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The PIPEs ConferenceThe PIPEs Conference
November 2006November 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
3
Investment ConsiderationsInvestment Considerations
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
Blastomere Program – Significant AdvancementsBlastomere Program – Significant Advancements
Transfer of 2 Blastomere Lines into GMP for Product Development
Scientific Work Under-way to Produce Additional Blastomere Lines
Planning to Seek Approval for NIH Federal Funding for hESC
Research Utilizing Blastomere Lines
Set-up Collaboration with Wi-Cell to Make Blastomere Lines Widely
Available if Eligible for Federal Funding
7
Blastomere divided outgrowth
first passage second passage established line
1.Derivation of hES cells from single blastomeres
single blastomere
Initial outgrowthcocultured blastomereblastomere isolation
Why Human Embryonic Stem
Cells?
Why Human Embryonic Stem
Cells?
9
Stem Cells are a Proven TherapyStem Cells are a Proven Therapy
First successful bone marrow transplant (BMT) in 1968
Tens of thousands of patients have been cured with BMT
Human embryonic stem cells first isolated in 1998
hESC’s have significant potential to treat diseases
10
The Significant Advantage of Embryonic Stem CellsThe Significant Advantage of Embryonic Stem Cells
11
ES Cells Form Complex TissuesES Cells Form Complex Tissues
Science 2002 295: 819
PNAS 2003, 100 Suppl 1:11911
Intestine Muscle Retina
Hair / Skin Cartilage Bone
12
Regenerative Medicine
Curing Devastating Disease
Regenerative Medicine
Curing Devastating Disease
800,000
Parkinson's patients 750,000
liver disease patients
20,000,000
heart disease patients
20,000,000 diabetes
patients
5,000,000
epilepsy patients
4,000,000
Alzheimer's patients
2,000,000
stroke patients
300,000
kidney disease patients
13
Growing Legislative SupportGrowing Legislative Support
California & Proposition 71
Voters approve $3.0 billion in funding over next ten years
Formation of CA Institute of Regenerative Medicine (CIRM)
Recent awards of initial training grants
Court trial complete
CIRM public discussions – funding 2007
Other State Initiatives
MA, CT, NJ, NY, IL, FL
Federal Legislation
Bi-partisan House of Representatives Bill H.R. 810
Federal Funding for Alternative Methods of Deriving Human ES Cells
14
The Next Frontier in MedicineThe Next Frontier in Medicine
"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
“[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)
“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
“This is not a matter of using a human embryo that has the potential to produce life. Rather,
these otherwise discarded embryos have the potential to save lives.”
Senator Arlen Specter (R-PA)
Driving Human ES Cell
Therapies to the Clinic
Driving Human ES Cell
Therapies to the Clinic
16
Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
17
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
18
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
19
Our RPE ProgramOur RPE Program
Completed Build-out of GMP Facility
/ Commenced Scientific Work
Published Paper on Proof of Concept
RPE Study in RCS Rat
Hired Regulatory and RPE Clinical
Advisors
Developed Clinical Study Outlines /
Commenced Additional
Pharmacology Studies
Plan to Commence Toxicology
Studies in early 2007
Target IND Filing by Year-end 2007
20
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
Collaboration with Xgene Corp. to
test dermal cell line
24
2006 Corporate Milestones (How are we doing?)2006 Corporate Milestones (How are we doing?)
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
Still To Come
Achieve Listing on AMEX or NASDAQ Capital Market
Announce Corporate Partnership
25
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
26
Senior Management TeamSenior Management Team
Mr. William Caldwell, IV, Chief Executive Officer
30 Year Career in Management and Finance
Dr. Michael West, President and Chief Scientific Officer
Founder of Geron and ACT
Dr. Robert Lanza, VP of Medical & Scientific Development
25 Year Career in Biomedical and Scientific Research
Mr. Ivan Wolkind, VP of Finance, Chief Accounting Officer
25 Year Career in Accounting, Finance
Mr. Jon Atzen, SVP, General Counsel
15 Year Legal Career in Corporate and Securities Law
PIPEs Conference Presentations, November 2006

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PIPEs Conference Presentations, November 2006

  • 1. The PIPEs ConferenceThe PIPEs Conference November 2006November 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
  • 3. 3 Investment ConsiderationsInvestment Considerations 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
  • 4.
