Cell Therapy Commercialization Applying Stem Cell and Related Strategies

Table of Contents

Chapter 1: Executive Summary 1--1

Chapter 2: Introduction and Background to Cell Therapy 2--1

• Introduction 2--1
• Cells for Therapeutic Applications 2--2
• Epithelial Tissue 2--3
• Connective Tissue 2--3
• Muscle Tissue 2--4
• Nervous Tissue 2--5
• Basic and Advanced Cell-Based Therapy Approaches 2--5
• Single Cell Therapy 2--5
• Multiple Cell Therapy Strategies 2--5
• Cell Isolation Techniques 2--6
• Primary Cell Isolation Protocol 2--6
• Mononuclear Cell Isolation 2--6
• Immunologic Procedures for Cell Isolation 2--7
• Developments in Cell Selection 2--7
• Ex vivo cell expansion 2--8
• Peripheral Blood Stem Cell Harvesting 2--9
• Dendritic Cells 2--9
• Background 2--9
• Dendritic Cell-Based Cancer Therapy 2--10
• Dendritic Cells in Cancer Therapeutics 2--11
• Stem Cells 2--11
• Background 2--11
• Stem Cells: Classification and Characterization 2--12
• Embryonic Stem Cells: Classification and Characterization 2--14
• Adult Stem Cells: Classification and Characterization 2--14
• Stem Cell Therapy: Basic and Advanced Principles 2--18
• Embryonic Stem Cells for Therapeutic Transplants 2--19
• Advantages and Disadvantages of Embryonic Stem Cells 2--19
• Potential Uses of Human Embryonic Stem Cells 2--20
• Genetic Manipulation of Human Embryonic Stem Cells 2--21
• Transplantation Research--Restoring Vital Body Functions 2--21
• Basic Research Applications of ESCs 2--22
• Therapeutic Delivery Systems for SCs 2--23
• Other Applications of Stem Cells 2--23
• Critical Evidence and Questions about Stem Cell Research 2--23
• Known facts About Human Pluripotent Stem Cells? 2--24
• Comparisons of Adult Stem Cells and Embryonic Stem Cells 2--25
• Conclusions 2--25

Chapter 3: Market Analysis of Cell Therapy Space 3--1

• Overall Forecast for the Cell Therapy Field 3--6

Chapter 4: Cell Therapy in The News 4--1

• Stem Cell Injection Restores Ability to Walk 4--1
• Stem Cells Can Help Fight Against HIV 4--1
• Iowan Makes Strides After Spine Surgery 4--2
• Stem Cell Trigger Identified 4--2
• Successful Treatment of Stroke Victim Who Received Stem Cell Transplantation Therapy 4--2
• 'Long Road' for Stem Cell Research 4--3
• S.F. Clinic Recruiting Egg Donors for Stem Cell Research 4--3
• FDA Approves Brain Stem Cell Transplant 4--3
• Successful Trial of Gene Therapy in France Uses "Adult" Stem Cells 4--4
• Adult Stem Cells Reverse Diabetes in Mice 4--4
• Bone Marrow Stem Cell Transplant Used to Treat Children with Cartilage Defect 4--4
• Neural Stem Cells Able to Repair Damage Throughout Brain 4--4
• Corneal Stem Cells Help Restore Sight 4--4
• Human Bone Marrow Stem Cells Can Form Liver 4--5
• Adult Bone Marrow Stem Cells Can Generate Nerve Cells 4--5
• Large Numbers of Adult Bone Marrow Stem Cells Can Be Generated in Culture 4--5
• Adult Bone Marrow Cells Can Form Multiple Tissue Types 4--5
• Adult Stem Cells Reverse Ddiabetes in Mice 4--6
• Scientists Identify Human Adult Pancreatic Stem Cells 4--6
• Comprehensive Review Finds Adult Stem Cells Present Fewer Biological Problems for Producing Insulin Secreting Pancreatic Beta Cells 4--6
• Clinical Successes in Cancer Treatment Again Show Versatility, Pluripotent Abilities of Adult Stem Cells 4--6
• Adult Stem Cells Again Proved Capable of Numbers to Meet Clinical Demand 4--7
• Treating Heart Disease with adult Stem Cells 4--7
• Adult Stem Cells Advance Toward Treating Degenerative Neural Disorders 4--7
• Sonic Hedgehog" Paves Way for Potentially Unlimited Supplies of Adult Stem Cells; Improved Treatment for Cancer Patients 4--8
• Stem Cells from Skin Develop into Brain Cells and Other Tissues 4--8
• Stem Cell-Rich Cord Blood Successfully Treats Often Fatal Blood Disorder 4--8
• Scientists Purify Pluripotent Adult Neural Stem Cells from Brain 4--8
• Adult Stem Cells Show Success in Treating Autoimmune Disease 4--8
• Adult Stem Cells Treat Potentially Fatal Skin Disorder 4--9
• Adult Stem Cells Repair Heart Damage 4--9
• Adult Stem Cells Can "Be Cultured and Expand Indefinitely" 4--9
• Adult Stem Cells Offer Hope without Hype 4--9
• Successful Repair of Heart Damage Using Adult Stem Cells 4--9
• Japanese Scientists use Neural Stem Cells to Decrease Parkinson's Symptoms 4--10
• Human Adult Bone Marrow Stem Cells Can Form Kidney Tissue 4--10
• Treatment of Multiple Sclerosis Using the Patient's Own Cells 4--10
• Umbilical Cord Blood Effective At Treating Adult Blood Disorders 4--10
• Sickle Cell Anemia Treated with Umbilical Cord Blood Stem Cells 4--10
• Stroke Treatment Follow-up Shows Patient Improvement 4--11
• Tissue-Engineered Arterial Grafts from Adult Blood Cells 4--11
• Functional Brain Cells Generated From Adult Bone Marrow Stem Cells 4--11
• Spinal Cord Regeneration Using Adult Cells 4--11
• New Corneas Grown from Patients' Own Adult Stem Cells Treat Corneal Scarring 4--11
• Treating Parkinson's with Adult Stems Cell and Other Alternatives 4--12
• Retinal Cell Implants Improve Parkinson's 4--12
• Stimulating Adult Brain Stem Cells Decreases Parkinson's Symptoms 4--12
• Progenitor Cells Reverse Severe Parkinson's Symptoms in Rats 4--12
• Adult Liver Stem Cells Make Pancreatic Cells, Reverse Hyperglycemia in Diabetic Mice 4--12
• Adult Bone Marrow Stem Cells Transformed Into Functional Liver Cells 4--12
• Adult Muscle Stem Cells Form Multiple Tissues; Regenerate Dystrophic Muscle 4--13
• Chapter 5: Analysis of End-user Survey and Expert Opinions 5--1

• Interview with Dr. Nevenka Kregar Velikonja, Managing Director, Educell d.o.o. 5--1
• Interview with Stephen Bartelmez Ph.D., President and CEO, Ixion Biotechnology 5--5
• Survey Analysis 5--7
• Chapter 6: Company Profiles 6--1
• Aastrom Biosciences, Inc. 6--1
• Advanced Cell Technology, Inc. 6--4
• AllCells, LLC. 6--9
• Amcyte Inc. 6--10
• Argos Therapeutics 6--11
• Athersys Inc. 6--12
• BioE 6--13
• Bioheart, Inc. 6--14
• BioMark International 6--16
• BioTissue Technologies GmbH. 6--17
• Cambrex 6--18
• Cell Transplant International 6--21
• Cellartis AB 6--26
• CellECT Bio, Inc. 6--28
• Cellerant Therapeutics, Inc. 6--29
• Cellerix 6--30
• CepTor Corporation 6--31
• Chromos 6--32
• Cryosite Limited 6--34
• Cytomatrix LLC 6--35
• Cytori Therapeutics, Inc. 6--36
• Dendreon Corporation 6--37
• Educell, d.o.o. 6--39
• ES Cell International Pte Ltd 6--40
• Gamida Cell Ltd. 6--41
• GenOway 6--43
• Genzyme 6--44
• Geron 6--45
• Histostem, Ltd. 6--47
• Ixion Biotech Inc. 6--49
• Medra, Inc. 6--50
• Nephros Inc. 6--51
• Neuronyx Inc. 6--53
• Neurotech USA 6--54
• Northwest Biotherapeutics Inc. 6--55
• Orion Biosolutions 6--57
• Osiris Therapeutics, Inc. 6--58
• Progenitor Cell Therapy, LLC 6--61
• ReNeuron 6--62
• Stem Cell Technologies 6--64
• StemCells Inc. 6--67
• Theravitae Inc. 6--69

Chapter 7: Cell Therapy: Patent Analysis 7--1

Appendix: Basic Properties of an Embryonic Stem Cell Appx--1

• What are Some of the Questions that Need to be Answered about
• Stem Cells? Appx--1
• Timeline of Human Embryonic Stem Cell Research Appx--3
• Stem Cells Appx--5
• Web Resources Appx--5
• 1. Stem Cell Information and Background Appx--5
• 2. Stem Cell-related Organizations (US) Appx--5
• 3. International Stem Cell-related Organizations Appx--5

TABLE OF EXHIBITS

• Exhibit 1.1 Basic Principle of the Cell-based Therapy 1--1
• Exhibit 1.2 Basic Principle Behind Cell Therapy Strategy 1--2
• Exhibit 1.3 Number of Publications in the Cell Therapy area in the Past Decade 1--3
• Exhibit 1.4 Technologies Driving the Development of Stem Cell Therapy 1--4
• Exhibit 1.5 Lead Companies in the Cell Therapy Space 1--5
• Exhibit 2.1 Treatment of Human Diseases Using Dendritic Cells 2--10
• Exhibit 2.2 Stem Cell Hierarchy 2--12
• Exhibit 2.3 Classifying Embryonic and Adult Stem Cell Types 2--13
• Exhibit 2.4 Stem Cell Types and Their Characteristics 2--13
• Exhibit 2.5 Generating Autologous Ttissue Grafts Using the Therapeutic Cloning Strategy 2--18
• Exhibit 3.1 Market Forecast for Cell Therapy Treatments 3--2
• Exhibit 3.2 Distribution of Companies in the Cell Therapy Area 3--3
• Exhibit 3.3 Distribution of Companies Based on the Targeted Organ 3--3
• Exhibit 3.4 Distribution of Companies Based on Specific Therapeutic Areas 3--5
• Exhibit 3.5 Distribution of Companies based on Specific Disease Indication 3--6
• Exhibit 3.6 Expected Growth in the Number of Clinical Treatment Studies in Four Major Therapeutic Indications 3--7
• Exhibit 6.1 Production Cycle for Three Cellerix Products 6--30
• Exhibit 6.2 Applying Stem Cells in Developing Human Therapeutic Applications 6--46
• Exhibit 6.3 Stem cell discovery cycle 6--68