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Unlock Your Global Business Potential: UK Regenerative Medicine and Stem Cells

Unlock Your Global Business Potential: UK Regenerative Medicine and Stem Cells. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL.

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Unlock Your Global Business Potential: UK Regenerative Medicine and Stem Cells

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  1. Unlock Your Global Business Potential: UK Regenerative Medicine and Stem Cells

  2. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL 1960 Sir Peter Brian Medawar is awarded the Nobel Prize in Physiology or Medicine, with Sir Frank Macfarlane Burnet, for his work on graft rejection and the discovery of acquired immune tolerance was fundamental to the practice of tissue and organ transplants. • By placing your business in the UK, your company can benefit from a rich heritage in regenerative medicine and stem cells 1972 Sir Martin Evans, university of Cambridge (Nobel Prize for Medicine 2007), isolated mouse embryonic stem cells and showed that cells could be taken directly from a mouse embryo, cultured in a dish outside the mouse’s body, genetically manipulated, and then mixed with normal mouse cells to form an embryo. 1978First IVF babyborn after Cambridge university scientists: Sir Robert Edwards (Nobel Prize for Medicine 2010) and surgeon Patrick Steptoe fertilise human eggs outside the body. 1990 Human Fertilisation and Embryology Act passed by both Houses of Parliament. Human Fertilisation and Embryology Authority (HFEA) designated statutory body to enforce provisions of legislation and established in 1991. 1996Dolly the sheep is cloned by cell nuclear replacement (CNR) techniques at the Roslin Institute in Edinburgh, led by Professor Sir Ian Wilmut. 2002 UK Stem Cell Bank established as a repository for stem cells derived from adult, foetal and embryonic tissues and to be open to academics and industrialists from the UK and abroad. 2002 Sydney Brenner, a researcher at the Medical Research Council Unit in Cambridge is awarded the Novel Prize in Physiology or Medicine for his work on the genetic regulation of organ development and programmed cell death. 2003 Researchers at King’s College London, led by Professor Peter Braude, generate the UK’s first embryonic stem cell line. 2005The UK Government sets up the UK Stem Cell Initiative, with the aim of working with the public and private sectors to draw up a ten-year vision for UK stem cell research. • The UK has led the field through ground breaking innovation as well as supportive policy and initiatives, making it easy for you to tap into world class expertise and resources • International companies are already benefiting from working in the UK

  3. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL 2007Teams of researchers at Cambridge and Oxford universities independently discover a new type of stem cell in mice and rats that is very similar to human embryonic stem cells. • By placing your business in the UK, your company can benefit from a rich heritage in regenerative medicine and stem cells 2008A child suffering from gut failure was the first in the world to receive a life-saving haematopoietic stem cell transplant. Two hospitals in the UK now offer the adult stem cell therapy: Great Ormond Street and Great North Children’s. 2010ApaTech, a spin-out from Queen Mary University of London, and a world-leader in bone graft technologies is sold for $330M to Baxter. 2011UK approved Europe’s first clinical trial using human embryonic stem cells (hESCs). US based company Advanced Cell Technology (ACT) received approval from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin a trial to treat 12 patients with Stargardt's macular dystrophy. 2011ReNeuron’sPISCES study (Pilot Investigation of Stem Cells in Stroke) is conducted in the UK and is the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients. 2012the UK Regenerative Medicine Platform is established, with funding from Research Councils UK, to address the technical and scientific challenges associated with translating promising scientific discoveries in this area towards clinical impact. 2012The Cell Therapy Catapult is established to grow the cell therapy industry and support the development, delivery and commercialisation of cell therapies and advanced therapeutics in the UK. 2013The House of Lords Science and Technology Select Committee, under the Chairmanship of Lord Krebs, launched an inquiry into regenerative medicine focusing on UK’s expertise in the field, the applications of this research to treatments, the regulatory and financial barriers to translation and commercialisation of the findings of this research, and international comparisons. 2012Sir John Gurdon, of the Gurdon Institute in Cambridge, together with Shinya Yamanaka, is awarded the Nobel Prize for the discovery that mature cells can be converted to stem cells. • Recognising the challenges facing the application of regenerative medicine, the UK continues to invest in, and develop this sector, supporting world-first clinical trials and financially supporting innovative new companies with non-dilutive funding

  4. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • The UK recognises the need to support industry deliver patient benefit and achieve commercial viability

  5. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • The UK holds a leading position in the science and commercial translation of regenerative medicine. Resulting in world class resources that your company can tap into: • Cutting edge, well funded, research in cell therapy and stem cells, tissue engineering, biomaterials, medical technologies and nanotechnology • Connectivitythrough specialised networks: connecting you to the research, clinical and industry base and creating opportunities for collaboration • Talent and expertise: highly skilled researchers and clinicians and regenerative medicine experts • World-classcell and tissue banking facilities available for industry • Fundingfor UK based companies through government initiatives • Leveraged funding through collaboration with academia 1 • Collaborate and work in the UK to access world class resources that are delivering results Pfizer Regenerative Medicine opened a facility in Cambridge, UK, in 2010 to lead its move into the stem cell industry. In 2011, the operation embarked on a landmark collaboration with University College London (UCL) to develop a treatment for certain forms of blindness using stem cell therapy. Pfizer noted that “locating the new laboratory in the area will give us access to one of Europe's strongest biotech hubs”.

