unlock your global business potential: uk regenerative medicine and stem cells

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

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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

By placing your business in the UK, your company can benefit from a rich heritage in regenerative medicine and stem cells

1978 First IVF baby born after Cambridge university scientists: Sir Robert Edwards (Nobel Prize for Medicine 2010) and surgeon Patrick Steptoe fertilise human eggs outside the body.

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.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.

1996 Dolly 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.

2003 Researchers at King’s College London, led by Professor Peter Braude, generate the UK’s first embryonic stem cell line.

2005 The 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.

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.

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.

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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

By placing your business in the UK, your company can benefit from a rich heritage in regenerative medicine and stem cells

2010ApaTech, a spin-out from Queen Mary University of London, and a world-leader in bone graft technologies is sold for $330M to Baxter.

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.

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’s PISCES 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 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.

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.

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.


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.

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.

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The UK recognises the need to support industry deliver patient benefit and achieve commercial viability


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Collaborate and work in the UK to access world class resources that are delivering results

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

Connectivity through 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-class cell and tissue banking facilities available for industry

Funding for UK based companies through government initiatives

Leveraged funding through collaboration with academia


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”.

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Comprehensive supply chain and logistics allow you to deliver on time

and cost effectively You can tap into an established and accessible commercial supply chain that 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


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.

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Access existing manufacturing capabilities with proven ability to deliver

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 and supporting 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


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.

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Access a strong clinical research infrastructure allowing you to deliver clinical trials efficiently

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


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.

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The UK provides commercial support for all your activities

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 GMP and delivered cost effectively

Providing regulatory 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 opportunities so that commercially viable products are progressed and investable propositions are generated

The UK hosts numerous experienced commercial and business advisors and consultanciesThe UK regenerative medicine Industry has a united voice through the BioIndustry Association’s (BIA) Cell Therapy and RegenMed Advisory Committee


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.

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The UK is your gateway to Europe and the global markets

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).


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.

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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

Low corporation tax rate currently standing at 23 per cent decreasing further to 20 per cent by April 2015.

Patent box: 10% corporation tax on qualifying

profits

23%Corporation tax will

drop further to

20%

£180m

£300m

Biomedical Catalyst =

£180m programme of public funding

for growth To stimulate R&D

partnerships between

universities businesses &

charities

R&D tax credits for

SMEs worth approximately 25p on every

£1 (7p for large

companies)

Patent Box

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Clinical success: the UK is one of the leading European countries developing Advanced Therapy Medicinal Products

34 cell therapy clinical trials and 37 cell 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


You can work with leading clinicians and clinical centres to deliver your clinical trials easily, efficiently and to a high quality.

Trials by stage:

Figure 1: Clinical trials, in the UK, by therapeutic area

Data from Cell Therapy Catapult report

Oncology26%

Blood15%

Cardiovascular12%

Neurological12%

Gas-troen-terolgy

9%

Oph-thal-

mology9%

Diabetes9%

Bone and cartilage9%

Phase I

Phase 1/II

Phase II

Phase II/III

Phase III

0 2 4 6 8 10 12 14 16 18

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2 Trial in the UK by therapeutic areaOncology 9

Blood 5

Cardiovascular 4

Neurological 4

Gastroenterology 3

Ophtalmology 3

Diabetes 3

Bone and Cartilage 3

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Multi-disciplinary clinical successes in regenerative medicine from voice box reconstruction to tendon repair


You can work with leading clinicians and clinical centres to deliver your clinical trials easily, efficiently and to a high quality.

Ophthalmology

Diabetes

Digestive TractLiver cirrhosis

Voice-box ReconstructionHuntington Disease

Multiple Sclerosis

Stroke

Rheumatoid Arthritis

Cartilage repairCardiovascularTendon repair

Immunotherapy

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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

Your competition is already benefiting from the UK Offer in Regenerative Medicine


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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

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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


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

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Appendix: Success Stories

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How the UK environment helped ReNeuron be a leader in the field


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.

ReNeuron

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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


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.

Cell Medica


http://www.google.co.uk/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&docid=xV0PEQjG1-wUZM&tbnid=xWacFwhJZWE1WM:&ved=0CAUQjRw&url=http://www.bio.org/node/18766&ei=jlt6UqzyLMbO0QWr-IG4Dw&bvm=bv.55980276,d.ZG4&psig=AFQjCNFv0EIxB1-khCGxNxWiARQ9owEmrQ&ust=1383836939636244

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Intercytex: using a Progressive Translation approach to take product to market



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

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 development: A close partnership between client, contract developer and hospital allowing iterative development of new treatments

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 trial

completed 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

“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

Neusentis: working in partnership with academic researchers in Cambridge and across the UK


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.

Neusentis

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.

GE Healthcare Life Sciences chooses Wales for its new state of the art laboratories for cell therapy and cell bioprocessing


“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.”

GE Healthcare

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UK Approves Europe's First Embryonic Stem Cell Clinical Trial


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.

Advanced Cell TechnologyMoorfields Eye Hospital

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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

Athersys

EPSRC Centre for Innovative Manufacturing in Regenerative Medicine collaborating with industry


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 Centre for innovative manufacturing in regenerative

medicine

“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

A University of Swansea spin-out joins efforts with Biotec Services International to form 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.

TrakCel

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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’s dCELL® 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


Tissue Regenix

http://www.google.co.uk/url?sa=i&source=images&cd=&cad=rja&docid=Ntoxcpt969N2BM&tbnid=eD9P4FYEJYNFWM:&ved=0CAgQjRwwAA&url=http://www.leeds.ac.uk/info/125077/working_with_business/1572/who_we_work_with&ei=CF56UuTBLY6qhQeV0ICYDw&psig=AFQjCNHc6AbBNAFkiDxAtYLOX8ajnqd66g&ust=1383837576804541

http://www.google.co.uk/url?sa=i&source=images&cd=&cad=rja&docid=Ntoxcpt969N2BM&tbnid=eD9P4FYEJYNFWM:&ved=0CAgQjRwwAA&url=http://www.leeds.ac.uk/info/125077/working_with_business/1572/who_we_work_with&ei=CF56UuTBLY6qhQeV0ICYDw&psig=AFQjCNHc6AbBNAFkiDxAtYLOX8ajnqd66g&ust=1383837576804541

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Appendix Clinical successes

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Clinical Firsts: Windpipe Reconstruction


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.

