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Advanced Cell Technology
World Stem Cells and 
Regenerative Medicine Congress
~ May 2011 ~
This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology
Inc”, or “the Company”) salient business characteristics.
The information herein 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. Ropes Gray
Cautionary Statement Concerning Forward-Looking Statements
2
At the Forefront of Regenerative Medicine
3
• Patented Technology for Producing hESCs without Harm to Embryo
• Working with Roslin Cells to create GMP-compliant hESC bank
• Commencing 2 Human Clinical Trials utilizing hESC-derived RPE Cells
• Stargardt’s Disease, aka Stargardt’s Macular Dystrophy (SMD)
• Dry AMD – (Dry Age-Related Macular Degeneration)
• First Clinical Trial Site Announced – Jules Stein Eye Institute (UCLA)
• Treatment of First Patients – enrolling patients presently.
• Expecting Preliminary Safety and Engraftment Data by Year-End
• Commencing European Trials first half 2012
Therapeutic Programs Indication Clinical Stage
Retinal Pigment Epithelium (RPE) Program Stargardt’s Macular Dystrophy
Dry AMD
IND approved Nov. 2010
IRM Approval (UCLA) April 2010
Presently Enrolling Patients
IND approved Jan. 3, 2011
IRB Approval (UCLA) April 2010
Presently Enrolling Patients
Myoblast Program Heart Disease, Heart Attack and Heart Failure Phase I successfully completed
FDA-approved for Phase II
Hemangioblast Program Diseases and Disorders of Circulatory and
Vascular System
Preclinical
Anticipate IND filing late 2011/early 2012
4
5
Platform Technology for Generating 
Robust Human Embryonic Stem Cells
Without the Need to Destroy Embryos
Single Blastomere Technology
Single Blastomere ES Cell Technology
6
• Company scientists successfully generate human embryonic stem cell lines without
destruction of embryo
• Utilizes single cell biopsy similar to pre-implantation genetic diagnostics (PGD).
• PGD is routine - used in thousands of pregnancies every year in United States and
Europe.
• Resulting human ES cell lines are more robust and reproducible than traditional ICM-
derived lines.
• Head-to-head comparison with 24 NIH lines: Average 5X more efficient than
best NIH lines for producing cells from all three germ layers.
• Reproduced and peer-reviewed on several occasions.
• Feb. 23, 2011: Issued broad patent on technology
Generating Robust ES Cells without
Destruction of Embryo
First Proven Alternative hESC Method
• Enables Derivation of new hESC Lines via single cell biopsy
method  Does not change the fate of the embryo from which
the biopsy was taken
• Outside scope of federal court’s original injunction in Sherley vs.
Sebelius (U.S.) and Advocate General Yves Bot opinion to Court of
Justice in Brüstle vs. Greenpeace (Europe).
• Several hESC lines awaiting NIH approval for funding – embryos
from which these lines were derived were not destroyed.
• Roslin Cells and ACT plan to generate GMP-compliant bank of
human ES Cells for research and commercial uses.
• Technology was used to develop FDA approved hESC Master Cell
Bank for our clinical trials for Stargardt’s disease and Dry AMD
7
Human RPE Cells
Differentiated from
Blastomere hESC Lines
8
Retinal Pigmented Epithelial Cells
ACT Ocular Program
• The RPE layer is critical to the function and health of
photoreceptors and the retina as a whole.
RPE cells secrete trophic factors and impact on the chemical environment of the subretinal
space.
– recycle photopigments
– deliver, metabolize and store vitamin A
– transport iron and small molecules between retina and choroid
– maintain Bruch’s membrane
RPE malfunction may lead to photoreceptor loss and eventually blindness
Retinal Pigment Epithelium
9
Photoreceptor viability is
dependent on RPE cell layer
Retinal Pigment Epithelial Cells - Rationale
• Pigmented RPE cells are easy to identify (no need
for further staining)
• Small dosage vs. other therapies
• The eye is generally immune-privileged site, thus
minimal immunosuppression required, which may be
topical.
