MESENCHYMAL STEM CELLS ADVANCES & APPLICATIONS

Transcription

1 MESENCHYMAL STEM CELLS ADVANCES & APPLICATIONS TABLE OF CONTENTS 1. EXECUTIVE SUMMARY.p REPORT OVERVIEW..p Target Demographic 2.2 Key Findings 2.3 Statement of the Report 2.4 Purpose To Survey Recent MSC Advancements To Provide a Snapshot of the Global MSC Market To Assess Opportunities for Commercialization To Identify Major Market Players and Assess the Competitive Environment To Identify Existing and Emerging Trends To Identify Critical Opportunities and Threats within the MSC Market 3. RESEARCH METHODOLOGY..p Input Sources 3.2 Research & Analysis Methodologies 3.3 Criteria for Identiyfing MSC Market Leaders Size of Company Type of Product/Services Degree of Specialization Patent Positions Business Reputation Financial Strength Partnerships Certifications 4. MESENCHYMAL STEM CELL (MSC) DEFINITIONS & NOMENCLATURE...p Definition 4.2. Alternative Nomenclature Mesenchymal Stem Cell Marrow Stromal Cell (or Mesenchymal Stromal Cell) Multipotent Stromal Cell Colony-Forming Unit-fibroblasts 5. MSC CRITERIA & CHARACTERISTICS. p Discovery (FIGURE) Key Dates in the Discovery of Mesenchymal Stem Cells 5.2 Physical Assessment 5.3 Functional Attributes 5.4. Benefits of MSCs Relative to Other Stem Cells (FIGURE) Benefits of MSCs Relative to Other Stem Cells Well-Characterized Non-Controversial Diverse Differentiation Potential Ease of Growth in Culture Flexible Propagation Clinically Relevant Volumes Role as Regulatory Cells Delivery of Gene Products Favorable Immune Status Commercially Available Research Tools

2 6. MSC ISOLATION SOURCES.p Adult Sources (TABLE) Adult Sources for Mesenchymal Stem Cell (MSC) Isolation 6.2 Fetal Sources (TABLE) Fetal Sources for Mesenchymal Stem Cell (MSC) Isolation 7. COMMERCIAL INTEREST IN MSCs p.36 (TABLE) Differentiation Capacity of Mesenchymal Stem Cells 7.1 Cell Types Derived from MSCs 7.2 Therapeutic Potential of MSCs 8. INTRODUCTION TO REGENERATIVE MEDICINE (RM) INDUSTRY p Regenerative Medicine (RM) and Advanced Therapies Industry: A Brief Overview 8.2 Global Breakdown of Regenerative Medicine (RM) Industry by Region (TABLE) Global Breakdown of RM Companies by Region (FIGURE) Global Breakdown of RM Companies by Region 8.3 Breakdown of Global RM Companies by Type (TABLE) Global Breakdown of RM Companies by Type 8.4 Number of RM Clinical Trials by Phase of Development (TABLE) Number of RM Clinical Trials by Phase 8.5 Major Financial Events in the RM Industry (TABLE) Major Financial Events in RM Industry in 2014 (FIGURE) Major Financial Events in RM Industry in Major Partnerships and Acquisitions in RM Industry (TABLE) Major Partnerships and Acquisitions in RM Industry in 2014 (FIGURE) Major Partnerships and Acquisitions in RM Industry in Major Regulatory Milestones in RM Industry (TABLE) Major Regulatory Milestones in RM Industry in 2014 (FIGURE) Major Regulatory Milestones in RM Industry in Major Pharma and Biotech Companies Active in Advanced Therapies (TABLE) Major Pharma and Biotech Companies Active in Advanced Therapies (TABLE) Areas of Focus Among Large Pharmaceutical and Biotech Companies Involved with Advanced Therapies 8.9 Major Corporate Partnerships in RM Industry (TABLE) Major Corporate Partnerships in RM Industry in Anticipated Major RM Clinical Events (TABLE) Anticipated Major RM Clinical Events in INTRODUCTION TO THE STEM CELL THERAPY INDUSTRY.p Types of Stem Cells Used in Cell Therapy Human Embryonic Stem Cells Induced Pluripotent Stem Cells Mesenchymal Stem Cells (MSCs) Hematopoietic Stem Cells Adipose Stem Cells (ASCs) Neural Stem Cells (NSCs) (TABLE) Different Types of Stem Cells Used in Cell Therapy and their Characteristics 9.2 Types of Stem Cells Used in Cell Therapy, by Differentiation Potential (FIGURE) Five Types of Stem Cells, by Differentiation Potential Totipotent (or Omnipotent) Stem Cells Pluripotent Stem Cells Multipotent Stem Cells Oligopotent stem cells Unipotent Stem Cells

3 10. CELL THERAPY (CT) PRODUCTS: SUCCESSES AND FAILURES. p Cell Therapy Product Candidates in Late-Stage Clinical Development (TABLE) Mesenchymal Stem Cell Product Candidates in Late-Stage Clinical Development 10.2 Cell Therapy Products Being Developed for Cardiovascular Indication 10.3 Cell Therapy Products Being Developed for Other Indications 10.4 Major Commercially Available Cell Therapy Products 10.5 Cell Therapy Products Approved in South Korea (TABLE 13) Cell Therapy Products Approved in South Korea 10.6 Major Cell Therapies in Phase III Prochymal Mesenchymal Precursor Cell (MPC) MyoCell Ixmyelocel-T ELAD HP StemEx (Carlecortemcel-L) LaViv GSK Renew NT-501 (Renexus) Neocart DeNovo ET MACI 10.7 Clinical Trial Failures in Cell Therapy Failure of Phase II for MultiStem Failure of Cardio 133 Trial Termination of AlloCure s ACT-AKI Trial Failed Stroke Trial in India Failure of HeartiCellGram-AMI Trial Failure of Adipose MSCs in ARDS CD133+ in CLI Not Feasible Failed MSC Trial for Multiple Sclerosis Failure of Chinese Diabetes Trial Failed Efficacy Trial in AMI by Stempeucel Involvement of Multinational Companies (MNCs) in Cell Therapy Sector (TABLE) Major Companies and Products in Development within Cell Therapy Sector 11. THE MARKET FOR MESENCHYMAL STEM CELL THERAPIES..p Market Potential All Stem Cell Types Market Potential Mesenchymal Stem Cells 11.3 Global Market for Stem Cells by Disease Indication (TABLE) Global Market for Stem Cell Therapies by Disease Indication, Through 2021 (FIGURE) Global Market for Stem Cell Therapies by Disease Indication, Through RESEARCH AND THERAPEUTIC APPLICATIONS OF MSCs..p.78 (TABLE) MESENCHYMAL STEM CELL RESEARCH APPLICATIONS 12.1 Basic Research Applications MSC Morphology MSC Detection Differentiation Capacity Immuno-modulatory Effects 12.2 Cell Transplantation for Site-Specific Repair Osteochondral Repair Myogenic and Myocardial Repair Neural Repair Pancreatic Repair

4 12.3 Directed Differentiation of Mesenchymal Stem Cells Directed Differentiation of MSCs into Intra-Mesenchymal Lineages Directed Differentiation of MSCs into Extra-Mesenchymal Lineages 12.4 Drug Delivery Applications: Engineered MSCs as Carriers of Anti-Cancer Drugs 12.5 Drug Screening 12.6 Gene Transfer Studies 12.7 MSC-Mediated Inhibition of Immune Effector Cells 12.8 Supporting Hematopoietic Cell Applications 12.9 Use of 3-D Scaffolds for Tissue Engineering Topical Therapy and Use of MSCs in Wound Healing 13. MSC PRODUCT DEVELOPMENT VARIABLES ATTRIBUTES & ACQUISITION..p Biological Properties of MSCs Contributing to Therapeutic Effects MSCs Capacity to Migrate and Engraft MSCs Differentiation Potential (TABLE) Origin and Cell Types Derived from MSCs MSCs Potential to Secrete Multiple Bioactive Molecules (TABLE) Important Bioactive Molecules Secreted by MSCs and Their Functions MSCs Potential for Immunomodulatory Functions (TABLE) Immunomodulatory Effects of MSCs on Immune Cells Variable Immunophenotype of MSCs (TABLE) Cell Surface Antigen Expressions of MSCs Isolated from Different Sources 13.2 Factors Impacting MSCs Acquisition 13.3 Major Clinical Sources of MSCs (TABLE) Major Clinical Sources of MSCs 14. CLINICAL APPLICATIONS OF MESENCHYMAL STEM CELLS p Bone Marrow-Derived Mesenchymal Stem Cells (BMMSCs) BMMSCs and Kidney (TABLE) A Sample of Clinical Trials using BMMSCs for Kidney Diseases BMMSCs and Pancreas (TABLE) A Sample of Clinical Trials Using BMMSCs for Diabetes BMMSCs and Heart (TABLE) A Sample of Clinical Trials Using BMMSCs for Heart Disease BMMSCs and Liver (TABLE) A Sample of Clinical Trials Using BMMSCs for Liver Diseases BMMSCs and Brain (TABLE) Clinical and Experimental Therapies Using MSCs for Neural Diseases BMMSCs and Intestine (TABLE) A Sample of Clinical Trials Using BMMSCs for Intestinal Diseases BMMSCs and Bone (TABLE) A Sample of Clinical Trials Using BMMSCs for Musculoskeletal Diseases 14.2 Adipose-Derived Mesenchymal Stem Cells (ADSCs) Clinical Appplicationsof Adipose-Derived Mesenchymal Stem Cells (ADSCs) (TABLE) ADSCs Transplantation in Clinical Trials by Region/Country in Phase III Selected ADSC Secretomes and their Functions (TABLE) Selected ADSC Secretomes and their Functions 14.3 Mesenchymal Stem Cells (MSCs) Derived from Wharton s Jelly Clinical Application Properties of WJ-MSCs Immunoprivileged Status of WJ-MSCs Clinical Applications of WJ-MSCs (TABLE) A Summary of Clinical Trials Using WJ-MSCs 14.4 Umbilical Cord Blood-Derived MSCs (UCBMSCs) (TABLE) Clinical Trials Using UCBMSCs and Targeted Diseases 14.5 Dominance of MSCs in Cell Therapy Clinical Trials

