Promoting the Development of Advanced Therapies in the UK, a presentation by Cell Therapy Catapult Chief Clinical Officer Natalie Mount.

1. The Cell Therapy Catapult
Promoting the development of advanced therapies in the UK
Natalie Mount
Chief Clinical Officer
Cell Therapy Catapult
29th April 2013
natalie.mount@ct.catapult.org.uk
Catapult is a Technology Strategy Board programme

2. Catapults
Catapults bridge the gap between business, academia,
research and government
Exploiting the science base to create new manufacturing
industries for the UK
£200m+ assigned for 7 Catapults; Cell Therapy Catapult
is the only life science Catapult
Catapults use their critical mass of expertise to accelerate
projects
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3. 3
Cell Therapy Catapult
•
Significant unmet medical needs
•
Early stage in market with large
potential for growth
•
World class science base
supported by government and
charity research funders
•
Development and production of
cell therapies is complex
•
Majority of therapies from
academic researchers

4. Core Purpose
Growing a UK cell therapy industry
delivering health and wealth
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5. Vision
The Cell Therapy Catapult vision is for the UK to be
a global leader in the development, delivery and
commercialisation of cell therapy.
Where businesses can start, grow and confidently
develop cell therapies, delivering them to patients
rapidly, efficiently and effectively.
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6. Global cell therapy industry: ‘Cell-able targets’
Established medical practice:
e.g. haematopoietic stem cell transplantation
Commercial products:
Rapidly growing global market
Global turnover of $2.7bn in 2011; rising to $5bn in 2014; driven by
diversity of small products (Apligraf, Dermagraft, Provenge made >$100m
each in 2011)*
Therapies in development:
Rapidly growing ~ 250 products in clinical development
Global pharma involvement:
Already Teva, Shire, Sanofi, Pfizer, GSK, Novartis…
* Mason Regen Med 5(3) 2010, Mason Regen Med 6(3) 2011, Syed & Evans, Nature Drug Discovery, March 2013
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7. Diverse cell therapies in clinical trial in the UK;
but few are company sponsored
Category
Cell therapies currently in
clinical trial in the UK*
Number
34
Comment
Approx. 2/3 autologous and 1/3
allogeneic
Range of indications from
cardiovascular to oncology
Bone marrow derived cells still
predominate but rising diversity
Cell therapies currently in
clinical trial in the UK
sponsored by a commercial
company
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Majority of studies are Research
Council and EU consortia
funded
*verified studies currently on-going; excludes haematopoietic transplantation
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8. Bone marrow derived cells still predominate but Tcells are a rising class amongst greater diversity
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9. Field is growing beyond the traditional
transplant and oncology indications
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10. Building the Portfolio
hESC
iPS
MSC
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Immune
Cell
Other
Somatic
Blood
Bone and
cartilage
Cardiovascular
Dermatology/wound
healing
Diabetes
Gastroenterology
Immunology
Liver
Metabolic
Neurological
Oncology
Ophthalmology
Respiratory
Other
Manufacturability

11. 11
Addressing Barriers
Business
Manufacturing
and
Supply Chain
Clinical
And
Regulatory
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•
•
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Economics
Health Economics
Manufacture & Supply vs Service
Business Plans
•
•
•
•
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COGS & Scale up
GMP
Characterisation & Analytical
Comparability
Delivery
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•
•
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Regulatory Landscape
Pre Clinical Packages
Clinical trial design and conduct
NHS partnering
Business Development
Process Development
Clinical Operations &
Regulatory Affairs

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

13. Facilitating cell therapy development: Nationally
Flexible approach to partnership on projects:
• Collaborative and grant funded clinical, regulatory, manufacturing
and business activities; substrate from database
• In-licensing opportunities
• Contract activity
• Ad hoc advice
Strategic issues: Close relationship with MHRA, HRA, HTA, MRC, TSB
Guidance : Practical ‘how to’ notes and future workshops
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14. Influencing the environment: Globally
Participation in EU/CAT/EMA /EDQM workshops
Responses to public consultations, often in collaboration with other
thought leaders and industry bodies
Promoting the UK as the ‘go to’ place in Europe for clinical
development of cell based therapies:
• Building on our science base and strong regulatory, ethics and
clinical research infrastructure
• Attracting inward investment in manufacturing and clinical trials
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15. Industry
Researchers
Cell Therapy
Catapult
NHS
Catapult is a Technology Strategy Board programme
Investment