University College London

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Clinical Activities: Exploiting Stem Cells for Immunotherapy

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.

University of OxfordCell Therapy Ltd

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Clinical Firsts: Liver Failure


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.

Imperial College London

http://en.wikipedia.org/wiki/Image:Imperial_College_London.svg

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Clinical Firsts: Digestive Tract


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.

Great Ormond StreetHospital

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Clinical activities: Rheumatoid Arthritis


Professor John Isaacs (Consultant Rheumatologist and Professor of Clinical Rheumatology) and Dr Catharien Hilkens (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

Newcastle University

35

Clinical Activities: Huntington Disease


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

Cardiff UniversityUniversity of CambridgeImperial College LondonAddenbrooke’s Hospital

http://en.wikipedia.org/wiki/Image:Imperial_College_London.svg

36

Clinical successes: limbal epithelial stem cell transplantation


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

Newcastle UniversityThe Newcastle upon Tyne Hospitals

37

Cells For Sight


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'.

University College LondonMoorfields Eye Hospital

38

Clinical activities: Knee cartilage repair


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

Keele UniversityRobert Jones & Agnes Hunt

39

Clinical activities in 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

Scotland

40

Clinical activities: Cardiovascular disease

Randomised control trial to compare the effects of G-CSF and autologous bone marrow progenitor cells infusion in patients with ischaemic heart diseaseBarts Health NHS Trust, Queen Mary University of London Phase II, recruitment target of 90 patients

Expanded adult haematopoietic CD34+ stem cells for autologous infusion to patients with myocardial ischaemiaImperial College London Phase I/II, recruitment target of 42 patients

Autologous bone marrow derived mononuclear cells for dilated cardiomyopathy, delivered via intracoronary injectionBarts Health NHS Trust, Queen Mary University of London Phase I/II, recruitment target of 60 patients

Autologous bone marrow derived mononuclear cells for acute myocardial infarction. Combines stem cell delivery with primary angioplasty within 5 hours post eventBarts Health NHS Trust, Queen Mary University of London, University College LondonPhase I/II, recruitment target of 70 patients

4 1

3 2


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Clinical activities: multiple sclerosis


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 FoundationMultiple Sclerosis Society

42

Clinical Activities: Achilles Tendinopathy


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.

UK Stem Cell FoundationUniversity College London

43

Clinical successes: Allogeneic pancreatic islet cell transplantation

for type 1 diabetes mellitus


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 in 65 people in the UK (Diabetes UK)

DIABETES UK

44

Appendix Examples of leading research

45

World class research centres are leading thinking on regenerative medicine in the UK

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.

The MRC Centre for Regenerative Medicine (CRM) in Edinburgh: brings together world leading basic stem cell research with established clinical excellence to deliver a "bench-to-bedside" approach aimed at developing new treatments for major diseases.

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 Nanotechnology and the MRC National Institute for Medical Research (NIMR), with a common interest in all aspects of stem cells, tissue engineering, repair and regeneration and the development of their therapeutic and biotechnological potential

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 approach to 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.

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The Medical Technologies Innovation and Knowledge Centre, 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

University of Leeds

http://www.google.co.uk/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&docid=XBHAAwCw4Al9qM&tbnid=Di_iIQIift6R8M:&ved=0CAUQjRw&url=http://careerweb.leeds.ac.uk/&ei=qrSyUvKuLLSg0wW4l4BI&bvm=bv.58187178,d.ZG4&psig=AFQjCNGuHB5WoKrdZ-c3CN0YAPNQYBMbYA&ust=1387529762757385

47

Pioneering translational stem cell research for cardiovascular applications at the 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), the Wellcome 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, Biocompatibles and 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.

University of Bristol

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International research collaborations: The University of Durham


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 Jahoda at 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

Durham University

http://www.google.co.uk/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&docid=btL_mShyWHbgTM&tbnid=7iIHiurW-4758M:&ved=0CAUQjRw&url=http://mpilot.blogs.lincoln.ac.uk/baltic-seals/&ei=Ot18UvTgC6XG0QXSk4HwCA&bvm=bv.56146854,d.ZG4&psig=AFQjCNGoaWOCtb2-H2WLfJhtP8FS6w6hzg&ust=1384001204369349

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The Wound Healing Research Unit supports industry activities


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.

Cardiff University

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European collaboration: The University of Liverpool co-ordinates REDONTAP


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 i Teixits, 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.

University of Liverpool

http://www.google.co.uk/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&docid=UM_kkkUfXJes1M&tbnid=EvTV5SrPKrLGbM:&ved=0CAUQjRw&url=http://www.rtpi.org.uk/education-and-careers/information-for-universities/accredited-qualifications/university-of-liverpool/&ei=A7x8UsjeE6aq0QWki4CgCw&bvm=bv.56146854,d.ZG4&psig=AFQjCNHbtAPor2f50IwBMx7UWRctsDKruQ&ust=1383992704794975

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Cell/Tissue 3D Printing


The world’s first 3D printing technique, which produces viable clusters of stem cells has been an important milestone in Scotland’s ability to deliver effective drug development tools.

3D printing technologies could speed up progress towards creating artificial organs but has a more immediate application generating biopsy-like tissue samples for drug testing.

The valve-based printing technology developed by Will Shu from Heriot Watt University in Edinburgh will be refined by Scottish based Roslin Cellab and UK Company Reinnervate to create the next generation 3D stem cell printing products and services.

Once refined, the 3D printed stem cell derived tissue samples could drastically reduce the cost and need for donor tissue and cells in pre-clinical research.