• Ease of administration
– Doesn’t require separate approval by the FDA (universal applicator)
– Procedure is already used by eye surgeons; no new skill set required for doctors
RPE cell therapy may impact over
200 retinal diseases
10
• Established GMP-compliant process for the Reproducible Differentiation
and Purification of RPE cells.
– Virtually unlimited supply of cells
– Can be derived under GMP conditions pathogen-free
– Can be produced with minimal batch-to-batch variation
– Can be thoroughly characterized to ensure optimal performance
– Molecular characterization studies reveal similar expression of RPE-specific genes to controls
and demonstrates the full transition from the hESC state.
Manufacturing
11
Ideal Cell Therapy Product
• Centralized Manufacturing
• Small Doses that can be Frozen and Shipped
• Ease-of-Handling by Doctor
Differentiation Media is Not Permissive for hESCs
12
Harvest for 
Cryopreservation
Harvest for 
Cryopreservation
ES Cells
EB
Formation RPE isolation P1 P2
0 5 weeks 12 weeks 15 weeks 18 weeks
Up to 10 percent hESC spiked
No hESC’s in harvested cells
Limit of Detection for hESC
1:20,000,000  cells
RPE differentiation is the default pathway under
ACT’s Patented Culture Conditions
3 weeks
EB 
Outgrowth
30 Weeks
Safety Studies
13
Extensive Safety Studies Shows 
Lack of Tumorigenicity
Long‐term data (spanning 
the life of the animals) 
revealed no evidence of 
teratoma formation after 
subretinal hESC‐RPE 
transplantation. 
RPE Engraftment – Mouse Model
For each set: Panel (C) is a bright field image and
Panel (D) shows immunofluorescence with anti-
human bestrophin (green) and anti-human
mitochondria (red) merged and overlayed on the
bright field image. Magnification 400x
Human RPE cells engraft
and align with mouse RPE
cells in mouse eye
14
Animal Studies
RPE treatment in animal model of retinal dystrophy has slowed the
natural progression of the disease by promoting photoreceptor
survival.
RPE cells rescued photoreceptors and
slowed decline in visual acuity
treated control
Photoreceptor
layer
15
16
• No Observed Pathologies
– All eye slides were reviewed by a board certified veterinary
pathologists.
• Only typical retinal morphology was observed.
• No ectopic tissue or abnormal pathology was observed.
• Histological examination of the retinas demonstrated the presence of
human-specific nuclear marker.
• Staining with human-specific proliferating cell nuclear antigen (PCNA) was
negative, indicating that there was no proliferation of the human RPE cells.
16
Safety/Biodistribution Study was designed to determine the ability of the cells to
migrate or disseminate outside the area of the transplant as well as assess the
general safety of the cells in an animal model.
RPE Engrafted without Adverse Effects in the Eye
16
17
Functional Rescue observed in Stargardt Mouse Model
– Efficacious dose established
– Near-normal functional measurements were recorded at >60 days.
Chemically Immunosuppressed RCS Rat Study
– Photoreceptor Rescue
• Extensive photoreceptor rescue. Photoreceptor layer 5-7 cell layers deep in treated
animals versus 1 cell layer deep in untreated control.
– Improved Visual Acuity
• 100 percent improvement in visual performance over untreated control
• Restored to 70 percent of normal healthy animals.
– Survival of transplanted cells more than 220 days post-op
– No Tumor Formation
– Normal Pathology
• No indication of extraneous non-retinal cells in eyes.
17
RPE Cells Engraft and Function in Two Animal Models of Disease
Functional Integration of
RPE Cells into RPE layer
was Observed
17
• Also referred to as “Juvenile Macular Degeneration”
– Causes progressive vision loss beginning in childhood.
– Stargardt’s Macular Dystrophy afflicts approximately 25,000 people in the U.S.
