Unlock Your Global Business Potential:
UK Regenerative Medicine and Stem Cells
By placing your business in the UK, your company can benefit from a
rich heritage in regenerative medicine and stem cells
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.
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.
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.
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
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.
Sydney Brenner, a researcher at the Medical Research
Council Unit in Cambridge is awarded the Novel Prize
in Physiology or Medicine for his work on the genetic
regulation of organ development and programmed
cell death.
2003
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.


Researchers at King’s College London, led by Professor
Peter Braude, generate the UK’s first embryonic stem
cell line.
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
2
By placing your business in the UK, your company can benefit from a
rich heritage in regenerative medicine and stem cells
2007
Teams 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.
2008
A 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.
2010
ApaTech, a spin-out from Queen Mary University of
London, and a world-leader in bone graft technologies is
sold for $330M to Baxter.
2011
ReNeuron’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.
UK 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.
2012
2012
the 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.
The 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.
2011
2013
The 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.

2012
Sir John Gurdon, of the Gurdon Institute in Cambridge,
together with Shinya Yamanaka, is awarded the Nobel
Prize for the discovery that mature cells can be
converted to stem cells.
Recognising the challenges facing the application of regenerative medicine, the UK continues to invest in, and develop this sector, supporting worldfirst clinical trials and financially supporting innovative new companies with non-dilutive funding
3
The UK recognises the need to support industry deliver patient benefit
and achieve commercial viability
4
Collaborate and work in the UK to access world class resources that
are delivering results
1
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.
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 noted that “locating the new laboratory in the
area will give us access to one of Europe's strongest
biotech hubs”.
5
Comprehensive supply chain and logistics allow you to deliver on time
and cost effectively
2
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.
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
6
Access existing manufacturing capabilities with proven ability to deliver
3
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
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.

Support for product developers in manufacturing innovation

Expertise in Scaffold design, fabrication and analysis
7
Access a strong clinical research infrastructure allowing you to deliver
clinical trials efficiently
4
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

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.
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
8
The UK provides commercial support for all your activities
5
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.
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 consultancies
The UK regenerative medicine Industry has a united voice through
the BioIndustry Association’s (BIA) Cell Therapy and RegenMed
Advisory Committee
9
The UK is your gateway to Europe and the global markets
6
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.
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 Europewide.
–

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).
10
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
R&D tax credits
for SMEs worth
approximately 25p
on every £1 (7p
for large
companies)
23%
Corporation
tax will drop
further to
20%
Low corporation tax rate currently
standing at 23 per cent decreasing further
to 20 per cent by April 2015.
£180m
Patent
Box
Patent box: 10%
corporation tax on
qualifying profits
£300m
To stimulate R&D
partnerships between
universities
businesses &
charities
Biomedical
Catalyst = £180m
programme of
public funding for
growth
11
Clinical success: the UK is one of the leading European countries
developing Advanced Therapy Medicinal Products
You can work with leading clinicians and clinical centres to deliver your clinical trials easily, efficiently and to a high
quality.
Figure 1: Clinical trials, in the UK, by therapeutic area



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
Bone and
cartilage
9%
Diabetes
9%
Ophthalmology
9%
Gastroenterolgy
9%
Trials by stage:
Phase III
Neurological
12%
1
Phase II
12
Phase 1/II
17
Phase I
2
0
Blood
14%
Cardiovascular
12%
Trial in the UK by therapeutic area
2
Phase II/III
Oncology
26%
5
10
15
Oncology
9
Blood
5
Cardiovascular
4
Neurological
4
Gastroenterology
3
Ophtalmology
3
Diabetes
3
Bone and Cartilage
3
20
Data from Cell Therapy Catapult report
12
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.
Stroke
Voice-box Reconstruction
Huntington Disease
Multiple Sclerosis
Rheumatoid Arthritis
Ophthalmology
Tendon repair
Cardiovascular
Cartilage repair
Diabetes
Immunotherapy
Digestive Tract
Liver cirrhosis
13
Your competition is already benefiting from the UK Offer in
Regenerative Medicine




