Pluripotent stem cells (PSCs) can form any tissue or cell in the body, and are the ideal starting material to manufacture replacement organs and cellular therapies. Many groups have shown therapeutic proof-of-concept of PSC-derived cells or tissues in relevant animal models, but PSCs are still more of a promise than a reality because of manufacturing challenges.  The task of manufacturing a sufficient number of cells under Good Manufacturing Practices (GMP) to enable clinical trials and eventual commercialization is a substantial hurdle. Furthermore, all allogeneic therapies, including those derived from PSCs, must meet strict regulatory requirements. One such requirement is the need for extensive, costly characterization and safety testing of the parental cell line and the subsequent master and working cell banks (MCB/WCB). The time and expense required to complete this testing necessitates the MCB be of sufficient quality and size to meet not only the requirements of a Phase 1 regulatory filing (IND), but also for the lifetime of the product through late stage trials and commercialization. This presentation will focus on the “how and why” of integrating high quality ancillary raw materials into cell therapy product development and manufacturing workflow, beginning with the starting cell source and MCB.  Generation of a high-quality MCB is especially crucial for PSC-derived therapies because the first and most critically important step in product manufacturing is procurement of GMP-compliant starting material that meet regulatory requirements. Consequently, using high-quality raw materials from suppliers experienced in supporting cell therapy development and GMP manufacturing increase the probability of success and reduce the risk of costly surprises that could spell clinical or financial failure for a promising cell therapy product.  

 

Learning Objectives:

 

Watch our related webinars here: