Stem Cell Markets

A stem cell is an extraordinary type of cell that has the ability to self-renew for long periods of time and to differentiate into specialized cells under appropriate physiological or experimental conditions. Traditionally, stem cells have been classified as either embryonic, adult (tissue-specific or cord blood) stem cells. Recent understanding of stem cell biology may provide new approaches for the treatment of a number of diseases as well as tissue/organ injuries, including cardiovascular disease, neurological disease, musculoskeletal disease, diabetes and hematopoietic disorders. Stem cell fate is determined by both intrinsic regulators and the extra-cellular environment (niche), and their expansion and differentiation ex vivo are generally controlled by growing them in a specific configuration (monolayer or three-dimensional culture). This process, which is vital to enable stem cells to be used for therapeutic purposes, is called differentiation. Differentiation is a process involving unspecialized cells progressing to become specialized cells with restricted developmental potential. The purpose of this TriMark Publications report is to describe the specific market segments of the medical research space using stem cells for research and development (R&D) purposes. This study reviews all of the generally accepted analytical methods that are currently in use today for preparing and using stem cells. It examines the use of stem cells in developing new therapies for disease.

- Aastrom Biosciences
- Advanced Cell Technology
- Aeolus Pharmaceuticals (formerly Incara Pharmaceuticals)
- BioE
- Bioheart
- BioTransplant
- Cell Science Therapeutics
- Cryo-Cell International
- Diacrin
- ES Cell International
- Gamida Cell
- Geron
- NeuroGeneration
- Nexell Therapeutics
- Osiris Therapeutics
- Pluristem Therapeutics
- ReNeuron
- Spectrum Pharmaceuticals (formerly NeoTherapeutics)
- StemCells, Inc.
- Titan Pharmaceuticals
- ViaCell (now PerkinElmer)

1. Overview 4
1.1 Statement of Report 4
1.2 Scope of the Report 5
1.3 Methodology 5
1.4 Executive Summary 6

2. Biology of Stem Cells 8
2.1 Scope of this Chapter 8
2.2 Basic Properties of Stem Cells and Their Classification 8
2.3 Differentiation of Stem Cells 11
2.3.1 Neural and Neuronal Differentiation 11
2.3.2 Cardiomyogenic Differentiation of ESCs 11
2.3.3 Differentiation of Mesenchymal Stem/Progenitor Cells 12
2.4 Self-Renewal of Stem Cells 12
2.5 Dedifferentiation and Nuclear Reprogramming 13
2.6 Functional Proliferation of Adult Mature Cells 14
2.7 Nanog Promotes Transfer of Pluripotency After Cell Fusion 14
2.8 MicroRNAs in Stem Cell Development 15
2.9 Stem Cell Therapy 15

3. Market Analysis of the Stem Cell Space 16
3.1 Scope of this Chapter 16
3.2 The Overall Market Opportunity and Segmentation of the Total Stem Cell Marketplace 16
3.3 Potential Stem Cell Commercial Applications 19
3.3.1 Cord Blood Stem Cells and Cord Blood Stem Cell Banking: Current Market Environment and Opportunity 19
3.4 Results of Primary Market Survey to Characterize Qualitative and Quantitative Trends in the Stem Cells Space 22
3.4.1 Characteristics of the Respondent Pool 23
3.4.2 Utilization of the Different Classes of Stem Cells 25
3.4.3 Quantitative Metrics of the Stem Cell Research Marketplace: Number of Experiments Performed in Research 26
3.4.4 Evolution of Research Activities with the Different Types of Stem Cells: Current Usage Trends and Usage in 12 to 18 Months 30
3.4.5 Types of Research Activities the End-User Community Performs with the Different Types of Stem Cells 32
3.4.6 Challenges Facing Stem Cell Researchers 35
3.4.7 Market Drivers and Research Questions Being Addressed in the Stem Cell Space 39
3.4.8 Summary of Challenges and Opportunities in the Stem Cell Space Based on TriMark's Analysis 40
3.4.9 Unmet Product Needs in the Stem Cell Research Space and Licensing/IP Constraints 41
3.5 Competitive Landscape of the Stem Cell Marketplace 43
3.5.1 SWOT Analysis of the Major Stem Cell Market Segments 50
3.6 Market Size and Forecasts 53