Ease of doing business: a supportive business environment coupled with
Government commitment to support the sector
– Business incentives such as year on year reductions in corporation tax,
R&D tax credits and the Patent Box, culminating in an improved business
environment in the UK.
– Increased funding into the sector from Government as well as UK charities
Rich and Diverse Ecosystem to tap into
– World class researchers, centres of excellence and research infrastructure
– A strong and growing supply chain
– Established manufacturing facilities
– Innovative manufacturing approaches
– A single healthcare system that treats 60 million patients, generating vast
volumes of structured real world data.
– Unrivalled access to cell and tissue samples to enable discovery and
development.
– A Cell Therapy Catapult to support your activities from business models,
manufacturing to clinical and regulatory expertise
Market Access: the UK, European and global markets
– Clear, well established regulatory framework, harmonised for Europe
– New commissioning models available for specialised products and
services allowing direct access to the whole of the NHS
– Strategy being developed for NHS readiness and action plan to facilitate
adoption of regenerative medicine products
A track record of successful collaboration with industry
– The NHS and research base working closely with industry
– Available support and funding for UK based companies
14
Find out how UK Trade & Investment (UKTI) can help your business
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
For further details on the LSIO and the UK life sciences offer for industry,
visit: www.ukti.gov.uk/lifesciences
15
16
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















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
17
Appendix: Success Stories
18
How the UK environment helped ReNeuron be a leader in the field
ReNeuron






ReNeuron is a leading, clinical-stage stem cell business. The
company’s primary objective is the development of novel stem
cell therapies targeting areas of significant unmet or poorly met
medical need.
ReNeuron’s clinical trials in disabled stroke patients: The
PISCES study (Pilot Investigation of Stem Cells in Stroke) is the
world’s first fully regulated clinical trial of a neural stem cell
therapy for disabled stroke patients. The trial is being conducted
in Scotland at the Institute of Neurological Sciences, Southern
General Hospital, Greater Glasgow and Clyde NHS Board.
Phase II of the stroke trial is starting with support of a £1.5
million grant from the Technology Strategy Board (TSB)
Also received regulatory and ethical approvals to commence a
Phase I clinical trial in the UK with its ReN009 stem cell therapy
programme critical limb ischaemia (CLI). The trial will be
undertaken through NHS Tayside at Ninewells Hospital and
Medical School, Dundee, Scotland.
Cell manufacturing for the trial will be carried out by Roslin Cells
Completed significant £33 million fundraising, with £7.8 million
from the Welsh Government, to continue development and build
a new manufacturing facility.
19
How the UK environment helped Cell Medica succeed
Cell Medica





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.
“Fortunately for us the Government had identified areas of important
“Fortunately for us the Government had identified areas of important
innovationininindustry
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20
Intercytex: using a Progressive Translation approach to take
product to market
Intercytex

Intercytex is a regenerative medicine product and services company focused on
developing its lead product to treat a variety of skin related problems. These
treatments are being brought to patients through Intercytex’s innovative Progressive
Translation system which is available to third party clients through their Cell2Therapy
CMO service
Intercytex operates a fully licensed GMP manufacturing facility and has experience in
producing a variety of autologous and allogeneic 2D and 3D cell therapy products. Its
current product Vavelta/ICX-RHY, an injectable allogeneic fibroblast suspension, is a
good example of Intercytex applying the Progressive Translation approach:

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
Biocatalyst grant to
 Developed costing models
Improve shelf life and
for current system and scale
logistics to enable
up options
 Phase II clinical trial
pharmacy supply
completed
 Obtained full hospital
 Working with Cell Therapy
reimbursement to treat first
 Specials license obtained
Catapult to produce less
patients under Specials
for Intercytex
painful injection system
manufacturing facility
 Created a partnership with
 Working with charities to
“Fortunately for us the Government
identified
areasexamine
of important
University had
Hospital
of
reimbursement
South Manchester to
possibilities
innovation in industry - and cell
therapy
was
one
of
them…Through
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Technology Strategy Board'sTranslation
involvement,
we had the benefit of
recently announced the
collaboration with Great Ormond
Street
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and University College
expansion
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GMP
“The UK provides a unique
mix ofand the opportunity facility
onto the
London,
to exchange
ideas with other organisations
Manchester Airport City
highly networked world-class
scientists,
working
in the same field’. Karen
Hodgkin,
Senior Vice President of
Enterprise
Zone
a globally respected andClinical
helpful Development
 Working with suppliers on
scale-up options
Orphan designation
obtained in EU and US
regulatory system, a National Health
service and a succession of supportive
Governments”, Dr Paul Kemp, Chief
Executive and Chief Scientific Officer
21
Neusentis: working in partnership with academic researchers
in Cambridge and across the UK
Neusentis



