The role of small molecules in cell and gene therapy.

Autor:	Brayshaw LL; Cell & Gene Therapy Discovery Research, Medicinal Science & Technology, GlaxoSmithKline Medicines Research Centre Gunnels Wood Road Stevenage SG1 2NY UK takis.x.athanasopoulos@gsk.com cherring99@gmail.com., Martinez-Fleites C; Protein Degradation Group, Medicinal Science & Technology, GlaxoSmithKline Medicines Research Centre Gunnels Wood Road Stevenage SG1 2NY UK., Athanasopoulos T; Cell & Gene Therapy Discovery Research, Medicinal Science & Technology, GlaxoSmithKline Medicines Research Centre Gunnels Wood Road Stevenage SG1 2NY UK takis.x.athanasopoulos@gsk.com cherring99@gmail.com., Southgate T; Cell & Gene Therapy Discovery Research, Medicinal Science & Technology, GlaxoSmithKline Medicines Research Centre Gunnels Wood Road Stevenage SG1 2NY UK takis.x.athanasopoulos@gsk.com cherring99@gmail.com., Jespers L; Cell & Gene Therapy Discovery Research, Medicinal Science & Technology, GlaxoSmithKline Medicines Research Centre Gunnels Wood Road Stevenage SG1 2NY UK takis.x.athanasopoulos@gsk.com cherring99@gmail.com., Herring C; Cell & Gene Therapy Discovery Research, Medicinal Science & Technology, GlaxoSmithKline Medicines Research Centre Gunnels Wood Road Stevenage SG1 2NY UK takis.x.athanasopoulos@gsk.com cherring99@gmail.com.
Jazyk:	angličtina
Zdroj:	RSC medicinal chemistry [RSC Med Chem] 2020 Dec 24; Vol. 12 (3), pp. 330-352. Date of Electronic Publication: 2020 Dec 24 (Print Publication: 2021).
DOI:	10.1039/d0md00221f
Abstrakt:	Cell and gene therapies have achieved impressive results in the treatment of rare genetic diseases using gene corrected stem cells and haematological cancers using chimeric antigen receptor T cells. However, these two fields face significant challenges such as demonstrating long-term efficacy and safety, and achieving cost-effective, scalable manufacturing processes. The use of small molecules is a key approach to overcome these barriers and can benefit cell and gene therapies at multiple stages of their lifecycle. For example, small molecules can be used to optimise viral vector production during manufacturing or used in the clinic to enhance the resistance of T cell therapies to the immunosuppressive tumour microenvironment. Here, we review current uses of small molecules in cell and gene therapy and highlight opportunities for medicinal chemists to further consolidate the success of cell and gene therapies. Competing Interests: All authors are GSK shareholders. C. H. is an Adaptimmune Therapeutics shareholder. L. L. B. and C. M. F. are inventors on patent WO2020078925. L. L. B., C. M. F. and C. H. are inventors on patent WO2019007869. There are no other conflicts of interest to declare. (This journal is © The Royal Society of Chemistry.)
Databáze:	MEDLINE
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