Assessing a single-cell multi-omic analytic platform to characterize ex vivo-engineered T-cell therapy products
2024
Maryam Moshref | Jerry Hung-Hao Lo | Andrew McKay | Julien Camperi | Joseph Schroer | Norikiyo Ueno | Shu Wang | Saurabh Gulati | Somayeh Tarighat | Steffen Durinck | Ho Young Lee | Dayue Chen
Genetically engineered CD8+ T cells are being explored for the treatment of various cancers. Analytical characterization represents a major challenge in the development of genetically engineered cell therapies, especially assessing the potential off-target editing and product heterogeneity. As conventional sequencing techniques only provide information at the bulk level, they are unable to detect off-target CRISPR translocation or editing events occurring in minor cell subpopulations. In this study, we report the analytical development of a single-cell multi-omics DNA and protein assay to characterize genetically engineered cell products for safety and genotoxicity assessment. We were able to quantify on-target edits, off-target events, and potential translocations at the targeting loci with per-cell granularity, providing important characterization data of the final cell product. Conclusion: A single-cell multi-omics approach provides the resolution required to understand the composition of cellular products and identify critical quality attributes (CQAs).
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