1. The Cell Therapy Catapult
Growing a UK cell therapy industry delivering
health and wealth
Keith Thompson CEO
World Stem Cell and
Regenerative Medicine Conference,
London.
May 21st 2013
info@ct.catapult.org.uk
Catapult is a Technology Strategy Board programme

2. The Launch of Catapults
Hauser Report
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Creating new manufacturing industries for the UK
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Better exploiting the UK science base
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“Grow and stick”
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£200m+ assigned to TSB for 7 Catapults
Hauser Criteria
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> £10bn pa long term industrial revenue to UK
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Exploit a strong existing UK science base
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UK absorptive capacity, to capture and retain value
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Learn from other countries with successful Technology Institutes
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Translational activity to bridge investment gap
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Persistence of funding against a long term perspective and low
priority on becoming self-financing
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3. 3
Cell Therapy Catapult
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Significant unmet medical needs
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World class science base
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Complex development and
production
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NHS potential for both research
and exploitation
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Early enough for UK to establish a
strong market position

4. Mind the (translational funding) gap
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Little evidence yet that new cell therapies
can be developed, licensed and adopted
successfully
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Limited commercial investment
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Limited precedents for valuable exits via
IPO or acquisition
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Large corporates are watching and waiting
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Operational SME’s lack finance and
breadth of resources for rapid advance
Catapult

5. 6
Addressing Barriers
Business
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Health Economics
Business Models
Reimbursement
Partnering
Manufacturing
and
Supply Chain
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Robustness & Reliability
COGS & Scale up
Characterisation & Analytical
GMP
CMC
Delivery
Clinical
And
Regulatory
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Complex Regulatory Landscape
Pre Clinical Packages
Clinical trial design
NHS partnering
Business Development
Process Development
Clinical Operations &
Regulatory Affairs

6. Cell Therapy Catapult
-2012 objectives
• Identify premises, acquire and complete lease
• Write strategic and detailed business plan, secure 5 year GFA
• Establish company resource systems
• Design organisation, recruit a world-class management team and
Board
• Design Facilities
• Review UK GMP manufacturing capability
• Build stakeholder relationships and project the Catapult Image
•
(corporates/SMEs/medical and academic
researchers/technologists/DH/NHS/RCs/charities/patient groups)
• Establish database of projects and initiate dialogue
• Open for business November 2012
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7. 8
Building The Catapult
Catapult is a Technology Strategy Board programme

8. Assets
-Money
Finance
• £70m core grant from TSB to March 2018
• £10m pa from other grant funders
• £10m pa from industry contracts
We use our assets to accelerate innovation.
Not reliant upon IP for future funding
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9. Assets
-Facilities and Teams
• Facilities
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1200 sq m on 12th floor
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Capacity for 80-100 people
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Clinical research cluster
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• Business Team
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Business development
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Health economics
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Business models
• Translational Labs and Team
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Process development
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Analytical development
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GMP process proving
• Clinical Trial and Regulatory
Team
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Regulatory
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Clinical operations

10. Cell Therapy Catapult Board Members and
Executive Team
Dr John Brown, CBE, FRSE
Chairman of the Board
Keith Thompson
Chief Executive Officer
Tim Edwards
Non Executive Board Director
Matthew Durdy
Chief Business Officer
Nick Higgins
Non Executive Board Director
Professor Marc Turner
Non Executive Board Director
Professor Michael Whitaker
Non Executive Board Director
Dr Zahid Latif
Non Executive Board Director
Dr Natalie Mount
Chief Clinical Officer
Dr Stephen Ward
Chief Operating Officer
Professor Johan Hyllner
Chief Scientific Officer
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11. Cell Therapy Catapult Outputs
Investible therapies (Rounded Phase 2 data package)
Industry assisted over barriers
Novel technologies licenced to cell therapy companies
Contract research which enhances capability of the industry
Standards, quality systems, guidance & co-ordinated framework
Skilled, trained and experienced professionals
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12. 13
The UK ATMP Manufacturing
Landscape
Catapult is a Technology Strategy Board programme

13. ATMP GMP capability study
To document and analyse
• Physical Capacity
• Regulatory status
• Human capability
• Process capability
To allow
• Catapult to place work with appropriate facilities
• To direct inward investors
• To form the basis of a UK Strategy
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14. • 21 licensed facilities,
mostly academic or
public sector
•12 sites analysed
• 51 manufacturing
cleanrooms
• 55 parallel processes
• 94 full time staff
• 42 part time staff
• 44% availability 2013
Grade of
cleanroom
Grade B
Grade C
Grade B/C/D with
isolator
Number
24
10
17
Facility Process Experience
gene modification
viral vector
cell banking
cells from tissue
IPS
HESC
3D
2D
adherent
suspension
0
2
4
6
8
Number of Facilities
10
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15. ATMP GMP Manufacturing
Capability study
• Future capability
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Expansion planned in 4 academic facilities
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Licenses planned in 8 facilities
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2 commercial CMOs expressing interest in
expanding into Cell therapy
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Strategic planning for Phase 3 and in market
supply
• Catapult to collaborate on
establishment of CMO
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16. Regulatory Permissions
• Catapult building strong clinical and
regulatory team
• Interact with regulators to improve
clarity and speed
• Clinical access to NHS
• Clinical trial sponsor
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17. HRA vision and ambition
To make the UK a
great place to do
research, where
more money
invested in
research goes into
carrying out
relevant, good
quality research
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• Greater numbers of people can and
do take part in health research, and
continue to feel safe when they do
• Researchers find it easier to do highquality, ethical research
• Less resource is invested in getting
studies started
• Clinical trials are registered and
research gets published
• The NHS appreciates the benefits of
health research
Protecting and promoting the interests of patients and the public in health research