  6. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • You can tap into an established and accessible commercial supply chainthat is evolving and adapting to support regenerative medicine activities: • Ease of access to numerous specialist suppliers, and logistics providers, helping you deliver in a timely, cost effective manner • Access to storage solutions and established cold chain logistics experts • A well connected, efficient transport network connecting you nationally and internationally • Established expertise, accessible to industry, through the NHS Blood and Transplant (NHSBT) and Scottish National Blood and Transfusion Services (SNBTS) in cell and tissue collection and logistics 2 • Comprehensive supply chain and logistics allow you to deliver on time • and cost effectively Roslin Cells and the Cell Therapy Catapult partner to establish a source of clinical grade induced pluripotent stem cells (iPS) banked according to Good Manufacturing Practice (GMP) in the UK. The establishment of the cell bank with an initial £2m investment provides the UK with an important source of iPS cells acceptable to the regulatory authorities. These iPS cell lines will be available for clinical research in both academia and industry, with the initial six expected to be available by the end of 2014.

  7. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • Companies can work with established manufacturing facilities experienced in delivering for clinical trials • A large number of manufacturing and bioprocessing facilities • Established Good Manufacturing Practice (GMP) facilities with experience in working with industry • Advanced Therapy Medicinal Product (ATMP) manufacturing • The UK is also leading the innovative manufacturing revolution with investment into centres of excellence actively collaborating with andsupporting industry activities to develop processes and techniques for cost-effective scale up and manufacturing • Support for product developers in manufacturing innovation • Expertise in Scaffold design, fabrication and analysis 3 • Access existing manufacturing capabilities with proven ability to deliver ReNeuron is the first commercial customer for Roslin Cells’ new GMP Cellular Therapy Manufacturing Facility in Edinburgh. Under the contract, Roslin Cells will manufacture clinical-grade cell banks of ReNeuron’s CTX cell product, the basis of its ReN001 stem cell therapy for stroke and its ReN009 cell therapy for critical limb ischaemia. The work will be undertaken in Roslin Cells’ Edinburgh facility which secured its licence from UK authorities earlier this year.

  8. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • The UK is committed to delivering efficient clinical translation. Companies can access a strong health research infrastructure to deliver high quality clinical trials quickly and cost effectively • Access an infrastructure that can help you deliver • The UK is Investing £500 million annually in health research infrastructure • Support through the National Institute for Health Research (NIHR) Clinical Research Network (CRN) enabling over 25 clinical studies in regenerative medicine to take place within the NHS at the current time • Access to a single healthcare system that treats 60 million patients, generating vast volumes of structured real world data. • Access to specialist clinical expertise – Clinician led translation from bench to patient • Streamlined approvals for clinical research (Health Research Authority) • Simplified access for industry through the NIHR Office for Clinical Research Infrastructure (NOCRI), NHS Research Scotland (NRS) Permissions Coordinating Centre and Health Research Wales 4 • Access a strong clinical research infrastructure allowing you to deliver clinical trials efficiently An ongoing phase II trial to compare Autologous Chondrocytes Implantation (ACI) versus existing techniques for knee cartilage repair is being carried out over 27 UK sites (and 2 sites in Norway) with 400 patients recruited. The trial is sponsored by Keele University and is hosted by Jones & Agnes Hunt Orthopaedic Hospital NHS Trust.

  9. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • The Cell Therapy Catapult has been established to accelerate delivery of therapies to the patient and will work with industry by: • Taking products into clinical trial, de-risking them for further investment • Providing clinical expertise and access to NHS clinical partners • Providing technical expertise and infrastructure to ensure products can be made to GMPand delivered cost effectively • Providingregulatory expertise to ensure that products can get to the clinic safely in the shortest time • Providing opportunities for collaboration, nationally and globally • Providing access to business expertise networks and finance opportunitiesso that commercially viable products are progressed and investable propositions aregenerated • The UK hosts numerous experienced commercial and business advisors and consultancies • The UK regenerative medicine Industry has a united voice through the BioIndustry Association’s (BIA) Cell Therapy and RegenMed Advisory Committee 5 • The UK provides commercial support for all your activities The Cell Therapy Catapult and ReNeuron are collaborating on new cell therapy manufacturing technologies and assays, focusing on the development and optimisation of the processes required to scale up manufacture of the CTX cell line. The Catapult will contribute £1.3 million into the collaboration - in the form of expert knowledge, plus state-of-the-art laboratories, equipment and services, while ReNeuron will also provide facilities staff and relevant expertise.

  10. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • Accessing the UK market, and the National Health Service (NHS), is of strategic importance, increasing the probability of successful entry into the European market as evidenced by the number of European Headquarters in the UK: • A clear, well established regulatory framework making the UK a recognised route into the European market • Regulatory advice and support through the Medicines and Healthcare products Regulatory Agency (MHRA) and other organisations • Guidance and recommendations by the National Institute for Health and Care Excellence (NICE) are respected Europe-wide. • The Scottish Medicines Consortium (SMC), in Scotland • The NHS allows you to deliver robust clinical trial data to support market access • The NHS has the potential to be a great test bed for regenerative medicine products • NHS England directly commissions specialised services which allows consistent national clinical policies to be developed and implemented. The direct commissioning approach includes a process of Commissioning through Evaluation (CtE). 6 • The UK is your gateway to Europe and the global markets In April 2013, the MHRA approved Athersys’ application to expand its ongoing Phase 2 study evaluating the administration of MultiStem® therapy to patients who have suffered an ischemic stroke. Ethics Committee review was been successfully completed in August, 2013, and enrolment at multiple United Kingdom sites will commence following the completion of final preparations at participating clinical centres.

