1. Stem Cells USA RM Congress:
Cord Blood Banking and Therapeutics
Boston, Massachusetts
Gregory A. Bonfiglio
Proteus Venture Partners
September 12, 2011

2. Proteus: An Investment and Advisory
Firm Focused on Regenerative Medicine
Proteus, Inc.
Proteus Proteus Proteus
Management, LLC Insights, LLC Advisors, LLC
(Fund Management) (Consulting Services) (Investment
Banking Services)
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3. Agenda
I. The Regenerative Medicine Market
 Key Market Metrics
 RM Therapeutic Applications
II. The Cord Blood Market
 Key Market Metrics
 Banking Other Tissues
III. Therapeutic Applications for Cord Blood
 Key Transplant Metrics
 Current Treatments
 Therapies in Clinical Development
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4. Development of Regenerative Medicine
Current technologies build on 50 years of research
1960s 1970s 1980s 1990s 2000s
1973 – First 2001 –
unrelated 1997 – Dolly the RhBMP-7
1963 – Mouse bone marrow 1981 – Mouse Sheep cloned; – Approved
ASCs isolated transplant ESCs Isolated FDA Approves
Carticel (19997)
& Apligraf (1999) FDA Approves
Dermagraft (2001)
1986 – First
1968 – ALL
Mouse Cloned
patient
irradiated, 2005 Use of
infused with 1989 – First unrelated cord
identical twin Sibling Cord Blood blood in BMT
BMT Transplant
(Gluckman) 1998 – 2007 –
Human iPSCs
ESCs from
1993 – ViaCell isolated Humans
Begins Banking
Cord Blood
Source: Company websites, NIH, Pubmed
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5. RM Is Entering A New ERA
RM Market is Maturing: Key Metrics
Rapidly Expanding Market: Commercial Products
• $1.6B in 2010 • 400 on Market (Mostly Skin, Tools
Media, & Devices);
• $20.0B in 2025 – 900+ in Development
• CAGR of 18.34% • 44 Cell Therapies on Market
– $1B Revenues
Dramatic Revenue Growth – 400 in Development
• $130M in 2001 – 28 in PIII/Pivotal Trials
• $1.6B+ in 2010
1.2M+ Patients Treated with RM
Products.
Worldwide funding for research
Increasing
• 320K+ Cell Therapy Patients
• $2.5B Now RM Companies
• $14B in 10 Years • 700+ Co’s involved in RM
• 50+ Public Co’s;
Clinical Programs
• Over 3600 Clinical Trials
– $8-$10B Total Market Cap
• Over 400 ex-Oncology • 250+ Private Co’s
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6. RM Market: Expanding Rapidly
Dramatic Cell Therapy Revenue Growth
CTI Revenues: $410M (2008) - $5.1B (2014)
Cell Therapy Industry: Billion Dollar Global Business With Unlimited Potential;
Regenerative Medicine; Chris Mason, David Brindley, Emily J Culme-Seymour & Natasha L Davie
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7. RM Market:
Global Company Distribution
Canada UK
24 firms 133 firms Europe
(ex. UK)
3% 19%
14% 93 firms
Asia
56% 2% 32 firms
5%
Middle East
17 firms
USA
386 firms
700+ RM companies worldwide!
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8. Big Pharma is Actively Engaged
GSK & HSCI: $25M Deal
Merck, Pfizer & Lily Launch Enlight BioScience
Pfizer RM Division: $111M Deal with
Athersys (IBD); UCL (RPE); ViaCyte
(Diabetes) GSK, AZN & Roche Help Launch Stem Cells
for Safer Medicine in UK
Genzyme & Osiris: $1.25B Deal
Johnson & Johnson Invests in Tengion &
ViaCyte (NovoCell)
Cephalon & Mesoblast: $2B Deal
GE & Cytori: StemSource
Shire Acquires ABH for $750M
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9. RM Business Models: Autologous v. Allogeneic
Autologous Model Allogeneic Model
Patients Own Cells/Tissue Universal Cells in a Bottle
• Personalized Medicine • Big Pharma “Drug Model”
Advantages:
• Autologous Treatment for Prostate
Advantages:
Cancer Using Dendritic Cells
• Easier Regulatory Path • Scalable
(GTP) -Centralized Processing
• Low COGS
• No Immune Response
• $93K per Treatment Challenges:
Challenges: • More Difficult
-$1B+ Projected Revenues Regulatory
• Difficult to Scale Path
• High COGS• $5.0B Market Cap (Until Aug 3,Response
• Immune 2011)
Service vs. Product
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10. Agenda
I. The Regenerative Medicine Market
 Broad RM Market: Key Metrics
 RM Therapeutic Applications
II. The Cord Blood Market
 Cord Blood Market: Key Metrics
 Banking Other Tissues
III. Therapeutic Applications for Cord Blood
 Key Transplant Metrics
 Current Treatments
 Therapies in Clinical Development
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11. Cord Blood Market:
Key Metrics
Cord Blood Market Metrics
Market Size: Market Penetration
• $3.4B (2010) • 1-6% of All Births (varies by
• $14.9B (2015) country/region)
• CAGR: 27.9%
Average Fees: Private Banks
Cord Blood Banks: • $1,750 Initial Collection & Storage
• 150+ Private Banks – Range: $890 - $2300
• 44 Public Banks • $125 Annual Fee
• 26 Countries – Range: $85-$150
Total Cord Blood Units Stored Average Fee: Public Banks
• 500,000 Units in Public Banks • $35,000 per Unit (US/EU)
• 1M+ Units in Private Banks
Public Bank Utilization Rate:
