The purpose of this paper is to highlight the numerous challenges in validating cell and gene therapy (CGT), drug substance (DS) and drug product (DP) manufacturing processes and analytical methods. It is not intended to be a comprehensive overview of all stages of validation, and it does not address every CGT validation challenge; rather, it offers an initial insight into the topic. It showcases some of the challenges associated with validation of viral vector-mediated gene therapy and gene-modified cellular therapy manufacturing processes and analytical methods validation and offers potential solutions for these.
This paper is expected to make the reader more aware of the complexity and challenges associated with validating CGT products and the industry-wide consensus in addressing some of these to help drive regulatory acceptance. Addressing challenges as early as possible will help provide a clear and fast path for successful validation of the process and analytical methods. It should be noted that the recommendations in this paper are suggestions and organizations should devise strategies specific to their products in consultation with regulatory agencies based on process/product understanding, prior manufacturing experience, clinical safety and efficacy of the product and extent of unmet medical need.