  11. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • The UK business environment has changed; new fiscal incentives have been created to support life sciences and innovation • The UK government has introduced fiscal measures to stimulate innovation and growth for companies • There has never been a better time to develop and grow a commercial footprint in the UK in this sector 23%Corporation tax will drop further to 20% R&D tax credits for SMEs worth approximately 25p on every £1 (7p for large companies) Low corporation tax rate currently standing at 23 per cent decreasing further to 20 per cent by April 2015. £180m Biomedical Catalyst = £180m programme of public funding for growth Patent Box £300m Patent box: 10% corporation tax on qualifying profits To stimulate R&D partnerships between universities businesses & charities

  12. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL You can work with leading clinicians and clinical centres to deliver your clinical trials easily, efficientlyand to a high quality. • Clinical success: the UK is one of the leading European countries developing Advanced Therapy Medicinal Products Figure 1: Clinical trials, in the UK, by therapeutic area • 34cell therapy clinical trials and 37cell therapy pre-clinical studies (Cell Therapy Catapult) • Expertise in numerous therapeutic indications • The UK is in the top three leading European countries developing Advanced Therapy Medicinal Products (ATMPs) in Europe Trials by stage: Data from Cell Therapy Catapult report

  13. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL You can work with leading clinicians and clinical centres to deliver your clinical trials easily, efficiently and to a high quality. • Multi-disciplinary clinical successes in regenerative medicine from voice box reconstruction to tendon repair Stroke Huntington Disease Voice-box Reconstruction Multiple Sclerosis Rheumatoid Arthritis Ophthalmology Tendon repair Cardiovascular Cartilage repair Diabetes Immunotherapy Digestive Tract Liver cirrhosis

  14. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • Your competition is already benefiting from the UK Offer in Regenerative Medicine • Ease of doing business: a supportive business environment coupled with Government commitment to support the sector • Business incentives such as year on year reductions in corporation tax, R&D tax credits and the Patent Box, culminating in an improved business environment in the UK. • Increased funding into the sector from Government as well as UK charities • Rich and Diverse Ecosystem to tap into • World class researchers, centres of excellence and research infrastructure • A strong and growing supply chain • Established manufacturing facilities • Innovative manufacturing approaches • A single healthcare system that treats 60 million patients, generating vast volumes of structured real world data. • Unrivalled access to cell and tissue samples to enable discovery and development. • A Cell Therapy Catapult to support your activities from business models, manufacturing to clinical and regulatory expertise • Market Access: the UK, European and global markets • Clear, well established regulatory framework, harmonised for Europe • New commissioning models available for specialised products and services allowing direct access to the whole of the NHS • Strategy being developed for NHS readiness and action plan to facilitate adoption of regenerative medicine products • A track record of successful collaboration with industry • The NHS and research base working closely with industry • Available support and funding for UK based companies

  15. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • Our practical help and advice is free and confidential • UKTI’s professional trade and industry advisers operate in the UK alongside a network of experts based in all British diplomatic offices overseas. • UKTI can: • Provide tailored information on the UK offer and capabilities that match your business needs • Support your business journey to invest in and grow in the UK • UKTI’s Life Science Investment Organisation (LSIO) • The LSIO is here to help your business navigate the opportunity to partner with and invest in the UK and to connect you to the wider UK life science community • Find out how UK Trade & Investment (UKTI) can help your business For further details on the LSIO and the UK life sciences offer for industry, visit: www.ukti.gov.uk/lifesciences

  16. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • AHSC – Academic Health Science Centre • AHSN – Academic Health Science Network • ARUK – Arthritis Research UK • ATMP – Advanced Therapy Medicinal Product • BBSRC - Biotechnology and Biological Sciences Research Council • BRC – Biomedical Research Centre • BRU – Biomedical Research Unit • CPRD – Clinical Practice Research Datalink • CtE – Commissioning through Evaluation • EMA - European Medicines Agency • EPSRC – Engineering and Physical Sciences Research Council • KTN – Knowledge Transfer Network • LRMN – London Regenerative Medicine Network • LSIO– Life Science Investment Organisation • MRC – Medical Research Council • MHRA – Medicines and Healthcare products Regulatory Agency • NHSBT – NHS Blood and Transplant • NHS– National Health Service • NICE – National Institute for Health and Care Excellence • NIHR – National Institute for Health Research • NISCHR - National Institute for Social Care and Health Research • NOCRI– NIHR Office for Clinical Research Infrastructure • OSCI – Oxford Stem Cell Institute • SNBTS – Scottish National Blood and Transfusion Services • TSB– Technology Strategy Board • UKTI– UK Trade and Investment • UKRMP – UK Regenerative Medicine Platform • WT – the Wellcome Trust • Acronyms

  17. Appendix: Success Stories

  18. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL ReNeuron • ReNeuron is a leading, clinical-stage stem cell business. The company’s primary objective is the development of novel stem cell therapies targeting areas of significant unmet or poorly met medical need. • ReNeuron’s clinical trials in disabled stroke patients: The PISCES study (Pilot Investigation of Stem Cells in Stroke) is the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients. The trial is being conducted in Scotland at the Institute of Neurological Sciences, Southern General Hospital, Greater Glasgow and Clyde NHS Board. • Phase II of the stroke trial is starting with support of a £1.5 million grant from the Technology Strategy Board (TSB) • Also received regulatory and ethical approvals to commence a Phase I clinical trial in the UK with its ReN009 stem cell therapy programme critical limb ischaemia (CLI). The trial will be undertaken through NHS Tayside at Ninewells Hospital and Medical School, Dundee, Scotland. • Cell manufacturing for the trial will be carried out by Roslin Cells • Completed significant £33 million fundraising, with £7.8 million from the Welsh Government, to continue development and build a new manufacturing facility. • How the UK environment helped ReNeuron be a leader in the field

