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Translating Stem Cell Research into
Commercial Products
Translating Stem Cell Research into
Commercial Products
December 2006December 2006
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.
Cautionary Statement Concerning
Forward-Looking Statements
Cautionary Statement Concerning
Forward-Looking Statements
Advanced Cell TechnologyAdvanced Cell Technology
4
BackgroundBackground
Scientific Leader in Regenerative Medicine
First Proven Alternative Method for Deriving Human Embyronic Stem Cells
Driving Human ES Cell Therapies to the Clinic
Focus on RPE, Hemangioblast and Dermal Product Programs
Proprietary Human ES Cell Technology
Over 300 Patents and Patent Applications
Award Winning Team of Scientists and Management
Dr. Mike West and Dr. Robert Lanza are Recognized Thought Leaders
6
The Vision of Advanced Cell
Technology
The Vision of Advanced Cell
Technology
8
Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
9
Potential of ACT Embryonic Stem CellsPotential of ACT Embryonic Stem Cells
Unlimited Expansion of Stem Cell Source
Master Cell BankMaster Cell Bank
Working CellWorking Cell
BankBank
Bank of Differentiated CellsBank of Differentiated Cells
Bank of Differentiated CellsBank of Differentiated Cells
Bank of Differentiated CellsBank of Differentiated Cells
Single BlastomereSingle Blastomere
10
Commercialization of ACT TherapiesCommercialization of ACT Therapies
DermalDermal
RetinalRetinal
PigmentedPigmented
EpitheliumEpithelium
HemangioblastHemangioblast
Cryopreserved Cell BankCryopreserved Cell Bank
Working Cell BankWorking Cell BankCryopreserved Cell BankCryopreserved Cell Bank
Cryopreserved Cell BankCryopreserved Cell Bank
11
Our Product Development PathwayOur Product Development Pathway
Cell Discovery and Validation
Finding Cell Types with Therapeutic Potential
Select Target Indications
“GMP” Cell Production
Derive Human ES Cells under GMP
Differentiate Target Cell Type under GMP
Preclinical and Clinical Testing
Functional and Safety Preclinical Testing
Develop Plan with FDA for Human Clinical Trials
target cell typestarget cell types
target cell linestarget cell lines
20062006
20072007
20042004
20052005
Driving Human ES Cell
Therapies to the Clinic
Driving Human ES Cell
Therapies to the Clinic
13
Our Clinical Testing PathwayOur Clinical Testing Pathway
Research
Development
Process Validation
Process Understanding
Pre-Clinical Testing
Clinical Testing- Phase 1
Clinical Testing- Phase 2
Clinical Testing- Phase 3
Commercialization (Phase 4) Marketing
BLA
IND
Amendments
Updates
Letters
Submissions
14
Our GMP StrategyOur GMP Strategy
Worcester GMP Lab – utilize for pilot mfg of differentiated cell types
(RPE, Hemangioblasts, etc.) for pharm/tox studies and Phase I
studies
Alameda GMP Lab – utilize for larger scale mfg of differentiated cell
types for all phases of preclinical and human clinical studies
Nov 2005 – Commenced build-out of small GMP lab in Worcester
Feb 2006 – Opened research facility in Alameda, CA – 10,000 sq ft of
GMP capable lab
April 2006 – Completed build-out of GMP lab in Worcester
May 2006 – Commenced Scientific Work under GMP
15
Our Worcester GMP FacilityOur Worcester GMP Facility
16
Documentation
Training
Quality
Production and Process Controls
Materials (components, containers, closures)
In-process and Final Product Testing
Shipping/Stability
Labeling/Packaging Control
Equipment/Metrology
Facility
Laboratory Control
Systems for Phase I Human Clinical TrialsSystems for Phase I Human Clinical Trials
17
Blastomere Program – Taking Aim at the ClinicBlastomere Program – Taking Aim at the Clinic
Transfer of 2 Blastomere Lines into GMP Manufacturing
Creation of Master Cell Bank and Working Cell Banks
Derivation of RPE Cells under GMP from hESC Blastomere Lines
Creation of Cryopreserved Cell Product
Scientific Work Underway to Produce Additional Blastomere Lines
Seeking Approval for NIH Federal Funding for hESC Research Utilizing Blastomere
Lines
Announced Collaboration with Wi-Cell to Make Blastomere Lines Widely Available if
Eligible for Federal Funding
18
Our RPE Product Development ProgramOur RPE Product Development Program
Commenced GMP RPE Cell
Production
Published Paper on Proof of Concept
RPE Study in RCS Rat
Hired Regulatory and RPE Clinical
Advisors
Commenced Further Pharmacology
Studies
Commencing Toxicology Studies in
early 2007
Target IND Filing by Year-end 2007
19
20
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.