5 15. MAJOR DISEASES ADDRESSED BY MSCs IN CURRENT CLINICAL TRIALS p.112 (TABLE) Clinical Trials Using MSCs by Disease Type, 2014 (FIGURE) Clinical Trials Using MSCs by Disease Type, MSCs for Treating Liver Diseases (TABLE) A Sample of Clinical Trials Using MSCs for Liver Disorders 15.2 MSCs for Neurodegenerative Diseases (TABLE): A Sample of Clinical Trials Using MSCs for Neurodegenerative Diseases 15.3 Clinical Trials Using MSCs for Autoimmune Diseases (TABLE) A Sample of Phase I and II Clinical Trials for Autoimmune Diseases as of Clinical Trials Using MSCs for Diabetes (TABLE) A Sample of Clinical Trials Using MSCs for Diabetes 15.5 MSCs for Cardiovascular Repair (TABLE) A Sample of Clinical Trials Using MSCs for Cardiovascular Diseases 15.6 MSCs for Musculoskeletal Diseases (TABLE) Clinical Trials Involving MSCs for Bone and Cartilage Repair Studies Using MSCs for Musculoskeletal Indications by Leading Countries (TABLE) Studies Using MSCs for Musculoskeletal Indications by Country Application of MSCs in Joint Diseases (TABLE) A Sample of Current Clinical Trials Using MSCs for Osteoarthritis (TABLE) A Sample of Clinical Trials Involving MSCs for Bone and Cartilage Repair 15.7 MSCs in Neuron/Spinal Cord Diseases (TABLE) Sample of Clinical Trials in Different Countries Using MSCs for Neuron and Spinal Cord Diseases 15.8 MSC Infusion for GvHD (TABLE) Clinical Experience of MSCs in GvHD Treatment 15.9 MSCs for Crohn s Disease (TABLE) A Sample of Clinical Trials Using MSCs for Crohn s Disease MSCs in Wound Healing (TABLE) Clinical Studies using MSCs for Wound Healing Increasing Focus on Immunological Properties of MSCs (TABLE) Number of MSC Scientific Publications about Immune/Inflammation by Year (FIGURE) Number of MSC Scientific Publications about Immune/Inflammation by Year Selected MSCs Late-Stage Pipeline Cell Therapies (TABLE) Selected MSCs Late-Stage Pipeline Cell Therapies 16. MARKET OPPORTUNITIES FOR BIOBANKING OF MESENCHYMAL STEM CELLS...p Types of Mesenchymal Stem Cell Biobanking (TABLE) Types of Mesenchymal Stem Cell Biobanking 16.2 Dental Stem Cell Banking (MSC Biobanking) (TABLE) Companies Offering Dental Stem Cell Storage (Diversified Cord Blood Banks and Specialty Companies) 16.3 Umbilical Cord Tissue (MSC Biobanking) Definitions Cord Tissue Market Overview Comparison of Cord Blood & Cord Tissue Storage (FIGURE) Comparison of Umbilical Cord Blood vs. Umbilical Cord Tissue Clinical Trials Investigating Cord Tissue Derived Mesenchymal Stem Cells (CT-MSCs) (TABLE) Medical Conditions Being Investigated for Potential to be Treated with Cord Tissue Derived Mesenchymal Stem Cells (CT-MSCs) Market Penetration of Cord Tissue within the U.S. (TABLE) U.S. Cord Blood Banks Offering Cord Tissue Storage in 2013 (TABLE) U.S. Cord Blood Banks Offering Cord Tissue Storage in Pricing of Cord Tissue Storage (Global Analysis) (TABLE) Pricing Structures for Cord Tissue Storage

6 16.4 Adipose-Derived MSCStorage (TABLE) Companies Offering Adipose-Derived MSC Storage (Both Diversified Cord Blood Banks and Specialty Companies) 16.5 Other Types of MSCBio-Banking: Placental Tissue, Menstrual Stem Cells, Peripheral Blood Stem Cells, Bone Marrow Stem Cells, and More 17. KEY MARKET METRICS FORF MSCs..p Global Breakdown of Mesenchymal Stem Cell Clinical Trials Breakdown of MSC Clinical Trials, By Phase FIGURE: Percentage of Mesenchymal Stem Cell (MSC) Clinical Trials by Phase Additional Resources Breakdown of MSC Clinical Trials, By Geography (FIGURE) Geographical Assessment of MSC Clinical Trial Activity (TABLE) Geographical Breakdown of MSC Clinical Trial Activity Types of Sponsoring/Collaborating Organizations Supporting MSC Clinical Trials (TABLE) Breakdown of MSC Clnical Trial Activity by Sponsoring/Collaborating Organization (FIGURE) Breakdown of MSC Clinical Trial Activity by Sponsor/Collaborating Organization Specific Industry Participants Supporting MSC Clinical Trial Activity (TABLE) Industry Participants Sponsoring/Collaborating on MSC Clinical Trials (TABLE) Top Industry Participants Sponsoring/Collaborating on MSC Clinical Trials (FIGURE) Top Industry Participants Sponsoring/Collaborating on MSC Clinical Trials Summary of Findings for Mesenchymal Stem Cell Clinical Trials 17.2 Patent Analysis Role of Patent Analysis for Competitive Intelligence MSC Patent Landscape Methodology of MSC Patent Analysis MSC Patents Filed in 2015 (TABLE) Mesenchymal Stem Cell Patents Filed in Scientific Publication Analysis (TABLE) Analysis of MSC Scientific Publications per Year (FIGURE) Analysis of MSC Scientific Publications per Year (FIGURE) 5-Year Projections for MSC Scientific Publication Rates ( ) 17.4 Grant Rate Analysis All-Time Analysis of MSC Grants (1997 to Present) (TABLE) Number of MSC Grant Rates, by Year (RePORTer Analysis) Year-over-Year Trends in MSC Grants (TABLE) Number of MSC Projects Funded by NIH Support by Year (TABLE) Amount of Funding for MSC Projects Supported by NIH by Year Geographical Assessment of MSC Grants (TABLE) Geographical Assessment of MSC Grants Funded by NIH Support Assessment of MSC Grants By Subject Matter (1997 to Present) (GRAPH) Breakdown of MSC Grants Funded by NIH Suppport by Subject Matter Dominant Principal Investigators (TABLE) Leading Principal Investigators for MSC Research Analysis of MSC Grants List of MSC Grants Funded by NIH-Support in 2015 Fiscal Year (TABLE) MSC Grants Funded by NIH-Support in 2015 Fiscal Year Assessment of MSC Grants by Subject Matter (2015) (TABLE) Breakdown of MSC Grants Funded by NIH-Support by Subject

