Raw and starting materials for cellular and gene therapy products

One of the fastest growing classes of therapeutics is represented by the cellular and gene therapy (CGT) sector. Many non-genetically modified cell therapies, in vivo gene transfer therapies and therapies with gene modified cells are moving from investigational phases to marketed products at an increasing rate. The sourcing and quality of raw and starting materials becomes an essential element if support of this robust pipeline can continue to meet the needs of our patients. This review is intended to assist the developers of these therapies in better understanding the definitions and classification of raw and starting materials as interpreted by US or European regulators. Phase appropriate qualification of raw and starting materials critical to CGT manufacturing processes are described with examples of starting and ancillary materials, especially allogeneic and autologous cell sources subject to recent US FDA and European regulatory interpretation. Best practices are described to better address and mitigate sourcing, variability and supply continuity risks and those Chemistry, Manufacturing and Control (CMC) requirements to support IND clinical investigations. The reader is introduced to regulatory guidances recently issued by FDA, ISO and USP pertinent to raw and starting materials. The review concludes with points to consider in addressing the realities of the supply chain ecosystem.

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