Advanced Cell TechnologyAt The Forefront ofStem Cell TherapyFebruary 2011 Corporate Presentation
This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell TechnologyInc”, or “the Company”) sa...
At The Forefront of Regenerative Medicine3• Method of Producing human embryonic stem cells (hESCs)without Harm to Embryo• ...
Blastomere Program• Method for generating hESCs without harm tothe embryo• Uses routine method similar to pre-implantation...
Therapeutic Programs Indication Clinical StageRetinal Pigment Epithelium (RPE)ProgramStargardt’s Macular DystrophyDry AMDI...
RPE Program: Why the Eye? Why RPE Cells?• Eye is immune-privileged• RPE cells are pigmented so easy to identify(no need fo...
Animal StudiesRPE treatment in animal model of retinal dystrophy has slowedthe natural progression of the disease by promo...
Dry AMD– The most common form of Age-Related Macular Degeneration (as high as 90percent)– Affects 10-15 Million Americans–...
Myoblast ProgramAdult Stem Cells for Treatment of Heart FailureTarget MarketSufferers of Heart Failure, Chronic HeartFailu...
Hemangioblast ProgramSynthesizing Blood CellsThe HG cell is the precursor to all cell types in the circulatory andvascular...
Repair of Vascular Damage11Hemangioblasts were tested in animal models of heartdisease and peripheral vascular damageResul...
Hemangioblast Program• Investigating using HG cells to treatcardiovascular disease, stroke and cancer• Can generate large ...
13Intellectual PropertyRPE Cells• Worldwide Patent Filings• Dominant Patent Position for Treating Retinal Degeneration• On...
14Solid Financial FootingMost Stable Financial Situation In Company History• 12/31 Announced $25 Million Funding Commitmen...
The Advanced Cell Technology TeamWorld Class Scientific TeamSeasoned Management TeamDr. Robert Lanza, M.D. – Chief Scienti...
Thank you for your timeFor more information, visit www.advancedcell.com16
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Presentation at BIO CEO & Investor Conference, New York City. February 2011

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2/15/2011

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Presentation at BIO CEO & Investor Conference, New York City. February 2011

