Gene Therapy Innovation and Manufacturing Centre (GTIMC)

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.

Current UK facilities for manufacturing viral vectors suitable for human treatment cannot meet demand, thus seriously hindering the translation stage of moving exciting gene therapies into clinical trials. Our aim is to establish a Gene Therapy Innovation and Manufacturing Centre (GTIMC) to address key challenges facing academic groups. The GTIMC will integrate multidisciplinary partners from the Midlands, Wales and North of England with the aim to bring complementary expertise that we have identified as essential components to unravel the existing knowledge gaps focused on adeno-associated virus (AAV) manufacturing, analytics, process innovation, safety, regulatory, clinical pathways, training and commercialisation. The GTIMC includes provision of a new build state-of-the-art GMP manufacturing facility (ShefVec). This new facility, alongside the existing excellent track record and exciting initiatives in gene therapy research within the hub, will cement the hub as a leader in innovation, manufacturing and human gene therapy translational research. ShefVec, to be GMP accredited, will utilise highly efficient purification processes to generate clinical grade AAV and provide all the necessary quality assurance, regulatory certification and governance. ShefVec will enable provision of clinical grade material for human trials at Advanced Therapies Treatment Centres, the NHS hospitals Trusts within the GTIMC and the National Network. Merit, which was a key supplier for the Catapult build in Stevenage, proposes ShefVec to be constructed using a modular design. GTIMC will also develop a translational team, including a solid regulatory platform, to support further development and commercialisation of the selected projects. The hub offers opportunities for expanding training and skills through existing MSc courses in advanced therapies, ATTC training, links to the apprenticeship community, and establishing new PhD programmes in this space.