/j.xphs.2019.05.027.
2. Citeline Informa. A total of 2602 trials were listed, of which 985 in Ph I, 683 in Ph II, 557 in Ph I/II, 140 in Ph III, 68 in Ph II/III, 129 as Ph IV and 20 as N/A or other. The following therapeutic classes were included in the analysis, with trial status as “open”: “Gene therapy; Gene delivery vector; Cellular therapy, chimaeric antigen receptor; Cellular therapy, tumor-infiltrating lymphocyte; Cellular therapy, T cell receptor; Cellular therapy, stem cell; Cellular therapy, other”; www.Informa.com. Accessed 8 Oct 2022.
3. ASCGT Clinical Trials Finder. A total of 2556 trials were listed, of which 2268 were “open”, 271 “not yet recruiting” and 17 “active, not recruiting”. https://asgct.careboxhealth.com/. Accessed 8 Oct 2022.
4. FDA. LUXTURNA (voretigene neparvovec-rzyl). https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna. Accessed 8 Oct 2022.
5. FDA. ZOLGENSMA (onasemnogene abeparvovec). https://www.fda.gov/vaccines-blood-biologics/zolgensma . Accessed 8 Oct 2022.