Cell & Gene Therapy
Cell and gene therapies are important novel approaches to activate immune response, repair pathogenic mutations, and to repair or replace damaged tissue or cells. Ensuring correct genetic modification of these products is key in ensuring safe and effective therapies, and is important to demonstrate unambiguously during the regulatory approval process.
Cergentis' TLA technology contributes to upstream and downstream stages of cell and gene therapy product development and production.
Quality control in the upstream process
TLA analyses support:
- Optimisation and validation of new cell line generation technologies
- Clone selection
- Cell line characterisation
- Clonality assessment
- Genetic stability analyses
Quality control in the downstream process
In downstream processing of gene and cell therapy products, TLA can map viral integration sites and quality control the results of targeted gene editing.
TLA-based targeted sequencing is used to:
- Quantify the number of correct and incorrect integration and/or gene editing events at an intended location
- Assess the targeting of a CAR to the TRAC locus
- Completely sequence the HIV genome and determine integration sites in a patient's blood sample
- Evaluate transfected/transduced immune cell samples (e.g. T- and B-cells). For example, towards the development of CAR-T cell therapies
- Application notes
- Application note on targeted complete NGS and QC of transgenes and integration sites in upstream cell and gene therapy manufacturing
- Application note on targeted complete NGS and QC of transgenes, integration sites and gene editing in heterogeneous cell and gene therapy products
Bae DH et al. (2020)
FDA & NIH
Straetemans T et al. (2019)
University Medical Center Utrecht, Utrecht University & Cergentis
Chen YH et al. (2020)
Sachdeva M et al. (2019)
Eyquem J et al. (2017)
Memorial Sloan Kettering Cancer Center & Sloan Kettering Institute
Improved transgene & integration site sequencing in cell and gene therapy manufacturing
Cergentis supports all leading pharmaceutical companies in their cell line development and quality control of genetically engineered cells.
Our team of PhD-level scientists has extensive experience characterising plasmid and viral integrations in for example viral producing cell lines and CAR-T products.
Through this, Cergentis has established its position as a trusted partner and leader in the genetic analysis of genome-engineered therapeutic cell lines.