  19. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL CellMedica • Founded in 2006 by Gregg Sando. Funded by the Wellcome Trust and Imperial Innovations • Three Technology Strategy Board (TSB) grants to develop and progress novel cell therapy to treat infections in children following bone marrow transplant. Partner: Great Ormond Street Hospital (GOSH) • Two other TSB grants to support manufacturing and reimbursement models • Currently running early phase trial at GOSH – with support from TSB. • Company has grown to 41 employees with offices and laboratories in London, Berlin and Houston. Three development projects – global clinical trials. • Founded in 2006 by Gregg Sando. Funded by the Wellcome Trust and Imperial Innovations • Three Technology Strategy Board (TSB) grants to develop and progress novel cell therapy to treat infections in children following bone marrow transplant. Partner: Great Ormond Street Hospital(GOSH) • Two other TSB grants to support manufacturing and reimbursement models • Currently running early phase trial at GOSH – with support from TSB. • Company has grown to 41 employees with offices and laboratories in London, Berlin and Houston. Three development projects – global clinical trials. • How the UK environment helped Cell Medica succeed “Fortunately for us the Government had identified areas of important innovation in industry - and cell therapy was one of them…Through the Technology Strategy Board's involvement, we had the benefit of collaboration with Great Ormond Street Hospital and University College London, and the opportunity to exchange ideas with other organisations working in the same field’. Karen Hodgkin, Senior Vice President of Clinical Development “Fortunately for us the Government had identified areas of important innovation in industry - and cell therapy was one of them…Through the Technology Strategy Board's involvement, we had the benefit of collaboration with Great Ormond Street Hospital and University College London, and the opportunity to exchange ideas with other organisations working in the same field’. Karen Hodgkin, Senior Vice President of Clinical Development

  20. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Intercytex • Intercytex is a regenerative medicine product and services company focused on developing its lead product to treat a variety of skin related problems. These treatments are being brought to patients through Intercytex’s innovative Progressive Translation system which is available to third party clients through their Cell2Therapy CMO service • Intercytex operates a fully licensed GMP manufacturing facility and has experience in producing a variety of autologous and allogeneic 2D and 3D cell therapy products. Its current product Vavelta/ICX-RHY, an injectable allogeneic fibroblast suspension, is a good example of Intercytex applying the Progressive Translation approach: • Intercytex: using a Progressive Translation approach to take product to market • Progressive development:A close partnership between client, contract developer and hospital allowing iterative development of new treatments Progressive licensing: Focus on unmet medical need and orphan products in order to allow access to the current legislative frameworks already in place of conditional approvals. Progressive reimbursement:Obtain early reimbursement from a variety of sources that will both help development finances as well as providing marketing information • Orphan designation obtained in EU and US • Phase II clinical trialcompleted • Specials license obtained for Intercytex manufacturing facility • Biocatalyst grant to Improve shelf life and logistics to enable pharmacy supply • Working with Cell Therapy Catapult to produce less painful injection system • Created a partnership with University Hospital of South Manchester to develop Progressive Translation system and recently announced the expansion of their GMP facility onto the Manchester Airport City Enterprise Zone • Working with suppliers on scale-up options • Developed costing models for current system and scale up options • Obtained full hospital reimbursement to treat first patients under Specials • Working with charities to examine reimbursement possibilities “Fortunately for us the Government had identified areas of important innovation in industry - and cell therapy was one of them…Through the Technology Strategy Board's involvement, we had the benefit of collaboration with Great Ormond Street Hospital and University College London, and the opportunity to exchange ideas with other organisations working in the same field’. Karen Hodgkin, Senior Vice President of Clinical Development “The UK provides a unique mix of highly networked world-class scientists, a globally respected and helpful regulatory system, a National Health service and a succession of supportive Governments”, Dr Paul Kemp, Chief Executive and Chief Scientific Officer

  21. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Neusentis: working in partnership with academic researchers in Cambridge and across the UK Neusentis • Neusentis is a Cambridge-based Pfizer research unit that delivers 'new science therapeutics', with a particular focus on pain and sensory disorders. It works in partnership with academic researchers in Cambridge and across the UK. • Within regenerative medicine, Neusentis is developing cell based therapies for ulcerative colitis, age related macular degeneration and a drug to regenerate neural circuit function in stroke patients. • A key partnership case study is the work being done on Cell Replacement Therapy for Age Related Macular Degeneration in the ‘London Project’. ‘The London Project’ was formed by Professor Pete Coffey (Institute of Ophthalmology, UCL) and Lyndon da Cruz (Moorfields Eye Hospital) to produce a cell replacement therapy for AMD. In 2008 The London Project teamed up with Pfizer to help move the project into the clinic so as to determine whether this cell replacement therapy was safe and whether it lead to an increase in vision in patients with AMD. The work has pulled in expertise from across the whole Pfizer organisation and the team have received approval from the authorities to proceed with the trial.

  22. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL GE Healthcare Life Sciences chooses Wales for its new state of the art laboratories for cell therapy and cell bioprocessing GE Healthcare • In 2012, GE Healthcare Life Sciences, a business unit of GE Healthcare, opened its new £3 million cell science laboratories in Cardiff , Wales. The state-of-the-art investment at the company’s Maynard Centre creates is a world-class facility for research and technology innovation in cell science, to help advance the rapidly emerging fields of cell therapy and cell bioprocessing, and to support the development of new, more effective and safer medicines. • The laboratories will focus on the development of novel technologies for the rapidly emerging field of cell therapy and on advancing new cellular based tools to help pharmaceutical companies develop better and safer medicines at lower cost. The facility is equipped with the latest technologies for cell bioprocessing and cell imaging, as well as an industry-leading clean-room manufacturing facility. In addition, GE’s customers and scientific collaborators will benefit from an advanced technical training centre. “Cellular research has an extraordinary potential to address some of the world’s biggest healthcare challenges. Our investment in these new laboratories is a great milestone and demonstrates our commitment to innovation and excellence in cellular science. Here in Cardiff we have established an outstanding team of scientists, many of whom are leaders in their field, and these new facilities will allow us to greatly expand our work.”