Neusentis is a Cambridge-based Pfizer research unit that
delivers 'new science therapeutics', with a particular focus on
pain and sensory disorders. It works in partnership with
academic researchers in Cambridge and across the UK.
Within regenerative medicine, Neusentis is developing cell
based therapies for ulcerative colitis, age related macular
degeneration and a drug to regenerate neural circuit function in
stroke patients.
A key partnership case study is the work being done on Cell
Replacement Therapy for Age Related Macular Degeneration in
the ‘London Project’. ‘The London Project’ was formed by
Professor Pete Coffey (Institute of Ophthalmology, UCL) and
Lyndon da Cruz (Moorfields Eye Hospital) to produce a cell
replacement therapy for AMD. In 2008 The London Project
teamed up with Pfizer to help move the project into the clinic so
as to determine whether this cell replacement therapy was safe
and whether it lead to an increase in vision in patients with AMD.
The work has pulled in expertise from across the whole Pfizer
organisation and the team have received approval from the
authorities to proceed with the trial.
GE Healthcare Life Sciences chooses Wales for its new state
of the art laboratories for cell therapy and cell bioprocessing
GE Healthcare


In 2012, GE Healthcare Life Sciences, a business unit of GE
Healthcare, opened its new £3 million cell science laboratories in
Cardiff , Wales. The state-of-the-art investment at the company’s
Maynard Centre creates is a world-class facility for research and
technology innovation in cell science, to help advance the rapidly
emerging fields of cell therapy and cell bioprocessing, and to support
the development of new, more effective and safer medicines.
The laboratories will focus on the development of novel technologies
for the rapidly emerging field of cell therapy and on advancing new
cellular based tools to help pharmaceutical companies develop better
and safer medicines at lower cost. The facility is equipped with the
latest technologies for cell bioprocessing and cell imaging, as well as
an industry-leading clean-room manufacturing facility. In addition,
GE’s customers and scientific collaborators will benefit from an
advanced technical training centre.
“Cellular research has an extraordinary potential to address some of
the world’s biggest healthcare challenges. Our investment in these
new laboratories is a great milestone and demonstrates our
commitment to innovation and excellence in cellular science. Here in
Cardiff we have established an outstanding team of scientists, many of
whom are leaders in their field, and these new facilities will allow us to
greatly expand our work.”
UK Approves Europe's First Embryonic Stem Cell Clinical Trial
Advanced Cell Technology
Moorfields Eye Hospital

Advanced Cell Technology, a US-based company received
permission, in 2011, by the UK Medicines and Healthcare products
Regulatory Agency (MHRA) to start Europe's first clinical trial involving
human embryonic stem (hES) cells.

The ongoing trial aims to treat 12 patients with Stargardt's macular
dystrophy. The disease strikes people between the ages of 10 and 20,
causing progressive vision loss. There is currently no treatment.

Eye surgeon James Bainbridge of Moorfields Eye Hospital and
University College London leads the UK trial.
24
MHRA Authorizes Athersys to Expand Ongoing Phase 2 Clinical
Trial of MultiStem Cell Therapy in Ischemic Stroke
Athersys




Athersys (Cleveland, OH, US) is a clinical stage biotechnology company
engaged in the discovery and development of therapeutic product
candidates designed to extend and enhance the quality of human life. The
Company is developing its MultiStem® cell therapy product, a patented,
adult-derived "off-the-shelf" stem cell product platform for disease indications
in the cardiovascular, neurological, inflammatory and immune disease
areas.
In April 2013, the Medicines and Healthcare products Regulatory Agency
(MHRA) approved Athersys’ application to expand its ongoing Phase 2 study
evaluating the administration of MultiStem® therapy to patients who have
suffered an ischemic stroke. Ethics Committee review was been successfully
completed in August, 2013, and enrolment at multiple United Kingdom sites
will commence following the completion of final preparations at participating
clinical centres.
The study is expected to enrol approximately 136 patients in total and is
currently being conducted at multiple centres throughout the United States.
Athersys is working with the Newcastle Cell Therapies Facility for production
of cells for the trials
"The MHRA authorization will enable us to bring several leading United Kingdom
stroke centres into the study, which will help us to speed the completion of the stroke
clinical trial. The authorization is also noteworthy as it marks the initiation of MultiStem
clinical development activity in the United Kingdom.”
Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys
25
EPSRC Centre for Innovative Manufacturing in Regenerative
Medicine collaborating with industry
The EPSRC Centre for innovative
manufacturing in regenerative
medicine





