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Human Embryonic Stem Cells:
Medicine’s Next Frontier
2
This presentation contains “forward-looking statements” as defined under
the federal securities laws. Actual results could vary materially. Factors
that could cause actual results to vary materially are described 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 time with the Securities and Exchange
Commission. The risks identified therein, as well as others not identified by
the Company, could cause the Company’s actual results to differ materially
from those expressed in any forward-looking statements.
Legal Notice
Cautionary Statement Concerning
Forward-Looking Statements
3
The Hope for New Cures
“Science has presented us with a hope called stem-cell
research, which may provide our scientists with answers
that have so long been beyond our grasp.”
Nancy Reagan
“[Stem cell research] is the most promising research in
healthcare perhaps in this history of the world and we
should not be left behind in this research.”
Senator Orin Hatch, (R-Utah)
“If the potential of stem cell research is realized, it would
mean an end to the suffering of millions of people. If stem
cell research succeeds, there isn’t a person in the country
who won’t benefit, or know somebody who will. ”
Michael J. Fox
4
Stem Cell Based Therapy
Scientists worldwide believe stem cell-
based therapies have the potential
to restore healthy function of
diseased or damaged tissues
Goal is to develop therapies that work
by transplanting stem cells into the
body, using the body’s natural
powers of regeneration and healing
5
Embryonic and Adult Stem Cells
Human Embryonic Stem Cells (hESCs)
First Isolated in 1998
Totipotent – can become any of the body’s 220 cell types
Virtually immortal self-replication
Found in early-stage embryo
Adult Stem Cells
Used in Bone Marrow Transplants since 1968
Multipotent – can differentiate into a few cell types
Limited capacity to self-replicate in lab
Found in certain tissues in fully developed humans
6
Embryonic vs. Adult Stem Cells
Differentiation Advantages
Far easier in the lab to push ES cells to differentiate to specific tissues,
significantly increasing successful development programs
Self-replication Advantages
Adult stem cells lose ability to self-replicate after a few generations. But
ES cells are virtually immortal in their self-replication
vs.
Adult Self-Replication
Embryonic
Self-Replication
7
Potential of Human Embryonic Stem
Cells
PANCREAS
LIVER
THYROID
LUNGS BRAIN
SPINAL CORD
HEART
FERTILIZED OVUM
8
Stem Cell Therapy: Marketplace
Estimated $30 billion market for successful
stem cell therapies
Near-limitless potential applications in fast-growing field
of 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, liver
and kidney disease, epilepsy, eye disease, and many, many
others.
9
Company Mission
Translating World Class hESC
Research into Proprietary Human
Therapeutic Programs
10
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
11
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
12
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 to
stem cell therapy
13
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
16
Blastomere Program–Most Recognized
17
Success in Human
2006 Milestone
Achieved:
Derivation of
Human hESC
Lines Published in
Nature
18
First Proven Alternative Method
Enables Derivation of New hESC Lines via
Preimplantation Genetic Diagnosis (PGD)
Method, Preserving Development Potential of
the Embryo
First Proven Alternative; Presents Solution to
hESC Controversy
Blastomere hESC Lines May Be Eligible for
Federal Funding; Company Plans to Seek
Ruling
HemangioblastsHemangioblasts
Differentiated fromDifferentiated from
BlastomereBlastomere hESChESC LinesLines
Key Development Milestones:
ACTCellerate Program
20
ACTCellerate – A Sleeping Beauty
2006 Milestone Achieved:
Announced proprietary high throughput
technology platform for discovering large
number of therapeutically useful, hESC-derived
cell types
21
ACTCellerate Program
Cell
Sorting
Gene Expression
Profile
Scaling in Rollers
hES Cell Line
ACTCellerate Cell Lines
LinesHuman Embryo-
Derived Cells
22
ACTCellerate Program
ACT162
Cardiac Progenitor
Cardiac
Muscle
ACTC
MYH7
MYL4
MYH3
TNNT2
Neuronal
NEF3
NEFL
MEIS1
CDH2
SILV
ACTCellerate Cell Lines
Lines
Key Development Milestones:
GMP Manufacturing Program
24
CENTRIF
CT03002
Microscope
BSC
SH-01002
BSC
SH-01001
Viewing Window
Sterilizer
OV-05001
CENTRIF
