Future Directions in Regenerative Medicine

• Publication date: February 2011

Big Pharma has begun investing in regenerative medicine: Genzyme in 2008, Pfizer and Novartis in 2009, and Cephalon in 2010. In 3-5 years, investment will reach a “tipping point”, after which no medical industry players will want to be left behind. This report covers the development of therapies to replace human cells or promote their regeneration: including cell therapies, tissue engineering, drugs, and medical devices. Each company p...

Big Pharma has begun investing in regenerative medicine: Genzyme in 2008, Pfizer and Novartis in 2009, and Cephalon in 2010. In 3-5 years, investment will reach a “tipping point”, after which no medical industry players will want to be left behind.

This report covers the development of therapies to replace human cells or promote their regeneration: including cell therapies, tissue engineering, drugs, and medical devices. Each company profiled has at least one project in preclinical or Phase I study. Many also have later stage or launched products, which are covered briefly to illustrate the potential of the next generation of candidates.

Scope of this research

• Understand the concepts behind regenerative medicine, its potential applications, and advantages over traditional medicine.
• Compare the opportunities and challenges presented by the different therapeutic modalities.
• Understand the hurdles that must be overcome by regenerative medicine candidates before they can successfully enter the market.
• Identify emerging technologies that will shape the regenerative medicine industry in the coming years.
• Identify companies that may be competitors for a market, or alternatively potential partners or investment opportunities.

Research and analysis highlights

The first regenerative cell therapies to generate significant market value (within the next five years) will be based on allogeneic adult cells, and will target tissues that are naturally sheltered from patients’ immune systems.

Autologous cells are not rejected by the patient’s immune system, so are potentially safer than allogeneic cells and more suitable for permanent tissue replacement. “High tech” therapies based on reprogrammed autologous cells (e.g. induced pluripotent stem cells) may still be 20 years from market.

Tissue engineering still faces technical hurdles, including vascularization of grafted tissue and safe breakdown of scaffold materials. Research and regulatory efforts must be coordinated internationally to develop a stronger “evidence base” for regenerative medicine that is accessible and acceptable to all stakeholders.

Key reasons to purchase this research

• What is regenerative medicine and why are so many companies and investors excited about it?
• Which companies are currently developing new regenerative treatments and what approaches are they pursuing?
• What partnerships have been formed to develop new therapies, and where are the opportunities for further deals?
• What will the next generation of regenerative medicine candidates look like, and which indications will they treat?
• What hurdles do these candidates face en route to the marketplace?

Biological Therapy Industry in the United States

TABLE OF CONTENTS

About the author 2
Disclaimer 2

Executive Summary 12
An introduction to regenerative medicine 12
Allogeneic regenerative cell therapies 12
Autologous regenerative cell therapies 13
Tissue engineering 14
Regenerative drug molecules 14
Regenerative devices and implants 15
The future of regenerative medicine 15

Chapter 1 An introduction to regenerative medicine 17
Summary 17
Introduction 17
Cell-based therapies 18
Tissue engineering 19
Acellular therapies 20

Chapter 2 Allogeneic regenerative cell therapies 21
Summary 21
Introduction 21
Case studies 23
Advanced Cell Technology 24
Altrika 25
Athersys 26
BetaLogics 29
BioTime – embryonic progenitor cells 29
California Stem Cell 31
Cardio3 Biosciences 32
Celgene Cellular Therapeutics 33
Effector Cell Institute 33
Gamida Cell 34
Genzyme and Osiris Therapeutics 35
Geron 36
International Stem Cell 39
ISTO Technologies 40
MediStem – universal donor cells and cord blood transplants 41
Mesoblast – mesenchymal precursor cells 43
NeuralStem – fetal neural stem cells 45
Pfizer 46
Pluristem Therapeutics 47
Promethera Biosciences 49
Proteonomix 50
ReNeuron Group 50
Saneron CCEL Therapeutics. 51
Stematix 52
StemCells 52
Stemedica Cell Technologies 54
Stempeutics Research 55
TiGenix – allogeneic MSCs 55
TissueGene 56
Summary of allogeneic cell therapies in development 57
Discussion 63
Technical issues 63
Regulatory issues 66
Logistical issues 66
Conclusions 67

