1. Proposal: Build The Leading International Cryo-
Banking and Stem Cell Therapeutics Company
September, 2011
Gregory A. Bonfiglio
Proteus Venture Partners

2. Proteus: An Investment and Advisory
Firm Focused on RM
Proteus, Inc.
Proteus Proteus Proteus
Management, LLC Insights, LLC Advisors, LLC
(Fund Management) (Consulting Services) (Investment
Banking Services)
CONFIDENTIAL 2

3. Agenda
I. The Market & Opportunity
 Broader Regenerative Medicine Market
 Cord Blood Market: Key Metrics & Market Leaders
 The Opportunity
II. Phase I: Build A Dominant International Bio-Banking Company
 Acquire A Market Leader & Roll-Up Small Underperforming Banks
 Drive Revenues with Aggressive Sales & Marketing
 Expand Tissue Offering
III. Phase II: Bring RM Therapeutics To Emerging Markets
 Initial Therapies Leverage Stored Tissue
 Proven Therapies That Address Unmet Regional Medical Needs
IV. Funding & Exit Strategy
 Funding Required
 Exit Strategy
CONFIDENTIAL 3

4. 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
CONFIDENTIAL 4

5. 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
CONFIDENTIAL 5

6. 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
CONFIDENTIAL 6

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!
CONFIDENTIAL 7

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
CONFIDENTIAL 8

9. 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 (2105) 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
CONFIDENTIAL 9

10. Cord Blood Market:
Key Metrics
Cord Blood Market Metrics
Total Cord Blood Transplants: Regulatory Framework
15,000 in 43 Countries • Regulated as a “Biologic” by
FDA/EMA
• 1,056 per year (2009) • By October 2011, All Cord Blood
• 10,000 per year (2015) Banks Must be Licensed (BLA)
• CAGR: 37.8%
Therapeutic Applications
50% of All Patients Seeking a • 70+ 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 530 FDA Clinical Trials
• 25% of All Cell Transplants in – 396 New Therapies
2010 – 50 Pivotal/PIII Trials
• 60% by 2015
CONFIDENTIAL 10

11. 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 14.6M 3-4 if donor
50% 8/8 18 Days 50% Difficult
Marrow Donors Months is
available
Confidential 11

12. 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)
CONFIDENTIAL 12

13. Banking Other Tissues
• Peripheral Blood/Bone Marrow
• Stem Cell Assurance, Inc. (www.stemcellassurance.com)
• Oristem (www.oristem.com/)
• 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)
CONFIDENTIAL 13

14. 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
CONFIDENTIAL 14

15. Agenda
I. The Market & Opportunity
 Broader Regenerative Medicine Market
 Cord Blood Market: Key Metrics & Market Leaders
 The Opportunity
II. Phase I: Build A Dominant International Cryo-Banking Company
 Acquire A Market Leader & Roll-Up Small Underperforming Banks
 Drive Revenues with Aggressive Sales & Marketing
 Expand Tissue Offering
III. Phase II: Bring RM Therapeutics To Emerging Markets
 Initial Therapies Leverage Stored Tissue
 Proven Therapies That Address Unmet Regional Medical Needs
IV. Funding & Exit Strategy
 Funding Required
 Exit Strategy
CONFIDENTIAL 15

16. Build A Dominant International Cryo-
Banking Company: Strategic Plan
#1: Acquire a Market Leader
• Acquisition Candidate: StemCyte (http://www.stemcytefamily.com/)
 3rd Largest Cord Blood Company
o Cord Blood Registry, and ViaCord Are Largest
 Public & Private Banks
 Proprietary Plasma Reduction Technology
 Active Therapeutics Program
o 1300+ Transplants to Date
o Clinical Trials in China (Spinal Cord; Stroke)
 Facilities in US, Taiwan, and India
CONFIDENTIAL 16

17. Build A Dominant International Cryo-
Banking Company: Strategic Plan
#2: Roll-Up Small Underperforming Banks
• 20-25 Possible Candidates Worldwide
 Small “Proprietary” Operations
 Struggling to Achieve Sustained Profitability
o 2500-7500 Units Under Management
 Limited Budget for Sales & Marketing
 Facing New Regulatory Environment
o October 2011 – New FDA Regs Become Effective
o Need Update Technology to cGMP/cGLP Standards
 Facing Potential Long-term Liability
o Storage Obligations for 20+ years
• Aggregate All Banking Operations Into One Facility in
Each Country/Region
CONFIDENTIAL 17

