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Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
Stem cells usa rm congress   cord blood (boston, september 12, 2011)v.1
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Stem cells usa rm congress cord blood (boston, september 12, 2011)v.1

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  • 10:00 Chairman’s opening remarks: where does cord blood fit into the RM market?• Overview of the regenerative medicine market• Understanding where cord blood fits in the regenerative medicine market• Update on cord blood storage and therapeutics: public, private and research Greg Bonfiglio, Managing Partner, Proteus Venture Partners
  • 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
  • Cell Therapy Revenues (2008–2014): $410M (2008) -- $5.1B (2014); 52.22% CAGR Graph showing the estimated CTI revenues from 2008–2014†, together with the value of the regenerative cell therapy market estimated for 2009–2010, based on actual sales of US FDA/EMA approved products‡.†Data taken from: Evers P: Advances in the Stem Cell Industry. Global Business Insights (2009).‡Data taken from: Mason C, Manzotti E: Regenerative medicine cell therapies: numbers of units manufactured and patients treated between 1988 and 2010. Regen. Med.5(3),307–313 (2010).
  • 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.
  • Dendreon (NASDAQ: DNDN) is a Seattle based biotechnology company. Its lead product, Provenge (known generically as sipuleucel-T), is an immunotherapy for prostate cancer. It consists of a mixture of the patient's own blood cells (autologous, with dendritic cells thought to be the most important) that have been incubated with the Dendreon PAP-GM-CSF fusion protein. Phase III clinical trial results demonstrating a survival benefit for prostate cancer patients receiving the drug were presented at the AUA meeting on 28 April 2009. After going through the approval process, Provenge was given full approval by the FDA on April 29, 2010. Dendreon's name derives from the "Dendritic Cell" which forms a major component of the company's product candidates that use the "Dendreon Cassette Technology" to insert a disease-specific target protein into a general platform. Their lead product, Provenge, is an example of their "rationally designed therapeutic process" intended to break immune tolerance to certain disease specific proteins. It is hypothesized that receptor mediated uptake of antigen by dendritic cells occurs when they are exposed to the Dendreonfusion protein which links the disease specific protein to a recognition protein. This approach is in contrast to other dendritic cell vaccines that use methods such as electroporation to get the DC's to present antigen related epitopes. In the case of Provenge, this disease related protein is Prostatic acid phosphatase and the signalling component is GM-CSF.Initial clinical results for Provenge in 2000 showed immune responses supporting the expected mode-of-action, as well as a PSA reduction which was thought to relate to clinical improvement. In 2006, Dendreon built a manufacturing facility in Morris Plains, New Jersey to accommodate production for a Phase III trial and possible 2007 drug approval by the U.S. Food and Drug Administration (FDA). In January 2007, the FDA accepted Dendreon'sBiologic License Application (BLA) filing for Provenge.On March 29, 2007, the FDA Office of Cellular, Tissue and Gene Therapies Advisory Committee voted 17-0 that Provenge is reasonably safe and 13-4 that the trial data showed substantial evidence that it is effective. However, on May 9, 2007, Dendreon received a letter from the FDA demanding more results and information before approval.On April 14, 2009, Dendreon announced that the results for the Phase III trial of Provenge were positive, saying there had been a reduction in the odds of death compared to the use of a placebo. On April 28, 2009, the full details of the study were released. The trial found that patients treated with Provenge lived an average of 4.1 months longer than patients treated with the control (autologous cells without the GM-CSF / PAP fusion protein).On April 29, 2010, the FDA approved Provenge for use in the treatment of advanced prostate cancer.
  • A search for “Cord Blood” on the FDA Website re Clinical Trials (http://clinicaltrials.gov/) nets 619 trials – 425+ involving cord blood therapeutics.
  • 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 619 trials – 425+ 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.
  • 400+ 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
  • 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.
  • 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)
  • 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.
  • 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.
  • Genzyme-Osiris Deal (November 2008): $1.25B value; $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 Dendreon’sProvenge was approved for Prostate Cancer in April 2010$97K – approved reimbursementProjected Revenues: $1-3B DNDN Market Cap: $5B+Cephalon & Mesoblast: $2B Deal in December 2010Cephalon buying 19.99% of Mesoblastfor $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‘s
  • Transcript