Chapter 3 Autologous regenerative cell therapies 69
Summary 69
Introduction 69
Case studies 71
Aastrom Biosciences 72
Aldagen 72
American Medical Systems Holdings 75
Amorcyte 75
Azellon 75
Bioheart 76
BioTime – induced pluripotent stem cells 77
BrainStorm Cell Therapeutics 79
Cytori Therapeutics 80
Ectycell 81
GeneGrafts 82
GlaxoSmithKline 83
Intercytex 84
LoneStar Heart – regenerative cell induction 86
MedCell Bioscience 86
MediStem – Tolerostem 86
MultiGene Vascular Systems 87
Novartis – monocyte derived islet cells 88
StemSave 88
TCA Cellular Therapy 89
Tissue Genesis Inc. 89
TriStem 90
Summary of autologous cell therapies in development 91
Discussion 96
Technical issues 96
Regulatory issues 98
Logistical issues 99
Conclusions 100

Chapter 4 Tissue engineering 102
Summary 102
Introduction 102
Case studies 103
Advanced BioHealing 104
CellSeed 104
Cerco Medical 105
Cytograft Tissue Engineering 106
Japan Tissue Engineering Company 107
Living Cell Technologies 108
Lonza Group and Regenicin 109
Organogenesis 109
Organovo 110
Pervasis Therapeutics 112
Tengion 114
ViaCyte 115
Summary of tissue engineering treatments in development 116
Discussion 119
Technical issues 119
Regulatory issues 120
Logistical issues 120
Conclusions 121

Chapter 5 Regenerative drug molecules 122
Summary 122
Introduction 122
Case studies 124
Angion Biomedica 124
Cardio3 Biosciences 124
DeveloGen 125
DiaMedica 125
ImmuneRegen BioSciences 126
Epistem 127
Fate Therapeutics 128
Juventas Therapeutics 128
LoneStar Heart – cardiac stem cell modulators 129
Mesoblast – SDF-1 130
NeuralStem – NSI-189 130
NeuroNova 131
Novartis – TherAtoh 131
Olympus Biotech 132
Sanofi-Aventis 133
Stryker 133
Stem Cell Therapeutics 134
Tissue Repair Company 134
Trillium Therapeutics 137
Summary of regenerative drug molecules in development 137
Discussion 140
Technical issues 140
Regulatory issues 141
Logistical issues 141
Conclusions 141

Chapter 6 Regenerative devices and implants 143
Summary 143
Introduction 143
Case studies 144
Alacer Biomedical 144
Kensey Nash 145
Mesynthes 146
Nanotope 146
Neopec 148
Neotherix 149
RepRegen 150
TheraGlass 151
TiGenix – biomaterials 152
Tissue Regenix Group 152
Summary of regenerative devices in development 153
Discussion 155
Technical issues 155
Regulatory issues 156
Logistical issues 156
Conclusions 156

Chapter 7 The future of regenerative medicine 158
Summary 158
Introduction 158
Investment in regenerative medicine 158
Reimbursement 160
Hope versus hype 161
Appendix 163
Scope 163
Methodology 163
Primary research 163
Secondary research 164
Abbreviations 164
References 172
Company websites 172
Journal and magazine articles 178
Patent documents 181

Table of figures
Figure 1: Allogeneic regenerative cell therapy 23
Figure 2: Autologous regenerative cell therapy 71
Figure 3: Tissue engineering 103
Figure 4: NovoGen bioprinting 111
Figure 5: Regenerative drug therapy 123
Figure 6: Regenerative medical device therapy 144

Table of tables
Table 1: Allogeneic regenerative cell therapies under development (part 1) 57
Table 2: Allogeneic regenerative cell therapies under development (part 2) 58
Table 3: Allogeneic regenerative cell therapies under development (part 3) 59
Table 4: Allogeneic regenerative cell therapies under development (part 4) 60
Table 5: Allogeneic regenerative cell therapies under development (part 5) 61
Table 6: Allogeneic regenerative cell therapies under development (part 6) 62
Table 7: Autologous regenerative cell therapies under development (part 1) 92
Table 8: Autologous regenerative cell therapies under development (part 2) 93
Table 9: Autologous regenerative cell therapies under development (part 3) 94
Table 10: Autologous regenerative cell therapies under development (part 4) 95
Table 11: Tissue engineering treatments under development (part 1) 117
Table 12: Tissue engineering treatments under development (part 2) 118
Table 13: Regenerative drug molecules under development (part 1) 138
Table 14: Regenerative drug molecules under development (part 2) 139
Table 15: Regenerative drug molecules under development (part 3) 140
Table 16: Regenerative devices under development (part 1) 154
Table 17: Regenerative devices under development (part 2) 155