Roslin Cellab, Heriot Watt UniversityReinnervate

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The Oxford Stem Cell Institute


The Oxford Stem Cell Institute (OSCI) comprises over 40 world-leading laboratories from backgrounds as diverse as physiology, transplantation immunology, medicinal chemistry, tissue engineering and cancer biology, but each with a focus on regenerative medicine.

Interdisciplinary collaboration lies at the heart of OSCI’s mission to realise the potential of regenerative medicine for addressing unmet medical needs of the 21st century. The OSCI unites scientists and clinicians to provide a pathway from basic science, through translational medicine to clinical trials.

The OSCI has significant critical mass in areas such as neural stem cell biology, pluripotency, cardiovascular biology and haematopoiesis. Recent initiatives to emerge from the OSCI include the Oxford Parkinson’s Disease Centre, StemBANNC and the BHF Oxbridge Regenerative Medicine Centre.

Oxford’s acknowledged world-leading status in the biomedical sciences, the availability of facilities within its various business and science parks and access to expertise in clinical trials has proven a magnet for companies operating within the regenerative medicine space.

The University of Oxford

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Translation in nanotechnology: The university of Swansea


The Centre for NanoHealth (CNH) at Swansea University combines nanotechnology with medical science to provide opportunities to benefit patients, healthcare providers and the healthcare industry both across Wales and globally.

Provides a technology and innovation base for SMEs in Wales and beyond.

To date CNH has initiated many collaborative projects with industry and other higher education institutions realising more than £15 Million further investment.

Within the CNH there are four regenerative medicine research strategy areas– Stem cells for studying molecular mechanism controlling

cell differentiation– novel tissue scaffolds with controlled micro and nanoscale

morphology– 3D tissue regeneration models– mechanism of immune responses against artificial organs

Collaborative projects with local companies include research and development into advanced wound dressings, cartilage-derived stem cells, and novel scaffolds for dental bone grafts (e.g. Osseo Regenerative Technologies and Pulse Medical)

University of Swansea

54

Cardiff Institute of Tissue Engineering and Repair: Cardiff University


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

Cardiff University

55

The MRC Centre for Regenerative Medicine has recently relocated to the new Scottish Centre for Regenerative Medicine (SCRM) Building. The £54M building has received substantial funding from the Scottish Executive and Scottish Enterprise. It contains state-of-the-art laboratory space and a ‘Good Manufacturing Practice’ (GMP) clinical research facility for the production of cells for future therapeutic applications.

The new building is part of the Edinburgh BioQuarter development at Little France. This location, with a large state-of-the-art teaching hospital, the University of Edinburgh’s world-renowned medical school and bespoke biomedical research and development facilities all on one site, provides a unique operating environment with substantial collaborative opportunities.

The Edinburgh BioQuarter has a wide range of capabilities making it a leading European destination for translational medical research. There currently hosts nearly 900 hospital beds and 1,200 researchers: by 2016, these numbers are expected to rise to more than 1,500 hospital beds and 2,000 researchers.

The campus has specialist facilities, such as ATMP GMP labs for stem-cell work, an imaging centre with PET, MRI and CT-slice scanners and an MHRA-accredited Phase I clinical trials unit on site.

The Edinburgh BioQuarter is a leading European destination for

translational medical research


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Arthritis Research UK (ARUK) funds leading centres of excellence accelerating regenerative medicine research in the field

Arthritis Research UK Tissue Engineering Centre, Newcastle, York, Keele, Aberdeen and Cambridge

Arthritis Research UK Biomechanics and Bioengineering Centre – Cardiff

Arthritis Research UK Centre for Osteoarthritis Pathogenesis, Oxford

Other ARUK centres in: Primary care Pain Musculoskeletal ageing research Exercise and osteoarthritis Rheumatoid Arthritis Pathogenesis


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From discovery science through to clinical and commercial

Application: UK Regenerative Medicine Platform Hubs

Cell behaviour, differentiation and manufacturing; University of Sheffield

Engineering and exploiting the stem cell niche; University of Edinburgh

Safety and efficacy, focussing on imaging technologies; University of Liverpool

Acellular (smart material) approaches for therapeutic delivery; University of Nottingham


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Examples of leading induced pluripotent stem cell (iPSC) initiatives

The Wellcome Trust (WT) and the Medical Research Council (MRC) invested £12.75 million to establish the human induced pluripotent stem cells initiative (HipSci). The research project aims to create a catalogue of high-quality adult stem cells and to understand the role that minor genetic variants play in defining cellular phenotypes and thereby disease development. The project is led by King’s College London and the Wellcome Trust Sanger Institute and will generate iPS cells from >1000 healthy individuals and those with disease

The university of Birmingham is acting as repository for the many research grade iPS lines being made in the Innovative Medicines Initiative (IMI) funded project STemBANCC, which has many of Europe’s pharmaceutical companies engaged

Newcastle University is aiming to generate induced Pluripotent Stem Cells as a bespoke service. The aim of this organisation will be to provide high quality iPS cell lines to industry and academia for applications in both clinical and biological research programmes.

Roslin Cells and the Cell Therapy Catapult have partnered to establish a source of clinical grade iPS cells 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.


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Appendix Further supporting information

60

The UK offers a well funded research infrastructure for industry to leverage funding for innovative research, proof-of-concept, and clinical delivery

The UK boasts a superior infrastructure optimised to support industry

Cell Therapy Catapult established to assist industry

and academic groups to develop key manufacturing

processes, design and execute clinical trials and navigate the regulatory

system.

£180 million Biomedical Catalyst is an integrated

translational funding programme supporting

academics and UK SMEs to develop innovative solutions

to healthcare challenges

£800 million NIHR funding for five years from April 2012 for 11 Biomedical

Research centres and 20 Biomedical Research Units

with £9 million per annum of world-leading

translational research in regenerative medicine

The TSB supports Regenerative Medicine

themed R&D competitions and in 2013 has already

committed a further £8m to a competition in this area.

