July 2007
Volume: TMRCTHER07-0701
CELL THERAPY MARKETS
(SAMPLE COPY, NOT FOR RESALE)
TABLE OF CONTENTS
1. Overview 9
1.1 Statement of Report 9 1.2 About this Report 9 1.3 Scope of the Report 11 1.4 Objectives 11 1.5 Methodology 11 1.6 Executive Summary 12
2. Overview of the Science of Cell Therapy 15 2.1 Cell Therapy Technologies 15
2.1.1 Stem Cell Therapies 16
2.1.2 Umbilical Cord Blood Stem Cell Transplant 18
2.1.3. Cord Blood Collection, Transfusion and Storage Market 18 2.1.4 Cell Therapy and Related Technologies 23
2.2 Organ Transplantation to Cell Therapy 23 2.3 Sources of Cells for Therapy 25
2.4 Types of Cells Available for Therapy 28 2.4.1 Cells and Protein/Gene Therapy 29 2.4.2 Cell Therapy and Regenerative Medicine 29 2.4.3 Cells Therapy and Tissue Engineering 29
2.5 Cell Therapy Compared to Small Molecule Drugs, Biologics and Tissue Engineered Products 29 2.6 What Conditions are Amenable to Cell Therapy and Why? 30
2.6.1 Wounds and Burns 30
2.6.2 Aesthetic Medicine: Botox and Collagen 33 2.6.3 Cell Therapy for Bones and Joints 34 2.6.4 Blood Component Therapy 35 2.6.5 Immunotherapy for Cancer 36 2.6.6 Cardiovascular Disorders 36 2.6.7 Diabetes Mellitus 38 2.6.8 Neurological Disorders 39 2.6.9 Urinary Incontinence 40 2.6.10 Cells for Drug Discovery 41
2.7 Properties of Stem Cells and Their Classification 41 2.8 Stem Cell Differentiation 45
2.9 Stem Cell Therapy 46
2.10 Market Opportunity in the Stem Cell Marketplace 47 2.11 Potential Stem Cell Commercial Applications 49
2.11.1 Cord Blood Stem Cells and Cord Blood Stem Cell Banking: Current Market Environment and Opportunity 49 2.11.2 Qualitative and Quantitative Trends in the Stem Cells Space 53
2.11.3 Utilization of the Different Classes of Stem Cells 56
2.11.4 Quantitative Metrics of the Stem Cell Research Marketplace: Number of Experiments Performed, Research Spending Trends and Growth 57
3. The Cell Therapy Market 62 3.1 Market Overview 62
3.2 The Overall Cell Therapy Market Opportunity 62 3.3 Stem Cell Therapies 62
3.3.1 Cell Therapy/Regenerative Therapy Markets 65 3.3.2 Market Size According to Geographical Areas 66 3.4 Competitive Landscape of the Stem Cells Marketplace 66
3.4.1 Evolution of Research Activities with the Different Types of Stem Cells: Current Usage Trends and Usage in 12 to 18 Months 67 3.4.2 Types of Research Activities the End-User Community Performing with the Different Types of Stem Cells 69
3.4.3 Challenges Facing Stem Cell Researchers 73 3.4.4 Market Drivers 77
3.4.5 Research Questions Being Addressed in the Stem Cells Space 78 3.4.6 Focus on Cancer Stem Cells 78
3.4.7 Summary of Challenges and Opportunities in the Stem Cells Space Based on TriMark's Analysis 79 3.4.8 Unmet Product Needs in the Stem Cells Research Space and Licensing/IP Constraints 79
3.5 Competitive Landscape 81 3.5 1 Cell Therapy Market 81 3.5.2 Stem Cells Marketplace 82
3.6 Market Shares of Companies Offering Different Stem Cells 82 3.7 SWOT Analysis of the Major Stem Cell Market Segments 87 3.8 Market Size and Forecasts 90
3.8.2 Cell Therapy/Regenerative Therapy Markets 91 4. The Technology of Cell Therapy 93
4.1 Growing Cells-An Overview from Source to Product 93 4.2 Cell Culture 94
4.2.1 Cell Culture Process 94 4.2.2 Cell Culture Applications 94 4.2.3 Types of Mammalian Cultures 95 4.2.4 Plant Cell Culture 95
4.2.5 Commercialization of Cell Culture Products 95 4.2.6 Companies in Cell Culture Technology 96 4.3 Cell Lines 96
4.4 Large Scale Manufacturing 97
4.5 Making Product, Shipping, Distribution and Storage 98 4.6 Quality Control and Safety 98
4.7 Delivery Systems for Cell Therapy 99 4.8 Basic Technologies for Cell Therapy 100 4.8.1 Flow Cytometry and Cell Sorting 100 4.9 Cell Seperation Techniques 105 4.9.1 Density-Based Isolation Methods 105
4.9.1.1 Ficoll-hypaque-based Density Gradient System NIM2 105 4.9.1.2 Lymphocyte Separation Medium 105
4.9.1.3 Density Gradient Media 105 4.9.2 Micro Sieves 105
4.9.3 Antibody-based Cell Separation Techniques 106 4.9.4 Magnet Sorting 107
4.9.5 Automated Seperation Procedures 109
4.9.6 Aldesorter System for Isolation of Stem Cells 109 4.10 Cell Preservation Technology 110
4.11 Selective Expansion of T Cells for Immunotherapy 110 4.12 Devices for Delivery of Cell Therapy 111
4.12.1 Artificial Cells 111 4.12.2 Cell Encapsulation 111
4.12.2.1 Range of Therapeutic Tretaments 112 4.12.2.2 Technical Barriers 112
4.12.2.3 Current Challenges 112
4.12.3 Therapeutic Applications of Encapsulated Cells 115 4.12.3.1 Cancer Therapy 115
4.12.3.2 Therapeutic Genes 118 4.12.3.3 Retinal Disease 119
4.12.4 Use of Microencapsulated Genetically Modified Cells 119 4.12.5 Nanoparticle-Aptamer Bioconjugates 120
5. Stem Cells 121
5.1 Basic Properties of Stem Cells and Their Classification 121 5.1.1 Embryonic Stem Cells 121
5.1.2 Embryonic Germ Cells 121 5.2 Sources of Stem Cells 122
5.3 Challenges Facing Stem Cell Researchers 123 5.4 Stem Cells for Drug Toxicity Screening 125 5.5 Market Analysis of the Stem Cells Space 126 6. Clinical Applications of Cell Therapy 127 6.1 Cell Therapy for Hematological Disorders 127
6.1.1 Hematopoietic Cells for Hematologic Diseases and to Prevent Immune Rejection 127 6.1.2 Hematopoietic Cytokines 128
6.1.3 Prochymal™ for the Treatment of Acute Graft versus Host Disease 129 6.1.4 Cell/Gene Therapy of Hemophilia B 130
6.1.5 Ex Vivo Cell/Gene Therapy of Hemophilia A 131 6.1.6 Therapies for the Hemophilia Market 133 6.1.7 Stem Cell Transplant for Sickle Cell Anemia 134
6.1.8 Modified Hematopoietic Stem Cells for Chronic Acquired Anemias 135 6.1.9 Stem Cell Therapy of Hemoglobinopathies 135
6.1.10 Future Prospects of Cell Therapy of Hematological Disorders 136 6.2 Cell Therapy for Immunological Disorders 136
6.2.1 Treatment of Crohn's Disease with Stem Cells 136 6.3 Cell therapy for Diabetes Mellitus 137
6.3.1 Islet Cells for Diabetes 137 6.4 Cell Therapy for Liver Disorders 137 6.4.1 About Liver Disease 137
6.4.2 Technology Summary 137 6.5 Cell Therapy for Renal Disorders 138
6.5.1 Human Renal Epithelial Cells Administered Ex Vivo 138 6.6 Cell Therapy for Disorders of Bones and Joints 138
6.6.1 Osteoblasts for Osteoporosis and Non-Union Bone Fractures 138 6.6.2 Chondrocytes for Osteoarthritis and the Use of hESCs 139
6.6.3 Knee Injury Treatment for Meniscectomy Patients Using Mesenchymal Stem Cells 139 6.7 Wound Healing: Skin and Soft Tissue Repair 139
6.7.1 Autologous Living Cell Therapy 139 6.7.2 Cell Therapy for Corneal Repair 140
6.7.2.1 Current Clinical Status for Cornea Disease and Transplant 140 6.7.2.2 Regenerative Cornea Product from Cellseed 141
6.8 Role of Cells in Tissue Engineering and Reconstructive Surgery 141 6.9 Cell Therapy for Cardiovascular Disorders 141
6.9.1 Introduction to Cardiovascular Disorders 141 6.9.1.1 Myocardial Infarction (Heart Attack) 141 6.9.1.2 Congestive Heart Failure (CHF) 142 6.9.1.3 Heart Failure Classification 142
6.9.1.4 Diagnosis and Management of Heart Failure 143 6.9.1.5 Drug Therapies 143
6.9.1.6 Bi-ventricular Pacers 143
6.9.1.7 Heart Transplantation and Other Surgical Procedures 144 6.9.1.8 Ventricular Assist Devices 144
6.9.1.9 Market Potential 145
6.9.1.10 Limitations of Current Therapies for Myocardial Ischemic Disease 145 6.9.2 Sector Overview of Cell Based Cardiac Repair 145
6.9.2.1 Categories of Cell Types for Cell Therapy 146 6.9.2.1.1 Autologous Skeletal Myoblasts 146 6.9.2.1.2 Cardiocytes 147
6.9.2.1.3 Mesenchymal Stem Cell (MSC) 148 6.9.2.1.4 Bone Marrow Derived Stem Cells 148 6.9.2.1.5 Fetal Cardiomyocytes 148
6.9.2.1.6 Smooth Muscle Cells 148 6.9.2.1.7 Immortalized Myoblasts 148 6.9.2.1.8 Syngeneic Skeletal Myoblasts 148 6.9.2.1.9 Fibroblasts 148
6.9.2.1.10 Embryonic Stem Cell 149
6.9.2.1.11 Bone Marrow Derived Stromal Cells 149
6.9.2.2 Current Cell Therapies for Myocardial Ischemic Disease 150 6.9.2.2.1 Cardiomyocytes for Heart Disease 150
6.9.2.2.2 Mesenchymal Stem Cells (MSCs) Provacel™, (Osiris Therapeutics) 150 6.9.2.2.3 Living Regenerative Cardiac Patch (Cellseed) 151
6.9.2.2.4 Bioheart's MyoCell 151
6.9.2.2.5 Viacell's Unrestricted Somatic Stem Cells (USSCs) 154 6.9.2.2.6 Mytogen's Autologous Myoblasts 155
6.9.3 Research Methods for Delivery of Cells to the Heart 155 6.9.3.1 Cell Transplantation for Cardiac Repair 155
