1/11/2012

1. Leading Regenerative Medicine
Regenerative Medicine Insight Track
~ Biotech Showcase - January 2012 ~

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
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3. ACT Ocular Programs

4. 4
Retinal Pigment Epithelial Cells
 Macular Degeneration - dry AMD, Stargardt’s Disease, MMD
 Retinitis Pigmentosa
 Photoreceptor protection
Hemangioblast cells
 Ischemic retinopathy
– diabetic retinopathy, vascular occlusions
Retinal Neural Progenitor cells
Isolated Protective Factors
 Photoreceptor Loss, Modulation of Müller Cells
 Protection of Retinal Ganglion cells (Glaucoma)
Corneal Endothelium, Corneal Epithelium,
Descemet’s Membrane
 Corneal Disease
Mesenchymal Stromal Cells
 Glaucoma, Uveitis
 Retinitis Pigmentosa
 Management of Ocular Surfaces
light
retina
RPElayer
Photoreceptors

5. 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 loss may lead to photoreceptor loss and eventually blindness, such as dry-AMD
– Loss of RPE layer and Bruch’s membrane is substantial feature underlying development
of dry-AMD, and may be involved in progression from dry-AMD to wet-AMD
• Discrete differentiated cell population as target
• Failure of target cells results in disease progression
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Retinal Pigment Epithelial Cells - Rationale
RPE cell as Target

6. • Pigmented RPE cells are easy to identify (no need
for further staining) – impacts manufacturing
• 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
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Retinal Pigment Epithelial Cells - Rationale

7. • 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.
GMP Manufacturing
Ideal Cell Therapy Product
• Centralized Manufacturing
• Small Doses that can be Frozen and Shipped
• Relative Ease-of-Handling by Doctor
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8. 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
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9. RPE Engraft and Function in 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
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10. • 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 are monitored - including high definition imaging of retina
High Definition Spectral Domain Optical Coherence Tomography (SD-OCT)
Retinal Autofluorescence
Phase I - Clinical Trial Design
50K Cells 100K Cells 150K Cells 200K Cells
Patient 1 Patients 2/3
DSMB Review DSMB Review
Engraftment and photoreceptor activity data
available early in Phase I study.
Permit comparison of RPE and
photoreceptor activity before
and after treatment
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11. • Stargardt’s (SMD) Disease
• IND approved in November 2010
• European CTA Approved – enrolling patients
• Orphan Drug Designation granted in U.S. and Europe
• The SMD patient is a 26 year old female with baseline best corrected visual acuity
of hand motion that corresponded to 0 letters in the ETDRS chart.
• Dry AMD
• IND approved in December 2010
• European CTA in preparation
• The dry AMD patient is a 77 year old female with baseline BCVA of 20/500, that
corresponded to 21 letters in the ETDRS chart.
RPE Program Summary
July 12, 2011: First Patients in each trial
were treated by Dr. Steven Schwartz, M.D
at Jules Stein Eye Institute (UCLA)
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12. Surgical Overview
• Prospective clinical studies to determine the safety and tolerability of
sub-retinal transplantation of hESC-derived RPE cells.
• Subretinal injection of 50,000 hESC-derived RPE cells in a volume of
150µl was delivered into a pre-selected area of the pericentral macula
• Vitrectomy including surgical induction of posterior vitreous separation
from the optic nerve was carried out
• 25 Gauge Pars Plana Vitrectomy
• Posterior Vitreous Separation (PVD Induction)
• Subretinal hESC-derived RPE cells injection
• Bleb Confirmation
• Air Fluid Exchange
Drs. Steven Schwartz and Robert Lanza
Straightforward surgical approach
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13. Surgical Overview
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Autofluorescence
images of retinas.
The dark spots in the
side panels show a
large area of atrophy in
the macular region.
First SMD Patient
First dry AMD Patient

14. Surgical Overview
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15. Surgical Overview
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Remove gel from inner
surface of retina
Injection with bleb
formation
Air fluid exchange
Injection bleb formed at
interface of atrophic
retina and normal retina

16. Ocular Program – Corneal Endothelium
• More than 10 million people with corneal blindness
• The cornea is the most transplanted organ (1/3 of all
transplants performed due to endothelial failure)
• Solutions include the transplantation of whole cornea
“Penetrating Keratoplasty” (PKP)
• More popular: Transplantation of just corneal
endothelium & Descemet’s membrane (DSEK/DSAEK).
hESC-derived corneal
endothelium resembles
normal human corneal
endothelium
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17. Ocular Program – Hemangioblasts
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The Hemangioblast cell is a multipotent cell, and a common
precursor to hematopoietic and endothelial cells.
Hemangioblast cells can be used to
produce all cell types in the circulatory
and vascular systems
• Hemangioblast cells can self-renew.
• Hemangioblast cells can be used to achieve
vascular repair.
• Hemangioblast activity could potentially be
harnessed to treat diseases such as myocardial
infarction, stroke, cancer, vascular injury and
blindness.