A number of charities support research activities in regenerative medicine. For examples the British Heart Foundation is focusing on

raising £50 million to fund research into the field

TSB funded Knowledge Transfer Networks (KTNs) connecting the regenerative medicine community to create

opportunities for collaboration with

industry

Research Councils invest £25m in the UK

Regenerative Medicine Platform to deliver on key

areas of regenerative medicine and act as key

access points for collaboration with

industry

UK Stem Cell Foundation has helped

over £14 million to be invested in stem cell research projects in the UK through a combination of fundraising,

co-funding and collaboration.


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£25 million UK Regenerative Medicine Platform (UKRMP), established by MRC, BBSRC and EPSRC

EPSRC, TSB and BBSRC jointly funded Innovation and Knowledge Centre in Regenerative Therapies and Devices

UK Stem Cell Bank, established with funding from the MRC and BBSRC, repository of human embryonic stem cell lines

Bioprocessing Research Industry Club (BRIC), funded by the BBSRC and EPSRC EPSRC has supported the establishment of several innovative research and doctoral

training centres in regenerative medicine Charity Funders: Charities constitute an integral part of the UK’s medical

research sector and have invested strongly in regenerative medicine. For example:– British Heart Foundation: focusing on raising £50 million to fund research into

regenerative medicine with an ambition to be “pioneers in regenerative medicine”– UK stem cell foundation: The only UK charity to focus specifically on stem cell

research as a potential treatment for a wide range of conditions and diseases. The Foundation helped over £14 million to be invested in stem cell research projects in the UK through a combination of fundraising, co-funding and collaboration.

– Arthritis Research UK (ARUK): funds a range of pre-clinical and clinical research in regenerative medicine including the ARUK Tissue Engineering Centre which brings together leading academic clinicians, with leading scientists in engineering, biology and material science with the aim of regenerating bone and cartilage by using the patients’ own stem cells to repair joint damage caused by osteoarthritis.

– Diabetes UK: awarded a £1.3M multicentre grant to foster collaborative UK research in severe hypoglycaemia, optimised analogue treatment, pump therapy and continuous glucose monitoring.

Tapping into a rich environment supported by backing from research funders


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The UK offers a well funded research infrastructure for industry to access expertise and leverage funding for innovative research, proof-of-concept, and clinical delivery

Basic and translational research activities already happening and easily accessible– UK Stem Cell Foundation has helped over £14 million to be invested in stem cell

research projects in the UK through a combination of fundraising, co-funding and collaboration.

– A number of charities support research activities in regenerative medicine. For example, the British Heart Foundation is focusing on raising £50 million to fund research into the field whereas Arthritis Research UK has invested in the establishment of several regenerative medicine and tissue engineering centres across the UK

– £800 million NIHR funding for five years from April 2012 for 11 Biomedical Research centres and 20 Biomedical Research Units with £9 million per annum of world-leading translational research in regenerative medicine

– Research Councils invest £25m in the UK Regenerative Medicine Platform to deliver on key areas of regenerative medicine and act as key access points for collaboration with industry

Access to a talent pool and expertise– Engineering and Physical Sciences Research Council, through the Doctoral Training

Centre in Regenerative Medicine, is investing £7 million to equip 60 PhD students with cutting-edge skills to excel at the Life Science / Engineering interface

Networks that help you stay connected to the regenerative medicine community and access information and expertise– TSB funded Knowledge Transfer Networks (KTNs) connecting the regenerative

medicine community to create opportunities for collaboration with industry

A superior infrastructure optimised to support industry


63

Direct support for business led activities through: Direct funding accessible to UK based companies

– The TSB supports Regenerative Medicine themed R&D competitions and in 2013 has already committed a further £8m to a competition in this area.

– £180 million Biomedical Catalyst is an integrated translational funding programme supporting academics and UK SMEs to develop innovative solutions to healthcare challenges

Direct access to expertise and guidance in all areas– The Cell Therapy Catapult has been established to assist industry as well as

academic groups to develop key manufacturing processes, design and execute clinical trials and navigate the regulatory system

– An £8.3 million, ESPRC Centre for Innovation Manufacturing in Regenerative Medicine established to develop the products and systems to allow clinicians to administer new regenerative treatments. The centre, led by the University of Loughborough has over 28 partners from industry and the public sector.

– £25 million UK Regenerative Medicine Platform



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UK based companies are already benefiting from government support

Access to TSB funding with up to £4.5m investment from the TSB in the development of innovative technologies to enable and improve diagnostic analysis of samples from cells, tissues and tumour biopsy. This funding is available for industry-academic projects with a business focus. A consortium formed of Neotherix Ltd , Lorien Engineering Solutions Ltd, and Smith &

Nephew Advanced Wound Management received two rounds of funding from the TSB Regenerative Medicine programme to complete a proof-of-concept study for EktoTherix® . The consortium has since secured further TSB funding to take the therapy into the final development stage of clinical trials.

A tracheal replacement project, led by Videregen Ltd and involving a team of UK experts, has been awarded almost £2m by the TSB under its Regenerative Medicine and Cell Therapy competition. The consortium will use the funds to progress a promising clinical prototype through a rigorous development process and early clinical trials for the treatment of severe structural airway disease (SSAD)

Oxford BioMedica (UK) received funding for the development of a novel therapeutic treatment to engineer corneas to resist graft rejection

Asymptote Ltd received funding for the development of a regulatory compliant cassette for large volume cell culture, cryopreservation, thawing and perfusion

ReNeuron has succeeded in getting TSB regenmed funding for a Ph II trial in stroke disability which will be a landmark trial in the stem cell therapy field as well as TSB biomedical catalyst funding for a Ph I CLI trial and preclinical development of our retinal stem cell programme


65

You can access world-leading cell and tissue banking facilities to support your activities:

The UK Stem Cell Bank: The UK has led the world in setting up a stem cell bank, which supplies high-quality starting materials for the development of stem cell therapy and provides a centralised resource for academics and industry researchers from the UK and internationally

NHS Blood and Transplant, Tissue Services is the largest multi-tissue bank in Europe. Their role is to co-ordinate, recover, process, bank and supply human tissue grafts for use in surgery within the NHS and independent hospitals in the UK

A number of cord blood banks, public and private Funded by the Wellcome Trust (WT), Scottish National Blood and Transfusion Services

(SNBTS) has established a blood donor derived, anti-Epstein-Barr virus Cytotoxic T-Cell (EBV CTL) bank. SNBTS can grow cells which selectively destroy the EBV infected cells but do not attack the patient's other cells. The bank is licensed by the Medicines and Healthcare Products Regulatory Agency (MHRA) for the manufacture of unlicensed medicinal products under the terms of Manufacturer's ("Specials") Licence MS 3473.

