2B or Not 2B? Readiness for the PPQ of a Cell Therapy

As many ATMPs including genetically modified cell-based therapies move swiftly through the CMC process design and development to keep up with an accelerated clinical pathway, the planning to be ready for the critically important Process Performance Qualification (what some refer to as Stage 2B of the Process Validation Lifecycle) must begin early and must be appropriately managed. The employment of a science-driven and risk-based approach has never been more important for managing this process borrowing from lessons learned from previous biopharmaceutical products while also addressing the unique challenges associated with this novel type of product. Alignment of key CMC activities that are pre-requisites for the PPQ including process design and characterization, analytical methods validation, materials (raw/ancillary/starting) qualification and availability, and supply chain logistics (to name some but not all) is of utmost importance to success. In this presentation we will discuss the pre-requisites for the PPQ and how to address the unique challenges faced for the manufacture of a genetically modified cell-based therapy. Examples of custom tools and risk assessments to prepare for the PPQ of a genetically modified cell-based therapy within an ambitious overall project timeline will be detailed.