NYSSA Conference Presentation: B. Caldwell

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12/6/2006

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NYSSA Conference Presentation: B. Caldwell

  1. 1. Translating Stem Cell Research intoCommercial ProductsTranslating Stem Cell Research intoCommercial ProductsDecember 2006December 2006
  2. 2. 2This presentation contains “forward-looking statements” as defined under thefederal securities laws. Actual results could vary materially. Factors that couldcause actual results to vary materially are described in our filings with the Securitiesand Exchange Commission.You should pay particular attention to the “risk factors” contained in documents wefile from time to time with the Securities and Exchange Commission. The risksidentified therein, as well as others not identified by the Company, could cause theCompany’s actual results to differ materially from those expressed in any forward-looking statements.Cautionary Statement ConcerningForward-Looking StatementsCautionary Statement ConcerningForward-Looking Statements
  3. 3. Advanced Cell TechnologyAdvanced Cell Technology
  4. 4. 4BackgroundBackgroundScientific Leader in Regenerative MedicineFirst Proven Alternative Method for Deriving Human Embyronic Stem CellsDriving Human ES Cell Therapies to the ClinicFocus on RPE, Hemangioblast and Dermal Product ProgramsProprietary Human ES Cell TechnologyOver 300 Patents and Patent ApplicationsAward Winning Team of Scientists and ManagementDr. Mike West and Dr. Robert Lanza are Recognized Thought Leaders
  5. 5. 6
  6. 6. The Vision of Advanced CellTechnologyThe Vision of Advanced CellTechnology
  7. 7. 8Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
  8. 8. 9Potential of ACT Embryonic Stem CellsPotential of ACT Embryonic Stem CellsUnlimited Expansion of Stem Cell SourceMaster Cell BankMaster Cell BankWorking CellWorking CellBankBankBank of Differentiated CellsBank of Differentiated CellsBank of Differentiated CellsBank of Differentiated CellsBank of Differentiated CellsBank of Differentiated CellsSingle BlastomereSingle Blastomere
  9. 9. 10Commercialization of ACT TherapiesCommercialization of ACT TherapiesDermalDermalRetinalRetinalPigmentedPigmentedEpitheliumEpitheliumHemangioblastHemangioblastCryopreserved Cell BankCryopreserved Cell BankWorking Cell BankWorking Cell BankCryopreserved Cell BankCryopreserved Cell BankCryopreserved Cell BankCryopreserved Cell Bank
  10. 10. 11Our Product Development PathwayOur Product Development PathwayCell Discovery and ValidationFinding Cell Types with Therapeutic PotentialSelect Target Indications“GMP” Cell ProductionDerive Human ES Cells under GMPDifferentiate Target Cell Type under GMPPreclinical and Clinical TestingFunctional and Safety Preclinical TestingDevelop Plan with FDA for Human Clinical Trialstarget cell typestarget cell typestarget cell linestarget cell lines20062006200720072004200420052005
  11. 11. 12Stem Cell Therapy: The MarketplaceStem Cell Therapy: The Marketplace$30 billion estimated market for successful stem celltherapiesTotal costs of some major illnesses:Heart Disease - $175 billionDiabetes - $98 billionAlzheimer’s - $87 billionVision Disorders - $68 billionStroke - $43 billion
  12. 12. 13Regenerative MedicineCuring Devastating DiseaseRegenerative MedicineCuring Devastating Disease800,000Parkinsons patients 750,000liver disease patients20,000,000heart disease patients20,000,000 diabetespatients5,000,000epilepsy patients4,000,000Alzheimers patients2,000,000stroke patients300,000kidney disease patients
  13. 13. Driving Human ES CellTherapies to the ClinicDriving Human ES CellTherapies to the Clinic
  14. 14. 15Our Clinical Testing PathwayOur Clinical Testing PathwayResearchDevelopmentProcess ValidationProcess UnderstandingPre-Clinical TestingClinical Testing- Phase 1Clinical Testing- Phase 2Clinical Testing- Phase 3Commercialization (Phase 4) MarketingBLAINDAmendmentsUpdatesLettersSubmissions