The EPSRC Centre for Innovative Manufacturing in Regenerative Medicine
provides an integrated platform of fundamental and translational research as
a ‘go-to’ resource for the regenerative medicine product developer.
It is one of the few centres in the world providing academic and industrial
translational research in regulated regenerative medicine manufacturing, in
order to grow the industry, deliver benefit to patients, and develop the UK
research community.
It is a partnership of Loughborough, Keele and Nottingham Universities and
industry, with currently around 20 industry and clinical partners. Centre
institutions participate in three of the UK Regenerative Medicine Platform
hubs.
The Centre places an emphasis on cost-effective, robust manufacturing
system and process design with a focus on both regulatory compliance and
informing the regulators, in particular with respect to the implementation and
principles of good manufacturing practice.
An example of the collaborative work carried out at the centre is the
collaboration with Ruskinn Technology Ltd -one of the world’s leading
suppliers and manufacturers of anaerobic and modified atmosphere
workstations. Ruskinn was recently acquired by The Baker Company, USA around the development of HypoxyCOOLTM. Ruskinn
“The EPSRC project has provided the essential proof of principle for HypoxyCOOLTM,
and through such support has better ensured positive outcomes both in terms of the
application of autologous cell production and adding value to the Ruskinn business.”
Huw David Thomas, UK Commercial/Sales Manager, Ruskinn Technology Ltd
A University of Swansea spin-out joins efforts with Biotec
Services International to form TrakCel
TrakCel

TrakCel is an orchestration platform to safeguard patients being
treated with regenerative therapies. The platform ensures that the
right patient receives the right therapy at the right time and location.
Efficiently tracking, tracing and documenting the movement and
handling of multiple regenerative therapies with minimal resources
required, using state of art technologies including biometrics, Radio
Frequency Identification (RFID) and Global Positioning.

TrakCel’s was formed as a joint venture between AwenID (a university
of Swansea spin-out) and Biotec Services International (storage and
distribution of unlicensed, temperature-sensitive medicinal products
for use in clinical trials).

The company’s technology platform is going to be assessed by GSK
in Q1 2014. As part of a Department of Business Innovation and
Skills, Advanced Manufacturing Supply Chain Initiative (AMSCI) the
company has received financial assistance to develop its technology
for gene therapy applications.
How the UK environment supported Tissue Regenix
Tissue Regenix

Tissue Regenix is a medical technology company which uses a
proprietary technology platform, dCELL ®, which removes cells and
DNA to make replacement body parts from biological (animal)
materials.

The three priority markets for the application of the technology are:
Vascular; Cardiac; and Orthopaedics

A Technology Strategy Board (TSB) quality systems project led to
Tissue Regenix’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.
28
Appendix Clinical successes
29
Clinical Firsts: Windpipe Reconstruction
University College
London

In 2008, Professor Martin Birchall, in collaboration with Professor
Paolo Macchiarini, University of Barcelona, led the European Team
which successfully performed the World’s first stem-cell based, tissueengineered 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 antirejection 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.
30
Clinical Activities: Exploiting Stem Cells for Immunotherapy
University of Oxford
Cell Therapy Ltd

Researchers from the University of Oxford, led by Dr Paul Fairchild,
pioneered the differentiation of dendritic cells and their individual
subsets from pluripotent stem cells, both embryonic and induced
pluripotent stem cells (iPSC). The properties of this novel source of
dendritic cells promises to overcome many of the obstacles to their
use in immunotherapy.

In conjunction with collaborators at Great Ormond Street Hospital, the
group plans to scale up the production of dendritic cell subsets from
iPSC for use in a number of indications either requiring vaccination or
the induction of tolerance.

Future clinical trials will be conducted in collaboration with Cell
Therapy Ltd, for which Nobel laureate Sir Martin Evans serves as cofounder and director.
31
Clinical Firsts: Liver Failure
Imperial College London

Doctors at Imperial College, London, led by Professor Nagy Habib,
were the first to undertake phase I and II studies to treat patients with
liver failure with autologous adult bone-marrow derived cd34+ stem
cells.

It is planned to popularise the therapy in the immediate future and to
extend it to other indications. Clinical trials have been initiated in
patients with diabetes and stroke and will be started soon for patients
with cardiac disease and tibial fracture.

Clinical results to date have been encouraging. A new allogeneic stem
cell therapy for acute liver failure is anticipated in the foreseeable
future.
32
Clinical Firsts: Digestive Tract
Great Ormond Street
Hospital

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.
33
Clinical activities: Rheumatoid Arthritis
Newcastle University

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 immunomodulatory 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
34
Clinical Activities: Huntington Disease
Cardiff University
University of Cambridge
Imperial College London
Addenbrooke’s Hospital

A safety study for the transplantation of human fetal striatal tissue into
the striatum of five patients with Huntington’s disease has
demonstrated that the procedure is safe and feasible.