B
C
Photoreceptor Rescue in RCS Rat by transplanted H9-RPE cells
bb
cc
AA
21
Our Hemangioblast ProgramOur Hemangioblast Program
Significant Breakthrough in Scaled
Manufacture of Hemangioblasts Cells
Thoroughly 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 / early data
is extremely promising and expect to
publish results in 2007
Plan to generate Hemangioblasts
under GMP in 2007 and commence
additional preclinical studies for
target indications
40X
22
Our Hemangioblast ProgramOur Hemangioblast Program
Hemangioblasts Generated from
Single Blastomere Derived MA01s Human ES Cells
10X
23
Our Dermal ProgramOur Dermal Program
Produced scalable hESC-derived
dermal lines in Alameda facility
Collaborations under-way for proof of
concept studies
Currently considering regulatory
framework for dermal applications
Large applications in burns, wound
repair and surgery
Corporate MilestonesCorporate Milestones
25
2006 Corporate Milestones2006 Corporate Milestones
Achieved
Completed Build-out of GMP Facility
Began “GMP” Cell Production
Launched Preclinical and Clinical Testing Plans
Expanded Technology Platform with Scientific Breakthroughs
Raised Significant Additional Capital
26
2007 Corporate Milestones2007 Corporate Milestones
Setting the Bar High
Raise Additional Capital / Achieve Listing on National Exchange
Announce Significant Corporate Partnership
File IND for our RPE Program
Complete Substantial Preclinical Work for our Hemangioblast and
Dermal Programs
Build-out our Development Organization
Gain Approval from NIH for Federal Funding for hESC Research
utilizing Blastomere Lines
Announce Scientific Breakthrough on Solving Histocompatibility
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London Conference Presentation

  • 1. Translating Stem Cell Research into Commercial Products Translating Stem Cell Research into Commercial Products December 2006December 2006
  • 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. Cautionary Statement Concerning Forward-Looking Statements Cautionary Statement Concerning Forward-Looking Statements
  • 4. 4 BackgroundBackground Scientific Leader in Regenerative Medicine First Proven Alternative Method for Deriving Human Embyronic Stem Cells Driving Human ES Cell Therapies to the Clinic Focus on RPE, Hemangioblast and Dermal Product Programs Proprietary Human ES Cell Technology Over 300 Patents and Patent Applications Award Winning Team of Scientists and Management Dr. Mike West and Dr. Robert Lanza are Recognized Thought Leaders
  • 5.
  • 6. 6
  • 7. The Vision of Advanced Cell Technology The Vision of Advanced Cell Technology
  • 8. 8 Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
  • 9. 9 Potential of ACT Embryonic Stem CellsPotential of ACT Embryonic Stem Cells Unlimited Expansion of Stem Cell Source Master Cell BankMaster Cell Bank Working CellWorking Cell BankBank Bank of Differentiated CellsBank of Differentiated Cells Bank of Differentiated CellsBank of Differentiated Cells Bank of Differentiated CellsBank of Differentiated Cells Single BlastomereSingle Blastomere
  • 10. 10 Commercialization of ACT TherapiesCommercialization of ACT Therapies DermalDermal RetinalRetinal PigmentedPigmented EpitheliumEpithelium HemangioblastHemangioblast Cryopreserved Cell BankCryopreserved Cell Bank Working Cell BankWorking Cell BankCryopreserved Cell BankCryopreserved Cell Bank Cryopreserved Cell BankCryopreserved Cell Bank
  • 11. 11 Our Product Development PathwayOur Product Development Pathway Cell Discovery and Validation Finding Cell Types with Therapeutic Potential Select Target Indications “GMP” Cell Production Derive Human ES Cells under GMP Differentiate Target Cell Type under GMP Preclinical and Clinical Testing Functional and Safety Preclinical Testing Develop Plan with FDA for Human Clinical Trials target cell typestarget cell types target cell linestarget cell lines 20062006 20072007 20042004 20052005