7 18. SOCIAL ANALYTICS FOR MESENCHYMAL STEM CELLS p Marketing Cost Analysis: Search Term Mesenchymal Stem Cell Pay-Per-Click Cost for the Search Term Mesenchymal Stem Cell (FIGURE) Google Adwords Price-Per-Click for Term Mesenchymal Stem Cells (Plural Search Term; U.S. Only) (FIGURE) Google Adwords Price-Per-Click for Term Mesenchymal Stem Cell (Singular Search Term; U.S. Only) (FIGURE) Google Adwords Price-Per-Click for Term Mesenchymal Stem Cells (Plural Search Term; Geography: Worldwide) (FIGURE) Google Adwords Price-Per-Click for Term Mesenchymal Stem Cell (Singular Search Term; Geography: Worldwide) (TABLE) Google Adwords Pay-Per-Click (PPC) Costs for MSCs Analysis of Derivative Search Terms (FIGURE) Search Terms Containing or Relating to the Phrase Mesenchymal Stem Cell (Geography: USA Only) (FIGURE) Search Terms Containing or Relating to the Phrase Mesenchymal Stem Cell (Geography: Worldwide) 18.2 MSC Articles Receiving Most Social Media Shares over Past 12 Months MSC Specific Analysis (TABLE) Most Shared MSC Articles of the Past 12 Months Stem Cell Analysis (All Types) (TABLE) Most Shared Stem Cell Articles of the Past 12 Months (All Stem Cell Types) 18.3 Google Trend Data for MSCs Google Trend Data - Mesenchymal Stem Cells Only (FIGURE) Comparison of Google Trend Data for Mesenchymal Stem Cells (Plural) vs. Mesenchymal Stem Cell (Singular) over a 10-Year History (FIGURE) Google Trend Data for Mesenchymal Stem Cells over a 5-Year History (FIGURE) Google Trend Data Showing Regional Interest in Mesenchymal Stem Cells over a 5- Year History (GRAPH) Google Trend Data Showing City-by-City Interest in Mesenchymal Stem Cells over a 5-Year History (TABLE) Google Trend Data Showing Related Searches for Mesenchymal Stem Cells over a 5- Year History (FIGURE) Groundbreaking Years in Hematopoietic Stem Cell Transplant History Google Trend Data - All Types of Stem Cells (FIGURE) Google Trend Data for Stem Cells over a 10-Year History (FIGURE) Google Trend Data Comparison of All Types of Stem Cells over a 5-Year History (FIGURE) Google Trend Data Showing Regional Interest in Stem Cells over a 5-Year History (FIGURE) Google Trend Data Showing City-by-City Interest in Stem Cells over a 5-Year History (TABLE) Google Trend Data Showing Related Searches for Stem Cells over a 5-Year History 18.4 Twitter Hashtag Data (TABLE) Rates of Twitter Usage by Country Twitter Hashtag Data Mesechymal Specific Data (FIGURE) Top 10 Hashtags Related to #Mesenchymal on Twitter (TABLE) Top 10 Hashtags Related to #Mesenchymal on Twitter Twitter Hashtag Data Stem Cells (All Types) (FIGURE) Top 10 Hashtags Related to #StemCells on Twitter (TABLE) Top 10 Hashtags Related to #StemCells on Twitter Top Five MSC Influencers on Twitter (TABLE) Top 5 MSC Influencers on Twitter Top Five Stem Cell Influencers on Twitter (TABLE) Top Five Stem Cell Influencers on Twitter

