Manufacturing considerations for clinical uses of therapies derived from stem cells.

Manufacturing a therapeutic cell-based product from a stem cell source is far from simple. Regulatory authorities expect that the donor source of the stem cells is appropriately screened and tested for human pathogens. All of the synthetic or animal-derived ancillary materials must be appropriately qualified and tested before use in clinical manufacturing. The manufacturing process used to generate the therapeutic cells from the stem cells must be aseptic, consistent, and scalable. Finally, a robust quality program is necessary to ensure that appropriate quality procedures for the manufacturing process are in place and a quality assurance function that verifies the intended therapeutic cellular product has been prepared in a manner that is compliant with all regulatory expectations for clinical use in patients. This chapter discusses regulatory issues for manufacturing of therapeutic cells derived from stem cells and provides approaches for successfully addressing them.