Regenerative medicine includes both cell and gene therapies. Currently 672 regenerative medicine companies operate around the world and 20 products have been approved by the US Food and Drug Administration (FDA). Of 631 ongoing clinical trials by the end of 2015 (1), over 40% are in oncology, followed in prominance by cardiovascular and infectious diseases. Here I focus on gene and cell therapy bioprocessing in which the final product delivered to patients are cells.

Cell therapies are either autologous (derived from a single patient, for that patient) or allogeneic (coming from a banked donor source, for many patients). Autologous therapies do not face risks associated with cell rejection; however, they are much more expensive to manufacture and distribute. Although Dendreon’s Provenge autologous cell therapy was approved by the FDA, it ultimately failed commercially because of its high cost to patients due to a manufacturing and distribution model that was inefficient. Cost of goods (CoG), manufacturing processes, and logistics are all critical to successful cell therapy commercialization, so they need to be considered along with clinical science from the inception of a cell therapy company. Three key enablers for success are manufacturing automation and single-use technologies; a diverse pipeline in modularized facilities; and sophisticated data acquisition and logistics.

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Original Publication Date: 04/2019