Proposal: Build The Leading International Cryo-Banking and Stem Cell Therapeutics CompanySeptember, 2011Gregory A. Bonfigl...
Proteus: An Investment and AdvisoryFirm Focused on RM                       Proteus, Inc.       Proteus               Prot...
AgendaI.   The Market & Opportunity          Broader Regenerative Medicine Market          Cord Blood Market: Key Metric...
The Promise of Regenerative Medicine    To Cure Diabetes                      To Cure Vision Disorders        To Heal Acut...
Development of Regenerative Medicine Current technologies build on 50 years of research       1960s                       ...
RM Is Entering A New ERA                     RM Market is Maturing: Key Metrics     Rapidly Expanding Market:            C...
RM Market: Global Company DistributionCanada                   UK24 firms                 133 firms           Europe      ...
Big Pharma is Actively EngagedGSK & HSCI: $25M Deal                                      Merck, Pfizer & Lily Launch Enlig...
Cord Blood Market:Key Metrics                     Cord Blood Market Metrics      Market Size:                      Market ...
Cord Blood Market:Key Metrics                     Cord Blood Market Metrics     Total Cord Blood Transplants:      Regulat...
Cord Blood Therapeutics:Comparison with Bone Marrow                                  Cord Blood v. Bone Marrow            ...
Banking Other Tissues• Wharton’s Jelly/Entire Cord    • CHORI Technology (www.chori.org)• Placental Tissue (MSCs)    • Plu...
Banking Other Tissues• Peripheral Blood/Bone Marrow    • Stem Cell Assurance, Inc. (www.stemcellassurance.com)    • Oriste...
Current Therapeutic Applicationsfor Cord Blood Market                                    Key Metrics             • 25,000 ...
AgendaI.   The Market & Opportunity          Broader Regenerative Medicine Market          Cord Blood Market: Key Metric...
Build A Dominant International Cryo-Banking Company: Strategic Plan#1: Acquire a Market Leader         • Acquisition Candi...
Build A Dominant International Cryo-Banking Company: Strategic Plan#2: Roll-Up Small Underperforming Banks         • 20-25...
Build A Dominant International Cryo-Banking Company: Strategic Plan#3: Drive Revenues and Reduce Expenses         • Drive ...
Build A Dominant International Cryo-Banking Company: Strategic Plan#3: Drive Revenues and Reduce Expenses         • Reduce...
Build A Dominant International Cryo-Banking Company: Strategic Plan#4: Expand Tissue Offering    •   New Tissue Candidates...
AgendaI.   The Market & Opportunity          Broader Regenerative Medicine Market          Cord Blood Market: Key Metric...
Phase II: Bring RM Therapeutics toEmerging Markets               Selection Criteria for New RM Therapies• Leverage Existin...
Phase II: Bring RM Therapeutics toEmerging Markets                    Potential RM Therapies (Near Term)• Cord Blood      ...
Phase II: Bring RM Therapeutics toEmerging Markets               Potential RM Therapies - Aesthetic Medicine• MSCs (Adipos...
Phase II: Bring RM Therapeutics toEmerging Markets                    Potential RM Therapies (Mid Term)•   Engineered Skin...
Phase II: Bring RM Therapeutics toEmerging Markets                       Potential Therapies (Long Term)• Diabetes• Other ...
AgendaI.   The Market & Opportunity          Broader Regenerative Medicine Market          Cord Blood Market: Key Metric...
Funding & Exit StrategyFunding Required: $75M    • Use of Proceeds:          Acquisition of Market Leader          Execu...
Funding & Exit StrategyExit Strategy: IPO in 24-36 Months    • Drive Revenues to $100M-$150M          Revenue Ramp of 25%...
MAJOR RISKS• Risk: Decline In Market Price for Public Units Due to Increase in  Supply               • 550,000 Units now; ...
MAJOR RISKS• Risk: Failure to Execute               • Typical Roll-Up Execution Risk    o Risk Mitigation Strategy        ...