18. GTAC – Improvements
• Increased availability of review slots from 6 to 44 per
year
• Increased geographical spread – now four locations
• Improved timelines – 2012–2013:
• Pre-September 2012 – 8 studies: range 82–144
days
• Post-September 2012 – 7 studies: range 19–76
days
• KPI – HRA Business Plan – all studies < 60 days
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Protecting and promoting the interests of patients and the public in health research

19. 20
Business Development and
Project Financing
Catapult is a Technology Strategy Board programme

20. Funding for Cell Therapy in the UK
Govt.
Support
Medical
Charities
Commercial
Development
Plan
Financial
Investment
Corporate
Venturing
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21. Working models
Nature of project
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Paid for by:
Carried out
by:
Ownership
of outputs
Catapult
owned
Catapult
Catapult
Catapult
Contracted
Development
Client
Catapult
Client
Industry
Grant or
Collaboration Catapult
and
partner
Catapult Outputs
and
shared
partner

22. Collaboration
1+1 = 3
Client knowledge +
Catapult capabilities =
Better outcome
Up to 80% Technology Strategy Board
funding available for collaboration with SME
Partner
Project
Input
Funding
Ratio
TSB Grant
SME
£1m
60%
£0.6m
Catapult
£1m
100%
£1.0m
Total
£2m
£1.6m
80%

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Building a Project Portfolio
Catapult is a Technology Strategy Board programme

24. UK preclinical and clinical stage cell therapies
Category
Number
Comparision
More allogeneic therapies in
preclinical stage
Preclinical
(<2 yrs from clinic)
37
Larger variety of cell types in
preclinical stages
Larger range of indications for
preclinical projects
Clinical
(UK trial ongoing)
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Total
71
Few commercially sponsored
projects in both pre clinical and
clinical stages
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25. Larger variety of cell types in preclinical stage projects
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26. Larger variety of cell types in preclinical projects
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27. Opportunities for the Catapult
• Significant potential to grow the clinical translation of
therapies originating in the UK and to attract inward
investment for clinical development
• Significant potential to grow the number of UK based cell
therapy companies
• More and larger Phase 2 studies are required, statistically
designed and powered to demonstrate efficacy
Cell Therapy Catapult Confidential
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28. Identifying projects
• Pre-clinical and clinical databases
• Technology transfer offices
• Intermediaries
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Grant Funders
Industry Groups
Charities
Investors
• Direct contact
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Inward investors
EU entrants

29. Gateway Criteria

30. Projects
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Proof of Principle
• Scientific, clinical, regulatory, commercial
Suitability
Non-clinical
Suitability
• Commercial
Development
plan
• Safety, toxicology, GMP proving, assays
Clinical
• Safety and efficacy, investible data
Platform
• Generic issues and large collaborations

31. Project activity
Over the Next 5 Years, complete
• 150-200 short term suitability and assistance projects
• 11-15 proof of principle projects
• 5-9 non-clinical projects
• 4-6 clinical projects
Participate in the creation of 2-4 significant investible propositions
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32. Building the portfolio
hESC
Blood
Bone &
Cartilage
Cardiovascular
Dermatology/Woun
d Healing
Diabetes
Gastroenterology
Immunology
Liver
Metabolic
Neurological
Oncology
Ophthalmology
Respiratory
Other
iPS
MSC
Immune
Cell
Other
Somatic

33. Collaborations
Loughborough
University
• Cooperation on
manufacturing
platforms
UK Stem Cell
Foundation
• Project
sourcing and
funding
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34. Project Examples
GSK
• Share of expertise
• Support for in house projects
Intercytex
• New delivery device to reduce injection pain
Reneuron
• Phase 2 clinical trials
• Scale up, Assays, Freezing and distribution of
cells
Inward investor
• Manufacturing partner, Regulatory, Clinical trial
design and delivery
Kings
• Immunomodulation
• Regulatory, Clinical trial design, business models
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35. 36
What does success look like?
Catapult is a Technology Strategy Board programme

36. Strategic Goals
Pipeline
• Increased cell therapies in UK clinical trial and clinical use
Value
• Investible propositions created leading to cell therapy companies
that succeed and stay in the UK
Attractiveness
• Demonstrating that the UK is the place to do this work, with
increased inward investment
Goals
• Build a £10bn industry
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37. Industry
Researchers
Cell Therapy
Catapult
NHS
Catapult is a Technology Strategy Board programme
Investment

38. Contact us
Cell Therapy Catapult Limited
Biomedical Research Centre, R&D 16th Floor Tower
Wing, Guy’s Hospital
Great Maze Pond, London, SE1 9RT
Tel: +44 (0) 207 1883428
info@ct.catapult.org.uk
www.celltherapycatapult.org.uk
Catapult is a Technology Strategy Board programme
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