– Usually diagnosed in individuals under the age of twenty, when decreased central vision is first noticed.
• Animal Model
– 100 percent effectiveness of RPE injections.
– Treated animals retained 70 percent visual acuity of normal animal, whereas untreated went blind.
• ACT has obtained Orphan Drug Designation in United States
– 7 Years of Market Exclusivity for using RPE cells to treat Stargardt’s Disease
– Secured positive opinion on similar Orphan Status designation in Europe
Stargardt’s Macular Dystophy
18
FDA-approved for Phase I/II clinical trial
Patient Recruitment Underway
• Estimate over 30 Million patients in North America and Europe
• Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration
• Prevalence increases to 30% in patients 75 to 85 years of age.
Dry AMD (non-exudative)
– The most common form of AMD (estimates as high as 90 percent)
– No Currently Effective Therapy available
– Estimated $20-30 Billion market
Age Related Macular Degeneration
19
FDA-approved for Phase I/II clinical trial
Patient Recruitment Underway
• 12 Patients for each trial, ascending dosages of 50K, 100K, 150K and 200K cells.
– For each cohort, 1st patient treatment followed by 6 week DMSB review before remainder of cohort.
• Patients will be monitored weekly - including high definition imaging of retina
High Definition Spectral Domain Optical Coherence Tomography (SD-OCT)
Retinal Autofluorescence
Adaptive Optics Scanning Laser Ophthalmoscopy (AOSLO)
Phase I: Clinical Trial Design
20
50K Cells 100K Cells 150K Cells 200K Cells
Patient 1 Patients 2/3
DMSB Review DMSB Review
Anticipate engraftment and photoreceptor
activity data early in Phase I study.
Permit comparison of RPE and
photoreceptor activity before
and after treatment
21
• Stargardt’s Disease
• IND approved in November 2010
• Jules Stein Eye Institute (UCLA) announced as first clinical trial site
• European CTA filed.
• Orphan Drug Designation granted in U.S., Positive Opinion in Europe
• Ideal Candidate for compassionate use approval
• Would permit sales in certain European countries prior to Market Authorization
• Reimbursed at premium price in several countries
• Dry AMD
• IND: Approved in December 2010
• Jules Stein Eye Institute (UCLA)announced as first clinical trial site
• European CTA in preparation.
Program Summary
22
Intellectual Property
Retinal Pigment Epithelial Cells
• Worldwide Patent Filings
• Dominant Patent Position for Treating Retinal Degeneration
• US Patent 7,794,704 broadly cover methods for treating retinal degeneration using human RPE cells 
differentiated from human embryonic stem cells (hESCs). Includes, but is not limited to, Stargardt’s 
disease, retinitis pigmentosa, and macular degeneration.  
• Broad Coverage for Manufacturing RPE Cells from hESC
• U.S. Patents 7,736,896 and 7,795,025 are broadly directed to the production of retinal pigment 
epithelial (RPE) cells from human embryonic stem cells. Covers fundamental methods for 
manufacturing RPE cells from human ES cells suitable for use in generating transplantable cells for 
treatment of human patients.
Single Blastomere Technology
• Worldwide Patent Filings
• Broad Claims to use of Single Blastomeres
• U.S. Patent 7,893,315 broadly covers ACT’s proprietary single‐blastomere technology that provides a 
non‐destructive alternative for deriving human embryonic stem cell (hESC) lines.
Other Notables
• Controlling Filings (earliest priority date) to use of OCT4 relating to induced pluripotency (iPS).
• Pending and issued patent filings directed to significant protocols for transdifferentiation.
