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