Translational regenerative medicine 2014

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Translational Regenerative Medicine: Market Prospects 2014-2024

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Contents 1. 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. 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

1. Executive Summary

2. Introduction to Translational Regenerative Medicine

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Contents 2.6 Scope of this Report

2.6.1 Products Excluded from this Report

3. 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. 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?

3. Regenerative Medicine: World Market 2014-2024

4. Leading National Markets 2014-2024

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Contents 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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Contents

5. Leading Companies in 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

5. Leading Companies in the Regenerative Medicine Market

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Contents 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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Contents 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. Stem Cell Therapy: Leading Applications 2014-2024

6.1 Stem Cell Therapy: Currently Available Therapies

6. Stem Cell Therapy: Leading Applications 2014-2024

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Contents 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 2014-

2024

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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Contents 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 Pfizer’s Collaboration with The London Project to Cure Blindness

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Contents 7. 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

7. Tissue Engineering: Leading Applications 2014-2024

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Contents 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. 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

8. Gene Therapy: Leading Applications 2014-2024

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Contents 8.3.4 Gene Therapy for Neurological Diseases

8.3.5 Gene Therapy for Haemophilia B

9. Qualitative Analysis of the Regenerative Medicine Market 2014-2024

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

9. Qualitative Analysis of the Regenerative Medicine Market 2014-

2024

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Contents 9.5.3 Ethical Concerns Limiting R&D

9.5.4 Progress May be Slow

9.6 Porter’s 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

10.1 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

10. Research Interviews

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Contents 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

11.1 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?

11. Conclusions

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Contents

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


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

List of Tables

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Contents 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


by Region, 2012-2018


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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Contents 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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Contents 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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Contents

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, 2012-

2024

Figure 3.5 World Regenerative Medicine Market Forecast: CAGR (%) by Segment, 2012-2018





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

List of Figures

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Contents 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 4.9 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), 2012-

2024

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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Contents 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 Porter’s 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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Contents

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

Companies Listed

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Contents 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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Contents 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

www.visiongain.com

Contents 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)

www.visiongain.com

Contents 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

www.visiongain.com

Contents 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

www.visiongain.com

Contents 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

www.visiongain.com

Contents

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

Other Organisations Mentioned in This Report

www.visiongain.com

Contents Harvard Medical School

Haute Autorité de santé (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

www.visiongain.com

Contents 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 Autònoma de Barcelona

University College London

University of Edinburgh MRC Centre for Regenerative Medicine

University of Michigan

University of Modena Centre for Regenerative Medicine

www.visiongain.com

Contents University of Wisconsin

US Department of Defense

VA Northern California Health Care System

Weizmann Institute of Science (Rehovot, Israel)

Wellcome Trust

World Health Organization

www.visiongain.com Page 86

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

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

Source: visiongain 2013

• 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

Drivers Restraints

• Restrictive environment for human embryonic stem cell research

• Investors deterred by long and uncertain product development

• Falling R&D productivity and research expenditure


Translational Regenerative Medicine: Market Prospects 2014-2024 Table 5.17 Shire: Regenerative Medicine Clinical Trials, 2013

IdentifierStart and End Dates Intervention Indication Phase

NCT01181440 Sep 1994 - Jan 1997

Dermagraft Diabetic foot ulcer III

NCT01181453 Dec 1998 - Mar 2000

Dermagraft Diabetic foot ulcer III

NCT00399308Jan 2007 - Apr 2008

Celaderm (frozen cultured epidermal allograft)

Leg ulcer, varicose ulcer I/II

NCT00909870 Jun 2009 - Aug 2011

Dermagraft Venous leg ulcer III

NCT01099215 Apr 2010 - Oct 2012

PVS-10200 Peripheral artery disease I/II

NCT01891760May 2010 - Jun 2012 Dermagraft Venous leg ulcer III

NCT01450943 Oct 2011 - Oct 2014

Dermagraft or Oasis versus standard care

Diabetic foot ulcer (comparative efficacy)

NCT01749306Dec 2012 - Dec 2013 (terminated)

Dermagraft (ABH001) Epidermolysis bullosa III

NCT01806545 Mar 2013 - Aug 2014

Vascugel (SRM003) Arteriovenous fistula II

NCT01806584 Mar 2013 - Aug 2015

Vascugel (SRM003) Arteriovenous graft II

5.16.2 Shire: Products in Development

5.16.2.1 Vascugel

In April 2012, Shire acquired Pervasis Therapeutics, obtaining the rights to the company’s 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.

Source: visiongain 2013; ClinicalTrials.gov


Translational Regenerative Medicine: Market Prospects 2014-2024 Table 7.1 Selected Currently Available Tissue Engineering Products, 2013

Company Intervention Description IndicationAvita Medical ReCell Autologous cell treatment Wound repair

BioD BioDfactor Human allograft derived from amniotic tissue, morselised

Wound repair

BioD BioDfence Human allograft derived from amniotic tissue, patch

Wound repair

FibroCell Science LAVIV Autologous fibroblasts Nasolabial fold wrinkles

Japan Tissue Engineering Co.

(various tissue products)

Autologous cultured epidermis, cartilage and corneal epithelium

(various)

MacroCure CureXcell Activated allogeneic white blood cells

Wound repair

Organogenesis Apligraf Living bi-layered skin substitute Wound repair

Organogenesis GintuitAllogeneic cultured keratinocytes and fibroblasts in bovine collagen

Regeneration of gum tissue

Osiris Therapeutics Grafix Allograft cellular matrix Diabetic foot ulcers, burns, wound repair

Regenerys CryoSkinMonolayer of undifferentiated allogeneic keratinocytes

Burns, ulcers, non-healing wounds

Regenerys MySkinCultured autologous epidermal substitute

Burns, ulcers, non-healing wounds

Sanofi Carticel Autologous chondrocyte implantation

Cartilage repair

Sanofi Epicel Cultured epidermal autograft Wound repair in severe burns

Sanofi MACI Matrix-induced autologous chondrocyte implantation

Cartilage repair

Shire Dermagraft Fibroblast-derived skin substitute Diabetic foot ulcers

TiGenix ChondroCelect Autologous chondrocyte implantation

Cartilage repair

Zimmer DeNovo NT Juvenile cartilage allograft tissue Repair of focal cartilage defects

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.

Source: visiongain 2013; company reports

Translational regenerative medicine 2014

Technology

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