18. Build A Dominant International Cryo-
Banking Company: Strategic Plan
#3: Drive Revenues and Reduce Expenses
• Drive Revenues In Private Banks Thru Aggressive Sales &
Marketing Strategy
 Internet/Website; Call Center; Professional Sales Team (Nurses)
 Build Relationships with Maternity Hospitals & OBGYNs
• Drive Revenues in Public Banks Thru Education/Marketing and
Product Differentiation
 Focus on Transplant Physicians & Transplant Coordinators
 Led By Internal Transplant Surgeon & SAB Members
 Differentiate Product By Including Data to Improve Patient Match
o Genetic Data
o Ethnic Data
 Target Utilization Rate: 3-5%
CONFIDENTIAL 18

19. Build A Dominant International Cryo-
Banking Company: Strategic Plan
#3: Drive Revenues and Reduce Expenses
• Reduce Expenses By Improving Collection Technology and
Sourcing Materials from India
 Improved Collection Technologies: Closed Perfusions/Gravity
Systems
 Acquire Rights to Technology
 Source Materials & Manufacture in India
• Reduce Expenses By Leveraging Economies of Scale
 Single, Global Management Team
 Standardize Processes
 Common Sales & Marketing Materials
o Modified for Local Markets
CONFIDENTIAL 19

20. Build A Dominant International Cryo-
Banking Company: Strategic Plan
#4: Expand Tissue Offering
• New Tissue Candidates Include:
• Wharton’s Jelly/Entire Cord
• Placental Tissue (MSCs)
• Amniotic Fluid (MSCs & HSCs)
• Autologous Marrowized Bone
• iPS Cell Lines
• Peripheral Blood/Bone Marrow
• ALDH Cells (Hematopoietic Stem And Progenitor Cells)
• Adipose Derived Mesenchymal Cells (MSCs)
CONFIDENTIAL 20

21. Agenda
I. The Market & Opportunity
 Broader Regenerative Medicine Market
 Cord Blood Market: Key Metrics & Market Leaders
 The Opportunity
II. Phase I: Build A Dominant International Cryo-Banking Company
 Acquire A Market Leader & Roll-Up Small Underperforming Banks
 Drive Revenues with Aggressive Sales & Marketing
 Expand Tissue Offering
III. Phase II: Bring RM Therapeutics To Emerging Markets
 Initial Therapies Leverage Stored Tissue
 Proven Therapies That Address Unmet Regional Medical Needs
IV. Funding & Exit Strategy
 Funding Required
 Exit Strategy
CONFIDENTIAL 21

22. Phase II: Bring RM Therapeutics to
Emerging Markets
Selection Criteria for New RM Therapies
• Leverage Existing Tissues
 Cord Blood
 “Concentrated” MSCs & HSCs
• Autolgous Cells; Minimally Manipulated
 Simple Regulatory Path
• Established Record of Successful Treatments
 Proven Therapies
 Faster Time To Market & Revenues
 Build Track Record of Success
• Reasonable Market Size
CONFIDENTIAL 22

23. Phase II: Bring RM Therapeutics to
Emerging Markets
Potential RM Therapies (Near Term)
• Cord Blood
 Leukemia
 Thalassemia; Sickle Cell Anemia
 Hematopoietic Restoration
• MSCs
 Peripheral Vascular Disease
 Cardio
 GvHD
 Immune Disorders
• HSCs
 Hematopoietic Restoration
CONFIDENTIAL 23

24. Phase II: Bring RM Therapeutics to
Emerging Markets
Potential RM Therapies - Aesthetic Medicine
• MSCs (Adipose Derived)
 Skin Rejuvenation
 Anti-Wrinkle
 Reconstructive Surgery (Breast Implants)
• Fibroblasts
 Dermal Fillers
• Conditioned Media (Histogen; ReGen Biosciences)
 Hair Regrowth
 Skin Rejuvenation
CONFIDENTIAL 24

