Rodman Conference Presentation: William Caldwell
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Transcript of Rodman Conference Presentation: William Caldwell
Human Embryonic Stem Cells:
Medicine’s Next Frontier
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This 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 Notice
Cautionary Statement ConcerningForward-Looking Statements
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The Hope for New Cures“Science has presented us with a hope called stem-cell
research, 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
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Stem Cell Based Therapy
Scientists worldwide believe stem cell-based therapies have the potentialto restore healthy function ofdiseased or damaged tissues
Goal is to develop therapies that workby transplanting stem cells into thebody, using the body’s naturalpowers of regeneration and healing
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Embryonic and Adult Stem Cells
Human Embryonic Stem Cells (hESCs)
First Isolated in 1998
Totipotent – can become any of the body’s 220 cell typesVirtually immortal self-replicationFound in early-stage embryo
Adult Stem Cells
Used in Bone Marrow Transplants since 1968
Multipotent – can differentiate into a few cell typesLimited capacity to self-replicate in labFound in certain tissues in fully developed humans
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Embryonic vs. Adult Stem CellsDifferentiation Advantages
Far easier in the lab to push ES cells to differentiate to specific tissues,significantly increasing successful development programs
Self-replication AdvantagesAdult stem cells lose ability to self-replicate after a few generations. ButES cells are virtually immortal in their self-replication
vs.Adult Self-Replication Embryonic
Self-Replication
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Potential of Human Embryonic StemCells
PANCREAS
LIVER
THYROID
LUNGS BRAIN
SPINAL CORD
HEART
FERTILIZED OVUM
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Stem Cell Therapy: MarketplaceEstimated $30 billion market for successfulstem cell therapies
Near-limitless potential applications in fast-growing fieldof regenerative medicine
Current costs of some major illnesses:
Heart disease: $175 billion
Diabetes: $98 billion
Alzheimers: $87 billion
Stroke: $43 billion
Other potential disease applications include: parkinson’s, liverand kidney disease, epilepsy, eye disease, and many, manyothers.
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Company Mission
Translating World Class hESC
Research into Proprietary Human
Therapeutic Programs
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Key Investment Highlights
Three Proprietary Enabling Technology Platforms
Blastomere Program First Proven Alternative Method for hESC Derivation
ACTCellerate High Throughput Derivation and Differentiation of
Program Therapeutic Cell Types
GMP Manufacturing Producing GMP-Compliant, Pathogen-Free Banks
Program of hESCs and Therapeutic Cell Types
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Key Investment Highlights
Three Proprietary Human Therapeutic Programs Driving to The Clininc
RPE Program Treatment of AMD and Retinal Degenerative Diseases
Hemangioblast Treatment of Disease and Disorders of the Blood Program and Vascular System
Dermal Program Treatment of Wounds, Burns and Scars
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Key Investment Highlights
World Class Scientific Team Lead By:
Dr. Michael West President and Chief Scientific Officer
Dr. Robert Lanza VP of Research & Scientific Development
Dr. Pedro Huertas Chief Development Officer
Industry-Leading Intellectual Property Portfolio
More than 300 patents and patent applications related tostem cell therapy
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A Single Focused Business Strategy
Driving Proprietary
Human Therapeutic Programs
into the Clinic
Key Development Milestones:
Progress to the Clinic
Key Development Milestones:
Blastomere Program
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Blastomere Program–Most Recognized
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Success in Human
2006 Milestone Achieved: Derivation of Human hESC Lines Published in
Nature
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First Proven Alternative Method
Enables Derivation of New hESC Lines viaPreimplantation Genetic Diagnosis (PGD)Method, Preserving Development Potential ofthe Embryo
First Proven Alternative; Presents Solution tohESC Controversy
Blastomere hESC Lines May Be Eligible forFederal Funding; Company Plans to SeekRuling
HemangioblastsHemangioblastsDifferentiated fromDifferentiated from
BlastomereBlastomere hESChESC Lines Lines
Key Development Milestones:
ACTCellerate Program
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ACTCellerate – A Sleeping Beauty
2006 Milestone Achieved:
Announced proprietary high throughputtechnology platform for discovering largenumber of therapeutically useful, hESC-derivedcell types
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ACTCellerate Program
CellSorting
Gene ExpressionProfile
Scaling in Rollers
hES Cell Line
ACTCellerate Cell LinesLinesHuman Embryo-
Derived Cells
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ACTCellerate Program
ACT162Cardiac Progenitor
CardiacMuscleACTCMYH7MYL4MYH3TNNT2
Neuronal
NEF3NEFLMEIS1CDH2SILV
ACTCellerate Cell LinesLines
Key Development Milestones:
GMP Manufacturing Program
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CENTRIFCT03002
Microscope
BSCSH-01002
BSCSH-01001
Viewing Window
SterilizerOV-05001
CENTRIFCT-03001
Unclassified
PassThru
WarmingOven
