Scaling of cell and gene therapies to population

Cell and gene therapies (CGTs) are intended to address many different diseases, including widespread diseases with millions of patients. The success of CGTs thus depends on the practicability of scaling cell manufacturing to population. It is obvious that process integration and automation are key for the reproducibility, quality, cost-effectiveness, and scalability of cell manufacturing. Still, different manufacturing concepts can be considered depending on the characteristics of cell therapies such as the degree of ex vivo manipulation of cells, the intended treatment scheme for the underlying medical indication, the prevalence of the indication, and the cell dose per final drug product. Here, we explain the characteristics of cellular products and their implications from the perspective of a manufacturer. We outline and exemplify with a list of devices' different strategies and scaling options for CGT manufacturing considering technical and regulatory aspects in the early and late clinical development of cellular products. Finally, we address the need for appropriate in-process and quality controls and the regulatory requirements and options for improvements of a cellular product at different manufacturing stages.