CT-03001
Unclassified
Pass
Thru
Warming
Oven
IN-02001
-80
Freezer
CS-04002
2-8˚C
Refrigerator
CS-01001
Tri-Gas
Incubator
IN-03001
CO2
INCUBATOR
IN-01004 (top)
IN-01003 (bottom)
CO2
INCUBATOR
IN-01001 (top)
IN-01002 (bottom)
Microscope
with
Computer
Work
Station
SH-03001
BSC
SH-01003
FPRM198Revision5
13June06
Class 10.000
Room 198
Class 100.000
Room 199
GMP Manufacturing Program
2006 Milestone Achieved - Company Opens GMP
Facilities in Massachusetts and California
ADVANCED CELL TECHNOLOGY
GMP MANUFACTURING
LABORATORY Rms 198/199
25
GMP Manufacturing Program
2007 Milestone Achieved – Company Hires Dr. Pedro
Huertas as Chief Development Officer
Noted Biotech Industry Executive – Experience at
Genzyme, StemCells Inc., Novazyme, Amicus
Therapeutics
Harvard – MIT Trained Physician
M.D. from Harvard and MIT
Ph.D. from Harvard University in Cell and
Developmental Biology
M.B.A. from MIT’s Sloan School of Mgmt
Tasked with leading company’s push to the clinic
26
GMP Manufacturing Program
Design of Master and Working hESC Banks
Unlimited Expansion
of Stem Cell Line
Master
Cell Bank
Working
Cell Bank
Bank of Differentiated Cells
Bank of
Differentiated Cells
Bank of Differentiated Cells
Single Blastomere
27
GMP Manufacturing Program
2007 Milestone Achieved – Company Manufacturing RPE
Cells under GMP Compliant Conditions
Working Stem Cell Bank Cryopreserved RPE Cell Bank
Retinal
Pigmented
Epithelium
28
GMP Manufacturing Program
Future 2007 Milestone – Manufacture Hemangioblast Cells
under GMP Compliant Conditions
CryopreservedCryopreserved
Hemangioblast Cell BankHemangioblast Cell BankWorking Stem Cell BankWorking Stem Cell Bank
HemangioblastsHemangioblasts
Key Development Milestones:
RPE Program
30
RPE Anatomy and Function
RPE
Layer
Deteriorates
in Patients
with AMD
and other
Retinal
Degenerative
Diseases
 absorption of stray light
 barrier
 vitamin A metabolism and
transport
 phagocytosis of shed
photoreceptor segments
some functions of RPE
31
RPE Program
2006 Milestone
Achieved:
Published Data
Showing hESC-
derived RPE
Cells Rescue
Visual Function
in
RCS Rats
32
RPE Rescue in the RCS Rat
RPE: essential for the survival of photoreceptors in the retina;
believed to play a critical role in healthy vision
RCS retina at P100 with
RPE-9 injection:
A: low power view of retina
showing extensive rescue;
B: high power of outline b
showing rescued
photoreceptors;
C: high power of outline c
showing non-rescue area.
Photoreceptor
Rescue in RCS Rat
by transplanted
H9-RPE cells
33
RPE Program Collaboration
2007 Milestone Achieved - Preclinical Development Collaboration
with 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 and
Phase I Human Clinical Trials
Key Development Milestones:
Hemangioblast Programs
35
The Hemangioblast Cell
Precursor to all cell types of the blood and the
vascular system
36
Hemangioblast Program
2006 Milestone Achieved: Hemangioblast Cells
Generated from Blastomere Derived hESC Lines
40X 10X
37
Hemangioblast Program
2007 Milestone
Achieved:
Published Data
Showing
Hemangioblast
Function In Vivo in
Nature Methods
38
Hemangioblast Program
Repair of Ischemic Retinal Vasculatures
Mouse Rat
39
Hemangioblast Program
Restoration of Blood Flow in Ischemic Limbs (Rat)
40
Hemangioblast Program
Increase in Survival Rate in Infarcted Heart (Mouse)
41
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
43
Skin: Dermal Cells for Wound and Burn Repair
ES dermal cells possess unique
healing capacities of early skin cells
to heal wounds without scarring.
ACTC is researching ES dermal cell
technology that may someday
repair skin.
Large application for therapies to repair
damage from burns, wounds and surgical
procedures, quickly and without scarring
Cells successfully isolated in our California labs
44
Corporate Milestones
2007 Milestones Achieved So Far …
Hired Chief Development Officer
Manufactured Banks of GMP Compliant, Pathogen Free
hESCs
Manufactured Banks of GMP Compliant, Cryopreserved
RPE Cells
Announced RPE Program Collaboration with OHSU for
Preclinical Development
Acquired Infigen Intellectual Property Portfolio
Participated in 2007 CIRM Grants
45
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 and
Dermal Programs
Gain Approval for Federal Funding of Research Utilizing
Blastomere hESC Lines
Announce Scientific Breakthrough on Solving Histocompatibility
Thank you for your time
For more information, visit advancedcell.com
Advanced Cell Technology is traded on the OTC BB, symbol: ACTC.