Stereotaxic tissue placement following pre-operative magnetic
resonance imaging was performed in Cambridge, UK by investigators
from the National Institute for Health Research (NIHR) Cambridge
Biomedical Research Centre (BRC) as part of a broader UK network.

Cambridge-based protocols for the screening of human fetal tissue for
potential pathogens have also been developed, with clinical trials
planned in Parkinson’s disease.

This is part of a larger European collaboration with several expert
centres in the UK
35
Clinical successes: limbal epithelial stem cell transplantation
Newcastle University
The Newcastle upon Tyne Hospitals

Newcastle’s Royal Victoria Infirmary is a centre of excellence for the
treatment of mechanical, chemical or thermal damage to the Cornea.
The development of methods to grow limbal epithelial stem cells by
Prof Linda Lako and Prof Francisco Figueiredo of Newcastle
University made this treatment possible.

Professor Figueiredo (Consultant Ophthalmologist) has successfully
treated eight consecutive patients with total unilateral LSCD using
LSC autografts, resulting in significant improvements in visual
impairment and pain scores in all cases

The results of the phase I clinical trials show that limbal epithelial stem
cell transplantation is a robust method to treat corneal damage

A larger study, sponsored by the Medical Research Council, involving
24 new patients is underway to evaluate the long-term safety and
efficacy of cultured human LSC for the treatment of patients with
unilateral total LSCD.

Newcastle University and the Royal Victoria Infirmary are working with
the Cell Therapy Catapult to achieve European Market Authorisation
36
Cells For Sight
University College London
Moorfields Eye Hospital

The Cells for Sight Team is aiming to understand the biology and
therapeutic potential of stem cells (and the cells with which they
interact) to develop and deliver novel cell-based therapies and
anti-scarring strategies for patients with blinding corneal and
conjunctival disease.

The Cells for Sight Stem Cell Therapy Research Unit is a MHRA
licensed state-of-the-art GMP facility is available to internal and
external researchers (academic and commercial) for the
manufacture of Advanced Therapy Medicinal Products.

To date 25 patients with blinding ocular surface disease have
been treated with cultured limbal stem cell therapy. The Unit is
manufacturing human embryonic stem cell-derived retinal
pigmented epithelial cells for transplantation in a clinical trial
headed by Professor Pete Coffey 'The London Project to Cure
Blindness'.
37
Clinical activities: Knee cartilage repair
Keele University
Robert Jones & Agnes Hunt

Keele University (Sponsor) and Robert Jones & Agnes Hunt
Orthopaedic Hospital NHS Trust (Host organisation) are working
together to deliver the results of a phase II trial comparing
Autologous Chondrocytes Implantation (ACI) versus existing
techniques for knee cartilage repair

Multi-centre: 27 UK sites and 2 sites in Norway

400 patients were recruited onto this trial
38
Clinical activities in Scotland
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
39
Clinical activities: Cardiovascular disease
4
1
Autologous bone marrow derived mononuclear
cells for acute myocardial infarction. Combines
stem cell delivery with primary angioplasty within 5
hours post event
Randomised control trial to compare the effects of
G-CSF and autologous bone marrow progenitor
cells infusion in patients with ischaemic heart
disease
Barts Health NHS Trust, Queen Mary University of
London, University College London
Barts Health NHS Trust, Queen Mary University of
London
Phase I/II, recruitment target of 70 patients
Phase II, recruitment target of 90 patients
3
2
Autologous bone marrow derived mononuclear
cells for dilated cardiomyopathy, delivered via
intracoronary injection
Expanded adult haematopoietic CD34+ stem cells
for autologous infusion to patients with myocardial
ischaemia
Barts Health NHS Trust, Queen Mary University of
London
Imperial College London
Phase I/II, recruitment target of 42 patients
Phase I/II, recruitment target of 60 patients
40
Clinical activities: multiple sclerosis
UK Stem Cell Foundation
Multiple Sclerosis Society

UK Stem Cell Foundation and the Multiple Sclerosis (MS) society fund
several projects targeting multiple sclerosis. This includes clinical trials
such as the phase II trials looking at the use of autologous
mesenchymal stem cells to treat relapsing-remitting MS.

Led by Dr Paolo Muraro at Imperial College London, this clinical trial is
part of an international collaboration involving 150 to 200 people with
MS across Europe, America and Canada.

Edinburgh and London sites.

The London site aims to recruit 13 patients.