Proposal Re Cryo-Banking andStem Cell Therapeutics Business                     APPENIDX CONFIDENTIAL   32
Cord Blood Transplants Have Doubled InLast 5 Years (NMDP Data)CONFIDENTIAL   33
Cord Blood Is the Preferred Cell Sourcefor Pediatric Patients (NMDP Data)CONFIDENTIAL   34
Adult Patients Are the Fastest GrowingPopulation: 58% 0f 2010 Cord Transplants           Cord Blood Transplants By Patient...
Transplant Outcomes Have ImprovedDramatically in Last 5 Years       Report Year              Period              One-Year ...
Clinical Outcomes Related toQuality of Tissue & Match                               Key Metrics                 Quality of...
Cord Shipments By TNC Count for Adults1,2001,000 800                                                                      ...
Public Bank Utilization Rates: 2010 Inventory and Shipments     TNC of Shipments -                TNC of Inventory as of  ...
Current Therapeutic Applicationsfor Cord Blood                           60+ Diseases Treated             • Leukemias And ...
Diseases Treated:Hematologic Malignancies (NMDP Data)CONFIDENTIAL   41
Diseases Treated:Non-Malignant Disorders (NMDP Data)CONFIDENTIAL   42
Cord Blood Therapies in Clinical Development:426 Ongoing FDA Trials                     Source: ClinicalTrials.gov (www.cl...
FDA Trials Involving Cord Blood AddressNumerous Conditions & Diseases     •     Bacterial and Fungal Diseases         •   ...
NMDP DataCONFIDENTIAL   45
NMDP DataCONFIDENTIAL   46
NMDP DataCONFIDENTIAL   47
Adult Patients Are the Fastest GrowingPopulation: 58% 0f 2010 Cord Transplants  CONFIDENTIAL   48
Tissue Engineering: Skin Substitute/WoundHealing Products                     EpiCel®                CorMatrix ®          ...
RM Applications:Tissue Engineering – Skin & Wound Repair                                                                  ...
RM Applications: Orthopedic Company         Product      Product Type         Status      Indication      Other Informatio...
RM Applications: Cardiovascular Company             Product        Product Type          Indication            Status     ...
RM Applications: Cardiovascular                                                           Indicatio Company            Pro...
RM Applications: DiabetesCompany             Product      Product Type    Indication     Status       Other Information   ...
RM Applications: Diabetes Company              Product      Product Type         Indication        Status          Other I...
RM Applications: CNS Company            Product          Product Type       Indication     Status      Other Information  ...
RM Applications: CNS                                            Product       Indicatio                        OtherCompan...
RM Applications: OncologyCancer Vaccines• Vaccine that uses greater concentration of tumor antigens to  give a boost to th...
Cancer: Adoptive T-Cell Therapy(TIL & T-Cells)                      Source: J Clin Invest 2007 117:1466-76  CONFIDENTIAL  ...
RM Applications: Oncology/Hematology Company/                                                         Development         ...
RM Applications: Oncology/Hematology Company/                                                                Development  ...
RM Applications: Eye                                  Product Company          Product                        Indication  ...
RM Applications: Aesthetic MedicineCompany         Product        Product Type          Indication         Status         ...
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Proposal for cord blood banking and therapeutics business (roll up strategy september 2011)v 6

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  • TriMark: $3 billion in 2010; expected to reach $4.5 billion by 2014 = 10.67% CAGRScientia Advisors: $1.6 billion in 2010; expected to be $15-20 billion by 2025 = 18.34% CAGR Visiongain: $0.82B in 2010; excepted to reach $8.84B in 2021 = 24.13% CAGR Dendreon: $5.7B Market CapDr. Alain Vertes (Roche) RM Market: $410M (2008); $2.6B (2012); $5.0B (2014) [51.7% CAGR]Chris Mason Cell Therapy Market: $200M (2009); 323,000 Patients treated with Cell Therapies FDA Website (July 2010): 3100+ trials involving “stem cells” Vast majority are in cancer: 2270 lukemia (1129) + lymphoma (1149) 150+ studies in Cardio 2121 involve hematopoietic stem cell transplant
  • November 2008: Genzyme & Osiris: $1.25B Deal $130 million upfront with $100Ms in milestone payments; Covers Prochymal and Chondrogen;Osiris will commercialize both drugs in the U.S. and Canada, and Genzyme will sell the drugs in all other countries December 2009: Athersys & Pfizer/Neucentis: $111M Deal $6M Upfront; remainder in milestones License rights to develop Athersys' MultiStem cell therapy for inflammatory bowel disease. Dec 2010: Cephalon & Mesoblast: $2B Deal - - 2nd Largest Deal in Biotech in 2010Cephalon buying 19.99% of Mesoblast for $223 millionCephalon paying $130 million upfront fee $1.7 billion in milestone payments: RE MSC therapies for congestive heart failure, acute myocardial infarction, Parkinson's disease, and Alzheimer‘sCephalon now being Acquired by Teva, inpartot get access to Mesoblasts’s technologyMay 2011 Shire Acquires Advanced Biohealing for $750M ABH had $150M in revenue/$115M Gross Profit from Dermagraft; July 2010: JDRF and Sanofi announced a partnership to co-fund diabetes researchCytori & GE Healthcare: Partnership to distribute Cytori’s devices, and Stem Cell Banking Novo Nordisk: pursuing both Adult (Allocure – Acute Kidney Injury) & hESC (Cellartis - Diabetes)July 1, 2010 – Sanofi-aventis (EURONEXT: SAN and NYSE: SNY ) and the Juvenile Diabetes Research Foundation (JDRF) announced today a unique partnership to develop therapeutic treatments for people with type 1 diabetes at different stages of the disease – both those living with the disease and the newly diagnosed – as well as preventing diabetes in those at risk.  Toward those goals, the partnership will focus on therapeutics such as immune therapies and beta cell regeneration. Under the newly announced partnership, sanofi-aventis and JDRF will jointly provide academic investigators and non-profit medical research organizations with funding to conduct research projects in regeneration and immune therapy. This partnership will provide sanofi-aventis with options to the intellectual property developed by researchers who receive funding through the program.
  • A search for “Cord Blood” on the FDA Website re Clinical Trials (http://clinicaltrials.gov/) nets 538 trials – 396 involving cord blood transplants.
  • The National Marrow Donor Program estimates that by the year 2015, there will be 10,000 cord blood transplants worldwide per year using publicly banked cord blood. In 2009, 1,056 cord blood transplants were facilitated by the NMDP, which represents 22% of the total number of NMDP transplants in 2009. This is an 18% increase from 2008, when the NMDP facilitated 898 cord blood transplants. In the United States, the Food and Drug Administration regulates cord blood under the category of “Human Cells, Tissues, and Cellular and Tissue Based-Products.” (CFR: Title 21 Section 1271) Both public and private cord blood banks are eligible for voluntary accreditation with either the American Association of Blood Banks (“AABB”) or the Foundation for the Accreditation of Cellular Therapy (“FACT”). Other countries also have regulations pertaining to cord blood.In 2005, the National Academy of Sciences published an Institute of Medicine (IoM) report titled, "Establishing a National Cord Blood Stem Cell Bank Program". The IoM report recommended that expectant parents be given a balanced perspective on their options for cord blood banking, and options for donating, discarding or banking lifesaving newborn stem cells. Currently 17 states, covering two-thirds of U.S. births, have enacted legislation recommended by the IoM guidelines.
  • Bone Marrow/PBSC from a Matched Sibling Donor is the 1st Choice for most Therapeutic Applications (20% of cases). Cord Blood is rapidly becoming the preferred tissue if a Matched Sibling Donor is not available (80% of cases)
  • The placenta is a good source of stem cells since it contains up to ten times more stem cells than cord blood. Some placental blood may be returned to the neonatal circulation if the umbilical cord is not prematurely clamped. LifeLine Cryogenics (www.lifelinecryogenics.com) is a broad spectrum cryogenics company in business since 1991, offering storage of sperm, eggs, ovarian tissue, embryos, and cord blood.