18. Ocular Program – Hemangioblasts
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Hemangioblasts induce reparative
intraretinal angiogenesis is various
animal models of ischemic retinopathies
• Revascularization is observed in animals
injected either intravitreally or
intravenously with hESC-derived
hemangioblasts
• ischemia-reperfusion injury
• diabetic retinopathy
• GFP-labeling reveals incorporation of
injected cells into the vasculature of the
eye during angiogenesis
Repair of ischemic retinal vasculature in a mouse
after injection of hESC-derived hemangioblasts

19. • Generated various retinal neural progenitor cell types – or RNP cells
• From both embryonic and iPS cell sources.
• Discovered a new RNP cell type.
• Tested in mouse model for retinal degeneration - ELOVL4-TG2 mice
• Observed both structural and physiological consequences
After 2 months
• ERG - increases in both the a-wave and b-wave
• OCT - increases in central retinal thickness
Ocular Program – Retinal Neural Progenitors
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hESC-derived RNP cells reversed the progression of photoreceptor
degeneration– and appeared to promote regeneration
• Defined culture conditions
• High yield from hESC and iPS
• Homogeneous and highly pure
preparations

20. Ocular Program – Mesenchymal Stromal Cells
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Proprietary Large Scale
Manufacturing Process for
Generating “young” MSCs
from hESC and iPS lines
• hESC-MSCs and iPS-MSCs can be expanded to large
numbers in vitro
• Avoid premature senescence problem of “old” MSC’s
• Superior quality controls for a renewable cell source
• “Off-The-Shelf” therapy, available for immediate use
• hESC-derived MSCs are HLA I+, HLA II-
• MSCs can migrate to injury sites in eye – exert
immunosuppressive effects, and facilitate repair of
damaged tissues
Ocular Products in Development
▫ Treating inflammatory diseases of the eye
▫ Providing photoreceptor/neuron-protective activity
▫ Promoting tolerance to ocular grafts and devices
▫ Delivering therapeutic proteins to the eye.

21. Platform Technology for Generating
Robust Human Embryonic Stem Cells
Without the Need to Destroy Embryos
Single Blastomere Technology

22. 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
• Utilizes single cell biopsy similar to pre-implantation genetic diagnostics
(PGD).
• Roslin Cells and ACT plan to generate GMP-compliant bank of human
ES Cells for research and commercial uses.
• Head-to-head comparison with 24 NIH lines: Average 5X more efficient
than best NIH lines for producing cells from all three germ layers.
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Single Blastomere Technology

23. Intellectual Property Overview
Retinal Pigment Epithelial Cells
•Worldwide Patent Portfolio
•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).
•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.
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.
Hemangioblast Technology
•Worldwide Patent Filings
•U.S. Patent 8,017,393 - Dominant Patent Position for deriving hemangioblast cells from embryonic stem cells.
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.
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24. Financial Update – Strong Balance Sheet
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Most Stable Financial Situation In Company History
• The Company ended 2011 Q3 with $13.9 million cash on hand
• $17 million more equity available
• Virtually debt-free
• Able to self-fund both U.S. clinical trials and EU clinical trial
• Significantly deepened management team (and on-going)
• Put in place first organizational reporting lines in ACT history
• Robert Langer, Zohar Loshitzer and Greg Perry join ACT board, bringing
remarkable scientific, entrepreneurial and partnering skills
• One additional Board member to announce
• Unqualified audit opinion Continuing clinical trials with a
strong balance sheet

25. ACT Management 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. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research
Dr. Roger Gay, Ph.D. - Senior Director of Manufacturing
Dr. Matthew Vincent, Ph.D. – Director of Business Development
Gary Rabin – Chairman and CEO
Edmund Mickunas – Vice President of Regulatory Affairs
Kathy Singh - Controller
Rita Parker – Director of Operations
Bill Douglass – Director of Corporate Communications & Social Media
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26. Thank you
For more information, visit www.advancedcell.com