Containment considerations for the cryopreservation of cell and gene therapies

Background & Aim The commercialization of cells and genes for therapeutic use drives a critical need for the refinement of containment and storage procedures to ensure that the integrity of these sensitive biologics is maintained until administration to the patient. Development of many cell and gene therapies often begins in an academic setting with subsequent translation through the clinic into a commercial-grade product manufactured in a GMP environment. Along with the product itself, minimally-controlled processes using research grade consumables are also transferred and are often not revisited until most drug product characterization has already been completed. This practice can give rise to costly changes later, as the handling and storage of the products can have a direct impact on their quality. Using a science-based approach to understand and optimize advanced biologics in the context of their containers can help to mitigate risk. Methods, Results & Conclusion Various research studies were conducted to explore containment requirements in the context of cell and gene therapy products and processes. Container performance was evaluated independent of biologic material through analysis of particulate generation and container closure integrity testing measuring both oxygen and carbon dioxide ingress at temperatures down to -180°C. Cryogenic and low-temperature preservation of biologic material was evaluated through examining recovery of therapeutically relevant cells and viral vectors after storage in different container types and sizes. Together, the results generated provide a strong indication that the storage container and associated process parameters can affect the recovery of cell and gene therapy products. Insights will be provided, based on the present results and lessons learned from other biologics and combination products, to enable researchers and developers to consider how the critical quality attributes of their advanced biologic therapies could be affected by containment and processing.