Understanding raw material performance: quality and consistency of cytokines for translation to the clinic

Abstract

A deep understanding of origin, performance and quality of raw materials in cell and gene therapy development is crucial, but international standards for these raw materials are still missing in our industry. The challenge lies in the fact that raw biological materials are inherently variable, while batch-to-batch consistency is essential for successful and long-term commercialization of therapies. There is also the big debate of when to apply rigid standards, is commercial-scale manufacturing too late? One category of raw materials that require further standardization and characterization is cytokines, growth and differentiation factors (here named cytokines for simplicity). Cytokine quality and performance are directly linked to the clinical and commercial success of a therapy. However, there are important quality considerations to address during preclinical research to ensure your therapy is set up for regulatory and commercial success. Even cytokines that were originally developed for other uses, including those used as human therapeutics themselves, are not necessarily suited for use in cell and gene therapy manufacturing. Limited information on the potency and other critical attributes of the materials makes it difficult to define specifications for those reagents and to investigate the material. This article will explore ways of easing translation from preclinical development into the clinic, the importance of using animal-derived component-free (ADCF) cytokines, how to compare cytokines from different vendors and the value of international units of measurement.