SOUTH SAN FRANCISCO, Calif., May 2026
Mission Bio announced new data demonstrating the power of its Targeted Single-Cell DNA+RNA Assay to simultaneously analyze CRISPR genomic edits and downstream gene expression at the single-cell level in engineered T cell therapies. The findings, presented at the American Society of Gene & Cell Therapy Annual Meeting 2026, represent the first reported demonstration combining targeted DNA and RNA single-cell readouts for multiplex CRISPR-edited cell therapy characterization. The company believes the technology could significantly improve the safety evaluation and analytical characterization of next-generation gene-edited therapies as regulatory scrutiny around cell and gene therapy manufacturing continues to increase.
Single-Cell Multi-Omics Reveals Hidden CRISPR Editing Complexity
Mission Bio highlighted that conventional bulk sequencing approaches often fail to detect critical cellular heterogeneity because they average signals across millions of cells, masking rare off-target edits and variable editing patterns that may carry important safety implications. Using the company’s Tapestri Platform, researchers analyzed engineered Jurkat T cell models including PDCD1 knockout cells, TRAC knockout cells, and double PDCD1/TRAC knockout cells containing a known off-target edit. The engineered cells were mixed with healthy donor immune cells to mimic realistic cell therapy manufacturing conditions and evaluate the platform’s ability to distinguish edited populations within complex cellular backgrounds.
The study demonstrated that the Tapestri Platform could identify on-target edit co-occurrence, allelic zygosity, and off-target genomic events in individual cells, capabilities not achievable using legacy bulk analytical methods. Targeted DNA panels measured gene editing efficiency and confirmed off-target editing sites, while targeted RNA analysis simultaneously quantified expression changes in immune signaling pathways, lineage markers, and therapeutic target genes within the same single-cell workflow. According to Mission Bio, this integrated multi-omic approach enables researchers to directly connect genotype with functional cellular behavior, providing deeper insights into the biological consequences of CRISPR editing.
Data Supports Regulatory Demand for Advanced Cell Therapy Analytics
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The data also revealed a strong relationship between editing zygosity and downstream biological activity. Researchers observed that bi-allelic CRISPR knockouts generated significantly greater downstream pathway changes than mono-allelic edits, demonstrating the platform’s ability to characterize dose-dependent gene expression effects at single-cell resolution. In addition, the assay successfully separated cells carrying off-target edits from intended therapeutic cell populations and identified distinct aberrant signaling patterns associated with those unintended modifications.
Mission Bio emphasized that regulators including the FDA and European Medicines Agency (EMA) are increasingly focused on defining Critical Quality Attributes (CQAs) for advanced cell and gene therapies at much higher analytical resolution. The company believes single-cell multi-omics may become essential for establishing the orthogonal safety evidence required for future CRISPR-engineered therapeutics, particularly as multiplex gene editing strategies become more sophisticated and clinically complex.
Company executives stated that the platform was specifically developed to simultaneously resolve genotype and phenotype within the same individual cell because rare outlier cells can carry significant therapeutic or safety consequences in cell therapy manufacturing. The newly presented data reinforce Mission Bio’s positioning in the rapidly growing single-cell genomics and cell therapy analytics market, where demand continues to rise for technologies capable of improving quality control, therapeutic precision, and regulatory confidence in next-generation engineered cell products.
Source: Mission Bio press release
