A GMP cell therapy facility to test cell based therapies

Cell-based therapies represent the cutting edge of research for new treatments for a variety of conditions. The global commercial cell therapy industry was estimated to have an annual turnover of £600M in 2011, and is estimated to grow to £120BN by 2025. Currently there is a significant gap in the UK's clinical research capabilities and infrastructure to deliver trials involving cell-based therapy largely because cell therapies require specialised facilities to safely manage the cells for human administration.

The specific aim of this proposal is to develop a cell therapy facility to enable the testing of cell therapies in clinical trials. This project will facilitate the delivery of clinical trials to investigate cell-based therapies (currently under development by researchers at QUB) for both respiratory failure in the critically ill and vision loss.

This state of the art facility will provide capacity for management from the storage and subsequent preparation of cell therapies for patient administration, through to the manufacturing of cell based therapies. This will strategically enhance the capabilities of the UK in cell-based therapies and provide a foundation for future significant health and socio-economic benefits to the UK.

It will be established as a recognised UK centre for translational research in respiratory medicine.

The facility will be available for use by external universities, hospitals and companies.

The proposal is to build and equip a GMP cell therapy unit capable of manufacturing Advanced Therapy Medicinal Product (ATMP) Investigational Medicinal Products (IMPs) for clinical trials within the UK. This facility will provide capacity for management from the storage and subsequent preparation of cell therapies for patient administration, through to the manufacturing of cell based therapies. This will facilitate the delivery of clinical trials to investigate cell-based therapies (currently under development by researchers at QUB) for both respiratory failure in the critically ill and vision loss, as well as other conditions.

The steps to achieve our licensed GMP cell therapy facility are as follows:
1. User Requirement Brief (URB). This will detail what the facility is required to do in both technical (process, capacity) and business terms.
2. Conceptual Design. This will involve the translation of the URB into a detailed design.
3. Selection of a design & build specialist to detail and implement the concept design
4. Detailed Design Phase. A detailed design of build; specification of all process equipment; design qualification for all bespoke direct impact items will be undertaken.
5. Construction, Installation and Qualification
6. Inspection and Licensing
Performance Qualification (PQ) and Process Validation / Aseptic Validation will be performed prior to applying for and receiving the manufacturing license (MIA (IMP))
7. Pharmaceutical Quality System
In parallel with the capital project, we will develop our Pharmaceutical Quality System such that documentation in the form of Standard Operating Procedures and Work Instructions are available when required during the start-up of the facility, and that training of manufacturing staff has also taken place. The three critical posts will be filled i.e. Manufacturing Manager, Quality Control Manager, and Qualified Person.

Who will benefit from this research?
The scientific community including those in the commercial/private sector will be early and major beneficiaries with clinical, basic science and industry researchers gaining a better understanding of the potential for early phase cell-based therapy studies in human respiratory disease. The infrastructure and capabilities arising from the present application will be crucial in ensuring researchers at QUB lead in the delivery of innovative cell based therapy for acute and chronic respiratory disease. In the medium to longer term patients will benefit with a direct impact on the quality of patient care both in Northern Ireland and the wider UK which will foster international recognition of UK's position as a world leader in novel therapeutic strategies for respiratory disease.
Such is the interest in stem cell therapies the applicants envisage the wider public and third sector organisations representing other important beneficiaries, "Breakthroughs" are common-place in the popular press and although this can raise unrealistic expectation, the applicants will undertake a number of initiatives to raise public awareness of science and benefits to medical research.

How will they benefit from this research?
Cell-based therapies hold great promise for treating respiratory and degenerative diseases. Consequently the research and clinical trial capability arising from this application has the potential to improve health if the pre-clinical research is positive and the therapy shows no deleterious side effects. In the long term such regenerative treatments may become a mainstay of therapy improving the health and quality of life for patients with acute and chronic respiratory disease. A major route of dissemination for the research findings and clinical trial outcomes arising as a consequence of the cell based GMP facility will be published in high impact peer reviewed journals including open access publications which are freely available for interested members of the public to read. To make the wider group of beneficiaries aware of the research and to make it available in a more accessible format, the following steps will be taken. Firstly, a specific webpage will be created to communicate the ongoing research in a straightforward manner. Secondly, press releases summarising key findings will be prepared with the assistance of the University Marketing and Communications Office. Thirdly, the applicants will take all available opportunities to engage stakeholders through speaking at scientific meetings and in partnership with the media contribute to public interest articles catering for audiences beyond the academic community. Any potential IPR stemming from the work will be explored in line with MRC policy on intellectual property and channelled through the University's technology transfer program. Any commercialisation is likely to be pursued within the UK, which would increase wealth and foster economic competitiveness within a knowledge-based economy.