6.9.3.2 Using Nanofibers to Deliver Insulin Growth Factor 155 6.9.4 Role of Stem Cells in Repair of the Heart 156
6.9.4.1 Cardiac Autologous Stem Cells 156 6.9.4.1.1 Expansion of Stem Cells 156
6.9.4.1.2 Role of ESCs in Repair of the Heart 158 6.9.4.1.3 Transplantation of BMPCs 158 6.9.4.1.4 Transplantation of CMPCs 159 6.9.4.1.5 Transplantation of hESCs 159
6.9.4.1.6 Transplantation of Skeletal Myoblasts 160
6.9.4.1.7 Transplantation of Autologous Angiogenic Cell Precursors 160 6.9.4.1.8 Regeneration of Chronic Myocardial Infarcts by HSC Therapy 161 6.9.4.1.9 Transplantation of Adipose-Derived Stem Cells 162
6.9.4.1.10 Intracoronary Infusion of Bone Marrow-Derived Cells for AMI 163 6.9.4.1.11 Human Mesenchymal Stem Cells for Cardiac Regeneration 163
6.9.4.1.12 Simultaneous Transplantation of Autologous MSCs and Skeletal Myoblasts 163 6.9.5 Role of Genetically Modified Cells 164
6.9.5.2 Use of Cells Secreting Vascular Endothelial Growth Factor 166 6.9.6 Cell Therapy for Congestive Heart Failure 166
6.9.6.1 Angiocell Gene Therapy for Congestive Heart Failure 166 6.9.7 Role of Cell Therapy in Cardiac Arrhythmias 166
6.9.7.1 Genetically Modified Cell Grafts 167 6.9.7.2 Cardiomyocyte Cell Grafts 167
6.9.7.3 Antiarrhythmic Potential of Cell Therapy Strategies 168 6.9.7.4 Vectors for Gene Therapy 169
6.9.7.5 Methods of Gene Delivery 169
6.9.7.6 Gene Therapy in Cardiac Arrhythmias 169 6.9.7.7 Current Problems with Gene Therapy 169
6.9.8 Cell Therapy for Cardiac Tissue Engineering of Blood Vessels with Cells 170 6.9.8.1 Fetal Cardiomyocytes Seeding in Tissue-Engineered Cardiac Grafts 170 6.9.9 Competitive Landscape for Cell Therapy Based Cardiovascular Products 170 6.9.10 Evaluation of Cell Therapy as Therapy for Heart Disease 171
6.10 Cell Therapy for Cancer 171 6.10.1 Introduction 171
6.10.2 Cell Therapy Technologies for Cancer 171
6.10.3 Cellular Immunotherapy for Cancer Treatments for Cancer by ExVivo Mobilization of Immune Cells 172 6.10.4 Alternative Cancer Treatment Market 172
6.10.5 Autologous Tumor Cell Vaccines and Dendritic Cell Therapy 173 6.10.6 The Use of Dendritic Cells for Cancer Vaccination 177
6.10.6.1 Collection of Cells 177
6.10.6.2 Basics of Dendritic Cell Vaccines 178
6.10.6.3 Dendritic Cells Treated with Purified Tumor Antigen 179
6.10.6.4 Stimulation of Immature Dendritic Cells to Become Mature Dendritic Cells 179 6.10.6.5 Cell Fragments of Dendritic Cells Primed with Tumor Cell Antigens 180 6.10.7 Gene Therapy 181
6.10.8 Stem Cell-Based Anticancer Therapies 181 6.10.9 Other Cell Therapy Vaccines 181 6.10.10 Stem Cell Transplantation in Cancer 182 6.10.10.1 Peripheral Blood Stem Cell Transplantation 182 6.10.10.2 Autologous Stem Cell Transplantation 184
6.10.10.3 Complications of Stem Cell Transplants in Cancer 185 6.10.10.4 Mesenchymal Stem Cell Transplantation in Cancer 185 6.10.10.5 Umbilical Cord Blood Transplant for Leukemia 185 6.10.10.6 Hesc-Derived NK Cells for Treatment of Cancer 186 6.10.11 Innovations in Cell-Based Therapy of Cancer 186 6.10.11.1 Cancer Therapy Based on NK-92 Cells 186 6.10.11.2 Myoblast Mediated Gene Therapy 187 6.10.12 Cancer Stem Cells 187
6.10.13 Mesenchymal Stem Cells for the Treatment of Gliomas 187 6.10.14 Companies Involved in Cell-Based Cancer Therapy 188 6.10.15 Stem Cell-Based Anti-Cancer Therapies 191
6.11 Cell Therapy for Neurological Disorders 192
6.11.1 Oligodendrocytes for Spinal Cord Injury and Dopaminergic Neurons for Parkinson's Disease 192 6.11.2 Neural Stem Cells as a Treatment for Neuronal Ceroid Lipofuscinosis 192
6.11.3 Epilepsy and Neuropathic Pain Markets 192
6.11.4 Delivery of Naturally Occurring Neurotrophic Factors 192 6.11.5 Repairing the Nervous System 193
6.11.6 Genes Permanently Incorporated into Targeted Cells 193 6.11.7 Neurological Disorders Responding to Neurotrophic Factors 194 6.11.8 Ceregene's Products 194
6.11.9 Parkinson's Disease 195 6.11.9.1 Introduction 195 6.11.9.2 Market Opportunity 195
6.11.9.3 Stem Cell Line for Functional Dopaminergic Neurons 195 6.12 Cell Therapy for Stroke 197
6.12.1 Introduction 197 6.12.2 Market Opportunity 197
