This presentation is intended to present a summary of ACT’s (“ACT”, or “AdvancedCell Technology Inc”, or “the Company”) salient business characteristics.The information herein contains “forward-looking statements” as defined under thefederal securities laws. Actual results could vary materially. Factors that could causeactual results to vary materially are described in our filings with the Securities andExchange 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 Concerning Forward-LookingStatements2
State of the Company• Phase I/II ESC trial fully-funded (and notaffected by court ruling) and awaiting FDAapproval• “Embryo-safe” cell lines may qualify forfederal funding, despite recent court ruling• Actively pursuing alternatives toaccelerate development of programs3
ACT TherapeuticsACT Proprietary HumanTherapeutic ProgramsTreatment Clinical StageBlastomere ProgramDevelopment of embryonic stem cell lines withoutdestruction of embryoPre-ClinicalRetinal Pigment Epithelium(RPE) ProgramTreatment of Age-related Macular Degeneration(AMD) and Retinal Degenerative DiseasesClinical INDawaitingFDA approvalMyoblast ProgramTreatment of Heart Disease, Heart Attack andHeart FailurePhase IIHemangioblast programTreatment of Diseases and Disorders of theBlood, Circulatory and Vascular SystemsPre-Clinical4
Single Blastomere Technology• Company scientists successfully generate stem cell lineswithout destruction of embryo• Utilizes PGD extraction of single blastomere• PGD is safe and routine – has been used in thousands ofpregnancies in United States and Europe alone.• Cell lines retain potential to form all cells in the human body.• Resulting human ES cell lines have been demonstrated to bemore robust and reproducible than traditional ICM-derivedlines.• Technology has been reproduced and peer-reviewed onseveral occasions, and is currently being used in preclinicalstudies and a product awaiting FDA approval for IND study.5Blastomere Program:hESCs Without Embryo Harm
• Enables Derivation of New hESC Lines via Pre-implantation Genetic Diagnosis (PGD) Method,Preserving Development Potential of the Embryo• Offers source of autologous ES cells for donorduring his/her life, and closely matched allogeneicsource for blood relatives.• 4 hESC lines awaiting NIH approval for funding – embryosfrom which these lines were derived were not destroyed.• Technology is used to develop RPE cells for our clinicaltrials for Juvenile Macular Degeneration. (no FederalFunding)• Technology is used to develop Hemangioblasts –potential source for red blood cells and platelets.HemangioblastsDifferentiated fromBlastomere hESC Lines6Blastomere Program:Proven Alternative hESC Method with added Benefits
• The Sherley v. Sebelius case presents a major challengefor the regenerative medicine sector, without a doubt.• However, ACT has anticipated this development forsome time• ACT has been taking high-level, face-to-face meetings inDC with all the relevant players, in both houses and onboth sides of the aisle, as well as with NIH and HHS.• ACT‟s Single Blastomere technique for isolatinghESC‟s is likely not subject to the language JudgeLamberth used in Preliminary Injunction.• ACT stands ready to make these cells available tothe research community, if approved.7With Crisis, Comes OpportunityCurrent Challenge to hESC Federal Funding
Institutional CollaboratorsAdvanced Cell’s Institutional Collaborators include:Casey Eye Institute Mayo ClinicMoran Eye Institute UCSFHarvard Johns HopkinsStanford Sloan KetteringUniversity of Florida University of IowaUniversity of Illinois U.C. BerkeleyColorado State University8
Robust Patent Portfolio• RPE Program• Broad protection for production of RPE cells from human ES Cells• Includes two issued US Patents• Cover use of hESC-dervied RPE cells for treatment of retinaldegenerative disorders• Single Blastomere• Pending patent application for key technology• Induced Pluripotency (iPS)• ACT has earliest priority date to use of key regulatory factors required forgenerating iPS cells.• Transdifferentiation• Broad filings directed to transdifferentiation without viral vectors• SCNT• Dominant issued patents• Parthenogenesis• Aquired Infigen patents which are controlling in parthenogenesis9Realizing the fruits of more than adecade of important discoveries….
RPE Program: Why RPE?• Pigmented epithelium cells are easy to identify• Small dosage vs. other therapies• The eye is an immune-privileged site, thus no riskof rejection at the injection site• Ease of administration– Doesn‟t require a separate approval by the FDA (universal applicator)– Procedure is already used by eye surgeons; no new skill set requiredfor doctors• RPE cell therapy may positively impact over 200 retinal diseases10
ACT’s RPE Program IND - Status• Received Orphan Indication• Application for Phase I/II Trial BeingReviewed11• Initial IND for Stargardt’s Disease• Trial Design Dovetails Into Second IND, for Dry AMD• Represents $25-30 Billion Worldwide Market, With NoEffective Therapies Currently Available
Myoblast Program HighlightsTarget Market for Myoblast ProgramSufferers of Heart Failure, Chronic HeartFailure and patients with scarred orischemic (dead) heart tissue caused byor related to heart attackProgram StatusClearance from FDA to Proceed withPhase II Clinical Trials in the U.S.Charlestown, MA GMP Facility12
Myoblast Program: Clinical Trial SummaryPhase 1LVADPhase 1CABGPhase 1bCABGPhase 1bCatheterPatients 6 patients 12 patients 12 patients23 Patients(12 treated,11 control)IndicationLVAD Bridgeto Transplant+CABG +CABG Catheter InjectionDoseSingle Dose -300 MM CellsEscalating –10, 30, 100 and300 MM CellsSingle Dose -300 MM CellsEscalating -30, 100, 300 and600 MM CellsPrimaryEndpointSafety Safety Safety SafetyResultDemonstratedcell survival inhumansDoseescalation, Safeat all dosesSurvival of cells(cardiacimaging),improvedsymptoms andfunctionSafe at all doses,improvedsymptoms andfunction13
Myoblast Program: Phase II Clinical Trial 80 total patients (1:1 treatment vs. control) Double-blind, placebo-controlled (sham procedure) Catheter delivery of cells: percutaneous targeted delivery Three-month, six-month, and 12-month follow-up Endpoints: Improvement in HF symptoms and VentricularVolumes Primary endpoint: Improvement in heart failure symptomsmeasured by “Kansas City Cardiomyopathy Questionnaire” Supporting endpoints: Ventricular volumes, Six minute walk test Interim data analysis at six-months by Independent Review Board14
Hemangioblast Program: Partnership• Joint Venture with leading Korean stem celldeveloper CHA Biotech Co.• The J.V., „Stem Cell & Regenerative MedicineInternational‟ (SCRMI) is focused on thedevelopment of human blood cells and relatedproducts15• Developing IND submission for redblood cells and/or platelets derivedfrom iPS cells
Hemangioblast Program: OverviewThe HG cell is the precursor to all cell types in thecirculatory and vascular systems16
The Advanced Cell Technology TeamWorld Class Scientific Team Led BySeasoned Management TeamDr. Robert Lanza, M.D. – Chief Scientific OfficerDr. Jonathan Dinsmore, Ph.D. – Myoblast Project AdvisorMatthew Vincent, Ph.D. – Business Development and IP Strategy18William M. Caldwell IV – Chairman & CEOEdmund Mickunas – Vice President of RegulatoryRoger Gay, PhD – Senior Director of ManufacturingRita Parker – Director of OperationsBill Douglass – Director of Corporate Communications & Social Media
Thank you for your timeFor more information, visit www.advancedcell.comAdvanced Cell Technology is traded on the OTC BB, symbol: ACTC
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