Access to Cell and Tissue Banking


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UK Stem Cell Bank


The UK Stem Cell Bank was the first of its kind to be established in the world

It is a centre of expertise providing support to scientists and clinical projects through provision of well characterised stem cell lines.

It provides a repository for human stem cell lines of all types under appropriate and accredited quality systems.

It will supply well characterised cell lines both for basic research and for the development of clinical applications.

It is located at the National Institute for Biological Standards and Control (NISBC), which is now part of the Medicines and Healthcare Products Regulatory Agency (MHRA)

STATUS NUMBER OF LINES

Lines Available 22Due for Release (banked by the UKSCB and are awaiting QC release for distribution or are undergoing banking. ) 17

Accessioned by the Bank (currently stored in the Bank but have not yet been banked or released for distribution) 23

Accepted by the Steering Committee (approved by the Steering Committee for storage and distribution by the Bank) 17

Total 79

http://www.ukstemcellbank.org.uk/index.html

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SNBTS (Scotland) – already partner in public and private projects; developed close partnerships with the Universities of Aberdeen, Edinburgh and Glasgow and has supported the development of the Scottish Centre for Regenerative Medicine.

NHSBT infrastructure is pivotal to the effective manufacture and delivery of regenerative medicines e.g. Azellon partnering with NHSBT in cell production for the clinical trial of its platform technology using mesenchymal stem cells (MSCs) to repair damaged knee tissue

NHSBT deliver a diverse range of services in human tissues and cells and are key in the regenerative medicine environment

Location Clean Rooms

Development Labs

Cryostorage Facility

Liverpool 10 Yes YesOxford 2 Yes YesBirmingham 3 Yes YesBristol (Langford) 6 Yes YesBristol (Filton) 4 Yes YesSheffield 1 No YesLeeds 3 No YesSouthampton 2 No Yes

NHSBT manages clean room facilities across eight sites as shown in Table 1. Table 1. NHSBT GMP-Compliant Clean Room Facilities

Common features of the facilities are: Independent air handling

systems Flexibility of grade A

against B, or grade A against C/D environments

Dedicated space for development and closed processing technology

Controlled rate freezing and LN2 vapour phase storage. CO2 incubation

Dedicated QC laboratories including flow cytometry and environmental monitoring

GMP compliance.


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Stem Cell for drug discovery: Stem Cells for Safer Medicine


Stem Cells for Safer Medicines is an independent not-for-profit company.Initial research will enable the use of stem cells in early drug discovery by:

focusing on key scientific challenges and developing open standards and protocols.

This is aimed at providing standardised in vitro testing for toxicity of potential new drugs. The main focus of SC4SM is in developing systems for testing liver toxicity

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A strong and growing supply chain to support research, clinical and commercial activities In summary, the UK regenerative medicine sector is thriving in a rich ecosystem with a

supporting, well funded, infrastructure, leading basic as well as translational research and the backing provided by government, research councils and charities.

The UK also offers your company an established and evolving accessible commercial supply chain to support your activities at every stage and complement your expertise

>190 companies operating in the UK that consider themselves to have regenerative medicine as a feature of their business model

Enabling tools, technologies and

processesManufacturing

capabilitiesRegulatory advice

and guidanceClinical trial design

and delivery

Reagents, assays Cell production, processing Imaging tools Storage and transport Supply chain management Health economics Business planning Cell Therapy Catapult

High value manufacturing

Bioprocessing GMP facilities Scaffolds and matrices Devices Innovative approaches Cell Therapy Catapult

MHRA HTA Stem Cell toolkit Cell Therapy Catapult consultancy services,

CROs and CMOs

Cell Therapy Catapult Clinical Trial Toolkit route-

map NHIR, NOCRI NHS Research Scotland

Tissue and cell supply

UK Stem Cell Bank NHSBT/SNBTS SC4SM 16 banks and registries Transplant services


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Support for product developers in manufacturing innovation – EPSRC centre for innovative manufacturing in regenerative medicine provides an integrated platform of fundamental and translational research – a translational ‘go-to’ resource for the regenerative medicine product developer. The EPSRC Centre has a wide variety of commercial and clinical collaborators in addition to academic collaborators.

Scaffold design, fabrication and analysis - Wolfson Centre for Stem Cells, Tissue Engineering & Modelling, Nottingham- Prof Shakesheff and Drs Rose and Buttery focus on exploring interesting new ways to analyse and control the interactions between cells, inductive signals and scaffolds to promote the repair of tissues such as bone, cartilage, liver, cardiac muscle, gastro-intestinal tissues, pancreas as well as many other tissue types. These approaches are also used to develop in vitro tissue models, such as liver and thymus that can used to study developmental and disease processes and to test new drugs. The group has extensive expertise in scaffold design, fabrication and analysis and work with both natural and synthetic polymers to produce an array of scaffolds with controlled size, shape and structure.