  • Adipose Derived MSCs: Celution® 800/CRS Device
  • In 2010, more than 1,150 cord blood transplants were facilitated by the NMDP, which represents 22% of the total number of NMDP transplants in 2010.  Cord blood transplants grew at a rate of 9% compared to 1% for marrow, 11% for PBSC; and a 9% overall increase in the number of transplants.Approximately 25,000 allogeneic hematopoietic cell transplants (bone marrow, PBSC, or cord blood transplants — BMT) are performed annually worldwide, and approximately 5,200 patients are transplanted annually using unrelated donors or cord blood units through the NMDP.Since it began operations in 1987, the NMDP has facilitated more than 43,000 marrow and cord blood transplants. In 2010, the NMDP facilitated twice as many transplants as it did five years ago.
  • The New Economic Reality Public Markets: A World Without Biotech IPOs Venture Capital: Only “Pristine” Deals Need ApplyAlternate Funding SourcesII.Crossing the Valley Of Death With Friends The Relay Model Is Broken Capital Efficiency Is Critical A New Collaborative Model Macro Drivers Venture Capital: Only “Pristine” Deals Need Apply
  • The New Economic Reality Public Markets: A World Without Biotech IPOs Venture Capital: Only “Pristine” Deals Need ApplyAlternate Funding SourcesII.Crossing the Valley Of Death With Friends The Relay Model Is Broken Capital Efficiency Is Critical A New Collaborative Model Macro Drivers Venture Capital: Only “Pristine” Deals Need Apply
  • The New Economic Reality Public Markets: A World Without Biotech IPOs Venture Capital: Only “Pristine” Deals Need ApplyAlternate Funding SourcesII.Crossing the Valley Of Death With Friends The Relay Model Is Broken Capital Efficiency Is Critical A New Collaborative Model Macro Drivers Venture Capital: Only “Pristine” Deals Need Apply
  • This chart shows the growing use of PBSC and cord blood as graft sources in allogeneic transplantation. In 2010, 76% of adult donors – more than 3,100 – donated PBSC to patients through the NMDP. In 2010, more than 1,150 cord blood transplants were facilitated by the NMDP, which represents 22% of the total number of NMDP transplants in 2010. In 2010, cord blood transplants grew at a rate of 9% compared to 1% for marrow, 11% for PBSC; and a 9% overall increase in the number of transplants.
  • This chart shows that cord blood is used more often in pediatric patients than in adult patients. However, recent studies have demonstrated that this stem cell source can be successfully used in adults and so its use is growing in this patient population. BSC is used less frequently in pediatric patients undergoing allogeneic transplantation than in adult patients, which is due to poorer outcomes in children receiving PBSC transplants.
  • This increase is due in large part to the increased use of non-myeloablative or reduced-intensity transplants that have expanded transplant therapy to older patients who would otherwise be excluded from this therapy. This chart shows the increased number of older patients (>50 years) who have received cord blood transplants through the NMDP. In 2010, 58% of NMDP cord transplants.In 2000-2004, most transplants benefitted pediatric patients. Starting in 2005, adult patients have comprised an ever-growing percent of total cord transplant recipients, to represent 54% of all patients in both 2008 and 2009, and 58% in 2010.
  • A major reason for the continued increase in unrelated donor transplantation is the steady improvement in transplant outcomes. This is in turn due to several clinical advances such as more precise HLA typing and advances in patient care.  This chart shows that NMDP transplant outcomes have improved more than 10% in just five years.Recent studies have demonstrated that unrelated donor transplant outcomes are now comparable to related donor transplant outcomes in several patient populations. In addition, analyses of NMDP transplants from 1987-2006 have shown that survival has consistently, and sometimes dramatically, improved over time in each major disease category.
  • 77% of all shipments for adults in 2010 had TNC >150, compared to 65% in 2005.