25. Phase II: Bring RM Therapeutics to
Emerging Markets
Potential RM Therapies (Mid Term)
• Engineered Skin Products - Wound Repair
 Apligraf /Dermagraft
 Diabetic Foot Ulcers
• Dendritic Cells
 Prostate Cancer
• Cardio Products
 MSCs
 CHF or MI
• Fibroblasts
 ReCell - Wound Repair
• Vision Disorders
• Corneal Transplants; RPEs
CONFIDENTIAL 25

26. Phase II: Bring RM Therapeutics to
Emerging Markets
Potential Therapies (Long Term)
• Diabetes
• Other Autoimmune Diseases (MS; Lupus; Crohn’s)
• CNS Disorders
 Parkinson's
 Alzheimer's
 Stroke
• Spinal Cord Repair
• Tissue & Organs
 Bladder
 Ligaments; Veins; Valves
 Cartilage
CONFIDENTIAL 26

27. Agenda
I. The Market & Opportunity
 Broader Regenerative Medicine Market
 Cord Blood Market: Key Metrics & Market Leaders
 The Opportunity
II. Phase I: Build A Dominant International Cryo-Banking Company
 Acquire A Market Leader & Roll-Up Small Underperforming Banks
 Drive Revenues with Aggressive Sales & Marketing
 Expand Tissue Offering
III. Phase II: Bring RM Therapeutics To Emerging Markets
 Initial Therapies Leverage Stored Tissue
 Proven Therapies That Address Unmet Regional Medical Needs
IV. Funding & Exit Strategy
 Funding Required
 Exit Strategy
CONFIDENTIAL 27

28. Funding & Exit Strategy
Funding Required: $75M
• Use of Proceeds:
 Acquisition of Market Leader
 Execute Roll-Up Of Small Underperforming Banks
 Centralize Banking Operations in Country/Region
 Expand Sales & Marketing Program
o Website Design; Call Center Operations
o Expand Sales & Marketing Team
o Education/Conferences for Transplant Surgeons & Transplant
Coordinators
 Acquire New Collection Technologies
 Bring All Facilities Up To cGMP Standards
 Fund Research on Cord Blood Therapies
CONFIDENTIAL 28

29. Funding & Exit Strategy
Exit Strategy: IPO in 24-36 Months
• Drive Revenues to $100M-$150M
 Revenue Ramp of 25% per Quarter
 Gross Profit Margins: 60%
• List on Most Favorable Exchange
 Current Cord Bank Valuations: 4-6X Revenues
• Expected Valuation Range: $300M-$600M
 Expected ROI: 30+% (3 years; assumes 50% ownership)
• Use Proceeds of IPO to Fund Introduction of RM Therapies
CONFIDENTIAL 29

30. MAJOR RISKS
• Risk: Decline In Market Price for Public Units Due to Increase in
Supply
• 550,000 Units now; growing rapidly
o Risk Mitigation Strategy
 Product Differentiation: Store Higher Quality Product (TNC & CD34+ Counts) ;
Greater Genetic Diversity
 Expand Market: Increased Therapeutic Applications Drives Additional Demand
and Offsets Increases in Supply
• Risk: Cost of Roll-Up Increases Due to Market Pressures
• Acquisition Targets Increase Demands with Knowledge of Roll-Up
Strategy
o Risk Mitigation Strategy
 Close on Initial Acquisition Targets Simultaneously
CONFIDENTIAL 30

31. MAJOR RISKS
• Risk: Failure to Execute
• Typical Roll-Up Execution Risk
o Risk Mitigation Strategy
 Assemble World-Class Acquisition Team – Technical & Business (Chris
Mason; Melissa Carpenter)
 Leverage Proteus Team & Network
• Risk: Competition In Market
• Other Groups Looking at Roll-Up Strategy (DW Harper; Cord Blood
America)
o Risk Mitigation Strategy
 Move Quickly to Acquire Key Targets : 18 Month Window
CONFIDENTIAL 31