ATMP manufacturing - several MHRA licensed GMP manufacturing facilities across the UK. See table

Strong manufacturing capabilities supported by an innovative manufacturing environment

Some of the GMP manufacturing facilities in the UK:

Cancer Research UK, Biotherapeutics Development Unit (London)

Cellular Therapeutics Ltd (Manchester) Guy’s & St Thomas’ Hospital, GMP Facility

(London) Intercytex Ltd (Manchester) Imperial College London, John Goldman Centre

for Cellular Therapy (London) Kings College London, Rayne Cell Therapy

Suite (London) NHSBT – Speke (Liverpool) Newcastle University, Cellular Therapies

Facility (Newcastle upon Tyne) Roslin Cells Limited (Edinburgh) University College London, Great Ormond

Street Hospital Cellular Therapy Laboratories (London)

University College London, Royal Free Hospital, Paul O’Gorman Laboratory of Cellular Therapy (London)

University College London Hospital, Institute of Ophthalmology, Cells for Sight ATMP Manufacturing Unit (London)

University of Oxford, Clinical Biomanufacturing Facility (Oxford)


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The Cell Therapy Catapult, a national resource, supporting partners in achieving commercial success


The Cell Therapy Catapult was established as a centre of excellence in 2012 to create a world-leading cell therapy industry in the UK through innovation and collaboration. Supported by the UK Government, their mission is to drive the growth of the industry by helping cell therapy organisations across the world translate early stage research into commercially viable and investable therapies.

With one of the largest dedicated cell therapy teams anywhere, their scientists, business development, manufacturing and regulatory experts are collaborating with cell therapy organisations and other interested parties from across the globe. Together we are also finding solutions to industry-wide challenges including business models, logistics and reimbursement.

Building on the UK’s strength in scientific research and the healthcare industry, the Cell Therapy Catapult’s vision is to grow a cell therapy beyond critical mass, helping partners advance your therapies towards the market and tackle unmet medical needs.

Working with the Cell Therapy Catapult brings: – World-class laboratory facilities– Clinical expertise and a rapid trial and patient networks in the NHS and throughout

Europe– Infrastructure, supply chain management and manufacturing expertise– Regulatory knowledge and leadership– Financial and Industry networks – Health economics and reimbursement expertise– Business and commercialisation expertise – Reach into European markets

The Cell Therapy Catapult takes a consultative approach to collaborating on projects, with the aim of generating commercially viable and investable therapies.

https://ct.catapult.org.uk/

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Successful path to Commercialisation


Efficacy and safety hypothesis and evidence Definition and characterisation of cellular product

Patient population, unmet medical need, differentiation

Safety Robust evidence of efficacy Dose and dosing regimen

GMP manufacturing process; release; comparability assays

Supply logistics Scale-up / scale –out; Control of cost

Pricing and reimbursement plan Defensibility (IP, know-how…) Commercialisation partner

Science

Clinical

Manufacturing

Business

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The Cell Therapy catapult: supporting industry taking products to market


The Cell Therapy Catapult and…

…ReNeuron are collaborating on new cell therapy manufacturing technologies and assays and will focus on the development and optimisation of the processes required to scale up manufacture of the CTX cell line: rapid cell culture techniques, cryopreservation methodologies and the development of protocols for automated manufacturing processes. The Cell Therapy 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.

…the University of Loughborough , Centre for Biological Engineering, are collaborating to develop robust processes and new manufacturing and delivery techniques, removing the hurdles associated with turning cell-based therapies into products, and providing training and skills development. The collaboration will focus on tackling some of the challenges associated with scale-up and manufacturing of cell therapies e.g. ensuring reproducibility, purity, potency and efficacy

…the UK Stem Cell Foundation are collaborating to progress promising stem cell-based therapies in the UK. The two organisations are both focused on translational activities, moving potential therapeutic approaches on from early-stage research to the later stages of development and together will build a pipeline of projects for support and funding on the route to commercialisation

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Accessible clinical research infrastructure with leading activities in regenerative medicine

The UK is committed to making clinical translation more efficient through:

Investing in health research infrastructure £500 million annually to support experimental medicine research and clinical trials in the NHS in England

Introducing the Health Research Authority to streamline approvals for clinical research

Support, provided through the NIHR Clinical Research Network (CRN), to enable over 25 clinical studies in regenerative medicine funded by partners to take place within the NHS at the current time

Simplifying access for industry (through the NIHR Office for Clinical Research Infrastructure (NOCRI), NHS Research Scotland (NRS) Permissions Coordinating Centre and Health Research Wales)

NIHR supports several activities in regenerative medicine through the BRCs and BRUs but also through co-funding of specific initiatives in the field

NOCRI facilitates industry access

£500+ million annual spend

on infrastructure to deliver

60+ million patients in

the National Health Service


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Regulatory support: – You can seek regulatory advice and support from a number of organisations including the

Medicines and Healthcare products Regulatory Agency (MHRA), the Health Research Authority (HRA) , European Medicines Agency (EMA) and the Cell Therapy Catapult.

– The MHRA Innovation Office opened in March 2013 to foster and support innovation in the development and manufacture of medicines and the development of medical devices.

Gateway to Europe: The UK’s sound regulatory base is well understood by the EMA making the UK a recognised route into the European market, as evidenced by the number of European Headquarters in the UK.

Streamlined regulatory processes: – The HRA works closely with the MHRA and others to create a unified approval process and to

promote proportionate standards for compliance and inspection.– The regulatory pathway for stem cell research in the UK is well established, and is documented

for different research scenarios (human stem cells only) in the UK Stem Cell Toolkit, which has been established by the MRC and Department of Health (DH) to provide a one-stop-shop to orientate researchers to the regulatory needs in the stem cell area.

– The regulatory framework allows for procurement of hES cells from super-numary embryos by law and for the creation of human embryos for research purposes including cloning

On-going Regulatory Improvement: For example, the MRC and ESRC will co-sponsor workshops with field participants, design experts and regulators such as the MHRA, to explore clinical trial challenges to improve transparency of the regulatory framework and improve trial design.