  15. 15. 16Our GMP StrategyOur GMP StrategyWorcester GMP Lab – utilize for pilot mfg of differentiated cell types(RPE, Hemangioblasts, etc.) for pharm/tox studies and Phase IstudiesAlameda GMP Lab – utilize for larger scale mfg of differentiated celltypes for all phases of preclinical and human clinical studiesNov 2005 – Commenced build-out of small GMP lab in WorcesterFeb 2006 – Opened research facility in Alameda, CA – 10,000 sq ft ofGMP capable labApril 2006 – Completed build-out of GMP lab in WorcesterMay 2006 – Commenced Scientific Work under GMP
  16. 16. 17Our Worcester GMP FacilityOur Worcester GMP Facility
  17. 17. 18DocumentationTrainingQualityProduction and Process ControlsMaterials (components, containers, closures)In-process and Final Product TestingShipping/StabilityLabeling/Packaging ControlEquipment/MetrologyFacilityLaboratory ControlSystems for Phase I Human Clinical TrialsSystems for Phase I Human Clinical Trials
  18. 18. 19Blastomere Program – Taking Aim at the ClinicBlastomere Program – Taking Aim at the ClinicTransfer of 2 Blastomere Lines into GMP ManufacturingCreation of Master Cell Bank and Working Cell BanksDerivation of RPE Cells under GMP from hESC Blastomere LinesCreation of Cryopreserved Cell ProductScientific Work Underway to Produce Additional Blastomere LinesSeeking Approval for NIH Federal Funding for hESC Research Utilizing BlastomereLinesAnnounced Collaboration with Wi-Cell to Make Blastomere Lines Widely Available ifEligible for Federal Funding
  19. 19. 20Our RPE Product Development ProgramOur RPE Product Development ProgramCommenced GMP RPE CellProductionPublished Paper on Proof of ConceptRPE Study in RCS RatHired Regulatory and RPE ClinicalAdvisorsCommenced Further PharmacologyStudiesCommencing Toxicology Studies inearly 2007Target IND Filing by Year-end 2007
  20. 20. 21
  21. 21. 22RCS retina at P100 with RPE-9 injection:A: low power view of retina showing extensiverescue;B: high power of outline b showing rescuedphotoreceptors;C: high power of outline c showing non-rescuearea.BCPhotoreceptor Rescue in RCS Rat by transplanted H9-RPE cellsbbccAA
  22. 22. 23Our Hemangioblast ProgramOur Hemangioblast ProgramSignificant Breakthrough in ScaledManufacture of Hemangioblasts CellsThoroughly characterized - make allcells of the blood and vasculartissuesSignificant Collaborations underwayto generate proof of concept data in awide range of indications / early datais extremely promising and expect topublish results in 2007Plan to generate Hemangioblastsunder GMP in 2007 and commenceadditional preclinical studies fortarget indications40X
  23. 23. 24Our Hemangioblast ProgramOur Hemangioblast ProgramHemangioblasts Generated fromSingle Blastomere Derived MA01s Human ES Cells10X
  24. 24. 25Our Dermal ProgramOur Dermal ProgramProduced scalable hESC-deriveddermal lines in Alameda facilityCollaborations under-way for proof ofconcept studiesCurrently considering regulatoryframework for dermal applicationsLarge applications in burns, woundrepair and surgery
  25. 25. Corporate MilestonesCorporate Milestones
  26. 26. 27Vital StatisticsVital StatisticsAS OF NOVEMBER 27, 2006:Symbol: OTC BB: ACTCShare price: $0.8852-Week High: $2.4452-Week Low: $0.26Shares Outstanding: 27.7million*Float: 17.4 million*Market Cap: $24.4 million*Average Volume (90 days):980,733 shares**approximate
  27. 27. 282006 Corporate Milestones2006 Corporate MilestonesAchievedCompleted Build-out of GMP FacilityBegan “GMP” Cell ProductionLaunched Preclinical and Clinical Testing PlansExpanded Technology Platform with Scientific BreakthroughsRaised Significant Additional Capital
  28. 28. 292007 Corporate Milestones2007 Corporate MilestonesSetting the Bar HighRaise Additional Capital / Achieve Listing on National ExchangeAnnounce Significant Corporate PartnershipFile IND for our RPE ProgramComplete Substantial Preclinical Work for our Hemangioblast andDermal ProgramsBuild-out our Development OrganizationGain Approval from NIH for Federal Funding for hESC Researchutilizing Blastomere LinesAnnounce Scientific Breakthrough on Solving Histocompatibility

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