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In Vitro That Feels Like In Vivo 

We culture your human target cells in a 3D in vitro environment that mimics their natural biological state. Cellular and molecular events map more closely to those that could occur in patients, giving increased ability to predict vector safety and efficacy. 

Methylation analysis reveals gene therapy vector design influence on methylation changes in the host. We use probes to examine over 850,000 methylation sites in the human genome to profile vector interactions resulting in changes in the expression of cancer genes . 

The proliferation assay clonaly tracks vector integrations in cancer genes that drives the outgrowth of subclones in bulk populations. A proliferation index is determined for the vector under analysis over a 30 day period.

 

Insertion site selection in cancer genes has been shown to be important for vector characterisation. Likelihood/risk of insertional mutagenesis are used in IND submissions and post-market monitoring.

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Measurement of gene expression changes in genes provide outreads of unwanted vector effects that could lead to oncogenesis. Vector associated changes are made using i) RNASeq to measure differential gene expression or ii) q-RTPCR to measure the expression of cancer genes local to vector IS from clonal samples.

Gene truncation is known to be associated with cancer and genotoxicity by gene therapy vectors following interactions of the vector with the host genome. OMICS involving data from our proliferation assay, differential gene expression and novel truncations are essential to the mechanism of genotoxicity and vector design

  • Our patented in-vitro platform supports the growth of 3D human iPSC and differentiated liver cells to accurately map how your GT vector is likely to affect real human tissue 

  • Rapid molecular vector safety profiling is carried out using known vector-associated biomarker drivers of oncogenesis from vector-infected cells

  • Robust data is generated  to fuel your IND submission with clear and easy to understand outreads

  • Provides confidence in vector safety and valuable direction for safe and effective vector design

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Bioinformatic and Data Analytics

 

Our OMICS analyses uses the ʰInGeTox suite of assays to detect and measure a calibrated outread of the genotoxicity of the viral vector under test against standard genotoxic and non-genotoxic “control”  vectors to provide important information on vector side effects and ways to design safer vectors.

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