  • 12. Driving Human ES Cell Therapies to the Clinic Driving Human ES Cell Therapies to the Clinic
  • 13. 13 Our Clinical Testing PathwayOur Clinical Testing Pathway Research Development Process Validation Process Understanding Pre-Clinical Testing Clinical Testing- Phase 1 Clinical Testing- Phase 2 Clinical Testing- Phase 3 Commercialization (Phase 4) Marketing BLA IND Amendments Updates Letters Submissions
  • 14. 14 Our GMP StrategyOur GMP Strategy Worcester GMP Lab – utilize for pilot mfg of differentiated cell types (RPE, Hemangioblasts, etc.) for pharm/tox studies and Phase I studies Alameda GMP Lab – utilize for larger scale mfg of differentiated cell types for all phases of preclinical and human clinical studies Nov 2005 – Commenced build-out of small GMP lab in Worcester Feb 2006 – Opened research facility in Alameda, CA – 10,000 sq ft of GMP capable lab April 2006 – Completed build-out of GMP lab in Worcester May 2006 – Commenced Scientific Work under GMP
  • 15. 15 Our Worcester GMP FacilityOur Worcester GMP Facility
  • 16. 16 Documentation Training Quality Production and Process Controls Materials (components, containers, closures) In-process and Final Product Testing Shipping/Stability Labeling/Packaging Control Equipment/Metrology Facility Laboratory Control Systems for Phase I Human Clinical TrialsSystems for Phase I Human Clinical Trials
  • 17. 17 Blastomere Program – Taking Aim at the ClinicBlastomere Program – Taking Aim at the Clinic Transfer of 2 Blastomere Lines into GMP Manufacturing Creation of Master Cell Bank and Working Cell Banks Derivation of RPE Cells under GMP from hESC Blastomere Lines Creation of Cryopreserved Cell Product Scientific Work Underway to Produce Additional Blastomere Lines Seeking Approval for NIH Federal Funding for hESC Research Utilizing Blastomere Lines Announced Collaboration with Wi-Cell to Make Blastomere Lines Widely Available if Eligible for Federal Funding
  • 18. 18 Our RPE Product Development ProgramOur RPE Product Development Program Commenced GMP RPE Cell Production Published Paper on Proof of Concept RPE Study in RCS Rat Hired Regulatory and RPE Clinical Advisors Commenced Further Pharmacology Studies Commencing Toxicology Studies in early 2007 Target IND Filing by Year-end 2007
  • 19. 19
  • 20. 20 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. B C Photoreceptor Rescue in RCS Rat by transplanted H9-RPE cells bb cc AA
  • 21. 21 Our Hemangioblast ProgramOur Hemangioblast Program Significant Breakthrough in Scaled Manufacture of Hemangioblasts Cells Thoroughly 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 / early data is extremely promising and expect to publish results in 2007 Plan to generate Hemangioblasts under GMP in 2007 and commence additional preclinical studies for target indications 40X
  • 22. 22 Our Hemangioblast ProgramOur Hemangioblast Program Hemangioblasts Generated from Single Blastomere Derived MA01s Human ES Cells 10X
  • 23. 23 Our Dermal ProgramOur Dermal Program Produced scalable hESC-derived dermal lines in Alameda facility Collaborations under-way for proof of concept studies Currently considering regulatory framework for dermal applications Large applications in burns, wound repair and surgery
  • 25. 25 2006 Corporate Milestones2006 Corporate Milestones Achieved Completed Build-out of GMP Facility Began “GMP” Cell Production Launched Preclinical and Clinical Testing Plans Expanded Technology Platform with Scientific Breakthroughs Raised Significant Additional Capital
  • 26. 26 2007 Corporate Milestones2007 Corporate Milestones Setting the Bar High Raise Additional Capital / Achieve Listing on National Exchange Announce Significant Corporate Partnership File IND for our RPE Program Complete Substantial Preclinical Work for our Hemangioblast and Dermal Programs Build-out our Development Organization Gain Approval from NIH for Federal Funding for hESC Research utilizing Blastomere Lines Announce Scientific Breakthrough on Solving Histocompatibility