Gene Therapy Innovation and Manufacturing Centre (GTIMC)

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Gene therapies rely on engineered virus carriers as vehicles for the delivery of synthetic genes that allow correction of disease-altered changes in multiple organs of the human body. Viruses exploited in gene therapy approaches have been modified to remove harmful properties and carry the therapeutic gene of interest. Multiple gene therapy programmes are currently undertaken in research laboratories using relatively small-scale production of viruses which enables optimisation of the doses and administration routes as well as testing for the safety and therapeutic efficacy of interventions in animal models of disease. However, facilities required for the production of large quantities of clinical-grade viruses of consistent quality-controlled GMP grade are rare in the UK. Thus, it can easily take several years before clinical trials can be conducted. Currently, existing facilities cannot meet the escalating demand of academically-led research needs for clinical-grade virus carriers. This is significantly obstructing numerous UK-funded world-leading disease-modifying discoveries to be translated into clinical trials for human benefit. The lack of suitable GMP facilities seriously hinders the development of much-needed novel effective treatments for multiple incurable diseases which cannot be treated by conventional drug compounds. We propose to address the manufacturing shortage by creating a Gene Therapy Innovation and Manufacturing Centre (GTIMC) which includes provision of a new state-of-the-art GMP manufacturing facility to support gene therapy projects emerging from UK universities. GTIMC will also support the development of improved viral vectors, improved yield from the manufacturing process and will also provide essential regulatory and training support. Moreover, the GTIMC hub will allow new training and high-skilled employment opportunities through the ShefVec facility itself, and future start-up companies.