  1. 1. Advanced Cell TechnologyAt The Forefront ofStem Cell TherapyFebruary 2011 Corporate Presentation
  2. 2. This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell TechnologyInc”, or “the Company”) salient business characteristics.The information herein contains “forward-looking statements” as defined under the federal securitieslaws. Actual results could vary materially. Factors that could cause actual results to vary materially aredescribed in our filings with the Securities and Exchange Commission.You should pay particular attention to the “risk factors” contained in documents we file from time to timewith the Securities and Exchange Commission. The risks identified therein, as well as others notidentified by the Company, could cause the Company’s actual results to differ materially from thoseexpressed in any forward-looking statements.Cautionary Statement Concerning Forward-Looking Statements2
  3. 3. At The Forefront of Regenerative Medicine3• Method of Producing human embryonic stem cells (hESCs)without Harm to Embryo• Commencing 2 of 3 Human Clinical Trials using hESCs EverApproved by FDA in coming months• Stargardt’s Disease• Dry AMD – (Dry Age-Related Macular Degeneration)• Myoblast Program for Heart Failure approved for Phase II• Seeking to File IND for our Hemangioblast Program
  4. 4. Blastomere Program• Method for generating hESCs without harm tothe embryo• Uses routine method similar to pre-implantationgenetic diagnostics (PGD)• PGD is routine - used in thousands ofpregnancies every year• Published in Nature and Cell Stem Cell• NIH proposing expanded funding toaccommodate ACT’s blastomere-derived lines.4
  5. 5. Therapeutic Programs Indication Clinical StageRetinal Pigment Epithelium (RPE)ProgramStargardt’s Macular DystrophyDry AMDIND approved Nov. 2010Phase I to begin 1st Half 2011IND approved Jan. 3, 2011Phase I to begin 1st Half 2011Myoblast Program Heart Disease, Heart Attack andHeart FailurePhase I successfully completedFDA-approved for Phase IIHemangioblast Program Diseases and Disorders of Circulatoryand Vascular SystemPreclinicalAnticipate IND filing late 2011/early 20125
  6. 6. RPE Program: Why the Eye? Why RPE Cells?• Eye is immune-privileged• RPE cells are pigmented so easy to identify(no need for staining)• Small dosage vs. other therapies6• Ease of administration– Doesn’t require separate approval by the FDA– Procedure already used by eye surgeons; no new skill set required for doctorsRPE cell therapy could positivelyimpact over 200 retinal diseases
  7. 7. Animal StudiesRPE treatment in animal model of retinal dystrophy has slowedthe natural progression of the disease by promotingphotoreceptor survival.7RPE cells improve visual acuityand rescue photoreceptorstreated controlPhotoreceptorlayer
  8. 8. Dry AMD– The most common form of Age-Related Macular Degeneration (as high as 90percent)– Affects 10-15 Million Americans– No Approved Therapies available– $25-30 Billion market in US and Europe alone– FDA-approved for Phase I/II clinical trial; will start first half 2011Stargardt’s Disease– Affects at least 30,000 Americans– FDA Orphan Status; 7 years of Market Exclusivity– FDA-approved for Phase I/II clinical trial; will start first half 2011– Applying for similar Orphan Status in EuropeDry AMD and Stargardt’s Disease Programs8
  9. 9. Myoblast ProgramAdult Stem Cells for Treatment of Heart FailureTarget MarketSufferers of Heart Failure, Chronic HeartFailure and patients with scarred orischemic (dead) heart tissue caused by orrelated to heart attackProgram StatusClearance from FDA to Proceed with PhaseII Clinical TrialCharlestown, MA GMP Facility9
  10. 10. Hemangioblast ProgramSynthesizing Blood CellsThe HG cell is the precursor to all cell types in the circulatory andvascular systems10
  11. 11. Repair of Vascular Damage11Hemangioblasts were tested in animal models of heartdisease and peripheral vascular damageResults from treatment withhemangioblast cells• Restoration of blood flow toischemic limbs.• Survival after myocardialinfarction.Potential impact on a large number of vascular diseasesincluding myocardial infarction, vascular ischemic damage,ischemia-reperfusion injury, diabetic vascular disease andperipheral artery disease (PAD) that are leading causes ofdeath and/or disability worldwide.Hemangioblasts promoted repair in peripheral vascular damage
  12. 12. Hemangioblast Program• Investigating using HG cells to treatcardiovascular disease, stroke and cancer• Can generate large numbers, documentedin Nature Methods• Joint Venture with leading Korean stemcell developer CHA Biotech12
  13. 13. 13Intellectual PropertyRPE Cells• Worldwide Patent Filings• Dominant Patent Position for Treating Retinal Degeneration• One issued patent broadly covers methods for treating retinaldegeneration using human RPE cells differentiated from hESCs.• Broad IP Coverage for Manufacturing RPE Cells from hESCs• Two other patentsBlastomere Technology• Worldwide Patent Filings• Pending domestic patent applications
  14. 14. 14Solid Financial FootingMost Stable Financial Situation In Company History• 12/31 Announced $25 Million Funding Commitment• $15 Million on Balance Sheet• More Available• Virtually Debt-Free• Able to pay for both clinical trials, but that will likely notbe necessary:• Anticipate additional non-dilutive funding• Federal and state grants• Other grant and loan sources
  15. 15. The Advanced Cell Technology TeamWorld Class Scientific TeamSeasoned Management TeamDr. Robert Lanza, M.D. – Chief Scientific OfficerDr. Jonathan Dinsmore, Ph.D. – Myoblast Project AdvisorDr. Matthew Vincent, Ph.D. – Director of Business Development15Gary Rabin – Interim Chairman and CEOEdmund Mickunas – Vice President of RegulatoryDr. Roger Gay, PhD – Senior Director of ManufacturingRita Parker – Director of OperationsBill Douglass – Director of Corporate Communications & Social MediaStephen Price – Interim SVP – Corporate Development
  16. 16. Thank you for your timeFor more information, visit www.advancedcell.com16

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