6.12.3 Stem Cell Therapy for Chronic Stroke Disability 197 6.13 Cell Therapy for Dental Treatments 198
6.14 Gene Therapy 198
6.14.1 Vehicles for Gene Transfer 198 6.14.2 Viral Vectors for Gene Therapy 199 6.14.3 Nonviral Vectors: Liposomes 200
6.15 hESC-Derived Hepatocytes for Drug Screening and Toxicology 201 7. Ethics, Regulation and Funding 203
7.1 Ethics 203
7.2 The U.S. Position on hESC Research 205 7.3 Regulation and Legislation 206
7.3.1 Regulations on Stem Cell Research 206 7.3.2 Regulation of Tissue Engineered Products 208 7.4 Investment 209
7.5 Interdisciplinary and Public-Private Partnerships 210 7.6 Corporate Partnerships 210
7.6.1 ReNeuron and CellSeed Collaboration Using ReNcellTM Liver Cell Lines 210 7.6.2 Plureon Corporation in Agreement with BD 211
7.6.3 Icoria Inc. Research Agreement with Vesta Therapeutics 211 7.7 Corporate Mergers 211
7.7.1 GenVec's Acquisition of Diacrin, Inc. 211
7.7.2 Genvec Transferred its Cell Transplantation Assets to Mytogen, Inc. 211 7.8 European Regulation on Advanced Cell Therapies 212
7.9 Processing Autologous Cell Products-Discussing the Regulatory and Commercial Impact of Patient-Specific Therapies 213 7.10 Stem Cell Research Funding 214
8. Future Directions in Cell Therapy 215
8.1 Cell Therapy Future Markets According to Therapeutic Area 215 8.1.1 Brain and Central Nervous System Applications 215
8.1.2 Cardiology Applications 218 8.1.3 Diabetes Applications 221 8.1.4 Tissues and Organs 222 8.1.5 Other Applications 222
8.2 Unmet Market Needs in Cell Therapy 223 8.3 Future Prospects of Cell Therapy 223 8.3.1 Autologous Stem Cells 223 8.3.2 Pluripotential Stem Cells Lines 224 8.3.3 Angiogenesis 224
8.3.4 Reprogramming Stem Cells 224 8.4 Clinical Trials in Cell Therapy 224 8.5 Analytical Tools for Cell Therapy 226
8.6 Ethical Concerns of Cloning for Reproductive Purposes 226 8.6.1 Ethical Concerns 226
8.6.2 Policy and Regulation 226
8.7 Cloning for the Isolation of Human ES Cells 227 8.7.1 Ethical Concerns 227
8.7.2 Policy and Regulation 227
8.8 Somatic Cell Nuclear Transfer Generating Pluripotential Stem Cells 227 8.9 Funding of Stem Cell Research from Non-Federal Sources 228 8.10 Industry Challenges for Cell Therapy Manufacturing 228 8.10.1 Cost Challenges to Manufacturing Processes 228
8.10.2 Supply Challenges for Current Manufacturing Processes 228 8.11 Future Prospects of Cell Therapy 228
8.12 Challenges for Cell Therapy 229 8.13 Therapeutic Cloning 229
8.14 Future Market Potential of Adult Versus Embryonic Stem Cells 230
8.15 Challenges of Developing a Cell Therapy Product Compared to a Conventional Drug Product 231 8.16 Transition from a Techology Driven Company to a Product Driven Company 231
8.17 Exploiting Technology Platforms for Commercial Success 231
8.18 At What Point Do You Seek a Partner in Cell Therapy Development 232 8.19 Cell and Tissue Therapies Product Development and Manufacture 232
8.19.1 What Commercialization Models are Emerging to Cope with Complex Manufacturing Required by Cell Therapies? 232 8.19.2 Optimizing Product Development, Manufacture and Commercialization of Autologous and Allogeneic Cell Therapies 232 9. Company Profiles 234
9.1 Aastrom Biosciences 234 9.2 Advanced Cell Technology 235 9.3 Advanced Cell Therapeutics 235 9.4 Aldagen 235
9.5 AllCells, LLC. 236 9.6 AmCyte 236 9.7 Arthro Kinetics 236
9.8 AstraZeneca 236
9.9 Australian Stem Cell Centre 237 9.10 Axordia 237
9.11 BioE 238 9.12 Bioheart 238
9.13 BioTransplant Incorporated 239 9.14 BrainStorm Cell Therapeutics 239 9.15 Cambrex 240 9.16 Cardio3 240 9.17 Cartela 240 9.18 Cellartis 241 9.19 Cellerant Therapeutics 241 9.20 CellSeed Inc. 241
9.21 Cellular Dynamics International 241 9.22 Ceregene, Inc. 242
9.23 Cryo-Cell International 242 9.24 Cytori Therapeutics 243 9.25 ES Cell International 244
9.26 Fraunhofer Institute for Cell Therapy and Immunology 244 9.27 Gamida Cell Therapeutics 244
9.28 GE Healthcare 245 9.29 GenVec, Inc. 245 9.30 Geron 246 9.31 GlaxoSmithKline 247 9.32 Intercytex 249 9.33 Invitrogen 249
9.34 Institute for Stem Cell Research, University of Edinburgh 250 9.35 Isolagen 250
9.36 Lexicon Genetics 251
9.37 Lund Stem Cell Center, Lund University 252 9.38 MaxCyte 252