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Rodman Conference Presentation: William Caldwell

  • 1. Human Embryonic Stem Cells: Medicine’s Next Frontier
  • 2. 2 This presentation contains “forward-looking statements” as defined under the federal securities laws. Actual results could vary materially. Factors that could cause actual results to vary materially are described 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 time with the Securities and Exchange Commission. The risks identified therein, as well as others not identified by the Company, could cause the Company’s actual results to differ materially from those expressed in any forward-looking statements. Legal Notice Cautionary Statement Concerning Forward-Looking Statements
  • 3. 3 The Hope for New Cures “Science has presented us with a hope called stem-cell research, which may provide our scientists with answers that have so long been beyond our grasp.” Nancy Reagan “[Stem cell research] is the most promising research in healthcare perhaps in this history of the world and we should not be left behind in this research.” Senator Orin Hatch, (R-Utah) “If the potential of stem cell research is realized, it would mean an end to the suffering of millions of people. If stem cell research succeeds, there isn’t a person in the country who won’t benefit, or know somebody who will. ” Michael J. Fox
  • 4. 4 Stem Cell Based Therapy Scientists worldwide believe stem cell- based therapies have the potential to restore healthy function of diseased or damaged tissues Goal is to develop therapies that work by transplanting stem cells into the body, using the body’s natural powers of regeneration and healing
  • 5. 5 Embryonic and Adult Stem Cells Human Embryonic Stem Cells (hESCs) First Isolated in 1998 Totipotent – can become any of the body’s 220 cell types Virtually immortal self-replication Found in early-stage embryo Adult Stem Cells Used in Bone Marrow Transplants since 1968 Multipotent – can differentiate into a few cell types Limited capacity to self-replicate in lab Found in certain tissues in fully developed humans
  • 6. 6 Embryonic vs. Adult Stem Cells Differentiation Advantages Far easier in the lab to push ES cells to differentiate to specific tissues, significantly increasing successful development programs Self-replication Advantages Adult stem cells lose ability to self-replicate after a few generations. But ES cells are virtually immortal in their self-replication vs. Adult Self-Replication Embryonic Self-Replication
  • 7. 7 Potential of Human Embryonic Stem Cells PANCREAS LIVER THYROID LUNGS BRAIN SPINAL CORD HEART FERTILIZED OVUM
  • 8. 8 Stem Cell Therapy: Marketplace Estimated $30 billion market for successful stem cell therapies Near-limitless potential applications in fast-growing field of 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, liver and kidney disease, epilepsy, eye disease, and many, many others.
  • 9. 9 Company Mission Translating World Class hESC Research into Proprietary Human Therapeutic Programs
  • 10. 10 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
  • 11. 11 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
  • 12. 12 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 to stem cell therapy
  • 13. 13 A Single Focused Business Strategy Driving Proprietary Human Therapeutic Programs into the Clinic
  • 17. 17 Success in Human 2006 Milestone Achieved: Derivation of Human hESC Lines Published in Nature
  • 18. 18 First Proven Alternative Method Enables Derivation of New hESC Lines via Preimplantation Genetic Diagnosis (PGD) Method, Preserving Development Potential of the Embryo First Proven Alternative; Presents Solution to hESC Controversy Blastomere hESC Lines May Be Eligible for Federal Funding; Company Plans to Seek Ruling HemangioblastsHemangioblasts Differentiated fromDifferentiated from BlastomereBlastomere hESChESC LinesLines
  • 20. 20 ACTCellerate – A Sleeping Beauty 2006 Milestone Achieved: Announced proprietary high throughput technology platform for discovering large number of therapeutically useful, hESC-derived cell types
  • 21. 21 ACTCellerate Program Cell Sorting Gene Expression Profile Scaling in Rollers hES Cell Line ACTCellerate Cell Lines LinesHuman Embryo- Derived Cells
  • 23. Key Development Milestones: GMP Manufacturing Program
