Cell and gene therapies hold tremendous curative potential for the treatment of devastating diseases, such as Parkinson’s disease, Type 1 diabetes, and various cancers. Besides demonstrating the safety and efficacy of these therapies, it is important to establish robust, reliable, and scalable manufacturing processes to commercialize these therapies and meet the demand to treat large numbers of patients. Here, we introduce some of the bioprocessing best practices required to de-risk the commercial manufacturing of cell and gene therapies.
Lonza has established a standard step-by-step, late-stage development approach to address product life cycle requirements and commercialization readiness, including process improvements, process characterization, and assay validation. A failure mode and effects analysis (FMEA) can be utilized to identify modes of failure, risks, and mitigations, and to design appropriate process characterization studies prior to process validation. Appropriate process improvement studies may be needed to implement innovative technologies with proper in process control and to monitor analytical methods to ensure robustness and reproducibility of the manufacturing process. It may be necessary to eliminate open and variable manual unit operations while incorporating comprehensive characterization assays to verify long-term stability of the products. The implementation of an automated, scalable, computer-controlled 3D bioreactor with appropriate downstream processing technologies would be critical to minimize commercialization risks and produce high quality products. In this webinar, we will discuss how we have applied our unique approach to support the industrialization of cell and gene therapies using pluripotent stem cells and T cells.