4. Worldwide Landscape of Stem Cell Research 57
4.1 Scope of this Chapter 57
4.2 Global Embryonic Stem Cell Research 57
4.2.1 Top Research Institutions Worldwide in the Stem Cells Space and Global 'Hot Spots' for Research and Therapeutics 58
4.3 Stem Cell Research in the U.S. 60
4.3.1 Proposition 71 in the State of California 62
4.4 Intellectual Property (IP) Landscape Surrounding Stem Cells 63
4.5 Who Will Finance Stem Cell Research? 65
4.5.1 U.S. Government Policy on Stem Cell Research 65
4.5.2 Current Federal Policy 68
4.5.2.1 Moral Issues Affecting the Policy 69
4.5.2.2 Significance and Requirement of Federal Funding 71
4.5.2.3 Implementing the Current Policy 73
4.5.3 International Law 75
4.5.3.1 Australia 75
4.5.3.2 U.K. 75
4.5.3.3 Canada 75
4.5.3.4 China 76
4.5.3.5 Germany 77
4.5.3.6 Switzerland 77
4.5.3.7 Japan 77
4.5.3.8 Israel 77
4.5.3.9 Singapore 78
4.5.4 Technological and Economical Disadvantage for U.S. 78

5. Therapeutic Areas and Research Programs in the Stem Cell Space 80
5.1 Scope of this Chapter 80
5.2 Therapeutic and Other Markets Addressed by Stem Cells 80
5.2.1 Hematopoietic Stem Cells (HSCs) 81
5.2.2 Stem Cell Therapeutics for Cardiac and Ischemic Diseases 84
5.2.3 Stem Cell Therapeutics for Type I Diabetes 86
5.2.4 Stem Cell Therapeutics for Orthopedic Diseases 87
5.2.5 Stem Cell Therapeutics for Neurological Disease 87
5.3 Off-the-Shelf Stem Cells Being Developed 88

6. Company Profiles 91
6.1 Aastrom Biosciences 91
6.2 Advanced Cell Technology 92
6.3 Aeolus Pharmaceuticals (formerly Incara Pharmaceuticals) 93
6.4 BioE 94
6.5 Bioheart 94
6.6 BioTransplant 95
6.7 Cell Science Therapeutics 95
6.8 Cryo-Cell International 96
6.9 Diacrin 96
6.10 ES Cell International 97
6.11 Gamida Cell 97
6.12 Geron 97
6.13 NeuroGeneration 99
6.14 Nexell Therapeutics 99
6.15 Osiris Therapeutics 99
6.16 Pluristem Therapeutics 100
6.17 ReNeuron 101
6.18 Spectrum Pharmaceuticals (formerly NeoTherapeutics) 101
6.19 StemCells, Inc. 102
6.20 Titan Pharmaceuticals 103
6.21 ViaCell (now PerkinElmer) 103

