133356Establishment of a human iPS cell based cardiac gene therapv safety modelClosedFeasibility StudiesInnovate UKGene therapy (GT) has reached the clinic with substantial investment by pharmaceutical companies that include GSK, Novartis, Astra Zenica and Pfizer. Applications for clinical trials are increasing and virus based products such as ‘Stremvellis’ by GSK and ‘Glybera’ by Uniqure have been granted. In the past, GT has met with set-backs in trials in France and the UK, where children receiving treatment developed leukaemia due to virus associated side effects. No standardised safety model exists to test test GT vectors until now. Our technology won the SBRI, InMutaGene CRACK-IT Challenge to provide a human derived stem cell platform for personalised patient approach to test GT safety. With the set up of TestaVec Ltd, we will provide this to academia and industry alongside a profile of predicted GT performance. We have filed IP to also predict patient tolerance of GT products and have no competitors at present on this technology. With advice from interested regulators on set up we aim to move GT products more swiftly into the clinic. This project will introduce a cardiac cell model for GT safety to the heart to support our business plan to provide tissue specific GT genotoxicity profiling and with advice from interested regulators on set up we aim to move GT products swiftly into the clinic.