• 1-3% of Units per year
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12. Cord Blood Market:
Key Metrics
Cord Blood Market Metrics
Total Cord Blood Transplants: Regulatory Framework
25,000 in 43 Countries • Regulated as a “Biologic” by
FDA/EMA
• 1,500 per year (2005) • By October 2011, All Cord Blood
• 3,000 per year (2010) Banks Must be Licensed (BLA)
• 10,000 per year (2015)
Therapeutic Applications
50% of All Patients Seeking a • 60+ in Clinical Practice
BM/PBSC Transplant Cannot • Leukemia; Lymphoma; Blood
Find a Match Disorders; Hematopoietic
Restoration
Fastest Growing Segment of
Clinical Trials
Cell Transplant Market • Over 600 FDA Clinical Trials
• 22% of All Cell Transplants in – 425+ New Therapies
2010 – 57 Pivotal/PIII Trials
• 40% by 2015
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13. Cord Blood Therapies in Clinical Development:
600+ Ongoing FDA Trials
Source: ClinicalTrials.gov (www.clinicaltrials.gov)
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14. Banking Other Tissues
• Wharton’s Jelly/Entire Cord
• CHORI Technology (www.chori.org)
• Placental Tissue (MSCs)
• Pluristem Therapeutics, Inc. (www.pluristem.com) ; LifebankUSA
(www.lifebankusa.com)
• Amniotic Fluid (MSCs & HSCs)
• Biocell Center (www.biocellcenter.com)
• Autologous Marrowized Bone
• Arnie Caplan (Cell Bank Technologies)
• iPS Cell Lines
• Shinya Yamanaka (Japan)/Janet Rossant (Toronto); iPerian
(www.ipierian.com)
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15. Banking Other Tissues
• Peripheral Blood/Bone Marrow
• Stem Cell Assurance, Inc. (www.stemcellassurance.com)
• StemCor - Marrow-Miner
• ALDH Cells (Hematopoietic Stem And Progenitor Cells)
• Aldagen (www.aldagen.com)
• Adipose Derived Mesenchymal Cells (MSCs)
• Cytori (www.cytori.com); GE Healthcare
• Stem Cell Assurance, Inc. (www.stemcellassurance.com)
• Menstrual Blood
• CryoCell (www.cryo-cell.com)
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16. Agenda
I. The Regenerative Medicine Market
 Key Market Metrics
 RM Therapeutic Applications
II. The Cord Blood Market
 Key Market Metrics
 Banking Other Tissues
III. Therapeutic Applications for Cord Blood
 Key Transplant Metrics
 Current Treatments
 Therapies in Clinical Development
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17. Current Therapeutic Applications
for Cord Blood Market
Key Metrics
• 25,000 + Cord Blood Transplant To Date
• 3000+ Transplants in 2010
o 9% Increase over 2009
o 100% Increase (2X) over 2005
• 10,000 Transplants per year projected by 2015
o CAGR: 49.3%
• Multi-Cord Transplants Increased 25% in 2010
• 60+ Diseases Treated
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18. Cord Blood Therapeutics:
Comparison with Bone Marrow
Cord Blood v. Bone Marrow
HLA 2nd or
Donor Units Search Engraftment GvHD Ethnic
Match Double
Morbidity Available Time Time Risk Match
Required Graft
Cord 550,000
None 4/6 1 Day Yes 26 Days Minimal Easier
Blood Units
Possible
Bone 8/8 14.6M 3-4 if donor
50% 18 Days 50% Difficult
Marrow 10/10 Donors Months is
available
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19. Cord Blood Transplants Have Doubled In
Last 5 Years (NMDP Data)
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20. Cord Blood Is the Preferred Cell Source
for Pediatric Patients (NMDP Data)
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21. Adult Patients Are the Fastest Growing
Population: 58% 0f 2010 Cord Transplants
Cord Blood Transplants By Patient Age (NMDP Data)
0-17 18+
1,200
1,000
800
600
400
200
0
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010
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22. Transplant Outcomes Have Improved
Dramatically in Last 5 Years
Report Year Period One-Year Survival
2008 2002-2006 54.0%
2007 2001-2005 51.5%
2006 2000-2004 48.8%
2003 1996-2001 42.2%
• Improved HLA Matching;
• Advances in Conditioning Regimes; KEY FACTORS
• Advances in Post-Transplant Supportive Care
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23. Clinical Outcomes Related to
Quality of Tissue & Match
Key Metrics
Quality of Tissue
• TNC Count (150+)
• CD34+ Count
• Size of Unit
Matching Criteria
• HLA Match (4/6 Min)
• Race & Ethnicity Match
• Genetic Match (DNA tissue typing)
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24. Cord Shipments By TNC Count for Adults
1,200
1,000
800
150+
600 125-149
90-124
<90
400
200
0
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010
Most Units (77%) for Adults Had TNC >150 (NMDP Data)
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25. Public Bank Utilization Rates:
2010 Inventory and Shipments
TNC of Shipments - TNC of Inventory as of
CY 2010 June 30, 2010
Less than
125 Greater
18% than or
equal to
125
31% Less than
Greater 125
than or 69%
equal to
125
82%
82% of all 2010 shipments came from 31% of the available inventory.