  • Diseases treatableLeukemias and lymphomas, including:Acute myelogenous leukemia Acute lymphoblastic leukemia Chronic myelogenous leukemia Chronic lymphocytic leukemia Juvenile myelomonocytic leukemia Hodgkin lymphoma Non-Hodgkin lymphoma Multiple myeloma and other plasma cell disordersSevere aplastic anemia and other marrow failure states, including:Severe aplastic anemia Fanconi anemia Paroxysmal nocturnal hemoglobinuria (PNH) Pure red cell aplasiaAmegakaryocytosis / congenital thrombocytopenia 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 Hurler's syndrome and other inherited metabolic disorders, including:Hurler's syndrome (MPS-IH) AdrenoleukodystrophyMetachromaticleukodystrophyMyelodysplastic and myeloproliferative disorders, including:Refractory anemia (all types) Chronic myelomonocytic leukemia Agnogenic myeloid metaplasia (myelofibrosis)
  • This chart shows that the number of transplants for AML, ALL, MDS, and lymphoma have also increased significantly. The advent of reduced-intensity or non-myeloablative transplants is also a prime reason for this increase. In addition, improved HLA matching, advances in conditioning regimens, advances in post-transplant supportive care -- including improved management of GVHD, CMV disease, and infections -- have also contributed to the growing number of allogeneic transplants in general.
  • Figure 2 shows that NMDP transplants for non-malignant diseases has grown for most diseases, with the most rapid growth being seen in severe aplastic anemia (SAA). This follows the trend seen in the related-donor setting, where allogeneic transplantation is the preferred first-line treatment for SAA patients <40 years old who have a matched related donor. Survival of unrelated donor transplantation in patients with SAA is now approaching 60%. Use of unrelated donor transplantation in SAA may continue to grow following the 2007 publication of a study showing that allogeneic transplantation is better than immunosuppressive therapy as second-line treatment in pediatric SAA. The increase in allogeneic transplantation for many other non-malignant diseases can also be attributed to improved HLA matching, advances in conditioning regimens and advances in post-transplant supportive care.
  • Number of Studies Worldwide: 426Africa 15; Central America   7; East Asia   36; Japan 2;   Europe  72; Middle East 14; North America 387; Canada 33;   Mexico 3;   United States  373;  Pacifica   16; South America   6; South Asia 6; Southeast Asia   6 396 FDA Clinical Studies Involve New Therapies: (by Category)Bacterial and Fungal DiseasesBehaviors and Mental DisordersBlood and Lymph ConditionsCancers and Other NeoplasmsDigestive System DiseasesDiseases and Abnormalities at or before BirthEar, Nose, and Throat DiseasesGland and Hormone Related DiseasesHeart and Blood DiseasesImmune System DiseasesInjuries, Poisonings, and Occupational ConditionsMuscle, Bone, and Cartilage DiseasesNervous System DiseasesNutritional and Metabolic DiseasesParasitic DiseasesRespiratory Tract (Lung and Bronchial) DiseasesSkin and Connective Tissue DiseasesSymptoms and General PathologyUrinary Tract, Sexual Organs, and Pregnancy ConditionsViral Diseases
  • 2010 – 2015: Cord Blood Transplant CAGR = 49.3% The number of Cord Blood Transplants DOUBLED (2x) from 2005-2010The National Marrow Donor Program estimates that by the year 2015, there will be 10,000 cord blood transplants worldwide per year using publicly banked cord blood. In 2009, 1,056 cord blood transplants were facilitated by the NMDP, which represents 22% of the total number of NMDP transplants in 2009. This is an 18% increase from 2008, when the NMDP facilitated 898 cord blood transplants. A search for “Cord Blood” on the FDA Website re Clinical Trials (http://clinicaltrials.gov/) nets 601 trials – 426 involving cord blood therapeutics. In the United States, the Food and Drug Administration regulates cord blood under the category of “Human Cells, Tissues, and Cellular and Tissue Based-Products.” (CFR: Title 21 Section 1271) Both public and private cord blood banks are eligible for voluntary accreditation with either the American Association of Blood Banks (“AABB”) or the Foundation for the Accreditation of Cellular Therapy (“FACT”). Other countries also have regulations pertaining to cord blood.In 2005, the National Academy of Sciences published an Institute of Medicine (IoM) report titled, "Establishing a National Cord Blood Stem Cell Bank Program". The IoM report recommended that expectant parents be given a balanced perspective on their options for cord blood banking, and options for donating, discarding or banking lifesaving newborn stem cells. Currently 17 states, covering two-thirds of U.S. births, have enacted legislation recommended by the IoM guidelines.
  • This chart shows the increased number of older patients (>50 years) who have received cord blood transplants through the NMDP. In 2010, 58% of NMDP cord transplants.This increase is due in large part to the increased use of non-myeloablative or reduced-intensity transplants that have expanded transplant therapy to older patients who would otherwise be excluded from this therapy.
