Stem Cell Technologies 2013-2023


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Stem Cell Technologies 2013-2023

  1. 1. Stem Cell Technologies: World Market Outlook 2013-2023 ©notice This material is copyright by visiongain. It is against the law to reproduce any of this material without the prior written agreement of visiongain. You cannot photocopy, fax, download to database or duplicate in any other way any of the material contained in this report. Each purchase and single copy is for personal use only.
  2. 2. Contents 1. Executive Summary 1.1 Overview of Findings 1.2 Chapters in the Report 1.3 Research and Analysis Methods 2. Overview of the Stem Cells Market, 2013-2023 2.1 Strong Market Performance Expected 2013-2023 2.2 Cancer Segment Represents Bulk of Stem Cell Therapy Revenues, 2013 2.2.1 Other Therapeutic Areas to Gain Ground by 2023 2.2.2 Market Breakdown Focuses on Key Therapeutic Areas 2.3 Which Types of Stem Cells Matter for the Market? 2.3.1 The Discovery of Stem Cells 2.3.2 Embryonic Stem Cells (ESCs) Enter the Picture 2.3.3 Only ESCs and Induced Pluripotent Adult Cells (IPSCs) Have Pluripotency 2.3.4 MSCs Prominent in Clinical Trials 2.3.5 Autologous and Allogeneic Properties 2.3.6 Different Cell Advantages and Disadvantages 2.4 The Global Regulatory Environment for Stem Cells 2.4.1 US: No Morality Clause, Much Venture Capital ESC Controversy Now Settled? FDA Vs Regenerative Sciences 2.4.2 Japan: Liberal on hESCs First-in-Human iPSC Trial Announced in 2013 2.4.3 Europe: No Consistent Position Across the Continent The Brüstle Ruling: Dead and Buried? The UK Leads in European Stem Cells R&D 2.4.4 Rest of the World
  3. 3. Contents South Korea: Leading the Way in MSC Approvals China: Liberal Laws on Stem Cell Research Israel – Long-Term Research Activity ‘Stem Cell Tourism’ 2.8.1 The Vaccines Market 3. Stem Cell Cancer Therapeutics Segment 2013-2023 3.1 HSCT Will Underpin the Growth of the Cancer Segment in the 2013-2023 Period 3.2 Stem Cells: 45 Years as a Major Part of Blood Cancer Treatment 3.2.1 Autologous HSCT Operations Still Dominate Segment 3.2.2 Allogeneic HSCT: More Rejections, Fewer Relapses 3.2.3 GvHD: The Major Issue for HSCT 3.2.4 Peripheral Blood: More Anti-Tumour Effect, More GvHD Risk 3.2.5 The Rise of Umbilical Cord Blood 3.2.6 Around 60,000 HSCT Operations Performed Worldwide in 2012 3.2.7 HSCT Costly, Medicare Coverage Selective 3.2.8 Haematological Malignancies the Major Indication for HSCT 3.2.9 HSCT Addresses Cancers Representing a $20bn+ Market 3.2.10 From Successful Procedure to Successful Products? The Next Stage for the HSCT Sector 3.3 Pipeline Maturing, with First Approval in 2012 3.3.1 Osiris Therapeutics – Prochymal Finally Makes it to Market Osiris Leads the Pack in Stem Cell Therapeutics Using MSCs for Immunosuppression Positive Results in Acute Paediatric GvHD An Important Role to Play in the Future of HSCT 3.3.2 The Clinical-Stage Pipeline: Two More Candidates in Phase 3 3.3.3 Gamida Cell – StemEx: The Next Stem Cell Product to Market? Survival at 100 Days Improved by StemEx in Latest Trial FDA Remains Unconvinced NiCord Heads Up a Pipeline of New Cell Therapy Products
  4. 4. Contents NiCord Promising So Far 3.3.4 Mesoblast – Using Mesenchymal Precursors to Enrich Cord Blood Phase 3 Trial Measuring 100-Day Survival Rates and Engraftment Efficiency A Competitor for Gamida Cell? 3.3.5 Novartis Pharmaceuticals – Cord Blood Transplant Candidate Swiss Giant with Multiple Stem Cell Interests 3.3.6 Medipost – Promostem Uses Cord Blood MSCs to Prevent GvHD 3.3.7 CytoMedix – Improving Engraftment 3.3.8 Lentigen – LG631-CD34 for Chemoprotection 3.3.9 Athersys – MultiStem Trialled in Blood Cancers 3.3.10 HomeoTherapy – MSCs for GvHD 3.3.11 Pluristem Soon to Join the Hunt? 3.3.12 Other Stem Cell Oncology Prospects 3.3.13 Ancillary Products for the HSCT Setting 3.3.14 Targeting Cancer Stem Cells (CSCs) 3.3.15 Immunovative Therapies – Building on HSCT for a Cellular Anticancer Therapy 3.4 How Much Growth Potential Do Stem Cells Have in Cancer Indications? 4. Stem Cell Cardiovascular Therapeutics Segment, 2013-2023 4.1 Segment Set for 40%+ CAGR in 2013-2023 Period 4.2 Can Stem Cells Heal the Heart and Vasculature? 4.2.1 AMI, CLI and Stroke Major Ischaemic Disease Targets 4.2.2 Cerebrovascular Treatments for Stroke 4.2.3 What is the Heart’s Own Stem Cell? 4.2.4 MSCs Support Cardiac Stromal Lineages 4.2.5 The Mysterious Efficacy of Adult Stem Cells in the Heart 4.2.6 Cells Have Therapeutic Effects Despite Rapid Clearance 4.2.7 A New Generation of Cardiovascular Stem Cells May Need to Look Beyond MSCs 4.2.8 Are MSCs Safe and Efficacious?
  5. 5. Contents 4.2.9 Cardiovascular and Cerebrovascular Conditions Are World’s Most Fatal Diseases 4.2.10 Cardiovascular Drugs Market Exceeded $110bn in 2012 4.3 Cardiovascular Stem Cell Pipeline and Products, 2013-2023 4.3.1 FCB-Pharmicell – Hearticellgram-AMI, the First Approved Cardiovascular Stem Cell Treatment Performance So Far Will Hearticellgram Impress Regulators Outside Korea? 4.3.2 The Largest Pipeline in the Stem Cells Market 4.3.3 Baxter: Big Pharma Leader in Cardiovascular Stem Cells Exercise Tolerance and Angina Frequency Reduced Wider Potential for this Treatment 4.3.4 Cardio3 Bioscience – Cardiopoiesis Platform for Heart Regeneration Positive Phase 2 Data Phase 3 Trial Begins Amid Controversy 4.3.5 Bioheart – MyoCell in new MIRROR Trial Muscle Cells the Basis of MyoCell Seeking Expanded Access for Compassionate Use 4.3.6 NeoStem – Diversified Outfit with One of the Leading Candidates AMR-001 to Treat Acute Myocardial Infarction NeoStem Head-to-Head with Baxter in the HSC Cardiovascular Treatment Space 4.3.7 Aastrom Biosciences: Ixmyelocel-T for Cardiovascular Disease A Complex Multicellular Mixture Company Now Focusing on DCM 4.3.8 Athersys – MultiStem is a Potential Cardiovascular Treatment Addressing Ischaemic Stroke 4.3.9 CytoMedix – Three Clinical Candidates in the Cardiovascular Segment ALD-401 – Repairing Stroke Damage? ALD-301 – Entering Phase 2 in PAD Patients ALD-201 – Signs of Efficacy in Phase 1 Heart Failure Trial 4.3.10 TCA Cellular Therapy – Mesendo Autologous Treatments
  6. 6. Contents Preparing for Phase 3 with CLI Treatment 4.3.11 Medistem – The ‘Universal Donor’ Cell Trial in CLI the First Clinical Test for ERC Scalability a Major Advantage for Medistem 4.3.12 Stemedica Cell Technologies – Ischaemic Tolerant Stem Cell Platform Two Trials in Progress for Allogeneic MSCs 4.3.13 Mesoblast – Cell Therapy for Myocardial Infarction Teva Collaborating on Cardiovascular Stem Cell Therapy R&D 4.3.14 Pluristem – Cell Therapy for Myocardial Infarction Progress in PAD and CLI 4.3.15 Osiris – Prochymal Cell Therapy for Myocardial Infarction 4.3.16 Cytori Therapeutics – Adipose-Derived Cells for Acute Myocardial Infarction 4.3.17 Stempeutics – Emerging Bangalore Company Targets CLI 4.3.18 BioCardia – Comparing Autologous and Allogeneic MSCT Options 4.3.19 Apceth – Mesenchymal Stem Cells for Advanced Peripheral Arterial Occlusive Disease 4.3.20 Arteriocyte – Nanex Technology to Treat CLI 4.3.21 Capricor – Using Cardiosphere-Derived Cells for Heart Repair Phase 1/2 Trial Now Underway 4.3.22 Garnet BioTherapeutics – GBT009 for Cardiac Tissue Preservation 4.3.23 Biogenea-Cellgenea – Cardiogenea to Enter the Clinical Pipeline 4.3.24 Other Stem Cell Cardiovascular Prospects 4.4 Can Stem Cells Be a Game-Changer in the Cardiovascular Sphere? 5. Stem Cell CNS Therapeutics Segment, 2013-2023 5.1 The Biggest Stem Cell Therapeutic Revolution of All will Begin to Take Shape by 2023 5.2 Stem Cells the Best Hope for Many Serious CNS Conditions 5.2.1 Segment Awaits First Breakthrough Approval 5.2.2 Human NSCs Successfully Isolated 5.2.3 NSCs Reach the Clinic
  7. 7. Contents 5.2.4 CNS Segment the Major Focus for ESC Research 5.2.5 MSCs: Can Glial Cell and Astrocyte Formation Help Neurological Conditions? 5.2.6 Could ‘Resetting’ the Immune System Treat MS? 5.2.7 ALS: Rare Disease Could be the First Neurodegenerative Condition to Fall to Stem Cell Treatment 5.2.8 PD: Positive Signs from Early Experiments in Cellular Treatment 5.2.9 Dry AMD: A Major Global Unmet Need 5.2.10 Several Multi-Billion Dollar Potential Markets in the Sights of CNS Stem Cell Developers 5.3 Which Product Will Be First to Phase 3 Trials in the CNS? 5.3.1 BrainStorm Cell Therapeutics – NurOwn Cells for Spinal Cord Injury and ALS Validating the NurOwn Approach ALS Trial Ongoing 5.3.2 Neuralstem – Neural Stem Cells for Synaptic Repair and Neuroprotection Phase 2 Trial in ALS Enrolling by Invitation Chronic Spinal Cord Injury Treatment to Follow 5.3.3 Mesoblast – A New Option in Neovascular AMD? CNS Stem Cells from Dental Pulp 5.3.4 Advanced Cell Technology – Leading the Field in ESC Clinical Trials Continued Progress in Stargardt’s Macular Dystrophy in 2013 Early Signals of Efficacy? 5.3.5 StemCells – The Leading Player in the NSC Space? HuCNS-SC Product Proving Itself in Rare Indications Moving on to Spinal Cord Injury and AMD Alzheimer’s Disease and Stroke to Follow? 5.3.6 TCA Cellular Therapy – Mesendo for ALS 5.3.7 Corestem – Autologous Bone Marrow Stem Cells for ALS 5.3.8 ReNeuron – ReN001 is a Potential New Stroke Treatment Foetally-Sourced Cells Have Wide Potential Application 5.3.9 SanBio – SB623 for Ischaemic Stroke Future SanBio Programmes at Preclinical Stage in 2013
  8. 8. Contents 5.3.10 Pfizer – Collaborating on the London Project to Cure Blindness 5.3.11 Stemedica Cell Technologies- Ischaemic RPE Cells NSC Lines from Donated Brain Tissue 5.3.12 NeuroGeneration – Investigating Human NSCs 5.3.13 Companies at Preclinical Stage RhinoCyte – Stem Cells from the Nasal Passage to the Spinal Cord Theradigm - Neural Cells for CNS Disorders Biotime – Targeting AMD with ESCs 5.3.14 Other Companies with Technologies of Interest iPierian – Bringing iPSCs to the CNS Market? 5.4 Will CNS Stem Cells Fulfil Their Perceived Therapeutic Potential? 6. Stem Cell Therapeutics in Other Disease Areas, 2013-2023 6.1 A Diverse Range of Approvals will Drive Segment Growth to 2023 6.2 Other Indications for Stem Cell Treatment 6.2.1 Curing Orphan Diseases with HSCT 6.2.2 Genetically-Modified HSCs May Cure HIV, Among Other Diseases 6.2.3 Stem Cells for Osteogenesis 6.2.4 Several Stem Cell Osteobiologics Already Marketed 6.2.5 Perianal Fistula Treatment Indicates Stem Cells’ Potential in Tissue Repair 6.2.6 A New Option for Many Autoimmune Disorders 6.2.7 Can Stem Cells Cure Diabetes? 6.2.8 Mending the Liver 6.2.9 Long-Range Possibilities 6.3 Which Other Indications Will See the First Approvals? 6.3.1 Medipost - Cartistem, ‘World’s First Allogeneic Stem Cell Drug’ 6.3.2 Anterogen – Cupistem, Adipose-Derived Stem Cells for Anal Fistula 6.3.3 R&D Ranges Broadly Across Disease Areas 6.3.4 TiGenix – Phase 3 Data for Cx601 Expected In July 2014
  9. 9. Contents ‘First Ever Signal of Clinical Activity of a Cell Therapy in RA’ ChondroCelect Already on the Market 6.3.5 Osiris Take on Crohn's Disease - Another Possible Use for Prochymal Chondrogen: New Osteoarthritis Candidate 6.3.6 Mesoblast – Treating Back Pain and Disc Degeneration IV Precursor Cells Seeking Out New Indications 6.3.7 Athersys – Pfizer Collaboration in Ulcerative Colitis 6.3.8 Celgene Cellular Therapeutics – Targeting Multiple Inflammatory Conditions 6.3.9 AlloCure – AC607 for Anti-Inflammatory Effects 6.3.10 Gamida Cell – Potential Sickle Cell Anaemia Treatment Option 6.3.11 GlaxoSmithKline – GSK2696273 Stem Cell Gene Therapy 6.3.12 Alliancells Bioscience Corporation – Possible RA Treatment 6.3.13 ViroMed – Stem Cell Gene Therapy for Chronic Granulomatous Disease 6.3.14 S-Evans Biosciences – Menstrual Stem Cells for Hepatic Disease 6.3.15 Cytomedix – ALD-601 for Lysosomal Storage Diseases 6.3.16 Cellonis Biotechnology – Commercialising Research on Stem Cells in Diabetes 6.3.17 Other Companies in the Hunt for New Stem Cell Applications 6.4 Can We Expect A Clinical Breakthrough for Stem Cells in Other Therapeutic Areas by 2023? 7. Non-Therapeutic Applications of Stem Cell Technologies Segment, 2013-2023 7.1 Cell-Based Assays will Help Expand this Segment in 2013-2023 Period 7.2 A Diverse Range of Business Models Emerging 7.2.1 Stem Cell Banking: Growing Demand Worldwide 7.2.2 Stem Cell Supply and Processing: iPSCs the New Driver? 7.2.3 Stem Cell-Based Assays: Major Potential for Preclinical Screens 7.2.4 Research, Reagents, and Other Non-Therapeutic Stem Cell Revenue Streams 7.3 What Will the Main Growth Areas be for Non-Therapeutic Stem Cell Activities?
  10. 10. Contents 8. Qualitative Industry Analysis, 2013 8.1 Strengths: Stem Cells Are Finally Proving Their Worth 8.2 Weaknesses: Many Difficulties Still to Overcome 8.3 Opportunities: Stem Cells Have Unprecedented Clinical Potential 8.4 Threats: Regulatory and Commercial Instability Threatens the Field 8.5 Social Concerns: A Controversial Technology 8.6 Technological Developments: Stem Cell Science Still on the March 8.7 Economic Factors: Funding Gaps a Concern 8.8 Political Issues: State Attitudes Key to Research Environment 9. Research Interviews 9.1 Interview with Dr Antonio Lee, Associate Director Business Development, Medipost 9.1.1 Overview of Cartistem 9.1.2 Global Regulatory Variation in Cellular Cartilage Repair Field 9.1.3 Precedents and Competition for Cartistem 9.1.4 Cord Blood-Derived MSCs: Advantages and Challenges 9.1.5 Medipost’s Pipeline 9.2 Interview with Dr Alan Trounson, President, California Institute for Regenerative Medicine (CIRM) 9.2.1 Improved Funding in the Stem Cells Field 9.2.2 MSCs: Major Potential or Merely a Placeholder? 9.2.3 CIRM Investment Strategies 9.2.4 The Changing Regulatory Environment Worldwide 9.2.5 Key Trends for the Next Five to Ten Years 9.3 Interview with Dr Andras Nagy, Senior Scientist, Samuel Lunenfeld Research Institute, Mount Sinai Hospital 9.3.1 Overview of the Current State of the iPSC Field 9.3.2 Genomic Stability and Other Potential Issues for iPSCs
  11. 11. Contents 9.3.3 Potential for Treating and Modelling Diseases with iPSCs 9.3.4 Reprogramming Without Transgenes 9.3.5 Possibilities for Directly-Converted Cells 10. Conclusions 10.1 Stem Cell Technologies Will Begin to Break Through by 2017 10.2 Market to Record Double-Digit CAGR to 2023 10.3 Oncology and Non-Therapeutic Uses to Dominate To 2017 10.4 CNS and Cardiovascular Treatments to Gain Market Share by 2023 10.5 Strong R&D Pipeline to Drive Market Growth 10.6 Radical Clinical and Commercial Possibilities for Stem Cells in the Longer Term 10.7 Challenges for the Stem Cells Market 10.8 Concluding Remarks
  12. 12. Contents List of Tables Table 2.1 Stem Cells Market: Revenues ($m), AGR (%), CAGR (%), 2012-2017 Table 2.2 Stem Cells Market: Revenues ($m), AGR (%), CAGR (%), 2018-2023 Table 2.3 Stem Cells Market Breakdown by Segment: Revenues ($m), Market Share (%), 2012 Table 2.4 Stem Cells Market by Segment: Revenues ($m), AGR (%), CAGR (%), Market Shares (%), 2012-2017 Table 2.5 Stem Cells Market Breakdown by Segment: Revenues ($m), Market Share (%), 2017 Table 2.6 Stem Cells Market by Segment: Revenues ($m), AGR (%), CAGR (%), Market Shares (%), 2018-2023 Table 2.7 Stem Cells Market Breakdown by Segment: Revenues ($m), Market Share (%), 2023 Table 2.8 Potency and Source of Stem Cells Table 2.9 Germ Layers and Their Associated Types of Cells and Organs Table 2.10 Main Types of Stem Cells and Their Properties, 2013 Table 2.11 Sources of Stem Cells for Medical Applications, 2013 Table 3.1 Cancer Segment: Revenues ($m), AGR (%), CAGR (%), 2012-2017 Table 3.2 Cancer Segment: Revenues ($m), AGR (%), CAGR (%), 2018-2023 Table 3.3 Comparative Characteristics of Bone Marrow, Peripheral Blood and Cord Blood for HSCT, 2013 Table 3.4 Indications with Medicare Coverage for HSCT, 2013 Table 3.5 Malignancies and Other Haematological Diseases Treated with HSCT, 2013 Table 3.6 Selected Companies Developing Stem Cell Cancer Treatments, 2013 Table 4.1 Cardiovascular Segment: Revenues ($m), AGR (%), CAGR (%), 2012-2017 Table 4.2 Cardiovascular Segment: Revenues ($m), AGR (%), CAGR (%), 2018-2023 Table 4.3 Selected Companies Developing Stem Cell Cardiovascular Treatments, 2013 Table 5.1 CNS Segment: Revenues ($m), AGR (%), CAGR (%), 2012-2017 Table 5.2 CNS Segment: Revenues ($m), AGR (%), CAGR (%), 2018-2023 Table 5.3 Selected Companies Developing Stem Cell Cancer Treatments, 2013 Table 6.1 Other Therapeutic Areas Segment: Revenues ($m), AGR (%), CAGR (%), 2012-2017 Table 6.2 Other Therapeutic Areas Segment: Revenues ($m), AGR (%), CAGR (%), 2018-2023
  13. 13. Contents Table 6.3 Diseases Other than Cancers to be Treated with HSCT, 2013 Table 6.4 Processes Involved in Bone Healing Table 6.5 Selected Companies Developing Stem Cell Cancer Treatments, 2013 Table 7.1 Non-Therapeutic Applications Segment: Revenues ($m), AGR (%), CAGR (%), 20122017 Table 7.2 Non-Therapeutic Applications Segment: Revenues ($m), AGR (%), CAGR (%), 20182023 Table 7.3 Non-Therapeutic Applications of Stem Cells: Estimated Breakdown of Activities, Revenues ($m), Segment Share (%), 2012 Table 7.4 Selected US Stem Cell Banking Companies, 2013 Table 7.5 Selected Non-US Stem Cell Banking Companies, 2013 Table 7.6 Selected Stem Cell Supply and Processing Companies, 2013 Table 7.7 Selected Companies with Involvement in Stem Cell-Based Assays, 2013 Table 7.8 Selected Companies with Other Stem Cell-Related Activities, 2013 Table 8.1 World Stem Cells Market: Strengths and Weaknesses, 2013 Table 8.2 World Stem Cells Market: Opportunities and Threats, 2013 Table 8.3 World Stem Cells Market: STEP Analysis, 2013-2023 Table 10.1 Stem Cell Products: Approved and in Late-Stage Trials, 2013
  14. 14. Contents List of Figures Figure 2.1 World Biological Drugs Market: Total Revenues ($m), Figure 2.1 Stem Cells Market: Revenues ($m), 2012-2023 Figure 2.2 Stem Cells Market Breakdown by Segment: Revenues ($m), Market Share (%), 2012 Figure 2.3 Stem Cells Market Breakdown by Segment: Revenues ($m), Market Share (%), 2017 Figure 2.4 Stem Cells Market Breakdown by Segment: Revenues ($m), Market Share (%), 2023 Figure 2.5 Stem Cells Market Breakdown by Segment: Revenues ($m), 2012-2023 Figure 2.6 Stem Cells Market Breakdown by Segment: Revenues ($m), 2012, 2017, 2023 Figure 2.7 The Early History of Stem Cell Discovery and Development Figure 3.1 Cancer Segment: Revenues ($m), 2012-2023 Figure 3.2 Drivers and Restraints for Stem Cell Cancer Therapies, 2013 Figure 4.1 Cardiovascular Segment: Revenues ($m), 2012-2023 Figure 4.2 Drivers and Restraints for Stem Cell Cardiovascular Therapies, 2013 Figure 5.1 CNS Segment: Revenues ($m), 2012-2023 Figure 5.2 Drivers and Restraints for CNS Stem Cell Therapeutics, 2013 Figure 6.1 Other Therapeutic Areas Segment: Revenues ($m), 2012-2023 Figure 6.2 Drivers and Restraints for Stem Cell Therapeutics in Other Therapeutic Areas, 2013 Figure 7.1 Non-Therapeutic Applications Segment: Revenues ($m), 2012-2023 Figure 7.2 Non-Therapeutic Applications of Stem Cells: Estimated Breakdown of Activities, Revenues ($m), Segment Share (%), 2012 Figure 7.3 Drivers and Restraints for Non-Therapeutic Applications of Stem Cells, 2013 Figure 10.1 Stem Cell Technologies Market Revenues ($m), 2012-2017 Figure 10.2 Stem Cell Technologies Market Revenues ($m), 2018-2023 Figure 10.3 Stem Cell Market Segments: Revenues ($m), 2012-2017 Figure 10.4 Stem Cell Market Segments: Revenues ($m), 2018-2023
  15. 15. Contents Organisations Mentioned in the Report Abbott Laboratories 3DM Aastrom Biosciences Adistem Advanced Cell Technology Alder Biopharmaceuticals AllCells Alliancells Bioscience Corporation AlloCure Amgen Amorcyte Angel Biotechnology Angiostem Angiotech Anterogen Antria apceth Aristotle University of Thessaloniki Arteriocyte Athersys Auriga Ventures Axiogenesis Baxter Healthcare Beth Israel Deaconess Medical Center BioCardia BioE Biogenea-Cellgenea Bioheart Bio-Matrix
  16. 16. Contents BioMet Orthopedics Biotime Bluebird Bio BrainStorm Cell Therapeutics Bresagen California Institute for Regenerative Medicine (CIRM) California Stem Cells Capricor Cardio3 Bioscience Cardiogenesis Cedars-Sinai Heart Institute Celgene Cellular Therapeutics Cell Cure Neurosciences CellCentric Cellectis Cellerant Therapeutics Cellonis Biotechnology CellSeed Cellular Dynamics International Cephalon Cha General Hospital China Cord Blood Corporation ChondroCelect Clal Biotechnology Industries Cognate BioServices Cord Blood America Corestem Cryo-Cell International CryoCord Cryo-Save
  17. 17. Contents CXR Biosciences CyThera Cytomedix Cytori Therapeutics Denali Ventures Dendreon Duke University Elbit Imaging Eli Lilly EpiStem ES Cell International European Medicines Agency (EMA) FCB-Pharmicell FCB-Twelve Food and Drug Administration (US FDA) Forticell Bioscience Gamida Cell Garnet BioTherapeutics General BioTechnology Geron GlaxoSmithKline Global Stem GMP Unlimited Greenpeace Hadassah Medical Organization Harvard Stem Cell Institute Histostem HomeoTherapy Human Fertilisation and Embryology Authority (HFEA) Human Stem Cell Institute
  18. 18. Contents ImmunoCellular Therapeutics Immunovative Therapies InSCeption Biosciences Institute of Biomedical Research (Japan) IntelliCell Biosciences International Stemcell Services iPierian iPS Academia Japan Israel Healthcare Venture Israel Stem Cell Society Ixion Biotechnology Johnson & Johnson Lentigen Lifebank Cryogenics LifebankUSA MaRS Innovation Maxcyte MedCell Bioscience Medical Research Council (UK) Medipost Medistem Laboratories Merck & Co. Merck Millipore Mesoblast MetaCyte Business Lab Miltenyi Biotec Ministry of Food and Drug Safety, Korea National Marrow Donor Program (US) NeoStem Neuralstem
  19. 19. Contents NeuroGeneration NeuroSearch Northern Therapeutics Novartis NovoCell NsGene NuVasive OncoCyte Opexa Therapeutics OrbusNeich OrthoCyte Orthofix Osiris Therapeutics Pfizer Pharmaceutical Research and Manufacturers of America (PhRMA) Pharmicell Plasticell Pluristem Therapeutics Progenitor Cell Therapy Proneuron Biotechnologies ReCyte Therapeutics Regenerative Sciences Regeneron Regenetech ReNeuron Renovo Neural RhinoCyte Roslin Cellab Rutgers University Samuel Lunenfeld Research Institute, Mount Sinai Hospital
  20. 20. Contents SanBio Saneron CCEL Therapeutics Sangamo BioSciences Sanofi Schepens Institute Seoul National University S-Evans Biosciences Sirna Therapeutics Skye Orthobiologics South Korea Food and Drug Administration (KFDA) Spinesmith Partners Stem Cell Authority Stem Cell Sciences Stem Cell Therapeutics Stemcell Technologies StemCells StemCore StemCyte Stemedica Cell Technologies StemEx StemGen Stemina Biomarker Discovery Stempeutics Research Stemride International Swissmedic TCA Cellular Therapy Teva Pharmaceutical Industries Theradigm Theratechnologies TiGenix
  21. 21. Contents United States District Court for the District of Columbia United Therapeutics University College London University of California, San Diego University of Heidelberg University of Kyoto University of Minnesota University of Texas Health Science Center University of Wisconsin Alumni Research Foundation Vesta Therapeutics ViaCell ViaCyte ViroMed Laboratories Vitro Biopharma World Health Organization (WHO) Yonsei University
  22. 22. Stem Cell Technologies: World Market Outlook 2013-2023 Table 6.2 Other Therapeutic Areas Segment: Revenues ($m), AGR (%), CAGR (%), 2018-2023 2018 2019 2020 2021 2022 2023 Revenues ($m ) 992 1158 1436 1720 1953 2026 AGR (%) 18 17 24 20 14 4 CAGR (%, 2018-2023) 15 CAGR (%, 2012-2023) 23 Share of overall m arket (%) 8 8 9 10 10 10 Source: visiongain 2013 Figure 6.1 Other Therapeutic Areas Segment: Revenues ($m), 2012-2023 2500 Revenues ($m) 2000 1500 1000 500 0 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 Year Source: visiongain 2013 6.2 Other Indications for Stem Cell Treatment This segment includes all areas of therapeutic stem cell R&D outside the treatment of cancers, the heart and vasculature, and the CNS and eyes. This leaves a large range of disease targets. Some are more plausible than others, and only three are addressed by formally-approved stem cell therapies: various non-cancerous blood disorders treated by HSCT; osteobiologics; and tissue Page 101
  23. 23. Stem Cell Technologies: World Market Outlook 2013-2023 damage as a complication of Crohn’s disease. However, the potential for stem cells extends into many other indications. 6.2.1 Curing Orphan Diseases with HSCT The indications for HSCT outside oncology are broad and varied; for the most part, these are orphan diseases with small patient populations. Some are listed in Table 6.3. Table 6.3 Diseases Other than Cancers to be Treated with HSCT, 2013 Amegakaryocytic thrombocytopenia Red cell aplasia Angioimmunoblastic lymphadenopathy Aplastic anaemia Severe combined immunodeficiency syndrome - X1 Sickle cell anaemia Autoimmune cytopenia Sideroblastic anaemia Diamond Blackfan anaemia Sjogren's syndrome Fanconi's anaemia Thalassaemia major Haemophagocytic lymphohistiocytosis Waldenstrom's macroglobulinaemia X-linked hyper-immunoglobulin e-M syndrome X-linked lymphoproliferative syndrome Source: visiongain 2013 In rare cases, HSCT can cure these diseases: for example, HSCs from HLA-matched sibling donors can cure sickle cell disease, and, more problematically, beta-thalassemia. 6.2.2 Genetically-Modified HSCs May Cure HIV, Among Other Diseases One likely future extension of this principle will be autologous stem cell gene therapy: autologous HSCs will be modified to correct a genetic condition, then reimplanted. Studies have shown that Wiskott-Aldrich syndrome (an immunodeficiency disorder) can be cured by autologous HSCT if the HSCs are first virally modified to express the correct gene. Bluebird Bio has two clinical-stage programmes of this sort, addressing adrenoleukodystrophy and beta-thalassemia (they are not covered here since this is primarily a gene therapy platform; however, the distinction is somewhat subjective).This marks an important area of investigation for stem cell therapeutics. One of the possible extensions of this principle is a potential ‘host-targeting’ cure for HIV, whereby the CCR5 gene is disrupted in host blood cells, removing the receptor used by the virus to gain access to the Page 102