Transcript creating the european regenerative medicine

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 multiple technological
avenues of investigation.

United by a shared goal of stimulating,
directing or augmenting the body’s capacity
for self-repair and regeneration.

Our approach concentrates on technologies
using novel biomaterials – living cells,
genes and bioscaffolds.
DEFINING REGENERATIVE MEDICINE

Excludes small molecules and biologicals
(e.g. antibodies) that aim to stimulate in
vivo regenerative action.

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

Basic cell culture tools/media suppliers not
included unless have specialist focus.
DEFINING REGENERATIVE MEDICINE

‘European’ companies must be based
entirely in a European state, or have their
headquarters there.

Subsidiaries of non-European firms are not
included.

Company must have some publicly
available details (usually in the form of a
website).
CHARACTERIZING THE
EUROPEAN REGENERATIVE
MEDICINE FIRM UNIVERSE
FIRM TYPE BY PRIMARY ORIENTATION
Services/
other
14
Cell
Therapy
25
Bioscaffold
12
N = 59
Gene
Therapy
8
GEOGRAPHIC DISTRIBUTION OF FIRMS
Other
European
11
UK
21
Switz'land
3
France
4
Sweden
5
Germany
15
N =59
FIRMS BY SIZE (EMPLOYEE NOS)
No Data
11
SMALL
41
LARGE
3
MEDIUM
4
N = 59
SMALL = 0-99
MEDIUM = 100249
LARGE = 250+
FIRMS BY AGE (YEAR FOUNDED)
>20 years
8
No Data
1
0-5 years
8
6-10
years
17
11-20
years
25
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)

Wide range of company ages – many
remaining from 1990s and pre 2002 ‘crash’

UK and Germany are the main geographic
hubs of commercial RM activity
STRATEGIC ANALYSIS OF
EUROPEAN REGENERATIVE
MEDICINE FIRMS
AUTOLOGOUS VS ALLOGENIC CELL
THERAPY
25
25
20
18
15
10
3
5
4
0
Autologous
Allogenic
Both
No. of Companies
Total
STEM CELL VS SOMATIC CELL
THERAPY
25
25
20
16
15
9
10
5
0
Stem cell
Non-stem cell
No. of Companies
Total
THERAPEUTIC FOCUS OF CELL
THERAPY FIRMS
10
9
8
7
6
5
4
3
2
1
0
Cell therapy
Stem cells
Stem cells
Cell therapy
SALEABLE THERAPEUTIC PRODUCTS
Cell
therapy for
wound
healing 3
Cell
therapy for
cartilage
repair 13
Other cell
therapy 1
Bioscaffold
for tissue
repair 19
RM PRODUCTS IN DEVELOPMENT
RM Products in Development
Bioscaffolds
Preclinical
Phase I
Gene therapy
Phase II
Phase III
Cell therapy
0
5
10
15
20
25
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
Neurodegenerative, Ocular
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

Engagement with Advanced Therapy
Medicinal Products regulation:

Ark’s Cerepro rejected in 2009 but has
named-patient approval in France and
Finland

Amsterdam Molecular Therapeutics’
Glybera submitted for MAA in 2010

MolMed (Italy) have cell-based ex-vivo
gene therapy in phase III trials
FOCUS OF DEDICATED SERVICE
PROVISION FIRMS
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
Distribution of bioscaffolds for tissue
repair, wound healing etc
3
1
-
TOTAL
13
4
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 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