9.39 Miltenyi Biotec 253 9.40 NeuroNova 253
9.41 Norwegian Center for Stem Cell Research 254 9.42 NovaThera 254
9.43 NsGene 254
9.44 Osiris Therapeutics 255 9.45 Pfizer 255
9.46 Plureon 257
9.47 Pluristem Life Systems 257 9.48 Progenitor Cell Therapy, LLC 258
9.49 Regenerative Medicine Network (RegMedNet), University of Leipzig 258 9.50 RenaMed Biologics 258
9.51 ReNeuron 258 9.52 StemCells, Inc. 259 9.53 Stem Cell Innovations 259 9.54 Stem Cell Sciences 260 9.55 StemCell Technologies 260 9.56 Thermogenesis 260 9.57 TiGenix 261
9.58 Tissue Engineering and Regenerative Medicine Centre, Imperial College London 261 9.59 UK Stem Cell Bank 261
9.60 UK Stem Cell Foundation 262 9.61 ViaCell 262
9.62 Vesta Therapeutics, Inc. 262 9.63 VistaGen Therapeutics 263
9.64 Wolfson Centre for Age-Related Diseases, King's College, London 263 9.65 AlphaCord 263
9.66 Celgene 263
9.67 Cord Blood Registry 264 9.68 Angiogene 265
9.69 EntreMed 265 Appendix 266
Sources of Umbilical Cord Blood for Stem Cell Research 266 National Marrow Donor Program 266
Stem Cells from Frozen Embryos 267 Stem Cell Policy Statement 267 Glossary of Terms in Cell Biology 268
LIST OF TABLES
Table 2.1: U.S. Companies Involved in Cord Blood Banking 21 Table 2.2: U.K. Companies Involved in Cord Blood Banking 22 Table 2.3: Waiting List for Organ Transplants in U.S. 25
Table 2.4: Number of Organ Transplants Carried out in U.S., January-March 2006 25 Table 2.5: Sources of Cells for Therapy and Other Applications 28
Table 2.6: U.S. Wound Care Market, 2003-2012 31
Table 2.7: Stem Cell Therapeutics for Orthopedic Disease in Development 35 Table 2.8: Cellular Therapies in Orthopedics Trauma and Fracture Markets 35
Table 2.9: Stem Cell-based Therapeutics for Cardiac and Ischemic Diseases in Development 37 Table 2.10: Stem Cell-Based Therapeutics for Vascular Diseases in Development 38
Table 2.11: Stem Cell Therapeutics for Type I Diabetes in Development 39 Table 2.12: Stem Cell Therapeutics for Neurological Disease in Development 40 Table 2.13: Key Properties of Stem Cells 42
Table 2.14: Sources of Human Stem Cells 42
Table 2.15: The Different Types of Stem Cells and their Properties 44 Table 2.16: Adult Stem Cell (ASC) Differentiation Properties 45
Table 2.17: Characteristics of Different Stem Cell Types and Associated Market Opportunities 48 Table 2.18: Segmentation of the Stem Cell Market by Type/Lineage of Stem Cell 49
Table 2.19: Selected Cord Blood Banks in the U.S. 52
Table 2.20: Selected Companies in the Cord Blood Stem Cell Technology and Therapeutics Development Space 53 Table 3.1: Worldwide Cell Therapy Markets, 2005-2012 62
Table 3.2: Worldwide Stem Cell, Cytokine, and Growth Factor Cell Therapy Markets, 2005-2012 63 Table 3.3: The World Market for Stem Cell Products and Services, 2005-2012 65
Table 3.4: Breakout of the Stem Cell Research Market: Current Research Efforts with Different Types of Stem Cells 67
Table 3.5: Breakout of the Stem Cell Research Market: Research Efforts with Different Types of Stem Cells in 12 to 18 Months 67 Table 3.6 Publicly-Held Stem Cell Companies 83
Table 3.7: Market Shares of Companies/Institutions Offering Different Stem Cells to Researchers 83 Table 3.8: Market Shares of Companies/Institutions Offering Reagents for Stem Cell Research 84 Table 3.9: Privately-Held Stem Cell Companies 84
Table 3.10: Product and Custom Service Providers in the Stem Cells Space 85 Table 3.11: Cord Blood Stem Cell Companies 85
Table 3.12: Adult Stem Cell Companies 85
Table 3.13: Reagents, Media and Consumable Companies 86 Table 3.14: Stem Cells Research Market Forecast, 2006-2011 91
Table 4.1: Worldwide Market for Flow Cytometry Instruments and Reagents, 2000-2010 102 Table 4.2 Leading Players in Flow Cytometry Instrumentation 103
Table 5.1: Leading Stem Cell Companies 126 Table 6.1: Hematopoietic Cytokines 128
Table 6.2: Skin Rejuvination (Aesthetics) Markets, 2005-2012 140 Table 6.3: Burn Scars Markets, 2005-2012 140
Table 6.4: Acne Scars, 2005-2012 140
Table 6.5: NYHA Classifications for Stages of Heart Failure 142
Table 6.6: Worldwide Market for Cardiac Care Therapeutics, 1999-2010 143 Table 6.7: Examples of Cell Types Used in Cardiac Repair 146
Table 6.8: Clinical Trials of MyoCell 153
Table 6.9 : The Common Metrics Used to Evaluate the Efficacy of These Therapies Include 154 Table 6.10: Stemline™ Platform Media for Expansion and Maturation of Stem Cells 156 Table 6.11: Gene Transfer Vehicles 199
Table 7.1: Investment in Stem Cell Research in Different Countries 209 Table 7.2: International Location of hESC Lines 212
Table 7.3: Unpublished Lines (Not Peer-Reviewed) 212 Table 8.1: Delivery of Cells for Heart Repair Technologies 220
LIST OF FIGURES
Figure 2.1: Release of Leukocytes into the Bloodstream 17
Figure 2.2: Derivation of Embryonic Stem Cells (ESCs) from the Inner Cell Mass of Blastocysts 43 Figure 2.3: Classification of Stem Cells: Embryonic Stem Cells versus Adult Stem Cells 44 Figure 2.4: Somatic Cell Nuclear Transfer (SCNT) 46
Figure 2.5: Segmentation of the Stem Cells Marketplace Based upon Commercial Offerings-Products and Services 50 Figure 2.6: Geographical Breakout of Stem Cell End-user Survey Respondents 54
Figure 2.7: Affiliation (Pharma, Biotech, Academic/University) of Stem Cell End-user Survey Respondents 54 Figure 2.8: Most of the Survey Respondents are Studying Stem Cells or Using Stem Cells in Their Research 55
Figure 2.9 Length of Time Survey Respondent Pool Studying Stem Cells or Using Stem Cells in Their Research Activities 55 Figure 2.10: Utilization/Research with Different Stem Cell Types 56
Figure 2.11: Range of Number of Experiments Involving Stem Cells Conducted per Week 57
Figure 2.12: Do the two Sub-Populations of Researchers Occupying the Two Ends of the Throughput Scale Exhibit Differences with Respect to Usage of Different Stem Cell Types? 58
Figure 2.13: How Much Research Dollars Are Spent Monthly on Research/Studies with Different Stem Cell Types 59 Figure 2.14: Growth of Research Spending in Various Segments of the Stem Cells Space (Defined by Stem Cell Type) 60 Figure 3.1: Global Forecast of Revenues for Stem Cell and Cytokine Therapies in Regenerative Medicine, 2003-2010 63 Figure 3.2: Stem Cell Research Market, 2006-2011 64
Figure 3.3: Growth in End-User Utilization of the Various Different Stem Cell Types 66
Figure 3.4: Breakout of the Stem Cell Research Market: Current Research Efforts with Different Types of Stem Cells 68
Figure 3.5: Breakout of the Stem Cell Research Market: Research Efforts with Different Types of Stem Cells in 12 to 18 Months 69 Figure 3.6: Broad Classification of Research Activities Being Performed with Stem Cells by Researchers 70
Figure 3.7: Current End-User Utilization Category of Adult Stem Cells (ASCs) 71
Figure 3.8: Current End-User Utilization Category of Human Embryonic Stem Cells (hESCs) 71 Figure 3.9: Current End-User Utilization Category of Human Cord Blood Stem Cells 72 Figure 3.10: Current End-User Utilization Category of Animal Stem Cells 72
Figure 3.11: Current End-User Utilization Category of Cancer Stem Cells 73 Figure 3.12: Challenges in the Study or Utilization of Adult Stem Cells (ASCs) 74
Figure 3.13: Challenges in the Study or Utilization of Human Embryonic Stem Cells (hESCs) 75 Figure 3.14: Challenges in the Study or Utilization of Human Cord Blood Stem Cells 75 Figure 3.15: Challenges in the Study or Utilization of Animal Stem Cells 76
Figure 3.16: Challenges in the Study or Utilization of Cancer Stem Cells 76
Figure 3.17: Top Unmet Needs in Commercial Products in the Stem Cells Research Space 80 Figure 3.18: Stem Cells Research Market, 2006-2011 91
Figure 4.1: Cell Microencapsulation: Promise and Progress 111 Figure 6.2: Ceregene Pipeline of Products 194
Figure 7.1: FDA Review Centers 205
1. Overview
1.1
Statement of Report
The purpose of this report is to describe the specific market segments of the cell therapy sector. This study reviews
all of the generally accepted clinical and analytical methods that are currently in use today for implementing cell
therapy techniques. It examines this new scientific technique and their reagents and supplies as utilized in hospitals,
research laboratories and biotechnology companies.