The Advanced Cell Technology Team
World Class Scientific Team
Seasoned Management Team
Dr. Robert Lanza, M.D. – Chief Scientific Officer
Dr. Irina Klimanskaya, Ph.D. – Director of Stem Cell Biology
Dr. Matthew Vincent, Ph.D. – Director of Business Development
23
Bill Douglass – Director of Corporate Communications & Social Media
Rita Parker – Director of Operations
Dr. Roger Gay, PhD – Senior Director of Manufacturing
Edmund Mickunas – Vice President of Regulatory Affairs
Stephen Price – Interim SVP – Corporate Development
Gary Rabin – Interim Chairman and CEO
Thank you for your time
For more information, visit  www.advancedcell.com
24

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ACT Advanced Cell Therapy Clinical Trials

  • 2. This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology Inc”, or “the Company”) salient business characteristics. The information herein 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. Ropes Gray Cautionary Statement Concerning Forward-Looking Statements 2
  • 3. At the Forefront of Regenerative Medicine 3 • Patented Technology for Producing hESCs without Harm to Embryo • Working with Roslin Cells to create GMP-compliant hESC bank • Commencing 2 Human Clinical Trials utilizing hESC-derived RPE Cells • Stargardt’s Disease, aka Stargardt’s Macular Dystrophy (SMD) • Dry AMD – (Dry Age-Related Macular Degeneration) • First Clinical Trial Site Announced – Jules Stein Eye Institute (UCLA) • Treatment of First Patients – enrolling patients presently. • Expecting Preliminary Safety and Engraftment Data by Year-End • Commencing European Trials first half 2012
  • 4. Therapeutic Programs Indication Clinical Stage Retinal Pigment Epithelium (RPE) Program Stargardt’s Macular Dystrophy Dry AMD IND approved Nov. 2010 IRM Approval (UCLA) April 2010 Presently Enrolling Patients IND approved Jan. 3, 2011 IRB Approval (UCLA) April 2010 Presently Enrolling Patients Myoblast Program Heart Disease, Heart Attack and Heart Failure Phase I successfully completed FDA-approved for Phase II Hemangioblast Program Diseases and Disorders of Circulatory and Vascular System Preclinical Anticipate IND filing late 2011/early 2012 4
  • 6. Single Blastomere ES Cell Technology 6 • Company scientists successfully generate human embryonic stem cell lines without destruction of embryo • Utilizes single cell biopsy similar to pre-implantation genetic diagnostics (PGD). • PGD is routine - used in thousands of pregnancies every year in United States and Europe. • Resulting human ES cell lines are more robust and reproducible than traditional ICM- derived lines. • Head-to-head comparison with 24 NIH lines: Average 5X more efficient than best NIH lines for producing cells from all three germ layers. • Reproduced and peer-reviewed on several occasions. • Feb. 23, 2011: Issued broad patent on technology Generating Robust ES Cells without Destruction of Embryo
  • 7. First Proven Alternative hESC Method • Enables Derivation of new hESC Lines via single cell biopsy method  Does not change the fate of the embryo from which the biopsy was taken • Outside scope of federal court’s original injunction in Sherley vs. Sebelius (U.S.) and Advocate General Yves Bot opinion to Court of Justice in Brüstle vs. Greenpeace (Europe). • Several hESC lines awaiting NIH approval for funding – embryos from which these lines were derived were not destroyed. • Roslin Cells and ACT plan to generate GMP-compliant bank of human ES Cells for research and commercial uses. • Technology was used to develop FDA approved hESC Master Cell Bank for our clinical trials for Stargardt’s disease and Dry AMD 7 Human RPE Cells Differentiated from Blastomere hESC Lines
  • 9. • The RPE layer is critical to the function and health of photoreceptors and the retina as a whole. RPE cells secrete trophic factors and impact on the chemical environment of the subretinal space. – recycle photopigments – deliver, metabolize and store vitamin A – transport iron and small molecules between retina and choroid – maintain Bruch’s membrane RPE malfunction may lead to photoreceptor loss and eventually blindness Retinal Pigment Epithelium 9 Photoreceptor viability is dependent on RPE cell layer
