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Human Embryonic Stem Cells: Medicine’s Next Frontier

Rodman Conference Presentation: William Caldwell

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"Human Embryonic Stem Cells: Medicine’s Next Frontier" 5/14/2007

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Page 1: 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

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Key Development Milestones:

Progress to the Clinic

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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

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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

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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

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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

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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

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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

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Thank you for your timeFor more information, visit advancedcell.com

Advanced Cell Technology is traded on the OTC BB, symbol: ACTC.