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EXPORTS & INTERNATIONAL
        MARKETS
The UK is recognised as a world leader in stem cell research.

How can the UK’s strengths be translated into commercial opportunities in a global
market?

UKTI required an overview of the stem cell sector, both in the UK and worldwide, in
order to assess how they should support UK stem cell companies overseas.

What are the UK’s strengths in the area?

The supportive regulatory framework, allowing researchers to work with
embryonic stem cells within clear legislation, is a key advantage. This policy has
facilitated world-class research, and UK scientists have recognised expertise
What are the commercial opportunities for the stem cell
sector?

1. Therapeutics: The hope is that stem cells could be used to treat degenerative
   disorders or to generate replacement tissues and organs. It is important to be
   realistic: there are many challenges to overcome, including issues of safety,
   efficacy, clinical application and regulatory approval. Stem cell therapies
   remain a long-term prospect.

2. Research applications: Differentiated cell lines generated from stem cells
   could be used in drug discovery, for screening assays and toxicity testing. If
   pharma companies adopt this technology, it could offer real commercial
   potential in the short-term.

3. Enabling technologies: Research tools and consumables, cell banking,
   and technology for clinical production, will all be required to support
   stem cell work. This is likely to be a growing area with significant value in
   the medium-term.
What are the prospects in a global
              market?
The opportunities and threats posed by US, Asia, Europe
and Scandinavia are assessed. Asia, with significant
government investment, has impressive infrastructure and
world-leading expertise. The US, which had been
hampered by federal restrictions, is now contributing large
sums of money to the field and could become dominant.
Scandinavia also has strong research, combined with
government and commercial backing. The UK needs to
gain benefit from global collaborations and international
activities in order to maintain its competitive advantage.
KEY PLAYERS & THEIR PROFILE
Company           Description
                  Spin-out from University of
1.AXORDIA         Sheffield.
                  ‘Stem cell technologies for
www.axordia.com   discovery and therapy’
                  Aim: to develop hES technology, to
                  create cell lines, reagents and
Founded -1999     diagnostic
                  markers of stem cell propagation,
                  differentiation and safety to
                  generate shortterm revenue. In the
                  longer term they will develop cell
                  replacement therapies
Spin-out from the Paterson
2.EPISTEM      Institute for Cancer Research,
               Manchester.

FOUNDED 2000   Pre-clinical contract research
               company, specialising in
               epithelial tissue and stem cell analysis

               EpiStem offers a wide range of in vitro
               and in vivo assays, providing advice
               and analysis to biotech and
               pharmaceutical companies. Much of
               their business is international.
3. CereStem Ltd   Spin-out from King’s College
                  London.

 FOUNDED 2002     Aim: to identify and
                  characterise human growth
                  factors that promote
                  proliferation and
                  differentiation of
                  therapeutically important
                  stem cell populations,
                  including liver, retinal and
                  cardiac cell lines.
4.CELL CENTRIC   Virtual company, spin-out
                 from      University      of
                 Cambridge.
                 ‘Unlocking epigenetics’
FOUNDED 2003     Aim:commercial
                 exploitation of epigenetics
                 and mechanisms that control
                 cell functionality and fate.
Future prospects
Areas of agreement Owing to its accessibility, the cornea constitutes an easy
anatomical target for stem cell regeneration. On this basis, limbal epithelial
stem transplantation is the only ocular cell-based therapy already in use in the
clinical setting.

Areas of controversy Regeneration of the retina, a less accessible and
complex neural tissue, currently constitutes a major challenge. Investigations
into the potential use of stem cells for retina regeneration have generated
variable data and no therapies have yet been designed for human treatments.

Growing points Despite the present limitations, it has been progressively
accepted that various stem cells may have potential use for the development of
cell-based therapies to restore retinal function.

Areas for research development There is need to understand the cell
requirements and environmental conditions that may promote functional
integration and long-term survival of stem cells within the diseased retina. At
present, this constitutes a major area of research.

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Exports & international markets

  • 1. EXPORTS & INTERNATIONAL MARKETS The UK is recognised as a world leader in stem cell research. How can the UK’s strengths be translated into commercial opportunities in a global market? UKTI required an overview of the stem cell sector, both in the UK and worldwide, in order to assess how they should support UK stem cell companies overseas. What are the UK’s strengths in the area? The supportive regulatory framework, allowing researchers to work with embryonic stem cells within clear legislation, is a key advantage. This policy has facilitated world-class research, and UK scientists have recognised expertise
  • 2. What are the commercial opportunities for the stem cell sector? 1. Therapeutics: The hope is that stem cells could be used to treat degenerative disorders or to generate replacement tissues and organs. It is important to be realistic: there are many challenges to overcome, including issues of safety, efficacy, clinical application and regulatory approval. Stem cell therapies remain a long-term prospect. 2. Research applications: Differentiated cell lines generated from stem cells could be used in drug discovery, for screening assays and toxicity testing. If pharma companies adopt this technology, it could offer real commercial potential in the short-term. 3. Enabling technologies: Research tools and consumables, cell banking, and technology for clinical production, will all be required to support stem cell work. This is likely to be a growing area with significant value in the medium-term.
  • 3. What are the prospects in a global market? The opportunities and threats posed by US, Asia, Europe and Scandinavia are assessed. Asia, with significant government investment, has impressive infrastructure and world-leading expertise. The US, which had been hampered by federal restrictions, is now contributing large sums of money to the field and could become dominant. Scandinavia also has strong research, combined with government and commercial backing. The UK needs to gain benefit from global collaborations and international activities in order to maintain its competitive advantage.
  • 4.
  • 5. KEY PLAYERS & THEIR PROFILE Company Description Spin-out from University of 1.AXORDIA Sheffield. ‘Stem cell technologies for www.axordia.com discovery and therapy’ Aim: to develop hES technology, to create cell lines, reagents and Founded -1999 diagnostic markers of stem cell propagation, differentiation and safety to generate shortterm revenue. In the longer term they will develop cell replacement therapies
  • 6. Spin-out from the Paterson 2.EPISTEM Institute for Cancer Research, Manchester. FOUNDED 2000 Pre-clinical contract research company, specialising in epithelial tissue and stem cell analysis EpiStem offers a wide range of in vitro and in vivo assays, providing advice and analysis to biotech and pharmaceutical companies. Much of their business is international.
  • 7. 3. CereStem Ltd Spin-out from King’s College London. FOUNDED 2002 Aim: to identify and characterise human growth factors that promote proliferation and differentiation of therapeutically important stem cell populations, including liver, retinal and cardiac cell lines.
  • 8. 4.CELL CENTRIC Virtual company, spin-out from University of Cambridge. ‘Unlocking epigenetics’ FOUNDED 2003 Aim:commercial exploitation of epigenetics and mechanisms that control cell functionality and fate.
  • 9. Future prospects Areas of agreement Owing to its accessibility, the cornea constitutes an easy anatomical target for stem cell regeneration. On this basis, limbal epithelial stem transplantation is the only ocular cell-based therapy already in use in the clinical setting. Areas of controversy Regeneration of the retina, a less accessible and complex neural tissue, currently constitutes a major challenge. Investigations into the potential use of stem cells for retina regeneration have generated variable data and no therapies have yet been designed for human treatments. Growing points Despite the present limitations, it has been progressively accepted that various stem cells may have potential use for the development of cell-based therapies to restore retinal function. Areas for research development There is need to understand the cell requirements and environmental conditions that may promote functional integration and long-term survival of stem cells within the diseased retina. At present, this constitutes a major area of research.