stem cell technologies 2013-2023

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Stem Cell Technologies: World Market Outlook 2013-2023

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Contents

1.1 Overview of Findings

1.2 Chapters in the Report

1.3 Research and Analysis Methods

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

2.4.1.1 ESC Controversy Now Settled?

2.4.1.2 FDA Vs Regenerative Sciences

2.4.2 Japan: Liberal on hESCs

2.4.2.1 First-in-Human iPSC Trial Announced in 2013

2.4.3 Europe: No Consistent Position Across the Continent

2.4.3.1 The Brüstle Ruling: Dead and Buried?

2.4.3.2 The UK Leads in European Stem Cells R&D

2.4.4 Rest of the World

1. Executive Summary

2. Overview of the Stem Cells Market, 2013-2023

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Contents 2.4.4.1 South Korea: Leading the Way in MSC Approvals

2.4.4.2 China: Liberal Laws on Stem Cell Research

2.4.4.3 Israel – Long-Term Research Activity

2.4.4.4 ‘Stem Cell Tourism’

2.8.1 The Vaccines Market

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

3.3.1.1 Osiris Leads the Pack in Stem Cell Therapeutics

3.3.1.2 Using MSCs for Immunosuppression

3.3.1.3 Positive Results in Acute Paediatric GvHD

3.3.1.4 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?

3.3.3.1 Survival at 100 Days Improved by StemEx in Latest Trial

3.3.3.2 FDA Remains Unconvinced

3.3.3.3 NiCord Heads Up a Pipeline of New Cell Therapy Products

3. Stem Cell Cancer Therapeutics Segment 2013-2023

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Contents 3.3.3.4 NiCord Promising So Far

3.3.4 Mesoblast – Using Mesenchymal Precursors to Enrich Cord Blood

3.3.4.1 Phase 3 Trial Measuring 100-Day Survival Rates and Engraftment Efficiency

3.3.4.2 A Competitor for Gamida Cell?

3.3.5 Novartis Pharmaceuticals – Cord Blood Transplant Candidate

3.3.5.1 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?

4.1 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?

4. Stem Cell Cardiovascular Therapeutics Segment, 2013-2023

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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

4.3.1.1 Performance So Far

4.3.1.2 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

4.3.3.1 Exercise Tolerance and Angina Frequency Reduced

4.3.3.2 Wider Potential for this Treatment

4.3.4 Cardio3 Bioscience – Cardiopoiesis Platform for Heart Regeneration

4.3.4.1 Positive Phase 2 Data

4.3.4.2 Phase 3 Trial Begins Amid Controversy

4.3.5 Bioheart – MyoCell in new MIRROR Trial

4.3.5.1 Muscle Cells the Basis of MyoCell

4.3.5.2 Seeking Expanded Access for Compassionate Use

4.3.6 NeoStem – Diversified Outfit with One of the Leading Candidates

4.3.6.1 AMR-001 to Treat Acute Myocardial Infarction

4.3.6.2 NeoStem Head-to-Head with Baxter in the HSC Cardiovascular Treatment Space

4.3.7 Aastrom Biosciences: Ixmyelocel-T for Cardiovascular Disease

4.3.7.1 A Complex Multicellular Mixture

4.3.7.2 Company Now Focusing on DCM

4.3.8 Athersys – MultiStem is a Potential Cardiovascular Treatment

4.3.8.1 Addressing Ischaemic Stroke

4.3.9 CytoMedix – Three Clinical Candidates in the Cardiovascular Segment

4.3.9.1 ALD-401 – Repairing Stroke Damage?

4.3.9.2 ALD-301 – Entering Phase 2 in PAD Patients

4.3.9.3 ALD-201 – Signs of Efficacy in Phase 1 Heart Failure Trial

4.3.10 TCA Cellular Therapy – Mesendo Autologous Treatments

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Contents 4.3.10.1 Preparing for Phase 3 with CLI Treatment

4.3.11 Medistem – The ‘Universal Donor’ Cell

4.3.11.1 Trial in CLI the First Clinical Test for ERC

4.3.11.2 Scalability a Major Advantage for Medistem

4.3.12 Stemedica Cell Technologies – Ischaemic Tolerant Stem Cell Platform

4.3.12.1 Two Trials in Progress for Allogeneic MSCs

4.3.13 Mesoblast – Cell Therapy for Myocardial Infarction

4.3.13.1 Teva Collaborating on Cardiovascular Stem Cell Therapy R&D

4.3.14 Pluristem – Cell Therapy for Myocardial Infarction

4.3.14.1 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

4.3.21.1 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?

