Mission Bio, a leader in single-cell multi-omics solutions, has teamed up with Integrated DNA Technologies (
IDT), a prominent force in genomics, to unveil an innovative workflow designed to enhance precision in gene editing applications. This collaboration fuses IDT’s celebrated rhAmpSeq™ technology with Mission Bio’s Tapestri® Platform, offering a nuanced and comprehensive method for evaluating genome editing results at a single-cell level.
The field of genome editing is essential for the advancement of cell and gene therapies, which rely on accurate genetic modifications. However, one of the ongoing challenges is ensuring that these edits are both specific and efficient, particularly within the sphere of cell and gene therapy (CGT). Conventional bulk sequencing methods, while insightful, lack the detail needed to discern single-cell variations, zygosity, and the simultaneous occurrence of multiple edits in individual cells.
To address these issues, Mission Bio and IDT have combined IDT’s rhAmpSeq amplicon-based targeted resequencing with Mission Bio’s droplet-based single-cell DNA sequencing. This integrated workflow builds on established methodologies that researchers have previously utilized, enhancing the precision with which on-target efficacy and off-target edits are detected in a single-cell context. This allows researchers to achieve an unprecedented level of accuracy and confidence in genome editing analysis.
The integrated approach will be showcased at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. A proof-of-concept study used well-characterized cell lines, selected for their comprehensive bulk sequencing data, to validate the workflow. By focusing on loci with known indel variations, the study demonstrated the effectiveness of rhAmpSeq chemistry in single-cell applications. The targeted panel was compatible with both bulk and single-cell assays, facilitating seamless adoption across different research requirements.
Brian Kim, CEO of Mission Bio, highlighted the value of combining single-cell multi-omics with bulk sequencing to enhance specificity, saying that integrating their capabilities with IDT’s trusted bulk sequencing approach adds multiple layers of genetic insights in a single analysis.
The Tapestri Platform’s automated single-cell genome editing analytics pipeline quantifies editing efficiency, off-target editing, and zygosity, offering data resolution beyond what bulk sequencing provides. By merging rhAmpSeq’s advanced primer design with Mission Bio’s single-cell DNA resequencing, the new workflow delivers a cost-effective, scalable, and customizable solution for validating genome editing in CGT applications.
Sandy Ottensmann, VP/GM of Gene Writing and Editing at IDT, emphasized the collaboration's role in advancing safer and more efficient genome editing platforms. The combination of rhAmpSeq’s precision with Tapestri’s single-cell resolution aims to aid developers in creating safe, transformative therapies.
The presentation at the ASGCT Annual Meeting will further detail this innovative approach. Mission Bio and IDT will present a poster on the study’s findings, illustrating the robust capabilities of integrated single-cell sequencing for validating genome editing.
Mission Bio, founded in 2014, is renowned for its Tapestri Platform, which offers unmatched granularity and precision in single-cell DNA and multiomics analysis. This platform is pivotal in fields like
cancer research, pharmaceutical development, and advanced cell and gene therapies, enabling tailored and effective treatment strategies. Researchers worldwide rely on Tapestri for identifying rare cell populations, understanding therapeutic resistance and response, and setting quality metrics for cutting-edge medical treatments.
Mission Bio has received investment from notable firms, including Mayfield Fund,
Novo Growth,
Cota Capital, and
Agilent Technologies, and continues to set standards in personalized medicine and targeted therapies.
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