32. Proposal Re Cryo-Banking and
Stem Cell Therapeutics Business
APPENIDX
CONFIDENTIAL 32

33. Cord Blood Transplants Have Doubled In
Last 5 Years (NMDP Data)
CONFIDENTIAL 33

34. Cord Blood Is the Preferred Cell Source
for Pediatric Patients (NMDP Data)
CONFIDENTIAL 34

35. 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
CONFIDENTIAL 35

36. 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
CONFIDENTIAL 36

37. 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)
CONFIDENTIAL 37

38. 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)
CONFIDENTIAL 38

39. 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)
CONFIDENTIAL 39

40. 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
CONFIDENTIAL 40

41. Diseases Treated:
Hematologic Malignancies (NMDP Data)
CONFIDENTIAL 41

42. Diseases Treated:
Non-Malignant Disorders (NMDP Data)
CONFIDENTIAL 42

43. Cord Blood Therapies in Clinical Development:
426 Ongoing FDA Trials
Source: ClinicalTrials.gov (www.clinicaltrials.gov)
CONFIDENTIAL 43

44. 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)
CONFIDENTIAL 44

45. NMDP Data
CONFIDENTIAL 45

46. NMDP Data
CONFIDENTIAL 46

47. NMDP Data
CONFIDENTIAL 47

48. Adult Patients Are the Fastest Growing
Population: 58% 0f 2010 Cord Transplants
CONFIDENTIAL 48

49. Tissue Engineering: Skin Substitute/Wound
Healing Products
EpiCel® CorMatrix ®
Apligraf ®
CONFIDENTIAL 49

50. RM Applications:
Tissue Engineering – Skin & Wound Repair
Other
Company Product Product Type Status Indication Pros/Cons
Information
3 times faster
1st cell-based
healing in
Allogeneic neonatal regenerative
Organo- Diabetic skin difficult
Apligraf® fibroblasts/keratinoc Commercial product approved
genesis ulcers wounds/Most
ytes on scaffold in 1998 in the
expensive,
U.S.
short shelf-life
No
Can be grafted
Abdominal refrigeration or
simultaneously
LifeCell Acellular dermal wall freezing
AlloDerm® Commercial with epithelial
Corporation allograft repair/breast required/Poten
autografts; shelf
reconstruction tial for disease
life 2 yrs
transmission
Autologous
urothelial and 12-month data No
Neo- Phase II Bio-
muscle cells on presented at AUA immunosuppre
Tengion Bladder complete engineered
biodegradable 2009 annual ssive drugs/2
Augment™ (October 2008) organs
scaffold for conference safety events
implantation
Large-scale
Fast Track
production/P3
Osiris Allogeneic adult Phase III designation
Graft versus data: not
Therapeutics, Prochymal® mesenchymal stem complete (May (FDA), Orphan
host disease statistically
Inc. cell therapy 2009) Drug (FDA &
better than
EMEA)
placebo

51. RM Applications: Orthopedic
Company Product Product Type Status Indication Other Information Pros/Cons
More durable hyaline
Autologous
Chondro- EU approval Cartilage 1stapproved cell cartilage*/separate
TiGenix chondrocytes
Celect® (Oct 2009) repair therapy by EMEA periosteal** patch, 4-6
injection
wks culture time
Autologous 3-4 wks culture
Cartilage Cell therapy in use for
Genzyme Carticel® chondrocytes Commercial time/separate
repair 10+ yrs in the U.S.
injection periosteal patch
Aesculap
Autologous Will be a biologic- No periosteal patch/2-
Implant Novocart ® Commercial Cartilage
chondrocytes on device combination step process, biopsy &
Systems, 3D in EU repair
3D bioscaffold product in the U.S. implantation
Inc.
Off-the-shelf product
potential; preliminary
Osiris Allogeneic adult Phase I/II Osteoarthris
Alliance between data: significant pain
Therapeutics Chondrogen® mesenchymal complete and knee
Osiris and Genzyme improvement/in-vitro
, Inc. stem cell therapy (Feb 2008) injury
MSC proliferation
could be limited