(NMDP Data)
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26. Current Therapeutic Applications
for Cord Blood
60+ Diseases Treated
• Leukemias And Lymphomas, Including:
– Acute Myelogenous Leukemia
– Acute Lymphoblastic Leukemia
• Multiple Myeloma And Other Plasma Cell Disorders
• Severe Aplastic Anemia And Other Marrow Failure States,
Including:
– Severe Aplastic Anemia
– Fanconi Anemia
• SCID And Other Inherited Immune System Disorders, Including:
– Severe Combined Immunodeficiency (SCID, All Sub-types)
– Wiskott-aldrich Syndrome
• Hemoglobinopathies, Including:
– Beta Thalassemia Major
– Sickle Cell Disease
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27. Diseases Treated:
Hematologic Malignancies (NMDP Data)
CONFIDENTIAL 27

28. Diseases Treated:
Non-Malignant Disorders (NMDP Data)
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29. FDA Trials Involving Cord Blood Address
Numerous Conditions & Diseases
• Bacterial and Fungal Diseases • Mouth and Tooth Diseases
• Behaviors and Mental • Muscle, Bone, and Cartilage
Disorders Diseases
• Blood and Lymph Conditions • Nervous System Diseases
• Cancers and Other Neoplasms • Nutritional and Metabolic Diseases
• Digestive System Diseases • Parasitic Diseases
• Diseases and Abnormalities at • Respiratory Tract (Lung and
or before Birth Bronchial) Diseases
• Ear, Nose, and Throat Diseases • Skin and Connective Tissue
• Eye Diseases Diseases
• Gland and Hormone Related • Substance Related Disorders
Diseases • Symptoms and General Pathology
• Heart and Blood Diseases • Urinary Tract, Sexual Organs, and
• Immune System Diseases Pregnancy Conditions
• Wounds and Injuries • Viral Diseases
Source: ClinicalTrials.gov (www.clinicaltrials.gov)
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30. APPENIDX
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31. NMDP Data
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32. NMDP Data
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33. NMDP Data
CONFIDENTIAL 33

34. Adult Patients Are the Fastest Growing
Population: 58% 0f 2010 Cord Transplants
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35. The Promise of Regenerative Medicine
To Cure Diabetes To Cure Vision Disorders To Heal Acute Spinal Cord Injury
Paralyzed Rat Paralyzed Rat Walks
(double click to see video) (double click to see video)
To Replace Entire Organs Tools for Drug Discovery To Regenerate Heart Muscle
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36. Gartner’s Hype Cycle of Emerging Technologies
Visibility
Technology Peak of Inflated Trough of Slope of Plateau of Productivity
Trigger Expectations Disillusionment Enlightment
Time
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37. RM Market: On 2nd Half of the Gartner Curve
Visibility Peak of Inflated Trough of Slope of Plateau of
Expectations Disillusionment Enlightment Productivity
2001: 3300 jobs, 73 firms, mkt cap > $2.5B
2011 Shire Acquires ABH -$750M
2001 Ortec FDA approved
2000 Time Magazine: 2010 Cephalon & Mesoblast $2B Deal
TE No. 1 job
2001 Dermagraft FDA approved
2010 Dendreon’s Provenge Approved
1999 TE bladders in clinic 2001 Bush “partial ban” on HESCs
1999 First FDA approved 2009 Obama Ends Ban on hESCs
TE product (Apligraf)
1998 Human ESCs first derived Nov 2008 Genzyme-Osiris $1.25B Deal
1997 Dolly the sheep 2007 iPS Technology Developed
1997 First FDA approved cell
therapy (Carticel) 2007 Apligraf - 200,000 Patients Treated
2006 Carticel - 10,000 Patients Treated
2002 ISSCR Founded
1992 Geron founded
2005 CIRM Founded
1988 SyStemix founded
1986 ATS and Organogenesis founded 2003 UK Stem Cell Bank Formed
1980 Early TE research (MIT) 2002 ATS + Organogenesis file
Chapter 11
Technology Trigger
Stage of Development Time
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