Stem Cell-Based Therapeutic Delivery: Challenges & Opportunities

Table of Contents

KEY FINDINGS

• Stem Cells & Cell-Based Delivery
• Stem Cell-Based Therapies: Pipeline Analysis
• Stem Cell-Based Therapies for Cardiovascular Disease
• Stem Cell-Based Therapies for CNS Diseases
• Stem Cell-Based Therapies for Autoimmune, Inflammatory & Other Conditions
• Stem Cell-Based Therapies for Cancer, Graft versus Host Disease & Hematological Malignancies
• Challenges & Opportunities Challenges & Opportunities for Stem Cell-Based Therapies
• Future of Stem Cell-Based Therapeutics & Market Trends

CHAPTER 1 STEM CELLS & CELL-BASED DELIVERY

• Key Points
• Introduction
• An Introduction to Stem Cell Science
  • Embryonic stem cells
  • Adult stem cells
  • Creating pluripotent cells from adult tissue
  • Nuclear transfer/therapeutic cloning
  • Combining embryonic stem cell therapy with gene therapy
• Stem Cell Therapy - A Discovery Timeline
• Major Scientific Challenges for the Development of Stem Cell Therapies
• Report Structure

CHAPTER 2 STEM CELL-BASED THERAPIES: PIPELINE ANALYSIS

• Key Points
• Key Therapy Areas Covered by Stem Cell-Based Therapies
  • Product pipelines
• Stem Cells for Drug Discovery
• Stem Cell Culture, Expansion & Manufacturing
• Stem Cell Companies Analyzed in This Report

CHAPTER 3 STEM CELL-BASED THERAPIES FOR CARDIOVASCULAR DISEASE 41

• Key Points
• Introduction
• Autologous Cell Platforms
  • Case study: MyoCell (Bioheart Inc)
  • Case study: Myoblast (Advanced Cell Technology)
  • Case study: CardioCure (Gamida Cell Ltd)
  • Case study: C-Cure (Cardio3 BioSciences)
  • Case study: Celution System (Cytori Therapeutics)
• Allogeneic Cell Platforms
  • Mesenchymal stem cell therapies
  • Case study: Prochymal (Osiris Therapeutics)
  • Case study: Revascor (Angioblast Systems Inc)
  • Case study: PLX-PAD (Pluristem Therapeutics Inc)
  • Human embryonic stem cell therapies
  • Case study: ACT Cellerate (Advanced Cell Technology)
• Cell Delivery Technologies
• Our Opinion on Stem Cell-Derived Therapies for CV Diseases
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Activity in the market
  • Major players
  • Winners

CHAPTER 4 STEM CELL-BASED THERAPIES FOR CNS DISEASES

• Key Points
• Introduction
• Autologous Cell Platforms
  • Case study: NurOwn (BrainStorm Cell Therapeutics Inc)
  • Case study: NeuroGeneration
• Allogeneic Cell Platforms
  • Stem cells from donated tissue
  • Case study: HuCNS-SC (StemCells Inc)
  • Case study: ReNeuron
  • Case study: Neuralstem
  • Case study: Q-cells (Q Therapeutics Inc)
  • Case study: StemedicaMCT (Stemedica USA and Stemedica International)
  • Stem cells from umbilical cord blood
  • Case study: Beike Biotechnology
  • Human embryonic stem cells
  • Case study: GRNOPC1 (Geron)
  • Case study: hESCs from California Stem Cell Inc
• Cell delivery technologies
• Our Opinion on Stem Cell-Based Therapies for CNS Diseases
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Activity in the market

CHAPTER 5 STEM CELL-BASED THERAPIES FOR AUTOIMMUNE, INFLAMMATORY & OTHER CONDITIONS

• Key Points
• Introduction
• Crohn' s Disease
• Case study: Prochymal for Crohn' s disease (Osiris)
• Case study: Ontaril (Cellerix)
• Diabetes
  • Autologous stem cell transplants for diabetes
  • Allogeneic stem cell transplants for diabetes
• Osteoarthritis
• Lung Diseases
  • Chronic obstructive pulmonary disease
  • Pulmonary arterial hypertension
• Our Opinion on Stem Cell-Based Therapies for Autoimmune and Other Diseases
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Major players and winners

CHAPTER 6 STEM CELL-BASED THERAPIES FOR CANCER, GRAFT VERSUS HOST DISEASE & HEMATOLOGICAL MALIGNANCIES

• Key Points
• Introduction
  • Cancer stem cells
  • Stem cells as a delivery vehicle
• Graft versus host disease
• Expanded Umbilical Cord Blood Transplants for Malignant Diseases
  • Case study: StemEx (Gamida Cell)
• Stem Cells as Delivery Systems
  • Lung cancer
  • Metastatic cancer and intracranial tumors
  • Hematological malignancy
  • Multi-lineage progenitor cells (BioE)
• Conclusion
• Our Opinion on Stem Cell-Derived Therapies for Cancer, GvHD and Hematological Malignancies
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Activity in the market, major players and winners

