European Regenerative Medicine Firms & Their Strategic Approaches. Michael Morrison University of York

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1 European Regenerative Medicine Firms & Their Strategic Approaches Michael Morrison University of York

2 OVERVIEW Creating the European RM Universe of firms Characterizing the European RM Universe Strategic Analysis

3 CREATING THE EUROPEAN REGENERATIVE MEDICINE UNIVERSE

4 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, tissueand-bioscaffolds etc

5 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.

6 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.

7 CHARACTERIZING THE EUROPEAN REGENERATIVE MEDICINE FIRM UNIVERSE

8 FIRM TYPE BY PRIMARY ORIENTATION Services/ other 14 Bioscaffold 12 Gene Therapy 6 Cell Therapy 27 N = 59

9 GEOGRAPHIC DISTRIBUTION OF FIRMS Other European 11 Switz'land 3 UK 21 France 4 Sweden 5 Germany 15 N =59

10 FIRMS BY SIZE (EMPLOYEE NOS) LARGE 3 MEDIUM 4 No Data 11 SMALL 41 N = 59 SMALL = 0-99 MEDIUM = LARGE = 250+

11 FIRMS BY AGE (YEAR FOUNDED) >20 years 8 No Data years years years 17 N = 59

12 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

13 STRATEGIC ANALYSIS OF EUROPEAN REGENERATIVE MEDICINE FIRMS

14 AUTOLOGOUS VS ALLOGENIC CELL THERAPY Autologous Allogenic Both Total No. of Companies

15 STEM CELL VS SOMATIC CELL THERAPY Stem cell Non-stem cell Total No. of Companies

16 THERAPEUTIC FOCUS OF CELL THERAPY FIRMS Cell Stem cells Stem cells Cell therapy

17 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

18 GENE THERAPY COMPANIES Name Amsterdam Molecular Therapeutics (AMT) Locati on Size Found d Disease focus Notes NL Small 1998 Metabolic disorder Lead product Glybera submitted for EMA MAA in 2009 Ark Therapeutics UK Medium 1997 Vascular occlusion. Cancer Arthrogen BV NL Small 2005 Rheumatoid Arthritis Cerepro submitted to EMA for MAA in 2008, received rejection Trinam in phase III trials 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, 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

19 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

20 SALEABLE THERAPEUTIC PRODUCTS Cell therapy for wound healing 3 Other cell therapy 1 Cell therapy for cartilage repair 13 Bioscaffold for tissue repair 19

21 RM PRODUCTS IN DEVELOPMENT RM Products in Development Bioscaffolds Gene therapy Preclinical Phase I Phase II Phase III Cell therapy

22 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.

23 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 3 - Distribution of bioscaffolds for tissue 1 - repair, wound healing etc TOTAL 13 4

24 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.

25 CONCLUSIONS

26 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.

27 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

28 CONCLUSIONS RM Sub-sector Germany UK Cell Therapy 9 6 Gene Therapy 1 2 Bioscaffolds 2 4 Service/ other 3 9 TOTAL 15 21

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