The development of next-generation cell and gene therapies holds enormous promise for providing curative solutions to a host of genetic diseases. Yet, these therapies are inherently complex and difficult to characterize. The key to the continued success of gene and cell therapy development is deepening our understanding of the variation that exists in therapeutic agents and their effects early in discovery.
With Tapestri, you can simultaneously measure multiple genotypic and phenotypic parameters in the same cells, achieving a more comprehensive characterization of your ex vivo cell therapeutics. With rich analyses serving as a foundation, you can more robustly identify critical quality attributes and accelerate your path to commercialization.
For in vivo gene therapies, Tapestri offers comprehensive single-cell analyses of your model systems, providing valuable insights about the delivery, efficacy, and safety of your therapeutic.
Ex vivo and in vivo gene and cell therapy developmentEx vivo cell therapies (left) involve collecting cells from the patient or a donor. The cells may then be genetically modified before they are expanded and infused into the patient. In vivo gene therapies (right) are administered directly to the patient using a vector, such as adeno-associated virus (AAV) or lipid nanoparticles.
Genetic modifications during ex vivo cell therapy development are made to induce a therapeutic effect. Yet, these genetic alterations are not consistent across cells — some cells may contain the desired change, while others do not. Whether you are using viral integration of gene editing to alter DNA, Tapestri measures these modulations in thousands of individual cells per sample.
Similarly, Tapestri enables deeper analysis of in vivo gene therapies — such as cell-to-cell variability in the delivery of your vector within your target cells. With richer preclinical data, you can build a robust foundation for the clinical phases of your project.
Our multi-omics assays measure cell-surface markers as an additional layer of information so that you can track immunophenotype along with genotype in each cell.
For cell therapies that have an introduced transgene, Tapestri can measure the transduction/transfection efficiency, vector copy number (VCN), and integrated vs. episomal DNA at single-cell resolution. Unlike bulk PCR assays that report population averages, Tapestri measures cell-to-cell variation in these attributes with no cell culture required.
For therapies modified by CRISPR or other gene-editing tools, Tapestri can concomitantly measure on- and off-target editing in each cell. Additionally, multiplex editing, zygosity, and translocations can be assessed. In contrast to traditional bulk NGS analysis, Tapestri delivers data from individual cells so that you can understand cell-to-cell variability in your product.
Watch our video on Tapestri for Cell and Gene Therapies.
We are your strategic partner for advancing your gene and cell therapy development pipeline.
Our Pharma Assay Development team will help develop your custom assay — from experimental design and sample processing to data analysis and interpretation. We offer services internally or in partnership with qualified CROs and are invested in bringing more accurate characterization to your early discovery efforts and beyond. We are committed to helping you bring safe, efficacious, and successful therapies to patients faster.
Azadeh Golipour, PhD, AVROBIO
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The Tapestri Platform can be used to analyze a variety of genetic alterations at single-cell resolution. The following data illustrate the powerful ability of Tapestri to assess lentiviral transduction as well as on/off-target editing, multiplexed edits, zygosity, and translocations resulting from gene editing.
