Rodman Conference Presentation: William Caldwell


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"Human Embryonic Stem Cells:
Medicine’s Next Frontier"


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Rodman Conference Presentation: William Caldwell

  1. 1. Human Embryonic Stem Cells:Medicine’s Next Frontier
  2. 2. 2This presentation contains “forward-looking statements” as defined underthe federal securities laws. Actual results could vary materially. Factorsthat could cause actual results to vary materially are described in our filingswith the Securities and Exchange Commission.You should pay particular attention to the “risk factors” contained indocuments we file from time to time with the Securities and ExchangeCommission. The risks identified therein, as well as others not identified bythe Company, could cause the Company’s actual results to differ materiallyfrom those expressed in any forward-looking statements.Legal NoticeCautionary Statement ConcerningForward-Looking Statements
  3. 3. 3The Hope for New Cures“Science has presented us with a hope called stem-cellresearch, which may provide our scientists with answersthat have so long been beyond our grasp.”Nancy Reagan“[Stem cell research] is the most promising research inhealthcare perhaps in this history of the world and weshould not be left behind in this research.”Senator Orin Hatch, (R-Utah)“If the potential of stem cell research is realized, it wouldmean an end to the suffering of millions of people. If stemcell research succeeds, there isn’t a person in the countrywho won’t benefit, or know somebody who will. ”Michael J. Fox
  4. 4. 4Stem Cell Based TherapyScientists worldwide believe stem cell-based therapies have the potentialto restore healthy function ofdiseased or damaged tissuesGoal is to develop therapies that workby transplanting stem cells into thebody, using the body’s naturalpowers of regeneration and healing
  5. 5. 5Embryonic and Adult Stem CellsHuman Embryonic Stem Cells (hESCs)First Isolated in 1998Totipotent – can become any of the body’s 220 cell typesVirtually immortal self-replicationFound in early-stage embryoAdult Stem CellsUsed in Bone Marrow Transplants since 1968Multipotent – can differentiate into a few cell typesLimited capacity to self-replicate in labFound in certain tissues in fully developed humans
  6. 6. 6Embryonic vs. Adult Stem CellsDifferentiation AdvantagesFar easier in the lab to push ES cells to differentiate to specific tissues,significantly increasing successful development programsSelf-replication AdvantagesAdult stem cells lose ability to self-replicate after a few generations. ButES cells are virtually immortal in their self-replicationvs.Adult Self-ReplicationEmbryonicSelf-Replication
  8. 8. 8Stem Cell Therapy: MarketplaceEstimated $30 billion market for successfulstem cell therapiesNear-limitless potential applications in fast-growing fieldof regenerative medicineCurrent costs of some major illnesses:Heart disease: $175 billionDiabetes: $98 billionAlzheimers: $87 billionStroke: $43 billionOther potential disease applications include: parkinson’s, liverand kidney disease, epilepsy, eye disease, and many, manyothers.