IN-02001
-80Freezer
CS-04002
2-8˚CRefrigerator
CS-01001
Tri-GasIncubatorIN-03001
CO2INCUBATOR
IN-01004 (top)IN-01003 (bottom)
CO2INCUBATOR
IN-01001 (top)IN-01002 (bottom)
Microscopewith
Computer
WorkStation
SH-03001
BSCSH-01003
FPR
M 1
98 R
evis
ion
513
Jun
e 06
Class 10.000Room 198
Class 100.000Room 199
GMP Manufacturing Program2006 Milestone Achieved - Company Opens GMPFacilities in Massachusetts and California
ADVANCED CELL TECHNOLOGYGMP MANUFACTURINGLABORATORY Rms 198/199
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GMP Manufacturing Program
2007 Milestone Achieved – Company Hires Dr. PedroHuertas as Chief Development Officer
Noted Biotech Industry Executive – Experience atGenzyme, StemCells Inc., Novazyme, AmicusTherapeutics
Harvard – MIT Trained PhysicianM.D. from Harvard and MITPh.D. from Harvard University in Cell andDevelopmental BiologyM.B.A. from MIT’s Sloan School of Mgmt
Tasked with leading company’s push to the clinic
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GMP Manufacturing Program
Design of Master and Working hESC BanksUnlimited Expansionof Stem Cell Line
MasterCell Bank
WorkingCell Bank
Bank of Differentiated Cells
Bank ofDifferentiated Cells
Bank of Differentiated Cells
Single Blastomere
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GMP Manufacturing Program
2007 Milestone Achieved – Company Manufacturing RPECells under GMP Compliant Conditions
Working Stem Cell Bank Cryopreserved RPE Cell Bank
RetinalPigmentedEpithelium
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GMP Manufacturing Program
Future 2007 Milestone – Manufacture Hemangioblast Cellsunder GMP Compliant Conditions
CryopreservedCryopreservedHemangioblast Cell BankHemangioblast Cell BankWorking Stem Cell BankWorking Stem Cell Bank
HemangioblastsHemangioblasts
Key Development Milestones:
RPE Program
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RPE Anatomy and Function
RPE LayerDeterioratesin Patientswith AMDand otherRetinalDegenerativeDiseases
absorption of stray light barrier vitamin A metabolism and
transport phagocytosis of shed
photoreceptor segments
some functions of RPE
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RPE Program
2006 Milestone Achieved:
Published DataShowing hESC-derived RPECells RescueVisual FunctioninRCS Rats
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RPE Rescue in the RCS RatRPE: essential for the survival of photoreceptors in the retina;
believed to play a critical role in healthy vision
RCS 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
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RPE Program Collaboration
2007 Milestone Achieved - Preclinical Development Collaborationwith Casey Eye Institute at Oregon Health & Science University
Dr. Raymond Lund Leading Researcher in Retinal Degenerative Disease
Dr. Peter Francis Senior Researcher and Clinician
Dr. Richard Weleber Senior Clinician
OHSU Team is Conducting Dosage Studies in the RCS Rat
OHSU Team is Advising on RPE Program Safety Studies
Additionally, OHSU Team is Advising on Plans for IND andPhase I Human Clinical Trials
Key Development Milestones:
Hemangioblast Programs
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The Hemangioblast Cell
Precursor to all cell types of the blood and thevascular system
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Hemangioblast Program
2006 Milestone Achieved: Hemangioblast CellsGenerated from Blastomere Derived hESC Lines
40X 10X
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Hemangioblast Program
2007 Milestone Achieved:
Published Data Showing Hemangioblast Function In Vivo in Nature Methods
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Hemangioblast Program
Repair of Ischemic Retinal Vasculatures
Mouse Rat
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Hemangioblast Program
Restoration of Blood Flow in Ischemic Limbs (Rat)
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Hemangioblast Program
Increase in Survival Rate in Infarcted Heart (Mouse)
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Hemangioblast Program
Significant Breakthrough in Scaled Manufacture of Hemangioblasts Cells
Well characterized - make all cells of the blood and vascular tissues
Significant Collaborations underway to generate proof of concept data in a wide range of indications
Plan to generate Hemangioblasts under GMP in 2007 and commence additional preclinical studies for target indications
Key Development Milestones:
Dermal Program
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Skin: Dermal Cells for Wound and Burn Repair
ES 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 scarring
Cells successfully isolated in our California labs
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Corporate Milestones
2007 Milestones Achieved So Far …Hired Chief Development Officer
Manufactured Banks of GMP Compliant, Pathogen FreehESCs
Manufactured Banks of GMP Compliant, CryopreservedRPE Cells
Announced RPE Program Collaboration with OHSU forPreclinical Development
Acquired Infigen Intellectual Property Portfolio
Participated in 2007 CIRM Grants
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Corporate Milestones
2007-2008 Milestones To Come …Raise Additional Capital / Achieve Listing on National Exchange
Announce Significant Corporate Partnership
File IND for RPE Program
Complete and Publish Preclinical Work for Hemangioblast andDermal Programs
Gain Approval for Federal Funding of Research UtilizingBlastomere hESC Lines
Announce Scientific Breakthrough on Solving Histocompatibility
Thank you for your timeFor more information, visit advancedcell.com
Advanced Cell Technology is traded on the OTC BB, symbol: ACTC.