Appendix 1. Glossary of Terms in Stem Cell Biology 104

INDEX OF FIGURES

Figure 2.1: Derivation of Embryonic Stem Cells (ESCs) from the Inner Cell Mass of Blastocysts 9
Figure 2.2: Classification of Stem Cells: Embryonic Stem Cells Versus Adult Stem Cells 10
Figure 2.3: Somatic Cell Nuclear Transfer (SCNT) 13
Figure 3.1: Segmentation of the Stem Cell Marketplace Based Upon Commercial Offerings-Products and Services 18
Figure 3.2: Geographical Breakout of Stem Cell End-User Survey Respondents 23
Figure 3.3: Affiliation (Pharma, Biotech, Academic/University) of Stem Cell End-User Survey Respondents 23
Figure 3.4: Survey Respondents That Are Studying Stem Cells or Using Stem Cells in Their Research 24
Figure 3.5: Length of Time Survey Respondent Pool Has Been Studying Stem Cells or Using Stem Cells in Its Research Activities 24
Figure 3.6: Utilization/Research with Different Stem Cell Types 25
Figure 3.7: Range of Number of Experiments Involving Stem Cells Conducted Per Week 26
Figure 3.8: Differences with Respect to Usage of Stem Cell Types 27
Figure 3.9: Research Dollars Spent Monthly on Research/Studies with Different Stem Cell Types 28
Figure 3.10: Growth of Research Spending in Various Segments of the Stem Cells Space (Defined by Stem Cell Type) 29
Figure 3.11: Growth in End-User Utilization of the Various Different Stem Cell Types 29
Figure 3.12: Breakout of the Stem Cell Research Market: Current Research Efforts with Different Types of Stem Cells 31
Figure 3.13: Breakout of the Stem Cell Research Market: Research Efforts with Different Types of Stem Cells in 12 to 18 Months 31
Figure 3.14: Broad Classification of Research Activities Being Performed with Stem Cells by Researchers 32
Figure 3.15: Current End-User Utilization Category of Adult Stem Cells (ASCs) 33
Figure 3.16: Current End-User Utilization Category of Human Embryonic Stem Cells (hESCs) 33
Figure 3.17: Current End-User Utilization Category of Human Cord Blood Stem Cells 33
Figure 3.18: Current End-User Utilization Category of Animal Stem Cells 34
Figure 3.19: Current End-User Utilization Category of Cancer Stem Cells 34
Figure 3.20: Challenges in the Study or Utilization of Adult Stem Cells (ASCs) 36
Figure 3.21: Challenges in the Study or Utilization of Human Embryonic Stem Cells (hESCs) 36
Figure 3.22: Challenges in the Study or Utilization of Human Cord Blood Stem Cells 37
Figure 3.23: Challenges in the Study or Utilization of Animal Stem Cells 37
Figure 3.24: Challenges in the Study or Utilization of Cancer Stem Cells 38
Figure 3.25: Top Unmet Needs in Commercial Products in the Stem Cell Research Space 42
Figure 3.26: The U.S. Stem Cell Therapy Market Sales, 2005-2016 55
Figure 4.1: Stem Cell Research Worldwide 59

INDEX OF TABLES

Table 2.1: The Different Types of Stem Cells and Their Properties 10
Table 2.2: Adult Stem Cell (ASC) Differentiation Properties 11
Table 3.1: Characteristics of Different Stem Cell Types and Associated Market Opportunity 17
Table 3.2: Segmentation of the Stem Cell Market by Type/Lineage of Stem Cells 18
Table 3.3: Selected Cord Blood Banks in the U.S. 21
Table 3.4: Selected Companies in the Cord Blood Stem Cell Technology and Therapeutics Development Space 22
Table 3.5: Breakout of the Stem Cell Research Market: Current Research Efforts with Different Types of Stem Cells 30
Table 3.6: Breakout of the Stem Cell Research Market: Research Efforts with Different Types of Stem Cells in 12 to 18 Months 30
Table 3.7: Market Share of Companies/Institutions Offering Different Stem Cells to Researchers 45
Table 3.8: Market Share of Companies/Institutions Offering Reagents for Stem Cell Research 46
Table 3.9: The U.S. Stem Cell Therapy Market Forecast, 2005-2016 54
Table 4.1: Major Milestones in Stem Cell Research and Associated Timeline, 1981-2008 58
Table 4.2: Selected Stem Cell Hot Spots Worldwide Administering Non-FDA Approved Stem Cell Therapeutic Regimens 60
Table 4.3: NIH Funding of Stem Cell Research in the U.S., 2004-2009 61
Table 4.4: Snapshot of the Current Key Stem Cell Patents 64
Table 4.5: Stem Cell Derivations 73
Table 5.1: Estimate of Potential Market Opportunity for Stem Cell-based Therapeutics 80
Table 5.2: Autologous Versus Allogeneic Cell Therapy 81
Table 5.3: Diseases Treated with HSC Transplantation Today 82
Table 5.4: HSC Sources and Their Properties 83
Table 5.5: Stem Cell Therapeutics for Hematological Diseases in Development 84
Table 5.6: Stem Cell-based Therapeutics for Cardiac and Ischemic Diseases in Development 85
Table 5.7: Stem Cell-based Therapeutics for Vascular Diseases in Development 86
Table 5.8: Stem Cell Therapeutics for Type I Diabetes in Development 86
Table 5.9: Stem Cell Therapeutics for Orthopedic Disease in Development 87
Table 5.10: Stem Cell Therapeutics for Neurological Disease in Development 88
Table 5.11: Segmentation of the Allogeneic Cell Therapy Market 90