Global commercialisation of UK stem cell research Nicola Perrin

An internship project carried out for UK Trade & Investment, as part of an MPhil at the University of Cambridge April-June 2005 by Nicola Perrin, University of Cambridge Legal Disclaimer Whereas every effort has been made to ensure that the information given herein is accurate, UK Trade & Investment or its sponsoring Departments, the Departments of Trade & Industry and Foreign & Commonwealth Office, accept no responsibility for any errors, omissions or misleading statements in that information and no warranty is given or responsibility is accepted as to the standing of any firm, company or individual mentioned. Crown Copyright retained - this report maybe freely quoted by prior agreement. Attribution to UKTI / Nicola Perrin is required.

UK strengths 1 Regulatory framework - supportive, clear, comprehensive - allows research with adult and embryonic stem cells - one of only a few countries to allow therapeutic cloning Academic centres - world class expertise - Cambridge, Edinburgh, London, Newcastle, Sheffield Funding - Government, Research Councils and Charities Government support

UK strengths 2 UK Stem Cell Bank - world-first, opened in research and commercial use Stem cell companies

Global comparisons Permissive policy: various ES cell derivation techniques allowed, including SCNT Flexible policy: stem cells may be derived from human embryos donated by fertility clinics only Restrictive policy or no established policy (which may mean any research is permitted) Source: William Hoffman, MBBNet

United States Federal restrictions on funding with ES cells - has limited stem cell research - recent indications the situation may begin to change Range of State initiatives - California: Proposition 71, $3bn fund for stem cell research - New Jersey, Massachusetts, Connecticut also supporting research - some other States have tightened restrictions Active companies - eg Geron, ACT, Stem Cells Inc - significant resources and investment for commercialisation  could catch up

Asia Seen as a priority area Significant government investment, supportive regulations, impressive infrastructure, world- class expertise Main focus Chinaclinical translation South Korealeading SCNT expertise Singaporecommercial emphasis  Significant threat See Global Watch Mission Report 2004

Scandinavia Strong research, recognised expertise Government support Favourable legislation Clear routes for commercialisation  Similar position to UK Need to monitor progress in Sweden and Denmark

Europe Much more conservative to ESC work Banned:Italy, Austria, Ireland Restrictions: France, Germany, Greece, Netherlands Permitted: Belgium, Spain, Czech Republic Some highly-regarded adult SC research  Poses little threat

Other significant players Australia - leading centre of excellence - uncertain regulatory framework - appears to be losing strong position Canada - Canadian Stem Cell Network particularly strong - research with surplus embryos allowed but therapeutic cloning banned Israel - strong academic expertise - therapeutic cloning permitted - active spin-out companies

Commercial opportunities Therapeutics cell therapies growth factors Research applications disease models drug screening toxicity testing Enabling technologies research consumables manufacturing licensing cell lines cell banking clinical trials training

Time (years) Value Training Cell therapies Research tools Clinical trials Manufacturing Drug screening Cell banking Growth factors Toxicity testing Therapeutics Research applications Enabling technologies Bold: UK Strength Timescale for commercial opportunities

Challenges IP: lack of clarity Lack of venture capital investment Caution from Big Pharma Need for translational funding

Stages: Infrastructure: Stakeholders: Funding: ???? Basic ResearchApplied Development Clinical Therapies Universities, Research Institutes Biotech SMEs Pharma Clinicians Government / regulatory agencies Commercial funding VC, pharma Research Councils Charities Manufacturing Funding Gap Translation of stem cell research into commercial products

Conclusions Beware of hype UK needs to maintain competitive advantage Opportunities and threats from overseas Global collaborations crucial Important to raise profile of stem cell companies abroad

For further information A full version of the report is available on the UKTI portal at: Alternatively, please contact Nicola Perrin: Ian Bunker: