Translational Regenerative Medicine: Market Prospects 2014-2024

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1.1. Executive Summary Executive Summary
1.1 Overview of Findings 1.2 Structure of the Report 1.3 Research and Analysis Methods 1.4 Glossary of Terms in this Report

2.2. Introduction to Translational Regenerative Medicine Introduction to Translational Regenerative Medicine

2.1 What is Translational Regenerative Medicine? 2.2 Translational Regenerative Medicine: Market Segmentation 2.2.1 Stem Cell Therapy 2.2.1.1 Classifying Stem Cells by Potency 2.2.1.2 Categories of Stem Cells 2.2.1.3 Autologous versus Allogeneic Stem Cells: Towards Universal Stem Cell Products? 2.2.1.4 Stem Cell Therapeutic Applications 2.2.2 Tissue Engineering 2.2.3 Gene Therapy 2.2.3.1 Harnessing Infectivity: Viral Vectors in Gene Therapy 2.3 Other Areas of Regenerative Medicine 2.3.1 Small Molecules and Proteins Enhancing Regenerative Medicine 2.3.2 Cosmetic Applications of Regenerative Medicine 2.4 Regulation of Regenerative Medicine 2.4.1 Regulation of Regenerative Medicine Products in Europe 2.4.2 Regulation of Regenerative Medicine Products in the US 2.5 Phases of Clinical Trials

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2.6 Scope of this Report 2.6.1 Products Excluded from this Report

3.3. Regenerative Medicine: World Market 2014-2024 Regenerative Medicine: World Market 2014-2024
3.1 The World Regenerative Medicine Market in 2012 and 2013 3.2 Leading Regenerative Medicine Products 3.3 Leading Companies in the Regenerative Medicine Market 3.4 World Regenerative Medicine Market: Sales Forecast 2014-2024 3.4.1 Regenerative Medicine Market Segments: Sales Forecasts 2014-2024 3.4.2 Regenerative Medicine: CAGRs by Segment 2014-2024 3.4.3 Changing Market Shares by Segment 2014-2024 3.5 Stem Cell Therapies Lead the Regenerative Medicine Market 3.5.1 Stem Cells Therapy Market: Drivers and Restraints 2014-2024 3.6 Tissue Engineered Products Will Continue Strong Growth 3.6.1 Tissue Engineered Products Market: Drivers and Restraints 2014-2024 3.7 Gene Therapies Show High Growth Potential 3.7.1 Gene Therapy Market: Drivers and Restraints 2014-2024

4.4. Leading National Markets 2014-2024 Leading National Markets 2014-2024

4.1 Regional Breakdown of the World Regenerative Medicine Market 4.2 World Regenerative Medicine Market: Regional Forecast 2014-2024 4.2.1 Regenerative Medicine: CAGRs by Regional Market 2014-2024 4.2.2 How Will Regional Market Shares Change to 2024?

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4.3 The US Regenerative Medicine Market 2014-2024 4.3.1 US Regenerative Medicine Market: Drivers and Restraints 4.3.2 US Market Overview: The Leading Regenerative Medicine Market 4.3.2.1 Regulation of Regenerative Medicine in the US 4.3.2.2 Embryonic Stem Cell Research Remains Controversial 4.3.2.3 An End to Patents on Human Genes? 4.4 The European Regenerative Medicine Market 2014-2024 4.4.1 European Regenerative Medicine Market: Drivers and Restraints 4.4.2 European Market Overview: A Strong Science Base 4.4.2.1 On the Brink of a Unitary European Patent System 4.4.2.2 Regulation of Regenerative Medicine in Europe 4.4.2.3 European REMEDiE Project 4.4.2.4 A UK Perspective on the Regenerative Medicine Market 4.4.2.5 Developments in the Russian Regenerative Medicine Market 4.5 The Asia-Pacific Regenerative Medicine Market 2014-2024 4.5.1 Asia-Pacific Regenerative Medicine Market: Drivers and Restraints 4.5.2 Asia-Pacific Market Overview: Towards Rapid Commercialisation 4.5.2.1 Japan: Transforming the Regulatory Landscape 4.5.2.2 China: Encouraging Stem Cell R&D 4.5.2.3 India: An Emerging RM Industry 4.5.2.4 South Korea: First to Approve Stem Cell Treatments 4.5.2.5 Australia: Encouraging Translational Regenerative Medicine 4.6 The Regenerative Medicine Market in the Rest of the World 2014-2024 4.6.1 Brazil An Emerging Market in South America

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Leading Companies the Regenerative Medicine Market 5.5. Leading Companies inin the Regenerative Medicine Market
5.1 Aastrom Biosciences: Developing Cardiovascular Stem Cell Therapies 5.1.1 Aastrom Biosciences: Clinical Trials 5.1.2 Aastrom Biosciences: Products in Development 5.1.2.1 Ixmyelocel-T: Autologous Mesenchymal Stem Cell Therapy 5.2 Advanced Cell Technology: Stem Cells for Eye Diseases 5.2.1 Advanced Cell Technology: Clinical Trials 5.2.2 Advanced Cell Technology: Products in Development 5.2.2.1 Retinal Pigment Epithelial Cell Therapy 5.2.2.2 iPSC-derived Platelets 5.2.2.3 Hemangioblast Program 5.2.2.4 Mesenchymal Stem Cell Program 5.2.2.5 Other Products 5.3 Alphatec Spine: Bone Allgrafts 5.3.1 Alphatec Spine: Clinical Trials 5.3.2 Alphatec Spine: Products in Development 5.4 Anterogen: South Korean Stem Cell Company 5.4.1 Anterogen: Clinical Trials 5.4.2 Anterogen: Products in Development 5.5 Athersys: Developing Allogeneic Progenitor Cell Therapy 5.5.1 Athersys: Clinical Trials 5.5.2 Athersys: Products in Development 5.5.2.1 MultiStem: Allogeneic Multipotent Adult Progenitor Cells

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5.5.2.2 Serotonin Receptor Agonists 5.6 Avita Medical: Spray-On Skin Cell Therapy 5.6.1 Avita Medical: Clinical Trials 5.7 AxoGen: Nerve Regeneration Products 5.7.1 AxoGen: Clinical Trials 5.7.2 AxoGen: Products in Development 5.8 Genzyme (acquired by Sanofi): A Leader in Regenerative Medicine 5.8.1 Genzyme: Clinical Trials 5.8.2 Genzyme: Products in Development 5.9 Medipost: Cartilage Repair with Cartistem 5.9.1 Medipost: Clinical Trials 5.9.2 Medipost: Products in Development 5.10 Mesoblast: An Australian RM Leader 5.10.1 Mesoblast : Clinical Trials 5.10.2 Mesoblast: Products in Development 5.10.2.1 NeoFuse 5.10.2.2 Prochymal 5.10.2.3 Chondrogen 5.10.2.4 Mesenchymal Precursor Cells in Congestive Heart Failure 5.10.2.5 Other Pipeline Products 5.11 NuVasive: Spinal Surgery Bone Allografts 5.11.1 NuVasive: Clinical Trials 5.11.2 NuVasive: Products in Development 5.12 Organogenesis: a Tissue Engineering Pioneer

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5.12.1 Organogenesis: Clinical Trials 5.12.2 Organogenesis: Products in Development 5.13 Osiris Therapeutics: a Leading Stem Cell Company 5.13.1 Osiris Therapeutics: Clinical Trials 5.13.2 Osiris Therapeutics: Products in Development 5.14 Pharmicell: South Korean Stem Cell Leader 5.14.1 Pharmicell: Clinical Trials 5.14.2 Pharmicell: Products in Development 5.15 Regenerys: Manufacturing Skin Substitutes 5.15.1 Regenerys: Clinical Trials 5.15.2 Regenerys: Products in Development 5.16 Shire Regenerative Medicine: Entering the RM Market 5.16.1 Shire: Clinical Trials 5.16.2 Shire: Products in Development 5.16.2.1 Vascugel 5.17 TiGenix: European Cell Therapy Leader 5.17.1 TiGenix: Clinical Trials 5.17.2 TiGenix: Products in Development 5.18 UniQure: First Gene Therapy Approved in Europe 5.18.1 UniQure: Clinical Trials 5.18.2 UniQure: Products in Development

6.6. Stem Cell Therapy: Leading Applications 2014-2024 Stem Cell Therapy: Leading Applications 2014-2024
6.1 Stem Cell Therapy: Currently Available Therapies

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6.1.1 Haematopoietic Stem Cell Transplantation (HSCT) 6.1.2 Stem Cell Orthobiologics 6.1.2.1 Osteocel Plus (NuVasive): Sales Forecast 2014-2024 6.1.2.2 Trinity ELITE and Trinity Evolution (Orthofix): Sales Forecast 2014-2024 6.1.2.3 LiquidGen (Skye Orthobiologics) 6.1.2.4 AlloStem (AlloSource) 6.1.2.5 PureGen (AlphaTec Spine) 6.1.3 Other Approved Stem Cell Therapy Products 6.1.3.1 Prochymal (remestemcel-L, Osiris Therapeutics/Mesoblast): Sales Forecast 20142024 6.1.3.2 Hearticellgram-AMI (Pharmicell) 6.1.3.3 Cupistem (Anterogen) 6.1.3.4 Cartistem (Medipost) 6.2 Stem Cell Therapy Pipeline to 2024 6.2.1 Late-Stage Pipeline for Stem Cell Therapies 6.2.1.1 StemEx (carlecortemcel-L, Gamida Cell) 6.2.1.2 Mesenchymal Precursor Cell Therapies (Mesoblast) 6.2.1.3 Adult Autologous CD34+ Cells (Baxter) 6.2.1.4 MyoCell (Bioheart) 6.2.1.5 Cx601 (TiGenix) 6.2.1.6 Adipose-Derived Stem Cells (Cytori Therapeutics) 6.2.1.7 Stempeucel (Stempeutics Research) 6.2.1.8 C-Cure (Cardio3 BioSciences) 6.2.1.9 Other Late-Stage Stem Cell Therapies

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6.2.2 Diabetes and Stem Cell Therapy 6.2.2.1 Mesenchymal Precursor Cells (Mesoblast) 6.2.2.2 VC-01 (ViaCyte) 6.2.3 Cardiovascular Disease and Stem Cell Therapy 6.2.3.1 Endometrial Regenerative Cells (Medistem) 6.2.3.2 AMR-001 (NeoStem) 6.2.3.3 CAP-1002 (Capricor) 6.2.4 Stem Cell Therapy in Cancer 6.2.4.1 CLT-008 (Cellerant Therapeutics) 6.2.4.2 OncoCyte/BioTime Targeting Cancer with Gene and Stem Cell Therapy 6.2.5 Stem Cell Therapy for CNS Conditions 6.2.5.1 NurOwn (BrainStorm Cell Therapeutics) 6.2.5.2 NSI-566 (Neuralstem) 6.2.5.3 MultiStem (Athersys) 6.2.5.4 PDA-001 (cenplacel-L, Celgene) 6.2.5.5 ALD-401 (Cytomedix) 6.2.5.6 ReN001 (ReNeuron) 6.2.6 Stem Cell Therapy for Eye Diseases 6.2.6.1 MA09-hRPE (Advanced Cell Technology) 6.2.6.2 HuCNS-SC (StemCells, Inc.) 6.2.6.3 EC200 (EyeCyte) 6.2.6.4 Pfizers Collaboration with The London Project to Cure Blindness

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7.7. Tissue Engineering: Leading Applications 2014-2024 Tissue Engineering: Leading Applications 2014-2024
7.1 Tissue Engineering: Currently Available Therapies 7.1.1 Tissue Engineering for Wound Repair 7.1.1.1 Apligraf (Organogenesis): Sales Forecast 2014-2024 7.1.1.2 BioDfence and BioDfactor (BioD) 7.1.1.3 CureXcell (MacroCure) 7.1.1.4 Dermagraft (Shire): Sales Forecast 2014-2024 7.1.1.5 Epicel (Sanofi) 7.1.1.6 Grafix (Osiris Therapeutics) 7.1.1.7 MySkin and CyroSkin (Regenerys) 7.1.1.8 ReCell (Avita Medical): Sales Forecast 2014-2024 7.1.2 Tissue Engineering for Cartilage Repair: Autologous Chondrocyte Implantation 7.1.2.1 Carticel (Sanofi) 7.1.2.2 ChondroCelect (TiGenix) 7.1.2.3 DeNovo NT (Zimmer) 7.1.2.4 MACI (Sanofi) 7.1.3 Other Available Tissue Engineering Therapies 7.1.3.1 Autologous Cultured Cell and Tissue Products (Japan Tissue Engineering Co.) 7.1.3.2 Gintuit (Organogenesis) 7.1.3.3 LAVIV (azficel-T, Fibrocell Science) 7.1.3.4 Acellular Tissue Engineering Products 7.2 Tissue Engineering Pipeline to 2024 7.2.1 Autologous Chondrocyte Implantation Pipeline 7.2.2 Tissue Engineering for Skin

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7.2.3 Tissue Engineering for Liver Disease 7.2.3.1 Extracorporeal Bio-Artificial Liver Therapy (Vital Therapies) 7.2.4 Tissue Engineering for Cardiovascular Disease 7.2.5 Tissue Engineering for Eye Diseases 7.2.6 Prospects for Organ Transplantation

8.8. Gene Therapy: Leading Applications 2014-2024 Gene Therapy: Leading Applications 2014-2024
8.1 Gene Therapy: Currently Available Therapies 8.1.1 Gendicine (Benda Pharmaceutical) 8.1.2 Oncorine (Shanghai Sunway Biotech) 8.1.3 Neovasculgen (Human Stem Cells Institute) 8.1.4 Neovasculgen (HSCI): Sales Forecast 2014-2024 8.1.5 Glybera (UniQure) 8.2 Withdrawn From Sale: Rexin-G (Epeius Biotechnologies) 8.3 Gene Therapy Pipeline to 2024 8.3.1 Late-Stage Gene Therapy Pipeline 8.3.1.1 Collategene (beperminogene perplasmid, AnGes MG/Vical) 8.3.1.2 ProstAtak (AdV-tk, Advantagene) 8.3.1.3 Trinam (taberminogene vadenovec, Ark Therapeutics) 8.3.1.4 TVEC (talimogene laherparepvec, Amgen) 8.3.1.5 AAV2-hRPE65v2 (Spark Therapeutics) 8.3.1.6 CG0070 (Cold Genesys) 8.3.2 Gene Therapy for Diabetes 8.3.3 Gene Therapy for Cancer

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8.3.4 Gene Therapy for Neurological Diseases 8.3.5 Gene Therapy for Haemophilia B

Qualitative Analysis the Regenerative Medicine Market 20149.9. Qualitative Analysis ofof the Regenerative Medicine Market 2014-2024 2024
9.1 SWOT Analysis of the Regenerative Medicine Market 9.2 Strengths 9.2.1 Incremental Progress in Research 9.2.2 Regulatory Frameworks Improving 9.2.3 An Established Route to Market 9.2.4 Strong Support from National Governments 9.3 Weaknesses 9.3.1 Lack of Venture Capital 9.3.2 High Costs of RM Therapies 9.3.3 Commercialisation Requires New Business Models 9.3.4 Lack of Standardisation 9.4 Opportunities 9.4.1 Disruptive RM Therapies Can Create New Markets 9.4.2 3D Bioprinting and Other Technologies May Enable Rapid Scale-Up 9.4.3 Increasing Burden of Chronic Disease 9.4.4 Medical Tourism Can Drive the Market 9.5 Threats 9.5.1 Restraints on Reimbursement 9.5.2 Difficulty Establishing an Evidence Base

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9.5.3 Ethical Concerns Limiting R&D 9.5.4 Progress May be Slow 9.6 Porters Five Forces Analysis of the Regenerative Medicine Market 9.6.1 Rivalry Among Competitors [Medium] 9.6.2 Threat of New Entrants [Medium] 9.6.3 Power of Suppliers [Low] 9.6.4 Power of Buyers [Low] 9.6.5 Threat of Substitutes [High]

10. Research Interviews 10. Research Interviews

10.1 Interview with Dr Alan Trounson, President, California Institute for Regenerative Medicine (CIRM) 10.1.1 Improved Funding in the Stem Cells Field 10.1.2 MSCs: Major Potential or Merely a Placeholder? 10.1.3 CIRM Investment Strategies 10.1.4 The Changing Regulatory Environment Worldwide 10.1.5 Key Trends for the Next Five to Ten Years 10.2 Interview with Professor Francois Berthiaume, Associate Professor of Biomedical Engineering, Rutgers University, New Jersey 10.2.1 On the Regenerative Medicine Market 10.2.2 On the Future of Stem Cell Therapies 10.2.3 Challenges in the Tissue Engineering Market 10.2.4 Thoughts on Future Developments 10.3 Interview with Dr William Dolphin, CEO, Avita Medical, UK

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10.3.1 Future Prospects for the Regenerative Medicine Market 10.3.2 Is Regenerative Medicine Ready For Commercialisation? 10.3.3 On Emerging Regenerative Medicine Markets 10.3.4 Future Therapeutic Areas 10.3.5 Barriers to Developing Regenerative Medicine? 10.3.6 Thoughts on Future Developments

11. Conclusions 11. Conclusions

11.1 The World Regenerative Medicine Market in 2012 and 2013 11.1.1 Current Leading Regenerative Medicine Segments 11.1.2 Leading Regenerative Medicine Products 11.1.3 Leading Regenerative Medicine Companies 11.1.4 Leading Regional Markets 11.2 World Regenerative Medicine Market Forecast 2014-2024 11.3 Regenerative Medicine Late-Stage Pipeline 11.4 The Future of the Regenerative Medicine Market?

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List of Tables
Table 1.1 Currency Exchange Rates, 2012 Table 2.1 European Union Definitions of Regenerative Medicine Products, 2013 Table 2.2 Potency and Source of Stem Cells, 2013 Table 2.3 Germ Layers and Their Associated Cells, Tissues and Organs, 2013 Table 2.4 Stem Cell Categories, 2013 Table 2.5 Stem Cell Donor Terminology, 2013 Table 2.6 Clinical Trial Phases Table 3.1 World Regenerative Medicine Market: Revenues ($m) and Market Shares (%) by Segment, 2012 Table 3.2 Leading Regenerative Medicine Products: Revenues ($m), 2012 Table 3.3 Leading Companies in the Regenerative Medicine Market: Leading Products and Net Revenues ($m), 2012 Table 3.4 World Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 3.5 World Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%) by Segment, 2012-2018 Table 3.6 World Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%) by Segment, 2018-2024 Table 3.7 World Regenerative Medicine Market: CAGR (%) by Segment, 2012-2018, 2018-2024 and 2012-2024 Table 3.8 Market Shares (%) of the Regenerative Medicine Market by Segment, 2012, 2018 and 2024 Table 3.9 Stem Cell Market: Revenues ($m) and Market Shares (%) by Subsegment, 2012

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Table 3.10 Stem Cell Therapy Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 3.11 Tissue Engineered Products Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 3.12 Gene Therapy Market: Commercial-Stage Products, 2013 Table 3.13 Gene Therapy Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 3.14 Gene Therapy Market Forecast: Revenues ($m), AGR (%), CAGR (%) by Product, 2012-2024 Table 4.1 World Regenerative Medicine Market: Revenues ($m) and Market Shares (%) by Region, 2012 Table 4.2 World Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%) by Region, 2012-2018 Table 4.3 World Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%) by Region, 2018-2024 Table 4.4 World Regenerative Medicine Market: CAGR (%) by Region, 2012-2018, 2018-2024 and 2012-2024 Table 4.5 Market Shares (%) of the Regenerative Medicine Market by Region in 2012, 2018 and 2024 Table 4.6 US Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 4.7 European Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 4.8 Asia-Pacific Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 4.9 Rest of the World Regenerative Medicine Market Forecast: Revenues ($m), AGR (%), CAGR (%), 2012-2024 Table 5.1 Aastrom Biosciences: Regenerative Medicine Clinical Trials, 2013

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Table 5.2 Advanced Cell Technology: Regenerative Medicine Clinical Trials, 2013 Table 5.3 AlphaTec Spine: Regenerative Medicine Clinical Trials, 2013 Table 5.4 Anterogen: Regenerative Medicine Clinical Trials, 2013 Table 5.5 Athersys: Regenerative Medicine Clinical Trials, 2013 Table 5.6 Avita Medical: Regenerative Medicine Clinical Trials, 2013 Table 5.7 AxoGen: Regenerative Medicine Clinical Trials, 2013 Table 5.8 Genzyme: Products in the US Market, 2013 Table 5.9 Genzyme: Regenerative Medicine Clinical Trials, 2013 Table 5.10 Medipost: Regenerative Medicine Clinical Trials, 2013 Table 5.11 Mesoblast: Regenerative Medicine Clinical Trials, 2013 Table 5.12 NuVasive: Regenerative Medicine Clinical Trials, 2013 Table 5.13 Organogenesis: Regenerative Medicine Clinical Trials, 2013 Table 5.14 Osiris Therapeutics: Regenerative Medicine Clinical Trials, 2013 Table 5.15 Pharmicell: Regenerative Medicine Clinical Trials, 2013 Table 5.16 Regenerys: Regenerative Medicine Clinical Trials, 2013 Table 5.17 Shire: Regenerative Medicine Clinical Trials, 2013 Table 5.18 TiGenix: Regenerative Medicine Clinical Trials, 2013 Table 5.19 UniQure: Regenerative Medicine Clinical Trials, 2013 Table 6.1 Selected Currently Available Stem Cell Products, 2013 Table 6.2 FDA-Approved Cord Blood-Derived Stem Cell Products for HSCT, 2013 Table 6.3 Stages of Osteogenesis, 2013 Table 6.4 NuVasive: Osteocel Plus Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 6.5 Orthofix: Trinity ELITE Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 6.6 Mesoblast: Prochymal Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024

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Table 6.7 Stem Cell Therapy Pipeline: Therapies in Late-Stage Clinical Trials, 2013 Table 7.1 Selected Currently Available Tissue Engineering Products, 2013 Table 7.2 Organogenesis: Apligraf Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 7.3 Shire: Dermagraft Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 7.4 Avita Medical: ReCell Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 7.5 Vital Therapies: Extracorporeal Bio-Artificial Liver Therapy Clinical Trials, 2013 Table 8.1 Currently Available Gene Therapies, 2013 Table 8.2 Benda Pharmaceutical: Gendicine Clinical Trials, 2013 Table 8.3 Human Stem Cells Institute: Neovasculgen Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 8.4 UniQure: Glybera Revenue Forecast ($m), AGR (%), CAGR(%), 2012-2024 Table 8.5 Gene Therapy Pipeline: Therapies in Late-Stage Clinical Trials, 2013 Table 8.6 Advantagene: Gene Therapy Clinical Trials, 2013 Table 8.7 Amgen: TVEC Clinical Trials, 2013 Table 8.8 Spark Therapeutics: AAV2-hRPE65v2 Clinical Trials, 2013 Table 9.1 SWOT Analysis of the Regenerative Medicine Market, 2014-2024 Table 9.2 World 65+ Population Forecast: Size (m), AGR (%), CAGR (%), 2013-2024

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List of Figures
Figure 2.1 Brief History of Stem Cell Research, 1900-2000 Figure 2.2 Brief History of Tissue Engineering, 1900-2013 Figure 2.3 Examples of FDA-Approved Tissue Engineered Products Figure 2.4 Brief History of Gene Therapy, 1970-2013 Figure 3.1 World Regenerative Medicine Market: Market Shares (%) by Segment, 2012 Figure 3.2 Leading Regenerative Medicine Products: Market Shares (%), 2012 Figure 3.3 World Regenerative Medicine Market Forecast: Revenues ($m), 2012-2024 Figure 3.4 World Regenerative Medicine Market Forecast: Revenues ($m) by Segment, 20122024 Figure 3.5 World Regenerative Medicine Market Forecast: CAGR (%) by Segment, 2012-2018 Figure 3.6 World Regenerative Medicine Market Forecast: CAGR (%) by Segment, 2018-2024 Figure 3.7 World Regenerative Medicine Market Forecast: CAGR (%) by Segment, 2012-2024 Figure 3.8 World Regenerative Medicine Market: Market Shares (%) by Segment, 2018 Figure 3.9 World Regenerative Medicine Market: Market Shares (%) by Segment, 2024 Figure 3.10 Stem Cell Market: Market Shares (%) by Subsegment, 2012 Figure 3.11 Stem Cell Therapy Market Forecast: Revenues ($m), 2012-2024 Figure 3.12 Stem Cell Therapy Market: Drivers and Restraints, 2014-2024 Figure 3.13 Tissue Engineered Products Market Forecast: Revenues ($m), 2012-2024 Figure 3.14 Tissue Engineered Products Market: Drivers and Restraints, 2014-2024 Figure 3.15 Gene Therapy Market Forecast: Revenues ($m), 2012-2024 Figure 3.16 Gene Therapy Market: Market Shares (%) by Product, 2012 Figure 3.17 Gene Therapy Market: Market Shares (%) by Product, 2018

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Figure 3.18 Gene Therapy Market: Market Shares (%) by Product, 2024 Figure 3.19 Gene Therapy Market: Drivers and Restraints, 2014-2024 Figure 4.1 World Regenerative Medicine Market: Revenues ($m) by Region, 2012 Figure 4.2 World Regenerative Medicine Market: Market Shares (%) by Region, 2012 Figure 4.3 World Regenerative Medicine Market Forecast: Revenues ($m) by Region, 2012-2024 Figure 4.4 World Regenerative Medicine Market Forecast: CAGR (%) by Region, 2012-2018 Figure 4.5 World Regenerative Medicine Market Forecast: CAGR (%) by Region, 2018-2024 Figure 4.6 World Regenerative Medicine Market Forecast: CAGR (%) by Region, 2012-2024 Figure 4.7 World Regenerative Medicine Market: Market Shares (%) by Region, 2018 Figure 4.8 World Regenerative Medicine Market: Market Shares (%) by Region, 2024 Figure 4.9 US Regenerative Medicine Market Forecast: Revenues ($m), 2012-2024 Figure 4.10 US Regenerative Medicine Market: Drivers and Restraints, 2014-2024 Figure 4.11 European Regenerative Medicine Market Forecast: Revenues ($m), 2012-2024 Figure 4.12 European Regenerative Medicine Market: Drivers and Restraints, 2014-2024 Figure 4.13 Asia-Pacific Regenerative Medicine Market Forecast: Revenues ($m), 2012-2024 Figure 4.14 Asia-Pacific Regenerative Medicine Market: Drivers and Restraints, 2014-2024 Figure 4.15 Rest of the World Regenerative Medicine Market Forecast: Revenues ($m), 20122024 Figure 6.1 NuVasive: Osteocel Plus Revenue Forecast ($m), 2012-2024 Figure 6.2 Orthofix: Trinity ELITE Revenue Forecast ($m), 2012-2024 Figure 6.3 Mesoblast: Prochymal Revenue Forecast ($m), 2012-2024 Figure 7.1 Organogenesis: Apligraf Revenue Forecast ($m), 2012-2024 Figure 7.2 Shire: Dermagraft Revenue Forecast ($m), 2012-2024 Figure 7.3 Avita Medical: ReCell Revenue Forecast ($m), 2012-2024

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Figure 8.1 Human Stem Cells Institute: Neovasculgen Revenue Forecast ($m), 2012-2024 Figure 8.2 UniQure: Glybera Revenue Forecast ($m), 2012-2024 Figure 9.1 World 65+ Population Forecast: Size (m), 2013-2024 Figure 9.2 Porters Five Forces Analysis of the Regenerative Medicine Market, 2014-2024 Figure 11.1 World Regenerative Medicine Market: Revenues ($m) by Segment, 2012 Figure 11.2 World Regenerative Medicine Market: Revenues ($m) by Region, 2012 Figure 11.3 World Regenerative Medicine Market Forecast: Revenues ($m), 2012-2024

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Companies Listed
Aastrom Biosciences Adiposet Ltd (merged into Regenerys) Advanced BioHealing (acquired by Shire) Advanced Cell Technology (ACT) Advanced Tissue Sciences (ATS) Advantagene Allosource AlphaTec Spine Altrika (acquired by Regenerys) Amedica Amgen Amorcyte (acquired by NeoStem) Amorepacific Corporation Amsterdam Molecular Therapeutics (predecessor of UniQure) AnGes MG Angioblast Systems (acquired by Mesoblast) Anika Therapeutics AnorMED (acquired by Genzyme) Anterogen (subsidiary of Bukwang Pharm Co Ltd) Arcarios B.V. Ark Therapeutics Arteriocyte

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Athersys Avita Medical AxoGen AxoGen Corporation (merged into AxoGen) Baxter International Beike Biotechnology Benda Pharmaceutical BioD Bioheart BioMed Realty Trust, Inc. BioSante Pharmaceuticals BioTime BioTissue BioVex Group (acquired by Amgen) Blackstone Medical (acquired by Orthofix) Bluebird Bio BrainStorm Cell Therapeutics Inc. Bukwang Pharm Co Ltd California Stem Cell Capricor Cardio3 BioSciences CDI Celgene Cell Targeting

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CellCoTec Cellerant Therapeutics Cellerix (merged into TiGenix) Cephalon Ceregene CHA Biotech Chiesi Farmaceutici SpA Cipla Clinical Cell Culture (C3) (merged into Avita Medical) Clinimmune Labs co.don Cold Genesys Cook Biotech Incorporated CryoCenter Saint-Petersburg CytoMedix Cytori Therapeutics Daiichi Sankyo Dendreon DePuy Mitek (subsidiary of Johnson & Johnson) Epeius Biotechnologies EyeCyte Fibrocell Science Gamida Cell Ltd Gemabank

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Genpharm Genzyme (subsidiary of Sanofi) Geron Histogenics Humacyte Human Stem Cells Institute Insulete Inc Introgen Therapeutics ISTO Technologies Japan Tissue Engineering Co. (J-TEC) JCR Pharmaceuticals Co. Johnson & Johnson Kinetic Concepts Inc. (merging into KCI) LecTec Corporation (merged into AxoGen) LifeCell (merging into KCI) Lincoln Park Capital Lonza MacroCure Medipost Medistem (formerly Medistem Laboratories) Medtronic Mesoblast Mitsubishi Tanabe Musculoskeletal Transplant Foundation (MTF)

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Myriad Genetics Mytogen National Marrow Donor Program (NMDP) (US) NeoStem Neuralstem New York Blood Center Nippon Zoki Pharmaceutical NuVasive OncoCyte (subsidiary of BioTime) Organogenesis Organovo Oristem Orthofix Orthomimetics Ltd (acquired by TiGenix) Osiris Theraeutics Oxford BioMedica Parcell Laboratories Pervasis Therapeutics (acquired by Shire) Pfizer Pharmacells Pharmicell (formerly FCB-Pharmicell) Progenitor Cell Therapy (part of NeoStem) Protek Group Regenerys

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Reliance Life Sciences ReNeuron Reprobank Roslin Cellab RTI Surgical Rusnano Sangamo Sanofi Shanghai Qisheng Shanghai Sunway Biotech Shenzhen SiBiono GeneTech Shire Shire Regenerative Medicine (subsidiary of Shire) Skye Orthobiologics Sotex PharmFirm (subsidiary of Protek Group) Spark Therapeutics StemCells, Inc. Stempeutics Research Stratatech Corporation Systagenix (merging into KCI) TCA Cellular Therapy Tengion Teva Pharmaceutical Industries (Teva) TiGenix

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UniQure University of Colorado Cord Blood Bank ViaCyte (formerly NovoCell) Vical Vindon Scientific Visiomed Group (merged into Avita Medical) Vital Therapies, Inc. WKD Holding Oy Xcellerex Xenetic Biosciences Zentrales Knochenmarkspender-Register Deutschland (German National Registry of Blood Stem Cell Donors) (ZKRD) Zimmer

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Other Organisations Mentioned in This Report
Arthritis Research UK Associazione Infermieristica per lo Studio delle Lesioni Cutanee (Nursing Association for the Study of Cutaneous Wounds) (AISLeC) (Italy) Australian Regenerative Medicine Institute Australian Research Council Beijing Cancer Hospital Boston University British Heart Foundation California Institute of Regenerative Medicine (CIRM) Cambridge Stem Cell Biology Institute. Care Quality Commission (CQC) (UK) Catalan Institution for Research and Advanced Studies Cha General Hospital (Seoul, South Korea) Children's Hospital of Philadelphia China Food and Drug Administration (CFDA, formerly SFDA) Chinese Academy of Sciences Department of Biotechnology (India) Department of Health (UK) Duke University School of Medicine European Medicines Agency (EMA) European Patent Office Finnish Red Cross Blood Service Food and Drug Administration of the Philippines

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Harvard Medical School Haute Autorit de sant (HAS) (French National Authority for Health) Health Canada Heriot-Watt University Human Fertilisation and Embryology Authorityy (HFEA) (UK) Institute for Regenerative Medicine, Wake Forest University Baptist Medical Center Institute of Biomedical Research and Innovation Hospital (Kobe, Japan) International Society for Stem Cell Research Internet Archive Japan Patent Office Japanese Society for Regenerative Medicine Karolinska Institute King Fahad Medical City (Saudi Arabia) LifeSouth Community Blood Centers London Project to Cure Blindness Mayo Clinic Medical Research Council (UK) Medicines and Healthcare products Regulatory Agency (MHRA) (UK) Ministry of Economy, Trade and Industry (METI) (Japan) Ministry of Food and Drug Safety (MFDS, formerly KFDA) (South Korea) Ministry of Health (China) Ministry of Health, Labour and Welfare (MHLW) (Japan) Ministry of Science and Technology (China) Monash University

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Moorfields Eye Hospital National Health and Medical Research Council (Australia) National Institutes of Health (US) National Tissue Engineering Center (NTEC) (China) Northeast ALS consortium Pharmaceuticals and Medical Devices Agency (PMDA) (Japan) Riken Center for Developmental Biology Russian Ministry of Healthcare and Social Development Rutgers University Saudi Food and Drug Authority Scottish Centre for Regenerative Medicine Singapore General Hospital SSM Cardinal Glennon Children's Medical Center St. Jude's Children Research Hospital StemGen SUNY Upstate Medical University UCL Institute of Ophthalmology United Nations' Department of Economic and Social Affairs United States Food and Drug Administration (FDA) Universitat Autnoma de Barcelona University College London University of Edinburgh MRC Centre for Regenerative Medicine University of Michigan University of Modena Centre for Regenerative Medicine

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University of Wisconsin US Department of Defense VA Northern California Health Care System Weizmann Institute of Science (Rehovot, Israel) Wellcome Trust World Health Organization

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Translational Regenerative Medicine: Market Prospects 2014-2024

A large proportion of cell therapy trials being based in the US. The main restraints on growth are: Stringent restrictions on human embryonic stem cell research and difficulties with funding, despite recent improvements under the Obama administration; research involving the destruction of embryos remains a significant moral issue in the US Concerns among investors due to long and uncertain development times, in a market which is relatively dependent on venture capital to fund product development Falling R&D productivity and decreasing expenditure on scientific research in the US compared to Europe and the Asia-Pacific regions. Figure 4.10 summarises the drivers and restraints of the US regenerative medicine market from 2012 to 2024.

Figure 4.10 US Regenerative Medicine Market: Drivers and Restraints, 2014-2024

Drivers
Increasing demand for treatments for degenerative and chronic diseases High concentration of biotech and life-science funding available Large proportion of clinical trials based in the US Regulatory processes improving

Restraints
Restrictive environment for human embryonic stem cell research Investors deterred by long and uncertain product development Falling R&D productivity and research expenditure

Source: visiongain 2013

4.3.2 US Market Overview: The Leading Regenerative Medicine Market

As the leading market in regenerative medicine by virtue of the number of companies, clinical trials and product approvals and the funding available for companies and research, US dominance is set

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Table 5.17 Shire: Regenerative Medicine Clinical Trials, 2013
Start and Intervention End Dates Sep 1994 NCT01181440 Dermagraft Jan 1997 Dec 1998 NCT01181453 Dermagraft Mar 2000 Celaderm (frozen Jan 2007 NCT00399308 cultured epidermal Apr 2008 allograft) Jun 2009 NCT00909870 Dermagraft Aug 2011 Apr 2010 NCT01099215 PVS-10200 Oct 2012 May 2010 NCT01891760 Dermagraft Jun 2012 Oct 2011 - Dermagraft or Oasis NCT01450943 Oct 2014 versus standard care Identifier Indication Diabetic foot ulcer Diabetic foot ulcer Leg ulcer, varicose ulcer Venous leg ulcer Peripheral artery disease Venous leg ulcer Diabetic foot ulcer Phase III III I/II III I/II III (comparative efficacy) III II II

Dec 2012 NCT01749306 Dec 2013 Dermagraft (ABH001) Epidermolysis bullosa (terminated) Mar 2013 Vascugel (SRM003) Aug 2014 Mar 2013 NCT01806584 Vascugel (SRM003) Aug 2015 NCT01806545 Arteriovenous fistula Arteriovenous graft

Source: visiongain 2013; ClinicalTrials.gov

5.16.2 Shire: Products in Development 5.16.2.1 Vascugel

In April 2012, Shire acquired Pervasis Therapeutics, obtaining the rights to the companys cell therapy, Vascugel (SHP613, formerly SRM003). Vascugel consists of allogeneic human aortic endothelial cells cultured in a gelatine matrix (Gelfoam). The product is intended to facilitate the vascular access surgery that patients with end-stage kidney disease undergo so that they can receive haemodialysis. After this surgery, patients must wait several months on average for the access site to mature and even after maturation there is a high risk that vascular access will fail Shire claims that 60% fail within one year. Vascugel is placed outside the blood vessel at a vascular access site during surgery to promote maturation of the access site and reduce the risk of failure. The product has received Orphan Product and Fast Track designation from the FDA and Orphan Product designation from the European Medicines Agency. Phase II clinical trials of Vascugel are ongoing.

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Table 7.1 Selected Currently Available Tissue Engineering Products, 2013
Company Avita Medical BioD BioD FibroCell Science Japan Tissue Engineering Co. MacroCure Organogenesis Organogenesis Intervention ReCell BioDfactor BioDfence LAVIV (various tissue products) CureXcell Apligraf Gintuit Description Autologous cell treatment Human allograft derived from amniotic tissue, morselised Human allograft derived from amniotic tissue, patch Autologous fibroblasts Autologous cultured epidermis, cartilage and corneal epithelium Activated allogeneic white blood cells Living bi-layered skin substitute Indication Wound repair Wound repair Wound repair Nasolabial fold wrinkles (various) Wound repair Wound repair

Allogeneic cultured keratinocytes Regeneration of and fibroblasts in bovine collagen gum tissue Allograft cellular matrix Monolayer of undifferentiated allogeneic keratinocytes Cultured autologous epidermal substitute Autologous chondrocyte implantation Cultured epidermal autograft Matrix-induced autologous chondrocyte implantation Fibroblast-derived skin substitute Autologous chondrocyte implantation Juvenile cartilage allograft tissue Diabetic foot ulcers, burns, wound repair Burns, ulcers, nonhealing wounds Burns, ulcers, nonhealing wounds Cartilage repair Wound repair in severe burns Cartilage repair Diabetic foot ulcers Cartilage repair Repair of focal cartilage defects

Osiris Therapeutics

Grafix

Regenerys

CryoSkin

Regenerys Sanofi Sanofi Sanofi Shire TiGenix Zimmer

MySkin Carticel Epicel MACI Dermagraft ChondroCelect DeNovo NT

Source: visiongain 2013; company reports The gold standard of wound care remains the use of autologous skin grafts. This technique requires removal of healthy skin from one area of the patient to implant in another. The clear limitation of this treatment is the availability of healthy donor skin. For example, in burns victims, a large amount of donor skin is often required as burns can cover large areas.

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