Cell Therapy CMC Case Studies

Cell therapies represent an exciting and fast-growing field, offering opportunities for personalized therapeutic intervention in diseases with historically unmet medical needs. Given the small number of approved products and the broad types of therapies that fall under the Cell therapy umbrella, the road to development is rife with unique hurdles. As such, development efforts require phase-appropriate, scientifically-driven approaches to predict and circumvent these hurdles. Using two case studies (an autologous CAR-T therapy and an autologous cell activation technique), this abstract will illustrate one of the CMC challenges encountered and solutions provided during the development of cell therapies, specifically, flow cytometry. This important quality assurance tool is so general to Cell Therapies, that it impacts CMC areas such as: release specifications, stability, method validation, comparability, consistency and reproducibility of the manufacturing process, raw materials and supply chain issues, scalability, and cryopreservation and storage.