  • 10. Retinal Pigment Epithelial Cells - Rationale • Pigmented RPE cells are easy to identify (no need for further staining) • Small dosage vs. other therapies • The eye is generally immune-privileged site, thus minimal immunosuppression required, which may be topical. • Ease of administration – Doesn’t require separate approval by the FDA (universal applicator) – Procedure is already used by eye surgeons; no new skill set required for doctors RPE cell therapy may impact over 200 retinal diseases 10
  • 11. • Established GMP-compliant process for the Reproducible Differentiation and Purification of RPE cells. – Virtually unlimited supply of cells – Can be derived under GMP conditions pathogen-free – Can be produced with minimal batch-to-batch variation – Can be thoroughly characterized to ensure optimal performance – Molecular characterization studies reveal similar expression of RPE-specific genes to controls and demonstrates the full transition from the hESC state. Manufacturing 11 Ideal Cell Therapy Product • Centralized Manufacturing • Small Doses that can be Frozen and Shipped • Ease-of-Handling by Doctor
  • 12. Differentiation Media is Not Permissive for hESCs 12 Harvest for  Cryopreservation Harvest for  Cryopreservation ES Cells EB Formation RPE isolation P1 P2 0 5 weeks 12 weeks 15 weeks 18 weeks Up to 10 percent hESC spiked No hESC’s in harvested cells Limit of Detection for hESC 1:20,000,000  cells RPE differentiation is the default pathway under ACT’s Patented Culture Conditions 3 weeks EB  Outgrowth 30 Weeks
  • 14. RPE Engraftment – Mouse Model For each set: Panel (C) is a bright field image and Panel (D) shows immunofluorescence with anti- human bestrophin (green) and anti-human mitochondria (red) merged and overlayed on the bright field image. Magnification 400x Human RPE cells engraft and align with mouse RPE cells in mouse eye 14
  • 15. Animal Studies RPE treatment in animal model of retinal dystrophy has slowed the natural progression of the disease by promoting photoreceptor survival. RPE cells rescued photoreceptors and slowed decline in visual acuity treated control Photoreceptor layer 15
  • 16. 16 • No Observed Pathologies – All eye slides were reviewed by a board certified veterinary pathologists. • Only typical retinal morphology was observed. • No ectopic tissue or abnormal pathology was observed. • Histological examination of the retinas demonstrated the presence of human-specific nuclear marker. • Staining with human-specific proliferating cell nuclear antigen (PCNA) was negative, indicating that there was no proliferation of the human RPE cells. 16 Safety/Biodistribution Study was designed to determine the ability of the cells to migrate or disseminate outside the area of the transplant as well as assess the general safety of the cells in an animal model. RPE Engrafted without Adverse Effects in the Eye 16
  • 17. 17 Functional Rescue observed in Stargardt Mouse Model – Efficacious dose established – Near-normal functional measurements were recorded at >60 days. Chemically Immunosuppressed RCS Rat Study – Photoreceptor Rescue • Extensive photoreceptor rescue. Photoreceptor layer 5-7 cell layers deep in treated animals versus 1 cell layer deep in untreated control. – Improved Visual Acuity • 100 percent improvement in visual performance over untreated control • Restored to 70 percent of normal healthy animals. – Survival of transplanted cells more than 220 days post-op – No Tumor Formation – Normal Pathology • No indication of extraneous non-retinal cells in eyes. 17 RPE Cells Engraft and Function in Two Animal Models of Disease Functional Integration of RPE Cells into RPE layer was Observed 17