52. RM Applications: Cardiovascular
Company Product Product Type Indication Status Other Information Pros/Cons
Easy to use/vascular
Kensey Nash Vascular closure Class III device, in
Angio-Seal™ Arterial seal Commercial complications in 0.2-
Corporation device market since 1996
2% of patients*
Adipose-
derived stem No cell culture; P1
Autologous adult Phase I in EU Official data to be
Cytori and Heart failure met safety &
stem cell therapy (2007) reported in 1Q 2010
regenerative feasibility goal/
cells (ADRCs)
No in-vitro
differentiation; 1-yr
Injection via a data: significant
Advanced catheter,
Autologous Phase I improvement in
Cell
Myoblast skeletal myoblasts Heart failure completed 1-yr data presented tissue regrowth and
Technology,
stem cell therapy (2007) at 2007 AHA annual function/requires
Inc.
meeting tissue culture,
depends on in-vivo
transdifferentiation
*Published data from Kadner K; et. al, Vasc Endovascular Surg 2008; 42; 225
CONFIDENTIAL 52

53. RM Applications: Cardiovascular
Indicatio
Company Product Product Type Status Other Information Pros/Cons
n
No in-vitro
differentiation; 2-yr
P1 data: 6.6 point
Allogeneic adult Intravenous improvement in
Osiris infusion,
mesenchymal Heart Phase II LVEF*; reduction in
Therapeutics, Prochymal®
stem cell failure (May 2009) Phase I data in arrhythmias
Inc.
therapy JACC (May 2009) (p<0.006)/cell
culture needed,
limited in-vitro
MSC propagation
Successful
Intracardiac engraftment and
injection, heart function in
hESC-derived Heart
Geron GRNCM1 Pre-clinical 100% of rats/in-
cardiomyocytes failure Data published in vitro differentiation;
Nat Biot, 2007**
potential for
teratoma
*LVEF: Left Ventricular Ejection Fraction, a measure of overall heart function
**Laflamme MA, et. al, Nature Biotechnology, 2007, 25: 1015-1024
CONFIDENTIAL 53

54. RM Applications: Diabetes
Company Product Product Type Indication Status Other Information Pros/Cons
Phase I/II Results
Human islets Preliminary data
Allogeneic cell Type 1 complete marginal/regular
Novocell encapsulated in presented in 66th
therapy diabetes (started in insulin shots
PEG ADA
2006) needed
Insulin
2-yr drug discovery production in
hESC-derived Allogeneic cell Type 1and 2
Novocell R&D agreement w/ Pfizer mice/tumors
human islets therapy diabetes
(Dec 2008) developed in 5%
of treated mice*
*Nature Reports Stem Cells, 28 May 2009
CONFIDENTIAL 54

55. RM Applications: Diabetes
Company Product Product Type Indication Status Other Information Pros/Cons
Good safety
Collaborative
Allogeneic adult profile to
Osiris Phase II agreement with JCR
mesenchymal Type 1 date/in-vitro
Therapeutics, Prochymal® (started in Pharmaceuticals
stem cell diabetes MSC
Inc. Jun 2008) (2003), Japan &
therapy proliferation
JDRF (2007)
could be limited
Induced Differentiate
Harvard Stem pluripotent Type 1 and 2 Proc Natl Acad Sci mature cells
Cell therapy R&D
Cell Institute stem (iPS) diabetes USA (2009) into beta cells/
cells low efficiency**
** Proceedings of National Academic Sciences USA (Sep 15 2009, Vol 106, 15768-15773)
CONFIDENTIAL 55

56. RM Applications: CNS
Company Product Product Type Indication Status Other Information Pros/Cons
Pre-cut*; bio-
Integra Life Peripheral
Collagen sleeve absorbable; easy
Sciences NeuraWrapTM nerve Commercial
implant placement/repairs <
Corporation protection
30-40mm
55% reduction in
Autologous Phase I/II Increased baseline
Opexa Tovaxin® Multiple relapses/primary and
myelin-reactive T complete disease burden in all
Therapeutics vaccine Sclerosis secondary endpoints
cell therapy (Dec 2008) trial patients
not met
* http://www.podiatrytoday.com/article/8978

