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Development of conditionally immortalised cell lines as novel cell models of disease and for cell transplantation

Summary of the impact

Research by Professor Parmjit Jat (first at the Ludwig Institute for Cancer Research, then part of UCL; later at the UCL Institute of Neurology) established and applied the critically important scientific concept of conditional immortalisation to a wide variety of cell lines, enabling cells to be grown indefinitely in vitro but differentiate upon altering the growth conditions. Two companies were established in partnership with Jat to exploit this research, ReNeuron (now worth £63.5m and publicly traded on the London AIM market) and XCellSyz (now part of Lonza AG). More than 20 patents based on Professor Jat's work have been issued. Reagents based on his research have been evaluated, licensed and used by 17 companies worldwide: Amgen, Amylin, Boehringer Mannheim, Cell Genesys, Chiron, Eli Lilly, Genentech Inc., Genetics Institute, Immunex, Johnson & Johnson, Medarex, Novartis, Ortho Pharm., Pfizer Inc., Regeneron, ReNeuron, Takeda, EMD Serono, and XCellSyZ/Cambrex Bioscience/Lonza.

Submitting Institution

University College London

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology
Medical and Health Sciences: Oncology and Carcinogenesis

06_A portfolio of stem cell culture products is sold worldwide.

Summary of the impact

Impact on commerce: Five stem cell culture products derived from UoE research have been brought to a global market since 2009 through the US based company StemCells Inc. StemCells Inc strategically acquired Stem Cell Sciences plc (SCS), with its licensed portfolio of UoE patents, to position themselves as a world leader in cell-based medicine. This enabled them to develop media and reagent tools in order to pursue nearer-term commercial opportunities. These products include the gold standard media for embryonic stem cell culture, iSTEM.

Beneficiaries: Commercial companies and users of the stem cell culture products.

Significance and Reach: iSTEM is the gold standard media used worldwide by researchers for maintaining mouse ES cells in their basal, non-differentiated state. Products are sold worldwide through global life sciences companies.

Attribution: All research was carried out at UoE between 1994 and 2006 (published up to 2008), led by Prof Austin Smith. Collaboration with Prof Philip Cohen, University of Dundee, on one paper (2008).

Submitting Institution

University of Edinburgh

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics

15: Opening up the opportunities for stem cell therapies for neurodegenerative diseases

Summary of the impact

Neural stem cells offer enormous therapeutic potential for stroke but they require regulatory approval. Researchers at King's College London (KCL) devised a technology to immortalise stem cells, generated clinical-grade neural stem cell lines and demonstrated efficacy in an animal model of stroke. KCL research underpins the first approvals in the UK for a therapeutic stem cell product. This led to an industry-sponsored clinical trial of a stem cell therapeutic that has demonstrated vital improvement in all the first five stroke patients treated. KCL research has made a significant impact by considerably reducing the timetable for delivering potential therapies which will affect the life sciences industry and the process now in place acts as a model for other technology developments in this area.

Submitting Institution

King's College London

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Health

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology
Technology: Medical Biotechnology
Medical and Health Sciences: Neurosciences

Enabling Technology to Improve Cell Growth and Function In Vitro

Summary of the impact

3D Scaffold materials and synthetic retinoids for application in animal cell biology developed at Durham University have been commercialized by Reinnervate, a Durham spinout company, using a patent/licensing strategy. Reinnervate has raised £8m venture capital investment and has employed up to 25 people since 2008. Polystyrene-based highly porous polymers which act as 3D in vitro cell culture scaffolds were launched as Alvetex® in November 2010 and a retinoid derivative, designed to control cellular development including stem cell differentiation down neural pathways, was launched as ec23. Alvetex® was voted one of `The Scientist' magazine's Top 10 Life Science Innovations of 2010.

Submitting Institution

University of Durham

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology
Medical and Health Sciences: Neurosciences

V: Dolly the sheep - the first cloned mammal and a public icon for regenerative medicine

Summary of the impact

Impact: Public engagement and education, influence on public ethical and scientific policy.

Significance: The first demonstration of cloning from an adult mammalian somatic cell has stimulated rolling religious, ethical, cultural, political and scientific debates. Dolly has become a scientific icon entering the public and educational lexicons in addition to scientific ones.

Beneficiaries: Human society, culture, education.

Attribution: Wilmut and colleagues (Roslin Institute, UoE), undertook somatic cell nuclear transfer and used it to perform the first successful cloning of an adult mammal.

Reach: Worldwide: Dolly became a scientific icon that is recognisable all around the world, representing a major public engagement with bioscience. For example; cloning principles are part of high school education including the International Baccalaureate (implemented in >3600 schools on five continents).