The trial will test whether stem cells can treat the active MS lesions of
the participants, where damage is currently occurring on their brains.
41
Clinical Activities: Achilles Tendinopathy
UK Stem Cell Foundation
University College London

Led by Andrew Goldberg OBE of University College London (UCL)
and the Royal National Orthopaedic Hospital, this first-in-man pilot
study explores the treatment of Achilles Tendinopathy using a culture
of expanded autologous mesenchymal stem cells to repair damage.

The project is funded, in part, by the UK Stem Cell Foundation
(UKSCF)

Stems cells will be taken from the patient's own hip, expanded in the
laboratory then implanted into the damaged tendon.

UCL is one of the few centres in the world developing expertise into
the regeneration of tendons using stem cells, as well as translating
successful treatments used in horses into humans.

Achilles Tendinopathy causes pain in the heel and affects more than
70,000 people a year in the UK.
42
Clinical successes: Allogeneic pancreatic islet cell transplantation
for type 1 diabetes mellitus
DIABETES UK

Diabetes UK funded 12 islet transplants as part of a research project.
This was successful, with all patients achieving complete resolution of
severe hypoglycaemia.

On the basis of these results, the National Specialist Commissioning
Group provided central funding for the service.

Islet cell transplantation is now NICE Approved and available through
the NHS, and the UK benefits from having one of the only
government-funded islet cell Transplantation services in the world.

As of 2013, 95 islet transplants had been performed in 65 people in
the UK (Diabetes UK)
43
Appendix Examples of leading research
44
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 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 worldleading expertise in the fields of stem cell biology,
immunology, tissue engineering, materials science,
medicinal chemistry and cancer biology the OSCI aims to
create a seamless pathway from basic science, through
translational medicine to clinical trials.
The 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.
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 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
45
The Medical Technologies Innovation and Knowledge Centre, Leeds
University of Leeds



Medical Technologies IKC facilitates collaboration between
companies, engineers, scientists and clinicians to develop
innovative technologies that help the body repair and restore
function.
Work in partnership with the centre to:
• speed up your route to market
• work with world-leading experts to ensure products and
services are fit for purpose
• reduce the risk of late-stage failure
• access resources to support innovation for new product
development
Examples of capability areas include:
• Novel protein biosensors to diagnose disease and monitor
patients’ responses to treatment
• Use of minimally manipulated stem cells for
musculoskeletal and cardiovascular repair
• Regenerative biological scaffolds to replace damaged joint
and heart tissue
• Improved imaging to allow early diagnosis and targeted
treatment
• Longer lasting joint replacements for the hip, knee and
spine
• Enabling technologies
46
Pioneering translational stem cell research for cardiovascular
applications at the University of Bristol
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.
47
International research collaborations: The University of Durham
Durham University

Researchers at Durham University and Columbia University Medical
Center (CUMC), USA, have devised the method which is the first to
use cloned human cells to induce hair growth, rather than
redistributing hair from one part of the scalp to another.

These findings could significantly expand the use of hair
transplantation to women with hair loss, who tend to have insufficient
donor hair, as well as to men in early stages of baldness.

Previous research by Professor Colin 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
48
The Wound Healing Research Unit supports industry activities
Cardiff University

Headed by Prof Keith Harding, the Wound Healing Research Unit
(WHRU) is a world-class clinical, scientific and educational wound
healing resource for the benefit of people with wound problems,
providing expertise in biophysical and clinical science research.

Acts as an interface between healthcare providers, industry and the
academic community, to develop models of high quality care based on
sound scientific research.

WHRU has conducted 9 studies over the past 10 – 15 years for
ConvaTec (part of Bristol Myers Squibbs).

Clinical studies have included a randomised controlled trial of 131
patients, comparing AQUACEL ® Dressing versus an alginate
dressing.

In 2012, the WHRU undertook a clinical study commissioned by the
National Institute for Health and Care Excellence (NICE) to determine
the effectiveness of MIST ultrasound therapy compared to UK
standard care for the treatment of non-healing venous leg ulcers. In
this instance, the WHRU acted as the clinical experts in leading this
clinical trial, which was independent from NICE, and the MIST
manufacturer.
49
European collaboration: The University of Liverpool co-ordinates
REDONTAP
University of Liverpool




The Division of Clinical Engineering at the University of Liverpool,
home to the UK Centre for Tissue Engineering (UKCTE), has been at
the forefront of research in biomaterials, biocompatibility, tissue
engineering and regenerative medicine for over 30 years.
The University of Liverpool is coordinating a European collaboration,
REDONTAP.
The REDONTAP project aims to discover the conditions required for
the rapid and continuous creation of red blood cells from adult stem
cells. The European Commission, under the Nanosciences,
Nanotechnologies, Materials and new Production Technologies (NMP)
theme of the 7th Framework Programme, have granted the University
of Liverpool, the University of Leipzig, Applikon Biotechnology and the
Banc de Sang 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.
50
Cell/Tissue 3D Printing
Roslin Cellab,
Heriot Watt University
Reinnervate

