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Scientific presentation

A New Single-Cell Sequencing and Analysis Platform to Accelerate Cell and Gene Therapy Pipelines

Matt Cato & Shu Wang, Mission Bio

Cell and gene therapies (CGTs) hold promise for the treatment of a variety of diseases including cancer and rare genetic disorders such as sickle-cell disease. Many of these therapies involve the genetic engineering of cells in order to insert exogenous sequences or alter existing genes. After the cells are altered, they are introduced into the patient where they either replace dysfunctional cells or provide a desired immune response. The use of engineered cells for human therapies requires sensitive and robust characterization of genomic manipulations, which are typically achieved through viral transduction or the use of gene editors, such as CRISPR.

All methods of genetic engineering yield cell populations with genetic heterogeneity. For instance, there may be cell-to-cell variability in transduction efficiency and copy number (for viruses), or for on- and off-target editing efficiencies (for gene editors). Because unintended genomic alterations may have consequences to the efficacy and safety of therapeutic agents, it is imperative that engineered cells be characterized with a high degree of accuracy and sensitivity. Measuring genomic manipulations at a single-cell level provides the most robust characterization of CGT products.

Hear from Shu Wang, Ph.D., and Matthew Cato from Mission Bio about how highly sensitive single-cell DNA sequencing (scDNA-seq) on the Tapestri Platform accelerates CGT workflows. They discuss the advantages of Tapestri for the quantification of cell-specific alterations achieved through viral vectors and gene editors, and contrast this approach to conventional multi-assay processes. They also explain how integrating Tapestri into the CGT pipeline —from development to manufacturing and release testing — consolidates workflows and accelerates bringing breakthrough therapies to market. Lastly, Shu and Matthew discuss how Mission Bio’s Pharma Assay Development (PAD) service enables biopharmaceutical companies to seamlessly evaluate and integrate single-cell technology into their current CGT programs.


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