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)• “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
Blastomere Program: Synthesizing Stem Cellswithout Harm to the EmbryoSingle Blastomere Technology• Company scientists successfully generate stem celllines without destruction of embryo• Utilizes PGD extraction of single blastomere• PGD is routine - used in about 1000 pregnancies peryear in United States and Europe• Technology has been reproduced and peer-reviewed onseveral occasions.• Cell lines retain potential to form all cells in the humanbody.• Resulting human ES cell lines are more robust andreproducible than traditional ICM-derived lines.• National Institute of Health (NIH) proposing expandedfunding regulations to accommodate ACT‟s lines.5
First Proven Alternative hESC Method• 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• 4 hESC lines awaiting NIH approval for funding –embryos from which these lines were derived werenot destroyed. Outside scope of court‟s originalinjunction.• Technology is used to develop RPE cells for ourclinical trials for Stargardt‟s diseaseHemangioblastsDifferentiated fromBlastomere hESC Lines6
Current Challenge to hESC Federal FundingAugust 19, 2009 – Complaint filedOctober 27, 2009 – Suit dismissed by Judge Lamberth for lack of standing by PlaintiffsJune 25, 2010 – Court of Appeals upholds standing of two plaintiffs, remands to district courtAugust 23 – Judge Lamberth ruled that President Obama’s expansion of federal financing for hESCresearch violated federal lawIssued preliminary injunction enjoining federal funding of human ES cell research under the2009 guidelines promulgated by NIHSeptember 1 – Government requests stay of injunctionSeptember 7 – Judge denies request for stay. Indicates that Summary Judgment motions will beheard very soon – could lead to Permanent Injunction (Plaintiffs win without a trial)September 10 – Federal Court of Appeals stays injunction >> BUT, Summary Judgmentprocess marches on. Plaintiff’s file motions for permanent injunction; Government’s responseis due in 10 days.Has anything really changed to alter Lamberth’s thinking when he hearsthe summary judgment arguments?7Timeline: Sherley vs. Sebelius
8Should Scientists HaveBeen Surprised? "The NIH was frankly, I was stunned - as was virtuallyeveryone here at NIH - by the judicial decision yesterday".– Francis Collins, NIH DirectorACT was prepared …• Have been paying careful attention to this case• Have been meeting with members of Congressregarding the very issue of Dickey-WickerAmendment• Circulated a memo to members of Congress duringmeetings in DC on July 28th which specifically warned ofthis consequenceCurrent Challenge to hESC Federal Funding
The Federal Injunction Against hESC FundingACT’s Solutions andRecommendations forCongressional Action:•Amend Dickey-Wicker•Reinforce H.R. 4808 and other stemcell research bills9Each party using stem cell research as an election “LightningRod”Post-election legislation does not address reality of Dickey-Wicker
• The Sherley v Selebius 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.10With Crisis, Comes OpportunityCurrent Challenge to hESC Federal Funding
• Current situation: Lawyers controlling actions / trying tomaintain status quo• Mindful of this as a lightning rod for mid-term elections• The Reality: NIH is facing a permanent injunction onfederal funding, perhaps as early as end of month• ACT‟s Blastomere technique may be a fallback until theissue is resolved in Congress.• ACT is offering to make its hESC lines available toacademic laboratories11Action Plan: NIHCurrent 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 University12
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 parthenogenesis13Realizing the fruits of more than adecade of important discoveries….
ACT’s RPE Program IND - Status• Received Orphan Indication• Application for IND Being Reviewed14• 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
ACT’s RPE Program IND• Filed IND with FDA in November2009• FDA conveyed concerns in light ofGeron‟s hold15• Geron‟s hESC trial approved in August;established a bar showing that hESC can moveforward• FDA now focused on not replicating a “Geron-type” situation
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 Facility16
Hemangioblast Program: Partnership• Joint Venture with leading Korean stem celldeveloper CHA Biotech Co.• The J.V., „Stem Cell & Regenerative MedicineInternational‟, is focused on the development ofhuman blood cells and related products17• Developing IND submission for redblood cells and/or platelets derivedfrom iPS cells
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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