5.1 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

5. Stem Cell CNS Therapeutics Segment, 2013-2023

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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

5.3.1.1 Validating the NurOwn Approach

5.3.1.2 ALS Trial Ongoing

5.3.2 Neuralstem – Neural Stem Cells for Synaptic Repair and Neuroprotection

5.3.2.1 Phase 2 Trial in ALS Enrolling by Invitation

5.3.2.2 Chronic Spinal Cord Injury Treatment to Follow

5.3.3 Mesoblast – A New Option in Neovascular AMD?

5.3.3.1 CNS Stem Cells from Dental Pulp

5.3.4 Advanced Cell Technology – Leading the Field in ESC Clinical Trials

5.3.4.1 Continued Progress in Stargardt’s Macular Dystrophy in 2013

5.3.4.2 Early Signals of Efficacy?

5.3.5 StemCells – The Leading Player in the NSC Space?

5.3.5.1 HuCNS-SC Product Proving Itself in Rare Indications

5.3.5.2 Moving on to Spinal Cord Injury and AMD

5.3.5.3 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

5.3.8.1 Foetally-Sourced Cells Have Wide Potential Application

5.3.9 SanBio – SB623 for Ischaemic Stroke

5.3.9.1 Future SanBio Programmes at Preclinical Stage in 2013

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Contents 5.3.10 Pfizer – Collaborating on the London Project to Cure Blindness

5.3.11 Stemedica Cell Technologies- Ischaemic RPE Cells

5.3.11.1 NSC Lines from Donated Brain Tissue

5.3.12 NeuroGeneration – Investigating Human NSCs

5.3.13 Companies at Preclinical Stage

5.3.13.1 RhinoCyte – Stem Cells from the Nasal Passage to the Spinal Cord

5.3.13.2 Theradigm - Neural Cells for CNS Disorders

5.3.13.3 Biotime – Targeting AMD with ESCs

5.3.14 Other Companies with Technologies of Interest

5.3.14.1 iPierian – Bringing iPSCs to the CNS Market?

5.4 Will CNS Stem Cells Fulfil Their Perceived Therapeutic Potential?

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

6. Stem Cell Therapeutics in Other Disease Areas, 2013-2023

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Contents 6.3.4.1 ‘First Ever Signal of Clinical Activity of a Cell Therapy in RA’

6.3.4.2 ChondroCelect Already on the Market

6.3.5 Osiris Take on Crohn's Disease - Another Possible Use for Prochymal

6.3.5.1 Chondrogen: New Osteoarthritis Candidate

6.3.6 Mesoblast – Treating Back Pain and Disc Degeneration

6.3.6.1 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?

7.1 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?

7. Non-Therapeutic Applications of Stem Cell Technologies Segment,

2013-2023

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Contents

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

9.1 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

8. Qualitative Industry Analysis, 2013

9. Research Interviews

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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

10.1 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

10. Conclusions

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Contents

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 2.6 Stem Cells Market by Segment: Revenues ($m), AGR (%), CAGR (%), Market Shares

(%), 2018-2023


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 6.1 Other Therapeutic Areas Segment: Revenues ($m), AGR (%), CAGR (%), 2012-2017


List of Tables

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Contents Table 6.3 Diseases Other than Cancers to be Treated with HSCT, 2013

Table 6.4 Processes Involved in Bone Healing


Table 7.1 Non-Therapeutic Applications Segment: Revenues ($m), AGR (%), CAGR (%), 2012-

2017

Table 7.2 Non-Therapeutic Applications Segment: Revenues ($m), AGR (%), CAGR (%), 2018-

2023

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

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Contents

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 2.5 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

List of Figures

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Contents

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

Organisations Mentioned in the Report

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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

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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

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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

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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

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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

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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

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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

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) 23Share of overall

market (%)8 8 9 10 10 10

Source: visiongain 2013


Figure 6.1 Other Therapeutic Areas Segment: Revenues ($m), 2012-2023

0

500

1000

1500

2000

2500

2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023

Rev

enue

s ($

m)

Year


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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.

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

Amegakaryocytic thrombocytopenia Red cell aplasia

Angioimmunoblastic lymphadenopathy Severe combined immunodeficiency syndrome - X1

Aplastic anaemia 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

Table 6.3 Diseases Other than Cancers to be Treated with HSCT, 2013
