European Regenerative

Medicine Firms & Their

Strategic Approaches

Michael Morrison

University of York

OVERVIEW

Creating the European RM

‘Universe’ of firms

Characterizing the European

RM Universe

Strategic Analysis

CREATING THE EUROPEAN

REGENERATIVE MEDICINE

‘UNIVERSE’

DEFINING REGENERATIVE MEDICINE

Regenerative medicine is a heterogeneous domain

incorporating biological sciences, engineering,

materials science, medicine, and surgery.

Broadly, regenerative medicines act therapeutically

by stimulating, directing or augmenting the body’s

capacity for self-repair and regeneration.

Multiple technological avenues of investigation,

united by this overarching goal, but forming

identifiable sub-sectors of regenerative medicine

such as stem cell therapy, gene therapy, tissue-

and-bioscaffolds etc

DEFINING REGENERATIVE MEDICINE

Our approach concentrates on technologies

using novel biomaterials – living cells,

genes and bioscaffolds.

Excludes small molecules and biologicals

(eg antibodies) that aim to stimulate in vivo

regenerative action.

Alongside therapeutic approaches are cell

diagnostics for drug testing and platform

technologies, mainly tissue and biobanks.

DEFINING REGENERATIVE MEDICINE

Large pharmaceutical companies with an

interest in aspects of regenerative medicine

such as Pfizer, Sanofi-Aventis, Smith and

Nephew etc not included on the list

Company must have some publicly

available details (usually in the form of a

website).

Basic cell culture tools/media suppliers not

included unless have specialist focus.

CHARACTERIZING THE

EUROPEAN REGENERATIVE

MEDICINE FIRM UNIVERSE

FIRM TYPE BY PRIMARY ORIENTATION

Cell Therapy

27

Gene Therapy

6

Bioscaffold12

Services/ other

14

N = 59

GEOGRAPHIC DISTRIBUTION OF FIRMS

UK21

Germany15

Sweden5

France4

Switz'land3

Other European

11

N =59

FIRMS BY SIZE (EMPLOYEE NOS)

SMALL41

MEDIUM4

LARGE3

No Data11

N = 59SMALL = 0-99

MEDIUM = 100-

249

LARGE = 250+

FIRMS BY AGE (YEAR FOUNDED)

0-5 years8

6-10 years

1711-20 years

25

>20 years8

No Data1

N = 59

SUMMARY OF FIRM CHARACTERISTICS

Cell therapy is the largest single sector

Significant no. of bioscaffold and service

firms

Most firms are small (< 99 employees)

UK and Germany are the main geographic

hubs of commercial RM activity

Wide range of company ages – many

remaining from 1990s and pre 2002 ‘crash’

STRATEGIC ANALYSIS OF

EUROPEAN REGENERATIVE

MEDICINE FIRMS

AUTOLOGOUS VS ALLOGENIC CELL

THERAPY

0

5

10

15

20

25

Autologous Allogenic Both Total

18

34

25

No. of Companies

STEM CELL VS SOMATIC CELL

THERAPY

0

5

10

15

20

25

Stem cell Non-stem cell Total

9

16

25

No. of Companies

THERAPEUTIC FOCUS OF CELL

THERAPY FIRMS

Stem cells

Cell …

0

1

2

3

4

5

6

7

8

9

10

Stem cells

Cell therapy

CELL THERAPY LANDSCAPE

Strong preference for autologous cell

therapy

Stem cells a significant but not dominant

part of cell therapy landscape

Somatic cell therapies concentrated in

traditional T.E. domains

Stem cell therapies have much broader

therapeutic focus, generating new

applications

GENE THERAPY COMPANIES

Name Locati

on

Size Found’d Disease

focus

Notes

Amsterdam

Molecular

Therapeutics

(AMT)

NL Small 1998 Metabolic disorder Lead product Glybera submitted for

EMA MAA in 2009

Ark Therapeutics UK Medium 1997 Vascular

occlusion. Cancer

Cerepro submitted to EMA for MAA

in 2008, received rejection 2009.

Trinam in phase III trials

Arthrogen BV NL Small 2005 Rheumatoid

Arthritis

Lead product still at preclinical

stage

Diamyd Medical

AB

Sweden Small 1976 Chronic pain,

cancer

Main therapeutic programme is

therapeutic vaccines for diabetes.

Oxford BioMedica UK Small 1996 Neurodegenera-

tive, Occular

Lead product in phase I/II

Transgene France Small 1979 Cancer Several cancer vaccines in

development

Mol Med Italy Small 1996 Graft vs Host

disease

Lead product in Phase III trials

Mologen Germany Small 1998 Cancer Developing non- RM anti-cancer

therapeutics

GENE THERAPY LANDSCAPE

Small sub-sector, with small firms

No clear trends in firm age, location or

therapeutic focus

Engagement with Advanced Therapy

Medicinal Products regulation: Ark’s

Cerepro rejected but Amsterdam Molecular

Therapeutics’ Glybera submitted for MAA

MolMed (Italy) have cell-based ex-vivo

gene therapy in phase III trials

SALEABLE THERAPEUTIC PRODUCTS

Bioscaffold for tissue repair 19

Cell therapy for cartilage repair 13

Cell therapy for

wound healing 3

Other cell therapy 1

RM PRODUCTS IN DEVELOPMENT

0 5 10 15 20 25

Cell therapy

Gene therapy

Bioscaffolds

RM Products in Development

Preclinical

Phase I

Phase II

Phase III

THERAPEUTIC LANDSCAPE

Current market is dominated by Tissue

Engineering products – bioscaffolds and

autologous chondrocyte transplantation

therapies.

However, there are a much wider range of

products in development, including stem

cell and gene therapies, aimed at a broader

range of therapeutic applications.

Indicates an industry in transition.

Service Total Stem cell?

Cell and tissue based drug discovery /

screening services / toxicity testing

4 2

Cell and tissue supply including research

and clinical grade material

4 2

Contract manufacture of cells / cell line

expansion, cell culturing and scale up

1 -

Specialist tools and reagents 3 -

Distribution of bioscaffolds for tissue

repair, wound healing etc

1 -

TOTAL 13 4

FOCUS OF DEDICATED SERVICE

PROVISION FIRMS

ADDITIONAL SERVICE PROVISION

18 Cell therapy companies have additional

sources of revenue beyond lead therapeutic

product

Most common option is laboratory services,

including cell culturing, cell and tissue

supply, and cGMP facilities.

Bioscaffold products and regenerative

medicine consulting services are also

popular options.

CONCLUSIONS

CONCLUSIONS

Companies pursuing newer regenerative

medicine models appear to co-exist with

older tissue engineering and gene therapy

firms.

Strong industry preference for less risky

technologies – autologous cell therapy with

somatic cells or adult stem cells.

Little to no commercial investment in iPS

and embryonic stem cell technologies.

CONCLUSIONS

Commercial development concentrated in

Western Europe, and concentrated in hubs.

Importance of biotechnology infrastructure

and financial support.

Regulatory environment is also an

important shaping factor – as with hESCs

Can be illustrated by Germany – UK RM

firm breakdown

CONCLUSIONS

RM Sub-sector Germany UK

Cell Therapy 9 6

Gene Therapy 1 2

Bioscaffolds 2 4

Service/ other 3 9

TOTAL 15 21

http://www.york.ac.uk/res/remedie