Catapult is a Technology Strategy Board programme

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

Catapults 2

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

Cell Therapy Catapult

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• 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

Core Purpose

Growing a UK cell therapy industry

delivering health and wealth

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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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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…

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* Mason Regen Med 5(3) 2010, Mason Regen Med 6(3) 2011, Syed & Evans, Nature Drug Discovery, March 2013

Diverse cell therapies in clinical trial in the UK; but few are company sponsored

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*verified studies currently on-going; excludes haematopoietic transplantation

Category Number Comment

Cell therapies currently in

clinical trial in the UK* 34

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

Bone marrow derived cells still predominate but T-cells are a rising class amongst greater diversity

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9 Field is growing beyond the traditional transplant and oncology indications

Blood

Bone and cartilage

Cardiovascular

Dermatology/wound healing

Diabetes

Gastroenterology

Immunology

Liver

Metabolic

Neurological

Other

Ophthalmology

Respiratory

Oncology

hESC iPS MSC Immune Cell

Other Somatic

Building the Portfolio 10

Manufacturability

Addressing Barriers

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• Economics

• Health Economics

• Manufacture & Supply vs Service

• Business Plans

Business

• COGS & Scale up

• GMP

• Characterisation & Analytical

• Comparability

• Delivery

Manufacturing

and

Supply Chain

• Regulatory Landscape

• Pre Clinical Packages

• Clinical trial design and conduct

• NHS partnering

Clinical

And

Regulatory

Clinical Operations &

Regulatory Affairs

Process Development

Business Development

Su

ita

bili

ty

• Project assessment

Projects

Proof of Principle

• Scientific, clinical, regulatory, commercial

Non-clinical

• Safety, toxicology, GMP proving, assays

Clinical

• Safety and efficacy, investible data

Platform

• Generic issues and large collaborations

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Suitability

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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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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Catapult is a Technology Strategy Board programme

Industry

Cell Therapy Catapult

Investment

Researchers

NHS