  • Proposal for cord blood banking and therapeutics business (roll up strategy september 2011)v 6

    1. 1. Proposal: Build The Leading International Cryo-Banking and Stem Cell Therapeutics CompanySeptember, 2011Gregory A. BonfiglioProteus Venture Partners
    2. 2. Proteus: An Investment and AdvisoryFirm Focused on RM Proteus, Inc. Proteus Proteus Proteus Management, LLC Insights, LLC Advisors, LLC (Fund Management) (Consulting Services) (Investment Banking Services)CONFIDENTIAL 2
    3. 3. AgendaI. The Market & Opportunity  Broader Regenerative Medicine Market  Cord Blood Market: Key Metrics & Market Leaders  The OpportunityII. 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 OfferingIII. 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. 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. 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. 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. 7. RM Market: Global Company DistributionCanada UK24 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. 8. Big Pharma is Actively EngagedGSK & HSCI: $25M Deal Merck, Pfizer & Lily Launch Enlight BioSciencePfizer RM Division: $111M Deal withAthersys (IBD); UCL (RPE); ViaCyte(Diabetes) GSK, AZN & Roche Help Launch Stem Cells for Safer Medicine in UKGenzyme & Osiris: $1.25B Deal Johnson & Johnson Invests in Tengion & ViaCyte (NovoCell)Cephalon & Mesoblast: $2B Deal GE & Cytori: StemSourceShire Acquires ABH for $750M CONFIDENTIAL 8
    9. 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. 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. 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 GraftCord 550,000 None 4/6 1 Day Yes 26 Days Minimal EasierBlood Units PossibleBone 14.6M 3-4 if donor 50% 8/8 18 Days 50% DifficultMarrow Donors Months is available Confidential 11
    12. 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. 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. 14. Current Therapeutic Applicationsfor 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. 15. AgendaI. The Market & Opportunity  Broader Regenerative Medicine Market  Cord Blood Market: Key Metrics & Market Leaders  The OpportunityII. 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 OfferingIII. 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. 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 IndiaCONFIDENTIAL 16
    17. 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/RegionCONFIDENTIAL 17
    18. 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. 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 MarketsCONFIDENTIAL 19
    20. 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. 21. AgendaI. The Market & Opportunity  Broader Regenerative Medicine Market  Cord Blood Market: Key Metrics & Market Leaders  The OpportunityII. 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 OfferingIII. 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. 22. Phase II: Bring RM Therapeutics toEmerging 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 SizeCONFIDENTIAL 22
    23. 23. Phase II: Bring RM Therapeutics toEmerging 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 RestorationCONFIDENTIAL 23
    24. 24. Phase II: Bring RM Therapeutics toEmerging 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 RejuvenationCONFIDENTIAL 24
    25. 25. Phase II: Bring RM Therapeutics toEmerging 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; RPEsCONFIDENTIAL 25
    26. 26. Phase II: Bring RM Therapeutics toEmerging Markets Potential Therapies (Long Term)• Diabetes• Other Autoimmune Diseases (MS; Lupus; Crohn’s)• CNS Disorders  Parkinsons  Alzheimers  Stroke• Spinal Cord Repair• Tissue & Organs  Bladder  Ligaments; Veins; Valves  CartilageCONFIDENTIAL 26
    27. 27. AgendaI. The Market & Opportunity  Broader Regenerative Medicine Market  Cord Blood Market: Key Metrics & Market Leaders  The OpportunityII. 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 OfferingIII. 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. 28. Funding & Exit StrategyFunding 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 TherapiesCONFIDENTIAL 28
    29. 29. Funding & Exit StrategyExit 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 TherapiesCONFIDENTIAL 29
    30. 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 SimultaneouslyCONFIDENTIAL 30
    31. 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 WindowCONFIDENTIAL 31
    32. 32. Proposal Re Cryo-Banking andStem Cell Therapeutics Business APPENIDX CONFIDENTIAL 32
    33. 33. Cord Blood Transplants Have Doubled InLast 5 Years (NMDP Data)CONFIDENTIAL 33
    34. 34. Cord Blood Is the Preferred Cell Sourcefor Pediatric Patients (NMDP Data)CONFIDENTIAL 34