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 preclinical research.
51
The Oxford Stem Cell Institute
The University of Oxford

The Oxford Stem Cell Institute (OSCI) comprises over 40 worldleading 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.
52
Translation in nanotechnology: The university of Swansea
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)
53
Cardiff Institute of Tissue Engineering and Repair: Cardiff University
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
54
The Edinburgh BioQuarter is a leading European destination for
translational medical research




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.
55
Arthritis Research UK (ARUK) funds leading centres of excellence
accelerating regenerative medicine research in the field
Other ARUK centres in:

Primary care

Pain

Musculoskeletal ageing research

Exercise and osteoarthritis

Rheumatoid Arthritis Pathogenesis
Arthritis Research UK
Biomechanics and
Bioengineering Centre – Cardiff
Arthritis Research UK Tissue
Engineering Centre, Newcastle,
York, Keele, Aberdeen and
Cambridge
Arthritis Research UK Centre for
Osteoarthritis Pathogenesis,
Oxford
56
From discovery science through to clinical and commercial
Application: UK Regenerative Medicine Platform Hubs
Safety and efficacy, focussing on
imaging technologies; University of
Liverpool
Cell behaviour, differentiation and
manufacturing; University of
Sheffield
Engineering and exploiting the stem cell
niche; University of Edinburgh
Acellular (smart material) approaches for
therapeutic delivery; University of
Nottingham
57
Examples of leading induced pluripotent stem cell (iPSC) initiatives
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
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
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.
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.
58
Appendix Further supporting information
59
The UK boasts a superior infrastructure optimised to support
industry

The UK offers a well funded research infrastructure for industry to leverage funding for innovative
research, proof-of-concept, and clinical delivery
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.
£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
TSB funded Knowledge
Transfer Networks
(KTNs) connecting the
regenerative medicine
community to create
opportunities for
collaboration with
industry
Research Councils invest
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
The TSB supports
Regenerative Medicine
themed R&D competitions
and in 2013 has already
committed a further £8m to a
competition in this area.
£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
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.
60
Tapping into a rich environment supported by backing from
research funders






£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.
61
A superior infrastructure optimised to support industry




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
62
A superior infrastructure optimised to support industry
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
63
A superior infrastructure optimised to support industry
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
64
Access to Cell and Tissue Banking
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 highquality 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.
65
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
22
Due 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
66
NHSBT deliver a diverse range of services in human tissues and
cells and are key in the regenerative medicine environment
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.
Location
Liverpool
Clean Rooms Development
Labs
10
Yes
Cryostorage
Facility
Yes
Oxford
2
Yes
Yes
Birmingham
3
Yes
Yes
Bristol (Langford)
6
Yes
Yes
Bristol (Filton)
4
Yes
Yes
Sheffield
1
No
Yes
Leeds
3
No
Yes
Southampton
2
No
Yes
NHSBT manages clean room facilities across eight sites as shown in Table 1.
Table 1. NHSBT GMP-Compliant Clean Room Facilities
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
67
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
68
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
processes








Reagents, assays
Cell production, processing
Imaging tools
Storage and transport
Supply chain management
Health economics
Business planning
Cell Therapy Catapult
Tissue and cell
supply
Manufacturing
capabilities
 High value
manufacturing
 Bioprocessing
 GMP facilities
 Scaffolds and matrices
 Devices
 Innovative approaches
 Cell Therapy Catapult





UK Stem Cell Bank
NHSBT/SNBTS
SC4SM
16 banks and registries
Transplant services
Regulatory advice
and guidance





MHRA
HTA
Stem Cell toolkit
Cell Therapy Catapult
consultancy services,
CROs and CMOs
Clinical trial design
and delivery
 Cell Therapy Catapult
 Clinical Trial Toolkit routemap
 NHIR, NOCRI
 NHS Research Scotland
69
Strong manufacturing capabilities supported by an innovative
manufacturing environment

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
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)
70
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.
71
Successful path to Commercialisation
Science
Clinical
Manufacturing
Business

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
72
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-theart 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
73
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)
NOCRI facilitates
industry access
Clinical
Research
Network
£500+ million
annual spend on
infrastructure to
deliver
60+ million
patients in the
National Health
Service
 NIHR supports several activities in regenerative medicine
through the BRCs and BRUs but also through co-funding
of specific initiatives in the field
74
The UK has a clear and well established regulatory framework that
is harmonised for Europe




