USP 1046

Standards for Cell and Tissue-Based Products

The United States Pharmacopeia (USP) information chapter USP <1046> comprises a highly comprehensive set of considerations for the development of cellular and tissue-based products—i.e., those involving live cells to be administered to humans to “repair, replace, regenerate, or augment a recipient’s cells, tissue, or organs that are diseased, dysfunctional, or injured.”

Examples of cell-based therapy products include, among many others:

  • T cells, NK cells, dendritic cells, or macrophages used for treatment of cancers
  • Encapsulated β-islet cells used for diabetes therapies
  • Autologous keratinocytes or allogeneic dermal fibroblasts for wound healing
  • Mesenchymal stem cells used in bone repair
  • Neuronal progenitor cells used to treat spinal cord injury

USP <1046> covers the gamut of cell- and tissue-based therapy development and product manufacture, from the initial sourcing of cellular and tissue material, methods of extraction and validation of manufacturing components to the quality assessment of intermediate products.

Topics addressed include:

  • Organ and tissue dissection via cutting or enzymatic digestion
  • Cell-selective centrifugation; monoclonal antibody tagging
  • Ensuring manufacturing components are qualified
  • Data acquisition methods for ensuring safety and suitability
  • Specifications for determining product quality, safety and potency
  • Acceptance criteria for product tests such as sterility, mycoplasma and endotoxin

Perhaps most importantly for quality and safety assurances, USP <1046> outlines and elaborates in detail strategies for rigorous final product release testing for cell and tissue-based products. Table 1 below summarizes specific methods and approaches to determining accurate identity, purity, potency and dose information that must be known before a cell- or tissue-therapy product can be made available for patient administration.

USP <1046> addresses the need for stability testing as well, to ensure that packaging, shipping and storage conditions are adequate for maintaining all quality attributes of a cell- or tissue-based product.


References

“A New United States Pharmacopeia (USP) Chapter 1046: Cell and Gene Therapy Products - ScienceDirect.” ScienceDirect.Com | Science, Health and Medical Journals, Full Text Articles and Books., https://www.sciencedirect.com/science/article/abs/pii/S1465324900709194.

“Cellular and Tissue-Based Products.” Drug Future, https://www.drugfuture.com/pharmacopoeia/usp35/data/v35300/usp35nf30s0_c1046.html.

Potts, Ph.D., Rebecca. “Standards and Best Practices for Cell, Gene and Tissue-Based Therapies.” CASSS Sharing Science Solutions, https://www.casss.org/resource/resmgr/cmc_no_am_jul_spkr_slds/2017_CMCS_PottsRebecca.pdf.