Stem Cell-Based Therapeutic Delivery

Summary

This cutting edge & insightful report can be used to interpret & assess the potential of stem cell-based medicines. It provides opinions & market projections to:

• Assess the commercial potential of stem cell-based therapies in all of the key therapeutic areas: autoimmune diseases, cardiovascular, CNS & hematological malignancies;
• Identify key pharma & delivery specialists advancing the next generation of regenerative medicines;
• Gain an in-depth understanding of the technological manufacturing & delivery issues which face companies developing stem cell-based therapies;
• Evaluate the options available for delivering stem cell-based therapies now & in the future;
• Discover which companies are tackling the technological hurdles & are in a prime position to exploit new opportunities;
• Analyze how the market will evolve over the next decade highlighting key trends, opportunities & challenges.

""Stem cell-derived therapeutics are on the brink of a new era, with a handful of therapies expected on the market within the next 2 or 3 years. Big pharma and biotech companies such as Pfizer and Genyzme have recently invested large sums in this exciting and fast-moving area, further demonstrating its growing maturity. The results of numerous Phase 1/2 clinical trials that are underway now will provide a clearer picture of the potential of stem cell-derived therapies for the future.""- Dr Sara Sleigh

The pace of stem cell research is astonishing. It is less than 30 years since the discovery of the embryonic stem cell, and less than a decade since scientists determined the right conditions under which human embryonic stem cells (hESCs)could be directed to differentiate into a variety of cell types. Within the last few years, companies have built up sufficient data to support applications to start human clinical trials based on hESCs that have differentiated into oligodendrocytes for the treatment of spinal cord injury.

Embryonic stem cells are just part of the stem cell story. Adult stem cells, and those from other sources such as umbilical cord blood, have also been the subject of intense research over the past few years. While there has been a precedent for the use of bone marrow stem cells in the treatment of hematological malignancies, the development of new therapies based on cultured, expanded populations of specific cell types opens up a vista of new possibilities.

Regenerative medicine has a wide definition covering therapies that aim to repair, replace, restore and regenerate damaged or diseased cells, tissues and organs. Innovative therapies derived from stem cells fall within this definition and will, in the coming years, provide options for the treatment of a variety of diseases including: autoimmune diseases such as diabetes, Crohn's disease and osteoarthritis, CNS disorders and neurodegenerative diseases, heart diseases, as well as eye diseases.

Stem cell therapies are already offered at various locations across the globe, including China and Thailand, despite a lack of robust, randomized clinical trials demonstrating their safety and efficacy. The fact that stem cell therapies are available in these locations and that patients' travel across the globe to receive them illustrates the high unmet medical need associated with some of the targets being investigated. Clinical evidence from high quality clinical trials is now imperative in order to protect these vulnerable patient populations.

The challenges facing companies developing stem cell-derived therapies are high and range from ethical discussions surrounding the use of hESCs to difficulties associated with manufacture of cell-based products at scale and proving that products lack the potential to cause an immune response or cancer in a recipient. Nevertheless a growing pool of companies is investing heavily to bring this rapidly moving science to the clinic.

Contents

• Key Findings
• Stem Cells & Cell-Based Delivery
• The Market for Stem Cell-Based Therapies
• Stem Cell-Based Therapies for Cardiovascular Disease
• Stem Cell-Based Therapies for CNS Disorders
• Stem Cell Therapies for Diabetes
• Stem Cell Therapies for Cancer
• Stem Cell-Based Therapeutics: Challenges & Opportunities
• 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 and manufacturing
  • Stem cell companies analysed in this report
• Chapter 3 Stem Cell-Based Therapies for Cardiovascular Disease
  • 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
  • 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 Graft versus Host Disease, Hematological Malignancies and Cancer
  • 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 GvHD, hematological malignancies and cancer
  • 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 Cell-Based 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 Market size, Evolution & Prospects
  • Key Points
  • Key stem cell therapy companies
  • Recent deals and 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 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
  • Table10: 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 GvHD, hematological malignancies and cancer
  • Table 13: Other clinical trials of mesenchymal stem cells for the prevention or treatment of GVHD
  • 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)