    35. 35. Adult Patients Are the Fastest GrowingPopulation: 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. 36. Transplant Outcomes Have ImprovedDramatically 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. 37. Clinical Outcomes Related toQuality 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. 38. Cord Shipments By TNC Count for Adults1,2001,000 800 150+ 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. 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. 40. Current Therapeutic Applicationsfor 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. 41. Diseases Treated:Hematologic Malignancies (NMDP Data)CONFIDENTIAL 41
    42. 42. Diseases Treated:Non-Malignant Disorders (NMDP Data)CONFIDENTIAL 42
    43. 43. Cord Blood Therapies in Clinical Development:426 Ongoing FDA Trials Source: ClinicalTrials.gov (www.clinicaltrials.gov) CONFIDENTIAL 43
    44. 44. FDA Trials Involving Cord Blood AddressNumerous 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. 45. NMDP DataCONFIDENTIAL 45
    46. 46. NMDP DataCONFIDENTIAL 46
    47. 47. NMDP DataCONFIDENTIAL 47
    48. 48. Adult Patients Are the Fastest GrowingPopulation: 58% 0f 2010 Cord Transplants CONFIDENTIAL 48
    49. 49. Tissue Engineering: Skin Substitute/WoundHealing Products EpiCel® CorMatrix ® Apligraf ® CONFIDENTIAL 49
    50. 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 epithelialCorporation 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: notTherapeutics, Prochymal® mesenchymal stem complete (May (FDA), Orphan host disease statistically Inc. cell therapy 2009) Drug (FDA & better than EMEA) placebo
    51. 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 painTherapeutics Chondrogen® mesenchymal complete and knee Osiris and Genzyme improvement/in-vitro , Inc. stem cell therapy (Feb 2008) injury MSC proliferation could be limited
    52. 52. RM Applications: Cardiovascular Company Product Product Type Indication Status Other Information Pros/Cons Easy to use/vascularKensey 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 andTechnology, 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. 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 inTherapeutics, 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. 54. RM Applications: DiabetesCompany Product Product Type Indication Status Other Information Pros/Cons Phase I/II Results Human islets Preliminary data Allogeneic cell Type 1 complete marginal/regularNovocell 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 2Novocell 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. 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-vitroTherapeutics, Prochymal® (started in Pharmaceuticals stem cell diabetes MSC Inc. Jun 2008) (2003), Japan & therapy proliferation JDRF (2007) could be limited Induced DifferentiateHarvard Stem pluripotent Type 1 and 2 Proc Natl Acad Sci mature cells Cell therapy R&DCell 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. 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 allTherapeutics vaccine Sclerosis secondary endpoints cell therapy (Dec 2008) trial patients not met * http://www.podiatrytoday.com/article/8978
    57. 57. RM Applications: CNS Product Indicatio OtherCompany 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 AllogeneicNeuralstem, 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. 58. RM Applications: OncologyCancer 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 antigensAdoptive 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. 59. Cancer: Adoptive T-Cell Therapy(TIL & T-Cells) Source: J Clin Invest 2007 117:1466-76 CONFIDENTIAL 59
    60. 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 andBioSciences, 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. 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-MHCRoger William transduced to express Prostate Phase I started Retroviral Engineered T interaction/in-vivo antigen escape orMedical Center receptor for PSMA cancer April 2008 transduction cells poor expression; potential of genetic antibody mutagenesis CONFIDENTIAL 61
    62. 62. RM Applications: Eye Product Company Product Indication Status Other Information Pros/Cons Type 100% vision hESC-derived improvement in rats, noAdvanced 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. 63. RM Applications: Aesthetic MedicineCompany Product Product Type Indication Status Other Information Pros/Cons Significant wrinkle BLA review in improvementFibrocell Autologous cell Facial Laviv™ progress (due cGMP facility (p<0.0001)/exactScience therapy wrinkles Jan 2010) mechanism of action unknown Male baldness Phase II Looking for partner Increase in hair Autologous dermalIntercytex 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

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