57. RM Applications: CNS
Product Indicatio Other
Company Product Status Pros/Cons
Type n Information
IND put on Locomotive function
Spinal
hESC-derived Allogeneic 2nd clinical More preclinical in animal model/high
Geron cord
oligodendrocytes cell therapy hold (Aug studies done frequency of cysts in
injuries
2009)* one preclinical study
Preclinical data
Phase I in J of Comp Neuroprotective effect
Allogeneic
Neuralstem, approved Neu Jun 2009, seen in rats***/in-vitro
Neural stem cells fetal neural ALS
Inc. (December MSC propagation
stem cells Tumor-free
2009) could be limited
pigs**
* http://www.stemcellresearchnews.com/absolutenm/anmviewer.asp?a=1768&z=9
** http://money.cnn.com/2008/04/08/news/companies/stem_cell/index.htm
*** Journal of Comparative Neurology, 514: 297-309 (2009)
CONFIDENTIAL 57

58. RM Applications: Oncology
Cancer Vaccines
• Vaccine that uses greater concentration of tumor antigens to
give a boost to the immune system
Tumor-specific antigens
Cells loaded with tumor-specific antigens
Adoptive T Cell Therapy
• Isolation, ex-vivo expansion and activation, & infusion of T-
cells into patient
Tumor-infiltrating lymphocytes (TIL; lymchocytes inside the tumor)
Engineered/genetically modified T cells
CONFIDENTIAL 58

59. Cancer: Adoptive T-Cell Therapy
(TIL & T-Cells)
Source: J Clin Invest 2007 117:1466-76
CONFIDENTIAL 59

60. RM Applications:
Oncology/Hematology
Company/ Development Other
Product Product Type Indication Pros/Cons
Institute Stage Information
Off-the-shelf
potential; 9.5 mo
Bone-marrow- data: evidence of
Aastrom Data to be
Vascular derived adult stem Vascular Phase II started blood flow and
BioSciences, analyzed in 4Q
repair cells and progenitor regeneration in Apr 2007 wound healing/need
Inc. 2009
cells differentiation; in-
vitro propagation
could be limited
P3 data showed
Autologous
safety; reduced the
dendritic cells Prostate BLA submitted
Dendreon Provenge® GMP facility risk of death by
against prostatic cancer (Nov 2009)
22.5%/treatment
acid phosphatase
repeated 3 times

61. RM Applications:
Oncology/Hematology
Company/ Development
Product Product Type Indication Other Information Pros/Cons
Institute Stage
Tumor-specific T cells, T cell
Tumor-
U.S. National Phase II complete Treatment offered persistence post infusion/30-40%
infiltrating Autologous T cell from
Cancer Melanoma (started in Jun after a brief efficiency in producing TILs; long
lymphocytes melanoma tumor
Institute 2003) chemotherapy culture time (wks); multiple rounds of
(TIL)
T cell activation; requires IL-2 support
No tumor tissue needed; short culture
Designer/ Autologous T cell
time (days); independent of TCR-MHC
Roger William transduced to express Prostate Phase I started Retroviral
Engineered T interaction/in-vivo antigen escape or
Medical Center receptor for PSMA cancer April 2008 transduction
cells poor expression; potential of genetic
antibody
mutagenesis
CONFIDENTIAL 61

62. RM Applications: Eye
Product
Company Product Indication Status Other Information Pros/Cons
Type
100% vision
hESC-derived
improvement in rats, no
Advanced Cell retinal GMP-compliant
Allogeneic Adult macular IND filed ASE*, hESC line w/o
Technology, pigmented RPE cell line
cell therapy degeneration (Nov 2009) embryo
Inc. epithelial (cryopreserved)
destruction/potential for
(RPE) cells
teratomas
hESC-derived
retinal
Vision improvement, no
pigmented
Allogeneic Adult macular Phase I/II in UK in ASE in
Intercytex epithelial Preclinical
cell therapy degeneration late 2010 preclinical/potential for
(RPE) cells on
teratomas
a synthetic
matrix
*ASE = Adverse Safety Event

63. RM Applications:
Aesthetic Medicine
Company Product Product Type Indication Status Other Information Pros/Cons
Significant wrinkle
BLA review in improvement
Fibrocell Autologous cell Facial
Laviv™ progress (due cGMP facility (p<0.0001)/exact
Science therapy wrinkles
Jan 2010) mechanism of action
unknown
Male baldness Phase II Looking for partner Increase in hair
Autologous dermal
Intercytex ICX-TRC and female completed in for further clinical count in 78% of
papilla cell therapy
hair thinning UK (Mar 2008) development subjects/culture time
CONFIDENTIAL 63 Source: Intercytex