Abstract
Clinical trials over the last 15 years have demonstrated that cell and gene therapies for cancer, monogenic and infectious disease are feasible and can lead to long-term benefit for patients. However, these trials have been limited to proof-of-principle and were conducted on modest numbers of patients or over long periods of time. In order for these studies to move towards standard practice and commercialization, scalable technologies for the isolation, ex vivo manipulation and delivery of these cells to patients must be developed. Additionally, regulatory strategies and clinical protocols for the collection, creation and delivery of cell products must be generated. In this article we review recent progress in hematopoietic cell and gene therapy, describe some of the current issues facing the field and discuss clinical, technical and regulatory approaches used to navigate the road to product development.
Author Disclosure Statement: DD is a Scientific Advisory Board member for Genesis BioPharma and an External Advisor to Owl Biomedical Inc. HPK has no conflicts of interest to disclose. DD is supported by grants from the California Institute for Regenerative Medicine (Grant Numbers DR1-01490 and TR2-01771). The contents of this publication are solely the responsibility of the authors and do not necessarily represent the official views of CIRM or any other agency of the State of California. HPK is supported by funding from the National Institutes of Health, Bethesda, MD grants R01AI080326, 1U19AI096111-01, R01HL098489 and P30DK056465, The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health nor its subsidiary Institutes and Centers. In addition, H.P.K. is a Markey Molecular Medicine investigator and the recipient of the Jose Carreras/ E.D. Thomas Chair for Cancer Research.