8 19. SELECTED COMPANY PROFILES p American Type Culture Collection Inc. (ATCC) 19.2 Anterogen Co., Ltd Cupistem Injection Queencell 19.3 Apceth GmbH & Co. KG Apceth s Research Areas 19.4 BioCardia Inc. (TABLE) BioCardia s Product Pipeline Overview 19.5 BioRestorative Therapies Inc brtxdisc ThermoStem 19.6 Bone Therapeutics SA PREOB ALLOB (TABLE) Bone Therapeutics Product Pipeline 19.7 BrainStorm Cell Therapeutics Inc NurOwn 19.8 CellGenix Technologie Transfer GmbH 19.9 Celprogen Inc CellTherapies P/L Services Product Cesca Therapeutics Inc Surgwerks Cellwerks AutoXress (AXP) MarrowXpress (MXP) Res-Q BMC Cyagen Biosciences Inc Mesenchymal Stem Cells from Cyagen (TABLE) Cyagen s Mesenchymal Cells Adipose-Derived Mesenchymal Stem Cells from Cyagen (TABLE) Cyagen s Adipose-Derived MSCs Mesenchymal Stem Cells with GFP from Cyagen (TABLE) Gyagen s MSCs with GFP Adipose-Derived Mesenchymal Cells with GFP from Cyagen (TABLE) Adipose-Derived Mesenchymal Cells with GFP Stem Cell Culture Media from Cyagen (TABLE) Cyagen s Stem Cell Culture Media Stem Cell Differentiation Media from Cyagen (TABLE) Cyagen s Stem Cell Differentiation Media Primary Cells from Cyagen (TABLE) Primary Cells from Cyagen Cyagen s Cryopreservation Media (TABLE) Cyagen s Cryopreservation Media Cyagen s Primary Cell Culture Media (TABLE) Cyagen s Primary Cell Culture Media General Cell Culture Supplements and Specialty Reagents from Cyagen (TABLE) General Cell Culture Supplements and Specialty Reagents from Cyagen

9 19.13 Cynata Therapeutics Ltd Cymerus Platform Technology Cytori Therapeutics Inc Clinical Trial for Scleroderma Clinical Trials for Osteoarthritis Escape Therapeutics (TABLE) Escape Therapeutics Pipeline Products Genlantis Kite Pharma Inc Engineered Autologous Cell Therapy (eact) DC-Ad GM-CAIX (TABLE) Kite Pharma s Pipeline and Clinical Trials Life Technologies Corporation Lonza Group Ltd Selected Products Medipost Co. Ltd Cartistem Neurostem Pneumostem Mesoblast Ltd Mesoblast s Product Pipeline Overview (TABLE) Mesoblast s Product Pipeline Overview NuVasive Inc Osteocel Ocata Therapeutics Inc. (TABLE) Ocata s Pipeline of Therapeutic Programs Organogenesis Inc Apligraf Dermagraft Orthofix International N.V Trinity Elite Trinity Evolution Osiris Therapeutics Inc Grafix OvationOS Cartiform Pluristem Therapeutics Inc PLX Cells PromoCell Regeneus Ltd Products (TABLE) Regeneus Product Pipeline ScienCell Research Laboratories STEMCELL Technologies Inc Stemedica Cell Technologies Inc Stemedyne MSC Stemedyne NSC Stemedyne RPE Stempeutics Research Pvt. Ltd Stempeucel Stempeutron Stempeucare (TABLE) Stempeucel in Clinical Trials by Indication

10 19.34 TiGenix N.V Cx Cx Cx Vericel Corporation (TABLE) Vericel Corporation s Product Portfolio (TABLE) Summary Table for the Companies and Their Product Types