Assessing a single-cell multi-omic analytic platform to characterize ex vivo-engineered T-cell therapy products

• DOI: 10.3389/fbioe.2024.1417070
• Publication Date: 2024-08-20T05:07:42Z
• Authors: 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
• Keywords: Ex vivo; Cell therapy

Genetically engineered CD8 + T cells are being explored for the treatment of various cancers. Analytical characterization represents a major challenge in development genetically cell therapies, especially assessing potential off-target editing and product heterogeneity. As conventional sequencing techniques only provide information at bulk level, they unable to detect CRISPR translocation or events occurring minor subpopulations. In this study, we report analytical single-cell multi-omics DNA protein assay characterize products safety genotoxicity assessment. We were able quantify on-target edits, events, translocations targeting loci with per-cell granularity, providing important data final product. Conclusion: A approach provides resolution required understand composition cellular identify critical quality attributes (CQAs).