  • 24. 24 CENTRIF CT03002 Microscope BSC SH-01002 BSC SH-01001 Viewing Window Sterilizer OV-05001 CENTRIF CT-03001 Unclassified Pass Thru Warming Oven IN-02001 -80 Freezer CS-04002 2-8˚C Refrigerator CS-01001 Tri-Gas Incubator IN-03001 CO2 INCUBATOR IN-01004 (top) IN-01003 (bottom) CO2 INCUBATOR IN-01001 (top) IN-01002 (bottom) Microscope with Computer Work Station SH-03001 BSC SH-01003 FPRM198Revision5 13June06 Class 10.000 Room 198 Class 100.000 Room 199 GMP Manufacturing Program 2006 Milestone Achieved - Company Opens GMP Facilities in Massachusetts and California ADVANCED CELL TECHNOLOGY GMP MANUFACTURING LABORATORY Rms 198/199
  • 25. 25 GMP Manufacturing Program 2007 Milestone Achieved – Company Hires Dr. Pedro Huertas as Chief Development Officer Noted Biotech Industry Executive – Experience at Genzyme, StemCells Inc., Novazyme, Amicus Therapeutics Harvard – MIT Trained Physician M.D. from Harvard and MIT Ph.D. from Harvard University in Cell and Developmental Biology M.B.A. from MIT’s Sloan School of Mgmt Tasked with leading company’s push to the clinic
  • 26. 26 GMP Manufacturing Program Design of Master and Working hESC Banks Unlimited Expansion of Stem Cell Line Master Cell Bank Working Cell Bank Bank of Differentiated Cells Bank of Differentiated Cells Bank of Differentiated Cells Single Blastomere
  • 27. 27 GMP Manufacturing Program 2007 Milestone Achieved – Company Manufacturing RPE Cells under GMP Compliant Conditions Working Stem Cell Bank Cryopreserved RPE Cell Bank Retinal Pigmented Epithelium
  • 28. 28 GMP Manufacturing Program Future 2007 Milestone – Manufacture Hemangioblast Cells under GMP Compliant Conditions CryopreservedCryopreserved Hemangioblast Cell BankHemangioblast Cell BankWorking Stem Cell BankWorking Stem Cell Bank HemangioblastsHemangioblasts
  • 30. 30 RPE Anatomy and Function RPE Layer Deteriorates in Patients with AMD and other Retinal Degenerative Diseases  absorption of stray light  barrier  vitamin A metabolism and transport  phagocytosis of shed photoreceptor segments some functions of RPE
  • 31. 31 RPE Program 2006 Milestone Achieved: Published Data Showing hESC- derived RPE Cells Rescue Visual Function in RCS Rats
  • 32. 32 RPE Rescue in the RCS Rat RPE: essential for the survival of photoreceptors in the retina; believed to play a critical role in healthy vision RCS retina at P100 with RPE-9 injection: A: low power view of retina showing extensive rescue; B: high power of outline b showing rescued photoreceptors; C: high power of outline c showing non-rescue area. Photoreceptor Rescue in RCS Rat by transplanted H9-RPE cells
  • 33. 33 RPE Program Collaboration 2007 Milestone Achieved - Preclinical Development Collaboration with 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 and Phase I Human Clinical Trials
  • 35. 35 The Hemangioblast Cell Precursor to all cell types of the blood and the vascular system
  • 36. 36 Hemangioblast Program 2006 Milestone Achieved: Hemangioblast Cells Generated from Blastomere Derived hESC Lines 40X 10X
  • 37. 37 Hemangioblast Program 2007 Milestone Achieved: Published Data Showing Hemangioblast Function In Vivo in Nature Methods
  • 38. 38 Hemangioblast Program Repair of Ischemic Retinal Vasculatures Mouse Rat
  • 39. 39 Hemangioblast Program Restoration of Blood Flow in Ischemic Limbs (Rat)
  • 40. 40 Hemangioblast Program Increase in Survival Rate in Infarcted Heart (Mouse)
  • 41. 41 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
  • 43. 43 Skin: Dermal Cells for Wound and Burn Repair ES dermal cells possess unique healing capacities of early skin cells to heal wounds without scarring. ACTC is researching ES dermal cell technology that may someday repair skin. Large application for therapies to repair damage from burns, wounds and surgical procedures, quickly and without scarring Cells successfully isolated in our California labs
  • 44. 44 Corporate Milestones 2007 Milestones Achieved So Far … Hired Chief Development Officer Manufactured Banks of GMP Compliant, Pathogen Free hESCs Manufactured Banks of GMP Compliant, Cryopreserved RPE Cells Announced RPE Program Collaboration with OHSU for Preclinical Development Acquired Infigen Intellectual Property Portfolio Participated in 2007 CIRM Grants
  • 45. 45 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 and Dermal Programs Gain Approval for Federal Funding of Research Utilizing Blastomere hESC Lines Announce Scientific Breakthrough on Solving Histocompatibility
  • 46. Thank you for your time For more information, visit advancedcell.com Advanced Cell Technology is traded on the OTC BB, symbol: ACTC.