TuesdayGS - Bioassays for Cell and Gene Therapy

With increasing numbers of cell and gene therapy products in development and entering commercialization, more and more sponsors are facing a unique set of challenges of developing bioassays for cell and gene therapy products. Developing appropriate bioassays for cell and gene therapy products tends to be significantly more difficult than for conventional biologics due to a number of different factors, including- the difficulty of adequately modelling the clinical mode(s) of action in a simple cellular system; the challenge of defining, assessing suitability and generating sufficient quantity of, reference substances; non-linear or non-sigmoidal dose response relationships in some bioassays; qualitative or only semi-quantitative responses in others; a need for rapid analytical turnaround times in some cases; an evolving understanding of clinical mode(s) of action in some cases, which may require a close interplay between analytical and clinical development; and finally, the challenge of selecting the best bioassay(s) for release and stability testing vs. characterization/comparability testing. This session will focus on case studies and regulatory feedback that we hope will be beneficial to all sponsors faced with the daunting task of developing a comprehensive control strategy for a cell or gene therapy product.