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.
75
Supporting market access: facilitating adoption into the NHS
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 programme
Ease of access to the NHS for regenerative medicine treatments
Companies 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).
76
Progressive Licensing – Intercytex product examples (for
modifying table)
Progressive licensing:
Progressive development:
Progressive reimbursement:
Focus on unmet medical need and
orphan products in order to allow
access to the current legislative
frameworks already in place of
conditional approvals.
A close partnership between client,
contract developer and hospital
allowing iterative development of
new treatments
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
 Biocatalyst grant to Improve
shelf life and logistics to
enable pharmacy supply
 Developed costing models for
current system and scale up
options
 Working with Cell Therapy
Catapult to produce less
painful injection system
 Obtained full hospital
reimbursement to treat first
patients under Specials
 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 charities to
examine reimbursement
possibilities
 Phase II clinical trial completed
 Specials license obtained for
Intercytex manufacturing
facility
 Working with suppliers on
scale-up options
77
Appendix: Optional Introduction
78
The UK is committed to supporting Life Sciences and Healthcare
“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.”
David Cameron
This strategy makes the most of the UK’s core strengths:




Prime Minister
Basic science in universities
Translational and clinical research excellence
Industry and supply chain
The National Health Service (NHS)
Life Science Strategy and Innovation Health and Wealth are the
UK government’s response to a global sector under pressure
79
About the UKTI Life Science Investment Organisation
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:
1
Translating
2
Matching
3
Catalysing and empowering the UK Life Science community to work with international
benefits outlined in the Strategy for UK Life Science and Innovation, Health and
Wealth into opportunities for business
UK strengths with global business needs
business
+
=
80
The UK’s Regenerative Medicine Strategy will ensure that UK
leadership continues in the decades to come
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
81
The UK’s ambition is to be a global leader in regenerative medicine


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
Underpinning Research
Continued strategic investment in centres of
excellence and partnerships with industry
Focus
Achieve key goals such as clinical proof-ofconcept through focused efforts and critical
mass
Therapeutic Option
Continued support for translational science
through the MRC/TSB/EPSRC
Product Development
Linking early stage regenerative medicine
product development to the establishment of
manufacturing, transportation and delivery
solutions


Clinical Delivery & Evaluation
Establish the most effective trial designs and
improve the transparency of the regulatory
framework
Innovation & Value Systems
To address issues such as the evolution of new
business models, product development
mechanisms (including reimbursement &
adoption), and open innovation
Interdisciplinary Collaboration
Bringing together of strong complementary
skills, expertise and infra structure across
disciplines
International
Work with overseas partners (established and
new) to capitalise upon emerging opportunities
and provide access to complementary expertise
that will benefit UK science
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
82
There is a global market opportunity for regenerative medicine
Regenerative medicine has the potential to
address many of the challenges facing
Healthcare
 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 clinically
successful products
• Over 100 regenerative medicine products on the
market around the world focused on diverse
therapeutic areas
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
83
The UK market opportunity for your regenerative medicine business
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.
Department of Health
Social Services and
Public Safety,
Northern Ireland
£4.4bn
NHS Wales
£6.3bn
NHS Scotland
£11.5bn
Department of Health in
England
£106.6bn
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
84
Providing Access to Drive Healthcare Innovation:
Active Partnership Between Academia, Industry, and the NHS
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
IN NORTHERN IRELAND:
IN SCOTLAND:
 Connected Health and Prosperity Action Plan ensures
cross-sectorial support for R&D and technology
adoption
 Academic Health Sciences Collaboration
facilitates partnerships between universities
and health boards
 Invest Northern Ireland, the region’s business
development agency, provides a single access point
for industry investment interest.
 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 WALES:
IN ENGLAND:
 Academic Health Science Collaboration
maximises links between health boards,
trusts, universities and industry
 Biomedical Research Centres and Units link
universities and NHS trusts
 The Welsh Government gives you easy
access to key decision-makers and
private sector specialists, to help fasttrack your investment.
 Health Research Wales is one-stop shop
for industry access to NHS Wales
 Academic Health Science Centres open up
routes to market
The Cell Therapy Catapult is
a national resource acting as
a gateway to all of these
partners
 Academic Health Science Centres, university
and NHS partnerships of world-class
excellence, collaborate with industry across
research, education and patient care
85
Unrivalled data to drive innovation in regenerative medicine
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.
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
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”.
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
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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
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.
–
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
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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
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.
–
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
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