1.2
About this Report
The two most important areas where cell therapy is used are in the hospital and the clinic. Growth areas of interest
for cell therapy are biotechnology platforms. The emphasis in this analysis is on those companies and products that
are actively developing and marketing instrumentation, reagents and supplies for cell therapy. The reader should
consult other TriMark Publications reports at http://www.trimarkpublications.com for a detailed discussion of the
important individual market segments, which are related to the biotech.
The main objectives of this analysis are:
•
Identifying viable technology drivers through a comprehensive look at platform technologies for cell
therapy testing.
•
Understanding the different sectors of cell therapy testing, such as growing cells, cell sources and the
manufacturing segment; and, separately, looking at a description of the instruments, reagents and supplies
marketed by major companies in each segment. The report discusses the market size, growth rates and
market components for instruments and reagents, controls and consumables used in this area.
•
Obtaining a complete understanding of the individual cell therapy platforms from their basic principles to
their clinical applications.
•
Discovering feasible market opportunities by identifying high-growth applications in different therapeutic
areas, focusing on the biggest and expanding markets.
•
Examining on global industry development through an in-depth analysis of the major world markets for cell
therapy measurement technology, including growth forecasts.
•
Presenting market figures regarding the current value of cell therapy testing, market projections, market
share, key players and sector growth rates. The source of this information is the most current data derived
from the global pharmaceutical industry.
By purchasing this report, you will have:
•
An understanding of the most exciting cell therapy market segments, current and future.
•
The latest information on leading products and research and development (R&D) initiatives.
•
Familiarity with recent developments and their effects on selected markets.
•
Knowledge of the cell therapy market as an area of growth, research and investment.
•
An extensive review of the market for clinical cell therapy equipment and supplies used in the clinical
hospital market for testing cell therapy concentrations.
•
Details on the market for screening reagents and instruments for analysis of individual components in
blood, serum or plasma.
•
Defined dollar volume of sales, both worldwide and in the U.S. of the market, and analysis of the factors
that influence the size and the growth of the market segments.
Key questions answered in this study:
•
How can cell therapy measuring tools and technologies facilitate improved patient care?
•
What are the main types of cell therapy technologies that are currently available?
•
Who are the current key players in this marketplace?
•
Which cell therapy market areas have the greatest potential for growth?
•
What is the current state of the cell therapy market?
•
Which biotechnology and diagnostic companies are investing in new cell therapy technology platform
solutions?
•
What are the main cell therapy business strategies adopted by leading companies?
•
What are the benefits of various cell therapy technology platforms?
•
Who holds the proprietary rights to the cell therapy market technology platforms?
•
How is this technology currently being applied and utilized?
•
In the U.S., Japan and the E.U., what regulatory processes apply to cell therapy technologies?
•
How will new cell therapy technologies reduce healthcare expenditures?
•
How will new cell therapy technologies affect R&D spending?
This examination contains:
•
Detailed analysis of recent trends in the cell therapy marketplace.
•
In-depth profiles of the leading companies with cell therapy tools and technologies.
•
Forecasts for the cell therapy market in the biotechnology and diagnostic industries.
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Views and principles on the cell therapy industry from leading industry experts.
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Analysis of potential new cell therapy applications in the clinical sector.
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Market predictions and trends analysis concerning U.S. expenditures on cell therapy solutions.
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Projections of cell therapy market sizes for European and Asian markets.
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Projections for future applications of non-invasive tests in cell therapy related screening.
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Analysis of commercial cell therapy business strategies, such as co-branding.
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The latest news and M&A developments in the cell therapy marketplace.
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A comprehensive overview and insight into cell therapy business strategies for growth in foreign markets.
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An in-depth examination of the subsections of each cell therapy market segment is conducted.
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An overview that introduces the various kinds of stem cells, gives definitions, and discusses political,
regulatory aspects, and the major developments.
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Analysis of applications including current and potential cytokine and cell therapies.
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A discussion of current products, as well as many that are still in clinical trials.