  23. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Advanced Cell TechnologyMoorfields Eye Hospital • Advanced Cell Technology, a US-based company received permission, in 2011, by the UK Medicines and Healthcare products Regulatory Agency (MHRA) to start Europe's first clinical trial involving human embryonic stem (hES) cells. • The ongoing trial aims to treat 12 patients with Stargardt's macular dystrophy. The disease strikes people between the ages of 10 and 20, causing progressive vision loss. There is currently no treatment. • Eye surgeon James Bainbridge of Moorfields Eye Hospital and University College London leads the UK trial. • UK Approves Europe's First Embryonic Stem Cell Clinical Trial

  24. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Athersys • Athersys (Cleveland, OH, US) is a clinical stage biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem® cell therapy product, a patented, adult-derived "off-the-shelf" stem cell product platform for disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. • In April 2013, the Medicines and Healthcare products Regulatory Agency (MHRA) approved Athersys’ application to expand its ongoing Phase 2 study evaluating the administration of MultiStem® therapy to patients who have suffered an ischemic stroke. Ethics Committee review was been successfully completed in August, 2013, and enrolment at multiple United Kingdom sites will commence following the completion of final preparations at participating clinical centres. • The study is expected to enrol approximately 136 patients in total and is currently being conducted at multiple centres throughout the United States. • Athersys is working with the Newcastle Cell Therapies Facility for production of cells for the trials • MHRA Authorizes Athersys to Expand Ongoing Phase 2 Clinical Trial of MultiStem Cell Therapy in Ischemic Stroke "The MHRA authorization will enable us to bring several leading United Kingdom stroke centres into the study, which will help us to speed the completion of the stroke clinical trial. The authorization is also noteworthy as it marks the initiation of MultiStem clinical development activity in the United Kingdom.” Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys

  25. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL EPSRC Centre for Innovative Manufacturing in Regenerative Medicine collaborating with industry The EPSRC Centre for innovative manufacturing in regenerative medicine • The EPSRC Centre for Innovative Manufacturing in Regenerative Medicine provides an integrated platform of fundamental and translational research as a ‘go-to’ resource for the regenerative medicine product developer. • It is one of the few centres in the world providing academic and industrial translational research in regulated regenerative medicine manufacturing, in order to grow the industry, deliver benefit to patients, and develop the UK research community. • It is a partnership of Loughborough, Keele and Nottingham Universities and industry, with currently around 20 industry and clinical partners. Centre institutions participate in three of the UK Regenerative Medicine Platform hubs. • The Centre places an emphasis on cost-effective, robust manufacturing system and process design with a focus on both regulatory compliance and informing the regulators, in particular with respect to the implementation and principles of good manufacturing practice. • An example of the collaborative work carried out at the centre is the collaboration with Ruskinn Technology Ltd -one of the world’s leading suppliers and manufacturers of anaerobic and modified atmosphere workstations. Ruskinn was recently acquired by The Baker Company, USA - around the development of HypoxyCOOLTM. Ruskinn “The EPSRC project has provided the essential proof of principle for HypoxyCOOLTM, and through such support has better ensured positive outcomes both in terms of the application of autologous cell production and adding value to the Ruskinn business.” Huw David Thomas, UK Commercial/Sales Manager, Ruskinn Technology Ltd

  26. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL A University of Swansea spin-out joins efforts with Biotec Services International to form TrakCel TrakCel • TrakCel is an orchestration platform to safeguard patients being treated with regenerative therapies. The platform ensures that the right patient receives the right therapy at the right time and location. Efficiently tracking, tracing and documenting the movement and handling of multiple regenerative therapies with minimal resources required, using state of art technologies including biometrics, Radio Frequency Identification (RFID) and Global Positioning.  • TrakCel’s was formed as a joint venture between AwenID (a university of Swansea spin-out) and Biotec Services International (storage and distribution of unlicensed, temperature-sensitive medicinal products for use in clinical trials). • The company’s technology platform is going to be assessed by GSK in Q1 2014. As part of a Department of Business Innovation and Skills, Advanced Manufacturing Supply Chain Initiative (AMSCI) the company has received financial assistance to develop its technology for gene therapy applications.

  27. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Tissue Regenix • Tissue Regenix is a medical technology company which uses a proprietary technology platform, dCELL ®, which removes cells and DNA to make replacement body parts from biological (animal) materials. • The three priority markets for the application of the technology are: Vascular; Cardiac; and Orthopaedics • A Technology Strategy Board (TSB) quality systems project led to Tissue Regenix’sdCELL® vascular patch achieving European certification in August 2010. • The dCELL® technology, which reduces the immunological response so negating the need for immunosuppressants, is already benefitting patients worlwide. • Vital translation projects such as this can be difficult for an SME to fund privately. Further support, for Tissue Regenix’s knee meniscus material, allowed the company to successfully raise £6m through an AIM listing. • Tissue Regenix is founded on IP developed solely at the University of Leeds and they continue to work with those researchers to develop future products within the portfolio. The collaboration has also helped Tissue Regenix develop quality control methodologies for their products. • How the UK environment supported Tissue Regenix

  28. Appendix Clinical successes

  29. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL University College London • In 2008, Professor Martin Birchall, in collaboration with Professor Paolo Macchiarini, University of Barcelona, led the European Team which successfully performed the World’s first stem-cell based, tissue-engineered organ transplant, opening up new vistas for organ replacement in years to come • Claudia Castillo was given a new windpipe generated with tissue grown from her own adult stem cells, eliminating the need for anti-rejection drugs. This was a major breakthrough for science and technology • £1.2m funding from the MRC will now enable the team to take the research from one-off treatments for patients in exceptional circumstances to developing the first clinical trial of tissue-engineered larynx (voicebox) transplants; a project known as RegenVox. • Clinical Firsts: Windpipe Reconstruction

