Cell-based therapies present a new paradigm in drug development and have the potential to make a large contribution toward currently unmet medical needs. Manufacturing of high-quality, clinical-grade cell products through effective production processes and quality control strategies is essential to meet patients’ demands. Manufacturing and testing cell-based therapies is a complex process compared to standard medicinal products and biologics. Because cell-based therapies contain living cells, their manufacturing processes are complicated and they cannot be terminally sterilized, and so there must be a lot of emphases and additional efforts on quality control and quality assurance. The production of cell-based therapies generally consists on sample collection, cell isolation and purification, cell modification, differentiation and expansion, cell harvest and purification, and, finally, product formulation, cryopreservation, and storage. To guarantee product safety and quality, the entire manufacturing process and quality control strategy must be carefully designed using a risk-based approach. In this chapter, we summarize the manufacturing steps and review the state-of-the-art manufacturing technologies available, as well as the key issues for quality control and quality assurance of these novel therapies, with a focus on chimeric antigen receptor (CAR)-T cells.

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Manufacturing Cell-Based Products

  • Nuria López Lorenzo,
  • Lorena Boquete Vilariño,
  • Mariona Baliu-Piqué

摘要

Cell-based therapies present a new paradigm in drug development and have the potential to make a large contribution toward currently unmet medical needs. Manufacturing of high-quality, clinical-grade cell products through effective production processes and quality control strategies is essential to meet patients’ demands. Manufacturing and testing cell-based therapies is a complex process compared to standard medicinal products and biologics. Because cell-based therapies contain living cells, their manufacturing processes are complicated and they cannot be terminally sterilized, and so there must be a lot of emphases and additional efforts on quality control and quality assurance. The production of cell-based therapies generally consists on sample collection, cell isolation and purification, cell modification, differentiation and expansion, cell harvest and purification, and, finally, product formulation, cryopreservation, and storage. To guarantee product safety and quality, the entire manufacturing process and quality control strategy must be carefully designed using a risk-based approach. In this chapter, we summarize the manufacturing steps and review the state-of-the-art manufacturing technologies available, as well as the key issues for quality control and quality assurance of these novel therapies, with a focus on chimeric antigen receptor (CAR)-T cells.