Raw Materials: Perspectives on raw and starting materials risk assessment for cell and gene therapy (CGT) processes

The cell and gene therapy (CGT) field utilizes innovative manufacturing processes that bring distinctive requirements for raw or starting materials. The risk assessment of raw materials in the wider biopharmaceutical industry has been the subject of much study and review. The BioPhorum C&GT team has, therefore, sought to share some of the unique challenges of raw materials for CGT manufacturers when working with the suppliers of materials. Wide range of processes and materials in the CGT field makes it challenging for the team to provide detailed advice. Instead, it suggests areas where users should focus their effort and exercise flexibility when assessing individual situations. Topics considered include patient safety, product potency and adventitious agents, as well as the broader supply chain and material quality considerations.

Three case studies are included to provide practical examples and highlight some challenges of managing raw materials for CGT manufacturing. The first example is the frequently used transfection reagent polyethylenimine. The second is a plasmid that is used to transfect cells and generate viral vectors for (AAV) adeno-associated virus processes. These two items can be obtained from multiple sources, so a comparison between suppliers is possible. The final example is magnetic beads coated with antibodies, used as a cell selection reagent in cell therapy manufacturing, with only one available supplier.