  30. University of Oxford Cell Therapy Ltd • Researchers from the University of Oxford, led by Dr Paul Fairchild, pioneered the differentiation of dendritic cells and their individual subsets from pluripotent stem cells, both embryonic and induced pluripotent stem cells (iPSC). The properties of this novel source of dendritic cells promises to overcome many of the obstacles to their use in immunotherapy. • In conjunction with collaborators at Great Ormond Street Hospital, the group plans to scale up the production of dendritic cell subsets from iPSC for use in a number of indications either requiring vaccination or the induction of tolerance. • Future clinical trials will be conducted in collaboration with Cell Therapy Ltd, for which Nobel laureate Sir Martin Evans serves as co-founder and director. • Clinical Activities: Exploiting Stem Cells for Immunotherapy

  31. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Imperial College London • Doctors at Imperial College, London, led by Professor Nagy Habib, were the first to undertake phase I and II studies to treat patients with liver failure with autologous adult bone-marrow derived cd34+ stem cells. • It is planned to popularise the therapy in the immediate future and to extend it to other indications. Clinical trials have been initiated in patients with diabetes and stroke and will be started soon for patients with cardiac disease and tibial fracture. • Clinical results to date have been encouraging. A new allogeneic stem cell therapy for acute liver failure is anticipated in the foreseeable future. • Clinical Firsts: Liver Failure

  32. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Great Ormond StreetHospital • In 2008, Michael Wenman was the first child in the world to receive a life-saving hematopoetic stem cell transplant: • At age 5 he faced death after an overactive immune system destroyed his digestive tract. At 12, he was fed intravenously and suffered constant pain until the treatment. • His younger brother Matthew, then four, was offered the same procedure when he showed similar symptoms. • Both children have since returned to a normal life. They are two of ten children who have now received the treatment.  • Two hospitals in the UK, Great Ormond Street Hospital and Great North Children’s Hospital in Newcastle, now offer the adult stem cell therapy. • Clinical Firsts: Digestive Tract

  33. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Newcastle University • Professor John Isaacs (Consultant Rheumatologist and Professor of Clinical Rheumatology) and Dr CatharienHilkens (Reader in Immunotherapy) are using the Newcastle Biomedicine Cellular Therapies facility to derive tolerogenic dendritic cells (TolDC) for a Phase I clinical trial in patients with rheumatoid arthritis (AUTODECRA). • Autologous TolDC are derived ex vivo from monocytes using immuno-modulatory drugs and are introduced arthroscopically into the knees of patients to investigate the safety, feasibility and acceptability of the therapy. • The investigators aim to treat 12 patients in total, 9 with TolDC and 3 with a control treatment. • The study is funded by Arthritis Research UK • Clinical activities: Rheumatoid Arthritis

  34. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Cardiff UniversityUniversity of CambridgeImperial College LondonAddenbrooke’s Hospital • A safety study for the transplantation of human fetal striatal tissue into the striatum of five patients with Huntington’s disease has demonstrated that the procedure is safe and feasible. • Stereotaxic tissue placement following pre-operative magnetic resonance imaging was performed in Cambridge, UK by investigators from the National Institute for Health Research (NIHR) Cambridge Biomedical Research Centre (BRC) as part of a broader UK network. • Cambridge-based protocols for the screening of human fetal tissue for potential pathogens have also been developed, with clinical trials planned in Parkinson’s disease. • This is part of a larger European collaboration with several expert centres in the UK • Clinical Activities: Huntington Disease

  35. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Newcastle UniversityThe Newcastle upon Tyne Hospitals • Newcastle’s Royal Victoria Infirmary is a centre of excellence for the treatment of mechanical, chemical or thermal damage to the Cornea. The development of methods to grow limbal epithelial stem cells by Prof Linda Lako and Prof Francisco Figueiredo of Newcastle University made this treatment possible. • Professor Figueiredo (Consultant Ophthalmologist) has successfully treated eight consecutive patients with total unilateral LSCD using LSC autografts, resulting in significant improvements in visual impairment and pain scores in all cases • The results of the phase I clinical trials show that limbal epithelial stem cell transplantation is a robust method to treat corneal damage • A larger study, sponsored by the Medical Research Council, involving 24 new patients is underway to evaluate the long-term safety and efficacy of cultured human LSC for the treatment of patients with unilateral total LSCD. • Newcastle University and the Royal Victoria Infirmary are working with the Cell Therapy Catapult to achieve European Market Authorisation • Clinical successes: limbal epithelial stem cell transplantation

  36. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL University College LondonMoorfields Eye Hospital • The Cells for Sight Team is aiming to understand the biology and therapeutic potential of stem cells (and the cells with which they interact) to develop and deliver novel cell-based therapies and anti-scarring strategies for patients with blinding corneal and conjunctival disease. • The Cells for Sight Stem Cell Therapy Research Unit is a MHRA licensed state-of-the-art GMP facility is available to internal and external researchers (academic and commercial) for the manufacture of Advanced Therapy Medicinal Products. • To date 25 patients with blinding ocular surface disease have been treated with cultured limbal stem cell therapy. The Unit is manufacturing human embryonic stem cell-derived retinal pigmented epithelial cells for transplantation in a clinical trial headed by Professor Pete Coffey 'The London Project to Cure Blindness'. • Cells For Sight Add content – Moorfields, UCL, institute of ophthalmology

  37. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Keele UniversityRobert Jones & Agnes Hunt • Keele University (Sponsor) and Robert Jones & Agnes Hunt Orthopaedic Hospital NHS Trust (Host organisation) are working together to deliver the results of a phase II trial comparing Autologous Chondrocytes Implantation (ACI) versus existing techniques for knee cartilage repair • Multi-centre: 27 UK sites and 2 sites in Norway • 400 patients were recruited onto this trial • Clinical activities: Knee cartilage repair