CHAPTER 7 CHALLENGES & OPPORTUNITIES FOR STEM CELLBASED THERAPIES

• Key Points
• Introduction
• Ethical Issues for Stem Cell Research
• Funding Stem Cell Research
• Intellectual Property
• Manufacturing Stem Cell-Based Products
• Regulation
• Reimbursement
• Delivering Stem Cell-Based Therapies and Tissue Engineering
  • Challenges
  • Opportunities

CHAPTER 8 FUTURE OF STEM CELL-BASED THERAPEUTICS & MARKET TRENDS

• Key Points
• Key Stem Cell Therapy Companies
• Recent Deals & Alliances
  • The Global Stem Cell Market 2008-2014
  • Analysis parameters
  • Market forecast 2008-2014
  • Global stem cell-based therapy market in 2020
  • Drivers and market trends
• Summary & Conclusions
• Acknowledgements
• Bibliography & Endnotes

List of Figures

• Figure 1: Source of embryonic stem cells: the blastocyst
• Figure 2: Differentiation of embryonic stem cells
• Figure 3: Somatic cell nuclear transfer
• Figure 4: Combining gene therapy and cell therapy for sickle cell anemia in a mouse model
• Figure 5: Stem cell research: a timeline
• Figure 6: Stem cell therapeutics: defining therapeutic areas
• Figure 7: Number of projects in the different stages of pharmaceutical development
• Figure 8: Number of projects in each therapeutic area, by stage of development
• Figure 9: Proportions of autologous and allogeneic technology platforms
• Figure 10: Stem cell sources (embryonic, adult or cord blood/placenta)
• Figure 11: MyoCell: Heart failure treatment process
• Figure 12: Advanced Cell Technologies Myoblast Phase 1 programme; results
• Figure 13: The Celution system from Cytori Therapeutics
• Figure 14: Preclinical data demonstrating the effects of Prochymal therapy on the heart
• Figure 15: PLX cells growing in Pluristem' s PluriX bioreactor
• Figure 16: ACTCellerate technology
• Figure 17: Schematic of cell-based therapy administration to the myocardium
• Figure 18: Cells affected by different CNS diseases/disorders
• Figure 19: NeuroGeneration' s Parkinson' s disease therapy
• Figure 20: StemCells Inc technology platform
• Figure 21: ReNeuron' s process for generating therapeutic cells
• Figure 22: Stemedica' s established cell lines
• Figure 23: Results of animal studies with Geron' s oligodendroglial progenitor cells derived from human ESCs
• Figure 24: Stem cell sources under investigation to treat type 1 diabetes
• Figure 25: Stem cells applications in cancer and other malignant diseases
• Figure 26: Strategies for potentiating stem cells for tumor treatment
• Figure 27: Challenges and opportunities for stem cell science
• Figure 28: Ethical issues with stem cells from different sources
• Figure 29: Federal funding of embryonic stem cell research in the US: a timeline
• Figure 30: US patent ownership in the stem cell field
• Figure 31: Stem cell-based therapeutics: market drivers

List of Tables

• Table 1: Pros and cons of different stem cell types
• Table 2: Diseases targeted by stem cell-based therapeutics
• Table 3: Stem cell therapy companies discussed in this report
• Table 4: Leading companies evaluating stem cell-based CV therapies
• Table 5: Leading academic institutions researching the use of stem cells to treat CV diseases
• Table 6: Leading companies evaluating stem cell-based therapies for CNS diseases
• Table 7: Leading institutes evaluating stem cell-based therapies for CNS diseases
• Table 8: Geron' s stem cell-based product pipeline
• Table 9: Leading companies evaluating stem cell therapies for autoimmune and other diseases
• Table 10: Leading academic institutes evaluating stem cell therapies for autoimmune and other diseases
• Table 11: Leading companies evaluating stem cell therapies for GvHD and hematological malignancies
• Table 12: Academic institutions with an interest in cancer, GvHD and hematological malignancies
• Table 13: Other clinical trials of mesenchymal stem cells for the prevention or treatment of GvHD
• Table 14: Results of Phase 1/2 trial for StemEx
• Table 15: Global positions on research on embryonic stem cells derived from IVF procedures
• Table 16: Current funds available from individual US states for embryonic and adult stem cell research
• Table 17: Summary of leading stem cell-based therapeutics companies
• Table 18: Recent licensing deals and alliances with big pharma/biotech
• Table 19: Stem cell-based therapies included in the market forecast
• Table 20: Forecast of pipeline stem cell therapy products 2008-2014 (US$m)
• Table 21: Forecast of pipeline stem cell therapy products 2015-2020 (US$m)