  • 5. 5
  • 6. 6 Blastomere Program – Significant AdvancementsBlastomere Program – Significant Advancements Transfer of 2 Blastomere Lines into GMP for Product Development Scientific Work Under-way to Produce Additional Blastomere Lines Planning to Seek Approval for NIH Federal Funding for hESC Research Utilizing Blastomere Lines Set-up Collaboration with Wi-Cell to Make Blastomere Lines Widely Available if Eligible for Federal Funding
  • 7. 7 Blastomere divided outgrowth first passage second passage established line 1.Derivation of hES cells from single blastomeres single blastomere Initial outgrowthcocultured blastomereblastomere isolation
  • 8. Why Human Embryonic Stem Cells? Why Human Embryonic Stem Cells?
  • 9. 9 Stem Cells are a Proven TherapyStem Cells are a Proven Therapy First successful bone marrow transplant (BMT) in 1968 Tens of thousands of patients have been cured with BMT Human embryonic stem cells first isolated in 1998 hESC’s have significant potential to treat diseases
  • 10. 10 The Significant Advantage of Embryonic Stem CellsThe Significant Advantage of Embryonic Stem Cells
  • 11. 11 ES Cells Form Complex TissuesES Cells Form Complex Tissues Science 2002 295: 819 PNAS 2003, 100 Suppl 1:11911 Intestine Muscle Retina Hair / Skin Cartilage Bone
  • 12. 12 Regenerative Medicine Curing Devastating Disease Regenerative Medicine Curing Devastating Disease 800,000 Parkinson's patients 750,000 liver disease patients 20,000,000 heart disease patients 20,000,000 diabetes patients 5,000,000 epilepsy patients 4,000,000 Alzheimer's patients 2,000,000 stroke patients 300,000 kidney disease patients
  • 13. 13 Growing Legislative SupportGrowing Legislative Support California & Proposition 71 Voters approve $3.0 billion in funding over next ten years Formation of CA Institute of Regenerative Medicine (CIRM) Recent awards of initial training grants Court trial complete CIRM public discussions – funding 2007 Other State Initiatives MA, CT, NJ, NY, IL, FL Federal Legislation Bi-partisan House of Representatives Bill H.R. 810 Federal Funding for Alternative Methods of Deriving Human ES Cells
  • 14. 14 The Next Frontier in MedicineThe Next Frontier in Medicine "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 “[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) “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 “This is not a matter of using a human embryo that has the potential to produce life. Rather, these otherwise discarded embryos have the potential to save lives.” Senator Arlen Specter (R-PA)
  • 15. Driving Human ES Cell Therapies to the Clinic Driving Human ES Cell Therapies to the Clinic
  • 16. 16 Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
  • 17. 17 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
  • 18. 18 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
  • 19. 19 Our RPE ProgramOur RPE Program Completed Build-out of GMP Facility / Commenced Scientific Work Published Paper on Proof of Concept RPE Study in RCS Rat Hired Regulatory and RPE Clinical Advisors Developed Clinical Study Outlines / Commenced Additional Pharmacology Studies Plan to Commence Toxicology Studies in early 2007 Target IND Filing by Year-end 2007
  • 20. 20
  • 21. 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 Collaboration with Xgene Corp. to test dermal cell line
  • 22. 24 2006 Corporate Milestones (How are we doing?)2006 Corporate Milestones (How are we doing?) 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 Still To Come Achieve Listing on AMEX or NASDAQ Capital Market Announce Corporate Partnership
  • 23. 25 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
  • 24. 26 Senior Management TeamSenior Management Team Mr. William Caldwell, IV, Chief Executive Officer 30 Year Career in Management and Finance Dr. Michael West, President and Chief Scientific Officer Founder of Geron and ACT Dr. Robert Lanza, VP of Medical & Scientific Development 25 Year Career in Biomedical and Scientific Research Mr. Ivan Wolkind, VP of Finance, Chief Accounting Officer 25 Year Career in Accounting, Finance Mr. Jon Atzen, SVP, General Counsel 15 Year Legal Career in Corporate and Securities Law