  38. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Scotland Scotland has established an strong infrastructure to deliver cell therapy clinical trials with direct experience in bringing Advanced Therapy Medicinal Products (ATMPs) into the clinic that meet the UK regulatory agency’s (MHRA) Good Manufacturing Practice (GMP) standards. Cell Therapy Clinical Trials currently ongoing in Scotland: • Phase I ReN001 neural stem cell therapy for patients left disabled by ischemic stroke • Phase I/II corneal epithelial stem cell transplant for corneal blindness • Phase I/II CD133 cells for chronic liver failure • Phase I CTX stem cell line for critical limb ischemia • Phase I T-cell immunotherapy for melanoma • Clinical activities in Scotland

  39. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL 4 1 • Clinical activities: Cardiovascular disease Autologous bone marrow derived mononuclear cells for acute myocardial infarction. Combines stem cell delivery with primary angioplasty within 5 hours post event Barts Health NHS Trust, Queen Mary University of London, University College London Phase I/II, recruitment target of70 patients Randomised control trial to compare the effects of G-CSF and autologous bone marrow progenitor cells infusion in patients with ischaemic heart disease Barts Health NHS Trust, Queen Mary University of London Phase II, recruitment target of 90 patients 2 3 Autologous bone marrow derived mononuclear cells for dilated cardiomyopathy, delivered via intracoronary injection Barts Health NHS Trust, Queen Mary University of London Phase I/II, recruitment target of 60 patients Expanded adult haematopoietic CD34+ stem cells for autologous infusion to patients with myocardial ischaemia Imperial College London Phase I/II, recruitment target of 42 patients

  40. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL UK Stem Cell FoundationMultiple Sclerosis Society • UK Stem Cell Foundation and the Multiple Sclerosis (MS) society fund several projects targeting multiple sclerosis. This includes clinical trials such as the phase II trials looking at the use of autologous mesenchymal stem cells to treat relapsing-remitting MS. • Led by Dr Paolo Muraro at Imperial College London, this clinical trial is part of an international collaboration involving 150 to 200 people with MS across Europe, America and Canada. • Edinburgh and London sites. • The London site aims to recruit 13 patients. • The trial will test whether stem cells can treat the active MS lesions of the participants, where damage is currently occurring on their brains. • UK Stem Cell Foundation and the Multiple Sclerosis (MS) society fund several projects targeting multiple sclerosis. This includes clinical trials such as the phase II trials looking at the use of autologous mesenchymal stem cells to treat relapsing-remitting MS. • Led by Dr Paolo Muraro at Imperial College London, this clinical trial is part of an international collaboration involving 150 to 200 people with MS across Europe, America and Canada. • Edinburgh and London sites. • The London site aims to recruit 13 patients. • The trial will test whether stem cells can treat the active MS lesions of the participants, where damage is currently occurring on their brains. • Clinical activities: multiple sclerosis

  41. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL UK Stem Cell FoundationUniversity College London • Led by Andrew Goldberg OBE of University College London (UCL) and the Royal National Orthopaedic Hospital, this first-in-man pilot study explores the treatment of Achilles Tendinopathy using a culture of expanded autologous mesenchymal stem cells to repair damage. • The project is funded, in part, by the UK Stem Cell Foundation (UKSCF) • Stems cells will be taken from the patient's own hip, expanded in the laboratory then implanted into the damaged tendon. • UCL is one of the few centres in the world developing expertise into the regeneration of tendons using stem cells, as well as translating successful treatments used in horses into humans. • Achilles Tendinopathy causes pain in the heel and affects more than 70,000 people a year in the UK. • Clinical Activities: Achilles Tendinopathy

  42. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL DIABETES UK • Diabetes UK funded 12 islet transplants as part of a research project. This was successful, with all patients achieving complete resolution of severe hypoglycaemia. • On the basis of these results, the National Specialist Commissioning Group provided central funding for the service. • Islet cell transplantation is now NICE Approved and available through the NHS, and the UK benefits from having one of the only government-funded islet cell Transplantation services in the world. • As of 2013, 95 islet transplants had been performed in65 peoplein the UK (Diabetes UK) • Clinical successes: Allogeneic pancreatic islet cell transplantation • for type 1 diabetes mellitus

  43. Appendix Examples of leading research

  44. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL • World class research centres are leading thinking on regenerative medicine in the UK The MRC Centre for Regenerative Medicine (CRM) in Edinburgh: brings together world leading basic stem cell researchwith established clinical excellence to deliver a "bench-to-bedside" approach aimed at developing new treatments for major diseases. Cardiff Institute for tissue Engineering and Repair (CITER): an established interdisciplinary network, linking around 130 academic staff from 11 Schools across Cardiff University. CITER is internationally recognised for its expertise in basic, translational and clinical research in the field of tissue repair, regeneration and rehabilitation. Cambridge hub – one of the highest density of stem cell researchers in Europe - a total of 26 University laboratories are conducting research with stem cells - Cambridge clinicians have already begun clinical trials using cell based regenerative medicines to treat diseases such as Multiple Sclerosis and Parkinson’s Disease. The Oxford Stem Cell Institute (OSCI) unites over 40 laboratories from across the University’s Medical Sciences Division and offers a uniquely interdisciplinary approachto regenerative medicine. By harnessing world-leading expertise in the fields of stem cell biology, immunology, tissue engineering, materials science, medicinal chemistry and cancer biology the OSCI aims to create a seamless pathway from basic science, through translational medicine to clinical trials. The UCL Centre for Stem Cells and Regenerative Medicine brings together many different research groups from across UCL and it's partners, including the London Centre for Nanotechnologyand the MRC National Institute for Medical Research (NIMR), with a common interest in all aspects of stem cells, tissue engineering, repairand regeneration and the development of their therapeutic and biotechnological potential