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An industry structure analysis including companies in the field and their focus.
Analysis includes charts and graphs measuring product growth and trends within the marketplace. Company-specific
information, including sales figures, product pipeline status and R&D trends, is provided. Also, this study will:
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Assess cell therapy market drivers and bottlenecks, from medical and scientific community perspectives.
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Discuss the potential benefits of cell therapy for various sectors of the medical and scientific community.
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Establish the current total market size and future growth of the cell therapy market and analyze the current
size and growth of individual segments.
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Provide current and forecasted market shares by company.
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Discuss profit and business opportunities by segment.
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Provide strategic recommendations for near-term business opportunities.
1.3
Scope of the Report
This analysis emphasizes companies that are actively developing and marketing instrumentation, reagents and
supplies for performing cell therapy tests. The reader should consult other TriMark Publications reports at
http://www.trimarkpublications.com for detailed discussions of important individual market segments related to the
protein analysis market, such as clinical chemistry testing, high-growth diagnostic test markets, blood gas and
electrolytes, over-the-counter diagnostic testing markets, and point-of-care testing.
1.4 Objectives
The goal of this report is to review the market for cell therapy products using screening reagents and instruments for
analysis of individual components in tissue samples, blood, serum or plasma. It defines the dollar volume of sales,
both worldwide and in the U.S., and analyzes the factors that influence the size and the growth of the market
segments. Also examined are the subsections of each market segment, including the research labs, hospital labs and
commercial laboratories. Additionally, the numbers of institutions using this type of cell therapy and the factors that
influence purchases are discussed. The study surveys almost all of the companies known to be marketing,
manufacturing or developing instruments and reagents for the cell therapy market in the U.S. Each company is
discussed in extensive depth with a section on its history, product line, business and marketing analysis, and a
subjective commentary of the company’s market position.
1.5
Methodology
This examination is based upon interviews with sales and marketing professionals of companies in the cell therapy
marketplace. They were queried, some several times, about their companies’ products and marketing strategies, as
well as their overall thoughts about their industry segment. Information also was obtained from discussions with
founders, CEOs and vice presidents of some of the companies discussed in the report. Descriptions of the
laboratories and patient facilities were derived from interviews with laboratory directors and technologists in these
areas.
Other sources of information included trade association publications and meetings, product brochures and catalogs,
and company literature. We have also gathered statistical information from the U.S. government, the World Health
Organization, and private foundations. Annual reports, 10k filings, and financial reports were used as the basis for
data reported on publicly held companies. The author of this report is a Ph.D. in biochemistry with years of
experience in science writing and as a medical industry analyst. The editor is a doctoral level clinical scientist. He
has over thirty years of experience in laboratory testing and instrument and reagent development technology, as well
as extensive experience in senior level positions in biotech and medical service companies.
Some of the statistical information was taken from Biotechnology Associates’ databases and from TriMark’s private
data stores. The information set forth in this study was obtained from sources that we believe to be reliable, but we
do not guarantee the accuracy, adequacy or completeness of any information, omission or for the results obtained by
the use of such information.
Key information from the business literature was used as a basis to conduct dialogue with and obtain expert opinion
from market professionals with regard to commercial potential and market sizes. Senior managers from major
company players were interviewed for part of the information in this study. The information in this report is also
based upon direct experience with sales and marketing professionals of companies in the point of care instruments
and reagents market. People from many companies mentioned in this analysis were considered thoughtfully about
their companies’ products and marketing strategies, as well as their overall thoughts about their industry segment.
The structure of the laboratory facilities was derived from familiarity with scientists and technologists working in
these areas.
Primary Sources
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TriMark collects information from hundreds of database tables and many comprehensive
multi-client research projects and sector snapshots that we publish annually. We extract relevant data and analytics from
TriMark’s research of the past three years as part of this data collection. We also extract qualified data feeds from
e-questionnaire responses and primary research responses for this compilation.
Secondary Sources
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TriMark uses research publications, journals, magazines, newspapers, newsletters, industry
reports, investment research reports, trade and industry association reports, government affiliated trade releases, and
other published information as part of our secondary research materials.
The information is then analyzed and translated by the Industry Research Group into a TriMark study. The Editorial
Group reviews the complete package with product and market forecasts, critical industry trends, threats and
opportunities, competitive strategies and market share determinations. The report conclusions are verified through
intensive interviewing of top ranking companies in the industry.
TriMark Publications Report Research and Data Acquisition Structure
The general sequence of research and analysis activity prior to the publication of every report includes the following
items:
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Completing an extensive secondary research effort on an important market sector, including gathering all
relevant information from corporate reporting, publicly available databases, proprietary databases, direct
meetings and personal interviews with key personnel.
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Formulating a study outline with the assigned writer, including important items:
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Market and Product Segment grouping and evaluating their relative significance.
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Key competitors’ evaluations, including their relative positions in the business and other relevant facts
to prioritize diligence levels and assist in designing a primary research strategy.
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End-user research to evaluate analytical significance in market estimation.
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Supply chain research and analysis to identify any factors affecting the market.
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