Mission Bio, a leader in single-cell multiomics solutions for precision medicine, announced the release of data from scientists at Genentech’s Analytical Development Group, showcasing the successful utilization of Mission Bio’s Tapestri platform to enhance the characterization of gene-edited CD8+ T-cell therapies. Detailed in the journal Frontiers of Bioengineering and Biotechnology, Genentech scientists showed the value of Mission Bio’s DNA + protein multiomic assay for characterizing their triple gene-edited cell therapy, revealing heterogeneity in the combinations of on-target and off-target edits made in each cell as well as the correlation to cell subtype (immunophenotype).
In a significant advancement for cell therapy developers, the paper, titled “Assessing a single-cell multi-omic analytic platform to characterize ex vivo-engineered T cell therapy products,” demonstrates how Mission Bio’s analysis provided unparalleled resolution to identify critical quality attributes (CQAs) essential for CMC procedures.
“We are thrilled to share our impactful work published in Frontiers 2024, in which we assess the utility of single-cell analysis in characterizing multiplex editing of ex vivo cell therapies,” said co-author Maryam Moshref, PhD. “By leveraging single-cell DNA sequencing, we uncovered diverse editing outcomes, including variations in zygosity and the presence of off-target edits and translocations. Additionally, by integrating immunophenotypic information, we concluded that these editing events are evenly distributed across CD8+ T cell subpopulations. This comprehensive characterization, which surpasses the capabilities of bulk analysis, is crucial for truly understanding the outcomes of edited cell products.”
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Until now, analytical characterization represented a major challenge in the development of genetically engineered cell therapies, especially when assessing the potential for off-target editing and product heterogeneity. Tapestri addresses these hurdles, often missed by traditional bulk sequencing methods, ensuring a deeper understanding of cellular product composition and safety.
“Our publication underscores the critical importance of thorough characterization in the quality assurance of novel cell therapies developed through genome editing,” said co-author Ho-Young (Grace) Lee, PhD, founder of Elevalue Consulting. “Our study demonstrates the power of single-cell DNA multi-omic analysis in characterizing editing and correlating genotype and phenotype at the individual cell level. As we move into the next era of medicine, this meticulous level of analysis is essential for identifying CQAs and ensuring the safety and efficacy of these advanced therapies.”
“When we introduced translocation analysis to the Tapestri Genome Editing Solution earlier this year, we knew it would accelerate researchers’ understanding of genome-edited cells,” said Todd Druley, Chief Medical Officer, Mission Bio. “The work by the Genentech team is a powerful validation of Tapestri’s unmatched ability to ensure the next generation of critical cell therapies is both safe and effective.”
SOURCE: Businesswire