Cell and Tissue-based Regenerative Medicine Products: From Characterization to Compendial Assays

Cell therapy and tissue-engineered products represent a growing segment of the biotechnology industry. However, the development, clinical translation, and marketing authorization of these products faces many challenges, including the lack of established quality attributes to define and assess the finished products. Living cells, unlike classic pharmaceutical products, have the potential to change their properties during manufacturing—sometimes intentionally, such as through differentiation; in other cases unintentionally, such as through loss of functional activity. This creates a challenge for characterization and standardization.

Currently, USP–NF contains tissue monographs describing assays that have been developed and validated to ensure that a specific process yields a product with the necessary quality attributes. In some cases, these assays have not been correlated to clinical outcomes, since the majority of these products are not required to undergo clinical trials prior to marketing. Only a few cell therapy products are approved through the Biologic License Application pathway, with many more under development and pending regulatory approval. The quality attributes of both cellular and decellularized products vary from one product to another, and molecular markers are often used as a surrogate to determine identity, purity, or potency.

This workshop aims to bring together key stakeholders from industry, academia, and government to identify opportunities and challenges in defining relevant and appropriate characterization assays for cell and tissue-based products, to establish priorities, and to identify assays that are ready for inclusion in USP–NF.