  • 18. • Also referred to as “Juvenile Macular Degeneration” – Causes progressive vision loss beginning in childhood. – Stargardt’s Macular Dystrophy afflicts approximately 25,000 people in the U.S. – Usually diagnosed in individuals under the age of twenty, when decreased central vision is first noticed. • Animal Model – 100 percent effectiveness of RPE injections. – Treated animals retained 70 percent visual acuity of normal animal, whereas untreated went blind. • ACT has obtained Orphan Drug Designation in United States – 7 Years of Market Exclusivity for using RPE cells to treat Stargardt’s Disease – Secured positive opinion on similar Orphan Status designation in Europe Stargardt’s Macular Dystophy 18 FDA-approved for Phase I/II clinical trial Patient Recruitment Underway
  • 19. • Estimate over 30 Million patients in North America and Europe • Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration • Prevalence increases to 30% in patients 75 to 85 years of age. Dry AMD (non-exudative) – The most common form of AMD (estimates as high as 90 percent) – No Currently Effective Therapy available – Estimated $20-30 Billion market Age Related Macular Degeneration 19 FDA-approved for Phase I/II clinical trial Patient Recruitment Underway
  • 20. • 12 Patients for each trial, ascending dosages of 50K, 100K, 150K and 200K cells. – For each cohort, 1st patient treatment followed by 6 week DMSB review before remainder of cohort. • Patients will be monitored weekly - including high definition imaging of retina High Definition Spectral Domain Optical Coherence Tomography (SD-OCT) Retinal Autofluorescence Adaptive Optics Scanning Laser Ophthalmoscopy (AOSLO) Phase I: Clinical Trial Design 20 50K Cells 100K Cells 150K Cells 200K Cells Patient 1 Patients 2/3 DMSB Review DMSB Review Anticipate engraftment and photoreceptor activity data early in Phase I study. Permit comparison of RPE and photoreceptor activity before and after treatment
  • 21. 21 • Stargardt’s Disease • IND approved in November 2010 • Jules Stein Eye Institute (UCLA) announced as first clinical trial site • European CTA filed. • Orphan Drug Designation granted in U.S., Positive Opinion in Europe • Ideal Candidate for compassionate use approval • Would permit sales in certain European countries prior to Market Authorization • Reimbursed at premium price in several countries • Dry AMD • IND: Approved in December 2010 • Jules Stein Eye Institute (UCLA)announced as first clinical trial site • European CTA in preparation. Program Summary
  • 22. 22 Intellectual Property Retinal Pigment Epithelial Cells • Worldwide Patent Filings • Dominant Patent Position for Treating Retinal Degeneration • US Patent 7,794,704 broadly cover methods for treating retinal degeneration using human RPE cells  differentiated from human embryonic stem cells (hESCs). Includes, but is not limited to, Stargardt’s  disease, retinitis pigmentosa, and macular degeneration.   • Broad Coverage for Manufacturing RPE Cells from hESC • U.S. Patents 7,736,896 and 7,795,025 are broadly directed to the production of retinal pigment  epithelial (RPE) cells from human embryonic stem cells. Covers fundamental methods for  manufacturing RPE cells from human ES cells suitable for use in generating transplantable cells for  treatment of human patients. Single Blastomere Technology • Worldwide Patent Filings • Broad Claims to use of Single Blastomeres • U.S. Patent 7,893,315 broadly covers ACT’s proprietary single‐blastomere technology that provides a  non‐destructive alternative for deriving human embryonic stem cell (hESC) lines. Other Notables • Controlling Filings (earliest priority date) to use of OCT4 relating to induced pluripotency (iPS). • Pending and issued patent filings directed to significant protocols for transdifferentiation.
  • 23. The Advanced Cell Technology Team World Class Scientific Team Seasoned Management Team Dr. Robert Lanza, M.D. – Chief Scientific Officer Dr. Irina Klimanskaya, Ph.D. – Director of Stem Cell Biology Dr. Matthew Vincent, Ph.D. – Director of Business Development 23 Bill Douglass – Director of Corporate Communications & Social Media Rita Parker – Director of Operations Dr. Roger Gay, PhD – Senior Director of Manufacturing Edmund Mickunas – Vice President of Regulatory Affairs Stephen Price – Interim SVP – Corporate Development Gary Rabin – Interim Chairman and CEO