  9. 9. 9Company MissionTranslating World Class hESCResearch into Proprietary HumanTherapeutic Programs
  10. 10. 10Key Investment HighlightsThree Proprietary Enabling Technology PlatformsBlastomere Program First Proven Alternative Method for hESC DerivationACTCellerate High Throughput Derivation and Differentiation ofProgram Therapeutic Cell TypesGMP Manufacturing Producing GMP-Compliant, Pathogen-Free BanksProgram of hESCs and Therapeutic Cell Types
  11. 11. 11Key Investment HighlightsThree Proprietary Human Therapeutic Programs Driving to The ClinincRPE Program Treatment of AMD and Retinal DegenerativeDiseasesHemangioblast Treatment of Disease and Disorders of the BloodProgram and Vascular SystemDermal Program Treatment of Wounds, Burns and Scars
  12. 12. 12Key Investment HighlightsWorld Class Scientific Team Lead By:Dr. Michael West President and Chief Scientific OfficerDr. Robert Lanza VP of Research & Scientific DevelopmentDr. Pedro Huertas Chief Development OfficerIndustry-Leading Intellectual Property PortfolioMore than 300 patents and patent applications related tostem cell therapy
  13. 13. 13A Single Focused Business StrategyDriving ProprietaryHuman Therapeutic Programsinto the Clinic
  14. 14. Key Development Milestones:Progress to the Clinic
  15. 15. Key Development Milestones:Blastomere Program
  16. 16. 16Blastomere Program–Most Recognized
  17. 17. 17Success in Human2006 MilestoneAchieved:Derivation ofHuman hESCLines Published inNature
  18. 18. 18First Proven Alternative MethodEnables Derivation of New hESC Lines viaPreimplantation Genetic Diagnosis (PGD)Method, Preserving Development Potential ofthe EmbryoFirst Proven Alternative; Presents Solution tohESC ControversyBlastomere hESC Lines May Be Eligible forFederal Funding; Company Plans to SeekRulingHemangioblastsHemangioblastsDifferentiated fromDifferentiated fromBlastomereBlastomere hESChESC LinesLines
  19. 19. Key Development Milestones:ACTCellerate Program
  20. 20. 20ACTCellerate – A Sleeping Beauty2006 Milestone Achieved:Announced proprietary high throughputtechnology platform for discovering largenumber of therapeutically useful, hESC-derivedcell types
  21. 21. 21ACTCellerate ProgramCellSortingGene ExpressionProfileScaling in RollershES Cell LineACTCellerate Cell LinesLinesHuman Embryo-Derived Cells
  22. 22. 22ACTCellerate ProgramACT162Cardiac ProgenitorCardiacMuscleACTCMYH7MYL4MYH3TNNT2NeuronalNEF3NEFLMEIS1CDH2SILVACTCellerate Cell LinesLines
  23. 23. Key Development Milestones:GMP Manufacturing Program
  24. 24. 24CENTRIFCT03002MicroscopeBSCSH-01002BSCSH-01001Viewing WindowSterilizerOV-05001CENTRIFCT-03001UnclassifiedPassThruWarmingOvenIN-02001-80FreezerCS-040022-8˚CRefrigeratorCS-01001Tri-GasIncubatorIN-03001CO2INCUBATORIN-01004 (top)IN-01003 (bottom)CO2INCUBATORIN-01001 (top)IN-01002 (bottom)MicroscopewithComputerWorkStationSH-03001BSCSH-01003FPRM198Revision513June06Class 10.000Room 198Class 100.000Room 199GMP Manufacturing Program2006 Milestone Achieved - Company Opens GMPFacilities in Massachusetts and CaliforniaADVANCED CELL TECHNOLOGYGMP MANUFACTURINGLABORATORY Rms 198/199
  25. 25. 25GMP Manufacturing Program2007 Milestone Achieved – Company Hires Dr. PedroHuertas as Chief Development OfficerNoted Biotech Industry Executive – Experience atGenzyme, StemCells Inc., Novazyme, AmicusTherapeuticsHarvard – MIT Trained PhysicianM.D. from Harvard and MITPh.D. from Harvard University in Cell andDevelopmental BiologyM.B.A. from MIT’s Sloan School of MgmtTasked with leading company’s push to the clinic
  26. 26. 26GMP Manufacturing ProgramDesign of Master and Working hESC BanksUnlimited Expansionof Stem Cell LineMasterCell BankWorkingCell BankBank of Differentiated CellsBank ofDifferentiated CellsBank of Differentiated CellsSingle Blastomere
  27. 27. 27GMP Manufacturing Program2007 Milestone Achieved – Company Manufacturing RPECells under GMP Compliant ConditionsWorking Stem Cell Bank Cryopreserved RPE Cell BankRetinalPigmentedEpithelium
  28. 28. 28GMP Manufacturing ProgramFuture 2007 Milestone – Manufacture Hemangioblast Cellsunder GMP Compliant ConditionsCryopreservedCryopreservedHemangioblast Cell BankHemangioblast Cell BankWorking Stem Cell BankWorking Stem Cell BankHemangioblastsHemangioblasts
  29. 29. Key Development Milestones:RPE Program
  30. 30. 30RPE Anatomy and FunctionRPELayerDeterioratesin Patientswith AMDand otherRetinalDegenerativeDiseases absorption of stray light barrier vitamin A metabolism andtransport phagocytosis of shedphotoreceptor segmentssome functions of RPE
  31. 31. 31RPE Program2006 MilestoneAchieved:Published DataShowing hESC-derived RPECells RescueVisual FunctioninRCS Rats
  32. 32. 32RPE Rescue in the RCS RatRPE: essential for the survival of photoreceptors in the retina;believed to play a critical role in healthy visionRCS retina at P100 withRPE-9 injection:A: low power view of retinashowing extensive rescue;B: high power of outline bshowing rescuedphotoreceptors;C: high power of outline cshowing non-rescue area.PhotoreceptorRescue in RCS Ratby transplantedH9-RPE cells
  33. 33. 33RPE Program Collaboration2007 Milestone Achieved - Preclinical Development Collaborationwith Casey Eye Institute at Oregon Health & Science UniversityDr. Raymond Lund Leading Researcher in RetinalDegenerative DiseaseDr. Peter Francis Senior Researcher and ClinicianDr. Richard Weleber Senior ClinicianOHSU Team is Conducting Dosage Studies in the RCS RatOHSU Team is Advising on RPE Program Safety StudiesAdditionally, OHSU Team is Advising on Plans for IND andPhase I Human Clinical Trials
  34. 34. Key Development Milestones:Hemangioblast Programs
  35. 35. 35The Hemangioblast CellPrecursor to all cell types of the blood and thevascular system
  36. 36. 36Hemangioblast Program2006 Milestone Achieved: Hemangioblast CellsGenerated from Blastomere Derived hESC Lines40X 10X
  37. 37. 37Hemangioblast Program2007 MilestoneAchieved:Published DataShowingHemangioblastFunction In Vivo inNature Methods
  38. 38. 38Hemangioblast ProgramRepair of Ischemic Retinal VasculaturesMouse Rat
  39. 39. 39Hemangioblast ProgramRestoration of Blood Flow in Ischemic Limbs (Rat)
  40. 40. 40Hemangioblast ProgramIncrease in Survival Rate in Infarcted Heart (Mouse)
  41. 41. 41Hemangioblast ProgramSignificant Breakthrough in ScaledManufacture of Hemangioblasts CellsWell characterized - make all cellsof the blood and vascular tissuesSignificant Collaborations underwayto generate proof of concept data in awide range of indicationsPlan to generate Hemangioblasts underGMP in 2007 and commence additionalpreclinical studies for target indications
  42. 42. Key Development Milestones:Dermal Program
  43. 43. 43Skin: Dermal Cells for Wound and Burn RepairES dermal cells possess uniquehealing capacities of early skin cellsto heal wounds without scarring.ACTC is researching ES dermal celltechnology that may somedayrepair skin.Large application for therapies to repairdamage from burns, wounds and surgicalprocedures, quickly and without scarringCells successfully isolated in our California labs
  44. 44. 44Corporate Milestones2007 Milestones Achieved So Far …Hired Chief Development OfficerManufactured Banks of GMP Compliant, Pathogen FreehESCsManufactured Banks of GMP Compliant, CryopreservedRPE CellsAnnounced RPE Program Collaboration with OHSU forPreclinical DevelopmentAcquired Infigen Intellectual Property PortfolioParticipated in 2007 CIRM Grants
  45. 45. 45Corporate Milestones2007-2008 Milestones To Come …Raise Additional Capital / Achieve Listing on National ExchangeAnnounce Significant Corporate PartnershipFile IND for RPE ProgramComplete and Publish Preclinical Work for Hemangioblast andDermal ProgramsGain Approval for Federal Funding of Research UtilizingBlastomere hESC LinesAnnounce Scientific Breakthrough on Solving Histocompatibility
  46. 46. Thank you for your timeFor more information, visit advancedcell.comAdvanced Cell Technology is traded on the OTC BB, symbol: ACTC.