Submitting Institution

University of Edinburgh

Unit of Assessment

Clinical Medicine

Summary Impact Type

Societal

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics
Medical and Health Sciences: Paediatrics and Reproductive Medicine

Targeting treatment and improving lives in the diabetes community

Summary of the impact

Research from the University of Brighton's Diabetes Research Group (DRG) has underpinned the translation of beta-cell replacement therapy into clinical application for the treatment of type 1 diabetes (T1D) to establish the world's first government-funded islet transplant service at six new UK islet-transplant centres. As a consequence, the first 65 successful islet transplants were performed, reducing the incidence of hypoglycaemic events by >95% and providing a life- changing therapy for patients. Through a leadership role in Diabetes UK, BONE integrated the interests of stakeholders and professionals to establish a new model for diabetes care. With the Juvenile Diabetes Research Foundation, in 2013 he launched in Parliament the first UK T1D Research Roadmap.

Submitting Institutions

University of Brighton,University of Sussex

Unit of Assessment

Allied Health Professions, Dentistry, Nursing and Pharmacy

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Immunology

Improved efficiency for derivation of mouse embryonic stem cells: reducing use of animals and saving costs in life sciences

Summary of the impact

Mouse disease models provide an invaluable tool to the medical sciences, underpinning the understanding of disease mechanisms and the development of therapeutic interventions. A new cultivation protocol for deriving mouse embryonic stem (ES) cells was developed by Dr Nichols between 2006 and 2009. This has facilitated the production of ES cells from disease model mice that can be manipulated in vitro and used to establish modified transgenic mice with the required genetic profile, in a single generation. This method reduces the number of mice needed, as well as associated costs and staff time, by 90%. Dr Nichols has trained industry delegates from international transgenics companies and transgenic facility managers in the new technology. As a consequence, a minimum of 26820 fewer mice have been used in experiments, and a minimum of £536k have been saved since 2009.

Submitting Institution

University of Cambridge

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics
Technology: Medical Biotechnology

5 Chemical and Materials Technologies for Cell Biology

Summary of the impact

3D polyHIPE scaffold materials and synthetic retinoids developed at Durham University for applications in cell biology have been commercialized by Reinnervate, a Durham spin-out company, using a patent/licensing strategy. Reinnervate has raised £8m venture capital investment and has employed an average of 12 FTE staff since 2008, peaking at 27 in 2012. Polystyrene-based highly porous polyHIPE materials which act as 3D in vitro cell culture scaffolds were launched under the Alvetex® brand in November 2010 and a retinoid derivative, designed to control cellular development including stem cell differentiation down neural pathways, was launched as ec23®. The products have won several awards and Alvetex® was voted one of "The Scientist" magazine's top 10 Life Science Innovations of 2010.

Submitting Institution

University of Durham

Unit of Assessment

Chemistry

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology
Engineering: Biomedical Engineering

The Development of Stem Cells for Regenerative Medicine

Summary of the impact

Research on stem cells has led to an explosion of interest in the field of regenerative medicine, with the potential for new clinical interventions and treatments. Pioneering research in Sheffield led to the founding of a spin-out company, Axordia, in 2001, focussed on the applications of human embryonic stem cells (hESC) in medicine. Several hESC lines (including SHEF-1) were generated in Sheffield by Axordia, which was sold to Intercytex in 2008 for £1.68M. These Sheffield-derived hESC lines were then sold on to a major pharmaceutical company, Pfizer, for £0.75M in 2009. As a result, a clinical grade derivative of SHEF-1 has been developed and approved for clinical trials for treating age-related macular degeneration (AMD). In addition, Sheffield research has led to the licensing and sales of key hESC marker antibodies for stem-cell quality control. Finally, Sheffield researchers have informed emerging regulatory guidelines about the safety of hESC regenerative medicine applications by authoring reports and providing evidence to a Parliamentary committee. The case study has significant impact on commerce, health and welfare and public policy.

Submitting Institution

University of Sheffield

Unit of Assessment

Biological Sciences

Summary Impact Type

Health

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology
Technology: Medical Biotechnology

The development of HLA-peptide tetramers and their application as a novel form of cell therapy for immune suppressed patients suffering from cytomegalovirus infection

Summary of the impact

T lymphocytes recognise antigens in the form of an HLA-peptide complex. HLA-peptide tetramers consist of a fluorescent HLA protein and peptide which together bind to, and therefore identify, T cells that recognise this HLA-peptide complex. As such they have proven to be a revolutionary reagent in immunology. Professor Paul Moss at the University of Birmingham has played an integral role in the clinical and commercial application of tetramers, particularly around the cytomegalovirus (CMV)-specific immune response in the context of monitoring immune recovery after transplantation and pioneering a new approach for cellular immunotherapy. The impact of this research relates to the clinical management of CMV infection in immunosuppressed patients and the creation of Cell Medica, a UK Biotech company pioneering tetramer-based cell therapy, thus demonstrating impact on clinical practice and the UK economy.

Submitting Institution

University of Birmingham

Unit of Assessment

Clinical Medicine

Summary Impact Type

Technological

Research Subject Area(s)

Medical and Health Sciences: Immunology

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