The UK has a clear and well established regulatory framework that

is harmonised for Europe


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Accessing the UK market, and the National Health Service (NHS), is of strategic importance increasing the probability of successful entry into the European market Guidance and recommendations by the National Institute for Health and Care Excellence (NICE)

and the Scottish Medicines Consortium (SMC) are respected Europe-wide 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 – it allows you access to the whole 60 million patient population, clinical trials infrastructure and patient data

How is NICE supporting regenerative medicine treatment evaluation It’s evaluation methods can, and do, assess drugs and treatments with high upfront costs, and

they fully reflect all health gains to patients – including lifelong health impacts of curative treatments.

it keeps its methodologies under review to ensure that they remain fit for purpose it is currently developing the new value assessment system for products covered by value‐

based pricing it is supporting the design and evaluation of each CtE programmeEase of access to the NHS for regenerative medicine treatmentsCompanies with a single product with impact in the specialised commissioning space will have the ability to interact directly with NHS England for adoption. 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).

Supporting market access: facilitating adoption into the NHS


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Progressive Licensing – Intercytex product examples (for modifying table)

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 development: A close partnership between client, contract developer and hospital allowing iterative development of new treatments

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 trial completed 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


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Appendix: Optional Introduction

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David CameronPrime Minister

Life Science Strategy and Innovation Health and Wealth are the UK government’s response to a global sector under pressure

“In December 2011 I made a firm commitment to re-establish the UK's global leadership in the life science sector, announcing the Government's ten-year Strategy for UK Life Sciences.”

“By more closely integrating the UK's unique strengths, I believe that we can improve healthcare for patients, attract new investment to the UK, and create new jobs and business opportunities in an increasingly competitive and international industry.”

This strategy makes the most of the UK’s core strengths:

Basic science in universities Translational and clinical research excellence Industry and supply chain The National Health Service (NHS)

The UK is committed to supporting Life Sciences and Healthcare

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The Life Science Investment Organisation (LSIO) is a new UK Trade and Investment (UKTI) team that combines private-sector expertise with Government teams. It is charged with:

About the UKTI Life Science Investment Organisation

Matching UK strengths with global business needs

Translating benefits outlined in the Strategy for UK Life Science and Innovation, Health and Wealth into opportunities for business

Catalysing and empowering the UK Life Science community to work with international business


+ =

1

2

3

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Building on this strong heritage, the UK Government has put together the Regenerative Medicine Strategy to ensure that companies coming to the UK benefit from:

Access to cutting edge research innovation and infrastructure– In 2012, the UK Government invested £77m in regenerative medicine and has committed to

continued strategic investment in 2013 and beyond.

A collaborative, multidisciplinary and joined up network– By bringing together strong complementary skills, expertise and infrastructure across

disciplines through initiatives such as the UK Regenerative Medicine Platform, the UK will ensure that innovative research activities are aligned to clinical demand and deliver impact fast

Early stage regenerative medicine product development – Through the establishment of manufacturing, transportation and delivery solutions

A healthcare market ready for your product– Through establishing the most effective trial designs and improving the transparently of the

regulatory framework– Addressing issues such as the evolution of new business models, product development

mechanisms (including reimbursement & adoption), and open innovation– The Regenerative Medicine Expert Group is being established to develop an NHS regenerative

medicine readiness strategy and action plan

The UK’s Regenerative Medicine Strategy will ensure that UK leadership continues in the decades to come


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Regenerative Medicine recognised as one of the UK’s Eight Great Technologies The Strategy for Regenerative Medicine provides a roadmap for future regenerative medicine

research in the UK with eight industry relevant objectives

The UK Government continues to invest heavily into the field with £77m UK public sector investment in regenerative medicine in 2012

The Regenerative Medicine Expert Group is being established to develop an NHS regenerative medicine readiness strategy and action plan

The UK’s ambition is to be a global leader in regenerative medicine

Underpinning ResearchContinued strategic investment in centres of excellence and partnerships with industry

Therapeutic OptionContinued support for translational science through the MRC/TSB/EPSRC

Product DevelopmentLinking early stage regenerative medicine product development to the establishment of manufacturing, transportation and delivery solutions

Clinical Delivery & EvaluationEstablish the most effective trial designs and improve the transparency of the regulatory framework

Innovation & Value SystemsTo address issues such as the evolution of new business models, product development mechanisms (including reimbursement & adoption), and open innovation

InternationalWork with overseas partners (established and new) to capitalise upon emerging opportunities and provide access to complementary expertise that will benefit UK science

FocusAchieve key goals such as clinical proof-of-concept through focused efforts and critical mass

Interdisciplinary CollaborationBringing together of strong complementary skills, expertise and infra structure across disciplines


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A global opportunity in regenerative medicine

The regenerative medicine market is predicted to grow to over $35 billion by 2019

Three $1 Billion thresholds reached in 2012:– Monies raised– Sector transactions with large biopharma

and device companies– Revenues from approved products

The UK is your launch pad into Europe Europe is the second busiest territory for

regenerative medicine – >400 companies related to tissue

engineering or regenerative medicine. – The UK and Germany host the largest

number of such companies– There is an active, collaborative R&D

landscape in Europe with significant EU support

Nearly one in three (28%) of SME’s which are active in Europe’s Advanced Therapy Medicinal Product (ATMP) market are based in the UK.

The European market for stem cell products alone was $872 million in 2011 and is expected to reach nearly $1.5 billion by 2016

There is a global market opportunity for regenerative medicine

Regenerative medicine has the potential toaddress many of the challenges facingHealthcare Aging population and unmet medical

needs: Growing unmet medical needs for neurodegenerative diseases, stroke and heart failure.

Healthcare costs: Economic potential of regenerative medicine to save significant healthcare and development costs. Today 80% of healthcare costs go on treating the late stages of illnesses - in the future these could be cured early or better managed using regenerative medicine and cell therapies.