    • 1. Stem Cells USA RM Congress:Cord Blood Banking and TherapeuticsBoston, MassachusettsGregory A. BonfiglioProteus Venture PartnersSeptember 12, 2011
    • 2. Proteus: An Investment and AdvisoryFirm Focused on Regenerative Medicine Proteus, Inc. Proteus Proteus Proteus Management, LLC Insights, LLC Advisors, LLC (Fund Management) (Consulting Services) (Investment Banking Services) CONFIDENTIAL 2
    • 3. AgendaI. The Regenerative Medicine Market  Key Market Metrics  RM Therapeutic ApplicationsII. The Cord Blood Market  Key Market Metrics  Banking Other TissuesIII. Therapeutic Applications for Cord Blood  Key Transplant Metrics  Current Treatments  Therapies in Clinical DevelopmentCONFIDENTIAL 3
    • 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 CONFIDENTIAL 4
    • 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 CONFIDENTIAL 5
    • 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 CONFIDENTIAL 6
    • 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. 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. 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 CONFIDENTIAL 9
    • 10. AgendaI. The Regenerative Medicine Market  Broad RM Market: Key Metrics  RM Therapeutic ApplicationsII. The Cord Blood Market  Cord Blood Market: Key Metrics  Banking Other TissuesIII. Therapeutic Applications for Cord Blood  Key Transplant Metrics  Current Treatments  Therapies in Clinical DevelopmentCONFIDENTIAL 10
    • 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 CONFIDENTIAL 11
    • 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 CONFIDENTIAL 12
    • 13. Cord Blood Therapies in Clinical Development:600+ Ongoing FDA Trials Source: ClinicalTrials.gov (www.clinicaltrials.gov) CONFIDENTIAL 13
    • 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)CONFIDENTIAL 14
    • 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)CONFIDENTIAL 15
    • 16. AgendaI. The Regenerative Medicine Market  Key Market Metrics  RM Therapeutic ApplicationsII. The Cord Blood Market  Key Market Metrics  Banking Other TissuesIII. Therapeutic Applications for Cord Blood  Key Transplant Metrics  Current Treatments  Therapies in Clinical DevelopmentCONFIDENTIAL 16
    • 17. 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 17
    • 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 GraftCord 550,000 None 4/6 1 Day Yes 26 Days Minimal EasierBlood Units PossibleBone 8/8 14.6M 3-4 if donor 50% 18 Days 50% DifficultMarrow 10/10 Donors Months is available Confidential 18
    • 19. Cord Blood Transplants Have Doubled InLast 5 Years (NMDP Data)CONFIDENTIAL 19
    • 20. Cord Blood Is the Preferred Cell Sourcefor Pediatric Patients (NMDP Data)CONFIDENTIAL 20
    • 21. 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 21
    • 22. 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 22
    • 23. 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 23
    • 24. 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 24
    • 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) CONFIDENTIAL 25
    • 26. 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 26
    • 27. Diseases Treated:Hematologic Malignancies (NMDP Data)CONFIDENTIAL 27
    • 28. Diseases Treated:Non-Malignant Disorders (NMDP Data)CONFIDENTIAL 28
    • 29. 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 29
    • 30. APPENIDXCONFIDENTIAL 30
    • 31. NMDP DataCONFIDENTIAL 31
    • 32. NMDP DataCONFIDENTIAL 32
    • 33. NMDP DataCONFIDENTIAL 33
    • 34. Adult Patients Are the Fastest GrowingPopulation: 58% 0f 2010 Cord Transplants CONFIDENTIAL 34
    • 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 MuscleCONFIDENTIAL 35
    • 36. Gartner’s Hype Cycle of Emerging TechnologiesVisibility Technology Peak of Inflated Trough of Slope of Plateau of Productivity Trigger Expectations Disillusionment Enlightment Time CONFIDENTIAL 36
    • 37. RM Market: On 2nd Half of the Gartner CurveVisibility 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 CONFIDENTIAL 37

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