  45. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL University of Leeds • Medical Technologies IKC facilitates collaboration between companies, engineers, scientists and clinicians to develop innovative technologies that help the body repair and restore function. • Work in partnership with the centre to: • speed up your route to market • work with world-leading experts to ensure products and services are fit for purpose • reduce the risk of late-stage failure • access resources to support innovation for new product development • Examples of capability areas include: • Novel protein biosensors to diagnose disease and monitor patients’ responses to treatment • Use of minimally manipulated stem cells for musculoskeletal and cardiovascular repair • Regenerative biological scaffolds to replace damaged joint and heart tissue • Improved imaging to allow early diagnosis and targeted treatment • Longer lasting joint replacements for the hip, knee and spine • Enabling technologies • Integrated non-clinical/clinical research in repair, regeneration and rehabilitation • Cross-disciplinary involvement of the Schools of Biosciences, Chemistry, Dentistry, Engineering, Healthcare Studies, Medicine, Optometry & Vision Sciences, Pharmacy & Pharmaceutical Sciences, Physics, Social Sciences at Cardiff University • Projects funded by the Medical Research Council (MRC), the Engineering and Physical Sciences Research Council (EPSRC), the Wellcome Trust, European Framework 7 (FP7), Arthritis Research UK (funded Arthritis Research UK Biomechanics and Bioengineering Centre) and industry • Projects in musculoskeletal injury, vision loss, brain repair, kidney repair, tissue repair, degenerative disease, stem cell science, healthcare regulations, ethical research • Public-facing engagement and outreach activities • Tissue Engineering masters programme • The Medical Technologies Innovation and Knowledge Centre, Leeds

  46. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL University of Bristol • Pioneering cardiovascular stem cell therapy research is being successfully conducted at the University of Bristol (led by Prof. P. Madeddu and others) • Projects are funded by the Medical Research Council (MRC), the British Heart Foundation (BHF), theWellcome Trust (WT) and the National Institute of Health Research (NIHR), for a value of several million pounds. • The research groups are working on a number of industrial collaborations with national and international companies engaged in cell therapy, including ReNeuron, Biocompatiblesand StemMed. For example, Prof Madeddu collaborated with ReNeuron to support their proprietary cell line to be used in a clinical trial in limb ischemia. • Research supported by MRC (TSCR project) and BHF (special project grant – Mending broken heart scheme) covers the production of clinical grade pericytes which through the NHS Blood and Transplant (NHSBT) should be used first in a large animal model for final proof of efficacy before starting a first in man clinical trial in patients with refractory angina. • Pioneering translational stem cell research for cardiovascular applications at the University of Bristol

  47. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Durham University • Researchers at Durham University and Columbia University Medical Center (CUMC), USA, have devised the method which is the first to use cloned human cells to induce hair growth, rather than redistributing hair from one part of the scalp to another. • These findings could significantly expand the use of hair transplantation to women with hair loss, who tend to have insufficient donor hair, as well as to men in early stages of baldness. • Previous research by Professor Colin Jahodaat Durham University found that rodent dermal papillae – a small, group of cells at the base of the hair follicle which play a pivotal role in hair growth – could be easily harvested and transplanted back into rodent skin. • The UK side of the study (the other in the US) was funded by the Medical Research Council and the Biotechnology and Biological Sciences Research Council • International research collaborations: The University of Durham

  48. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Cardiff University • Headed by Prof Keith Harding, the Wound Healing Research Unit (WHRU) is a world-class clinical, scientific and educational wound healing resource for the benefit of people with wound problems, providing expertise in biophysical and clinical science research. • Acts as an interface between healthcare providers, industry and the academic community, to develop models of high quality care based on sound scientific research. • WHRU has conducted 9 studies over the past 10 – 15 years for ConvaTec (part of Bristol Myers Squibbs). • Clinical studies have included a randomised controlled trial of 131 patients, comparing AQUACEL ® Dressing versus an alginate dressing. • In 2012, the WHRU undertook a clinical study commissioned by the National Institute for Health and Care Excellence (NICE) to determine the effectiveness of MIST ultrasound therapy compared to UK standard care for the treatment of non-healing venous leg ulcers. In this instance, the WHRU acted as the clinical experts in leading this clinical trial, which was independent from NICE, and the MIST manufacturer. • The Wound Healing Research Unit supports industry activities

  49. UNLOCK YOUR GLOBAL BUSINESS POTENTIAL University of Liverpool • The Division of Clinical Engineering at the University of Liverpool, home to the UK Centre for Tissue Engineering (UKCTE), has been at the forefront of research in biomaterials, biocompatibility, tissue engineering and regenerative medicine for over 30 years. • The University of Liverpool is coordinating a European collaboration, REDONTAP. • The REDONTAP project aims to discover the conditions required for the rapid and continuous creation of red blood cells from adult stem cells. The European Commission, under the Nanosciences, Nanotechnologies, Materials and new Production Technologies (NMP) theme of the 7th Framework Programme, have granted the University of Liverpool, the University of Leipzig, Applikon Biotechnology and the Banc de Sang iTeixits, Barcelona, €2.75 M to pursue this research. • The global cost of blood donation services has been estimated to be more than $500M annually. Each year, this cost rises dramatically, mainly due to the cost of testing for infectious agents. The results of the REDONTAP Project will help create a device for generating red blood cells from adult stem cells at a commercially-viable scale, which will be free from risk of infectious diseases, and perfectly matched with the donor. Using this technology, it would be possible to create blood for patients with very rare blood types. • European collaboration: The University of Liverpool co-ordinates REDONTAP

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