Development costs: Stem cells could have a pivotal role as alternative models for screening candidate drugs and to identify novel targets, reducing expensive and time consuming failures in Phase III Trials,

We are now starting to see a transition from promise

to reality with many commercially and clinicallysuccessful products• Over 100 regenerative medicine products on

the market around the world focused on diverse therapeutic areas


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Your business can access the UK regenerative medicine market by engaging with a range of UK healthcare markets

The UK in 2011-12 collectively spent approximately £128bn on the National Health Service, a publicly funded healthcare system which is free at the point of service

The UK private sector healthcare market was valued at £30.4bn in 2010 and is estimated to reach £35.9bn by 2015.

NHS Scotland£11.5bn

Department of Health in England

£106.6bn

Department of Health

Social Services and Public Safety,

Northern Ireland£4.4bn

NHS Wales£6.3bn


The UK market opportunity for your regenerative medicine business

NHS spend: Breakdown by region (2011-12)

With a vibrant research base and growing industry activity, there is also a thriving market for consumables, services, and tools and technologies to support the regenerative medicine sector in the UK

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Industry can leverage the UK as an innovation partner to develop an evidence base, supporting market access, uptake and diffusion in the UK and around the world

Providing Access to Drive Healthcare Innovation:Active Partnership Between Academia, Industry, and the NHS

IN NORTHERN IRELAND: Connected Health and Prosperity Action Plan

ensures cross-sectorial support for R&D and technology adoption

Invest Northern Ireland, the region’s business development agency, provides a single access point for industry investment interest.

IN WALES: Academic Health Science

Collaboration maximises links between health boards, trusts, universities and industry

The Welsh Government gives you easy access to key decision-makers and private sector specialists, to help fast-track your investment.

Health Research Wales is one-stop shop for industry access to NHS Wales

IN SCOTLAND: Academic Health Sciences Collaboration

facilitates partnerships between universities and health boards

Health Sciences Scotland is a single industry access point for investing in health and life sciences in Scotland, supported by Scottish Development International and Scottish Enterprise

IN ENGLAND: Biomedical Research Centres and Units

link universities and NHS trusts Academic Health Science Centres open

up routes to market Academic Health Science Centres,

university and NHS partnerships of world-class excellence, collaborate with industry across research, education and patient care


The Cell Therapy Catapult is a national resource acting as a gateway to all of these partners

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Another benefit to your company is through access to patient data. The UK, enabled by the NHS, allows your business to access unrivalled, clinically-coded health data and bio-repositories, including linked datasets to understand care pathways and anonymised electronic patient records, offering a unique opportunity to understand care pathways.

Unrivalled data to drive innovation in regenerative medicine

More than 60 million people are served by a National Health System

Example data resources:– UK Biobank is a unique resource of data and samples

linked to medical histories and health records from 500,000 adult participants

– Clinical Practice Research Datalink (CPRD) enables observational studies, clinical trial feasibility and protocol optimisation, and post-market surveillance

– NIHR BioResource enables recall to clinical studies by genotype and phenotype and helps stratify and select patients for trials


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World class resources that drive innovation

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 from stem cells to

nanotechnology– Multiple academic centres of excellence in the field including

the Wellcome Trust—Medical Research Council (MRC) Cambridge Stem Cell Biology Institute and the University of Edinburgh MRC Centre for Regenerative Medicine, the European Cancer Stem Cell Research Institute at Cardiff University, amongst others

– UK Regenerative Medicine Platform funds ‘hubs’ across the UK Connectivity through specialised networks

– Knowledge transfer networks (KTN), London Regenerative Medicine Network (LRMN), Regener8, ATMP Manufacturing Community

Talent and expertise– Funding for training centres in regenerative medicine such as

the EPSRC doctoral training centres (Leeds and Loughborough) World-class cell and tissue banking facilities

– UK Stem Cell Bank, NHS Blood and transplant (NHSBT) Tissue Services, Scottish National Blood and Transfusion Service (SNBTS), public and private cord blood banks

Funding for UK based companies– Through the Technology Strategy Board (TSB); Devolved

administrations– Leverage funding through collaboration with the UK science

base


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”.

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Existing manufacturing capabilities with proven ability to deliver

Companies can work with established manufacturing facilities experienced in delivering for clinical trials A large numbers of manufacturing and bioprocessing facilities Established Good Manufacturing Practice (GMP) facilities with

experience in working with industry such as the Newcastle Cellular Therapies Facility, one of the largest of its kind in Europe, and Cell2Therapy

Advanced Therapy Medicinal Product (ATMP) manufacturing– E.g. The Scottish Centre for Regenerative Medicine has state-of-

the-art facility with 6 GMP-compliant clean rooms; operated by Roslin Cells (and SNBTS). The facility is designed - and approved – for clinical manufacture of ATMPs

The UK is also leading the innovative manufacturing revolution with investment into centres of excellence actively collaborating with and supporting industry activities to develop processes and techniques for cost-effective scale up and manufacturing Support for product developers in manufacturing innovation

– E.g. EPSRC centre for innovative manufacturing in regenerative medicine (Loughborough, Nottingham and Keele Universities); Innovation and Knowledge Centre in Regenerative Therapies and Devices (University of Leeds)

Expertise in Scaffold design, fabrication and analysis – E.g. Wolfson Centre for Stem Cells, Tissue Engineering &

Modelling, Nottingham

Athersys, an international biopharmaceutical company with operation in the UK and Belgium, is working with the Newcastle Cellular Therapies facility as part of its clinical trials in stroke currently being conducted in the UK.


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A strong clinical research infrastructure allowing to design and deliver clinical trials efficiently

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 to support experimental medicine research and clinical trials in the NHS in England

– Support, provided through the National Institute for Health Research (NIHR) Clinical Research Network (CRN), to enable over 25 clinical studies in regenerative medicine funded by partners to take place within the NHS at the current time

– Access to specialist clinical expertise

– Clinician led translation from bench to patient

– NIHR supports several activities in regenerative medicine through the Biomedical Research Centres (BRCs) and Biomedical Research Units (BRUs)

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, Health Research Wales

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 NIHR Cambridge Biomedical Research Centre 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.


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