Eligibility Module - DNA Damage Repair Assay

This gene therapy eligibility assay measures the (patient´s) target cell´s capacity for repair of DNA damage caused by vector integration. This assay will be applied to patient iPSC or differentiated cells, next to a range of cell standards that are either proficient in non-homologous DNA damage repair or have mutations preventing repair following exposure to integrative gene therapy vectors. The predictive assay will enable patient cells to be identified as susceptible or not to gene therapy vector induced genome damage and if a normal DNA damage repair profile is determined these cells will then be subject to comprehensive genotoxicity profiling with the intended gene therapy vector for therapy.