5/21/2012

1. Leading Regenerative Medicine
World Stem Cells & Regenerative Medicine Congress
May 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. Multiple Pluripotent Cell Platforms
• Single Blastomere-derived Embryonic Stem Cells
• Generating hESC without Destruction of Embryo
• Utilizes a single cell biopsy
• Our hESC lines exhibit all the standard characteristics and the
ability to differentiate into the cells of all three germ layers
both in vitro and in vivo.
• Induced Pluripotency Stem Cells (iPS)
• Early Innovator in Pluripotency (before iPS was even a term!)
• Recipient of National Institutes of Health Director's Opportunity Award
• Seminal paper identifying replicative senescence issue for vector-derived iPS cells
• Leading publication on protein induced iPS lines - avoids genetic manipulation with nucleic acid vectors
• Controlling Filings (earliest priority date) to use of OCT4 for inducing pluripotency
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Final Product Definition: hESC-derived
products will be manufactured using a cell
line made in 2005 from single cell isolated
without the destruction of any embryos

4. RPE Clinical Program

5. The RPE layer is critical to the function and health of photoreceptors and the
retina as a whole.
– RPE cells recycle photopigments, deliver, metabolize and store vitamin A, transport iron
and small molecules between retina and choroid and maintain Bruch’s membrane
– RPE loss leads to photoreceptor loss and eventually blindness, such as dry-AMD
– Loss of RPE layer and appears to lead to decline of Bruch’s membrane, leading
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 – aids manufacturing
• Small dosage vs. other therapies
• The eye is generally immune-privileged site, thus minimal
immunosuppression required
• Ease of administration, with no separate device approval and
straightforward surgical procedure
RPE cell therapy may impact over 200
retinal diseases
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Retinal Pigment Epithelial Cells - Rationale

7. Risk: Middle-aged adults have about a 2% risk of developing AMD - the risk increases to almost 30% in adults over age 75.
Expense: In 2012, the worldwide financial burden of vision loss due to AMD is estimated at >US$350 billion, with >US$250
attributed to direct health care expenditures.
On the Rise: Population demographics (“baby boomers”) combined with increased longevity predicts an increase of 50
percent or more in the incidence rate of AMD.
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The Unmet Medical Need
Early Stage AMD
(10-15M)
Intermediate AMD
(5-8M)
Late Stage AMD
(1.75M)
Few Drusen Deposits
Small in size
Geographic Atrophy: 1M
CNV Occurrence: 1.2M
Large Drusen Deposits
Pigment Change
Grade 0 0.5%
Grade 1 3%
Grade 2 9%
Grade 3 27%
Grade 4 43%
Probability of progression
to late stage AMD within 5 yrsU.S. Patent Population
We process 80 percent of
all our information
through our eyes. AMD
represents a huge impact
on QOL in later life

8. RPE Engraftment and Function – Pre-clinical
Human RPE cells engraft
and align with mouse RPE
cells in mouse eye
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treated
RPE cells rescued
photoreceptors and
slowed decline in acuity
in RCS rat
controltreated

9. • Established GMP process for differentiation and purification of RPE
– Virtually unlimited supply
– Pathogen-free GMP conditions
– Minimal batch-to-batch variation
– Characterized to optimize performance
– Virtually identical expression of RPE-specific genes to controls
GMP Manufacturing
Ideal Cell Therapy Product
• Centralized Manufacturing
• Small Doses
• Easily Frozen and Shipped
• Simple Handling by Doctor
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10. Characterizing Clinical RPE Lots
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Normal female (46 XX) karyotype
of the clinical RPE lot.
Up-regulation of RPE markers and
down-regulation of hESC markers

11. Characterizing Clinical RPE Lots
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Quantitative Potency Assay
Each lot is assessed by phagocytosis (critical
function in vivo) of fluorogenic bioparticles.
Flow cytometry histogram showing
phagocytosis of pHrodo bioparticles
4°C 37°C

12. Effects of Pigmentation
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Melanin content can be measured spectrophotometrically and used to determine the
optimal time to harvest and cryopreserve RPE.
y = 0.0141x + 0.0007
0.00
0.50
1.00
1.50
2.00
0 20 40 60 80 100120Absorbanceat475nm
µg/mL Melanin

13. Phase I - Clinical Trial Design
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SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K.
• 12 Patients for each trial, ascending dosages of 50K, 100K, 150K and 200K cells.
• Patients are monitored - including high definition imaging of retina
High Definition Spectral Domain Optical Coherence Tomography (SD-OCT)
Retinal Autofluorescence
50K Cells 100K Cells 150K Cells 200K Cells
Patient 1 Patients 2/3
DSMB Review DSMB Review
RPE and photoreceptor activity
compared before and after surgery

14. Phase I – Endpoints
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PRIMARY ENDPOINTS:
Safety Assessment acceptable, in the absence of:
 Adverse event related to cell product
 Any contamination with an infectious agent
 Cells exhibiting tumorigenic potential
SECONDARY
ENDPOINTS
Successful engraftment via:
 OCT, fundus and other similar imaging evidence
 ERG showing enhanced activity
Evidence of rejection:
 Imaging evidence that cells are no longer in the correct location
 ERG showing that activity has returned to pre-transplant conditions

15. RPE Clinical Program – to date
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World-leading eye surgeons and retinal
clinics participate in clinical trials, DSMB
and Scientific Advisory Board
• US Clinical Trial Sites
• Jules Stein Eye (UCLA)
• Wills Eye Institute
• Bascom Palmer Eye Institute
• Massachusetts Eye and Ear Infirmary
• Stargardts: 1st cohort complete, cleared to treat next cohort
• Dry AMD: 1st cohort complete, will submit data on patients 2
& 3 to DSMB in late May
• European Clinical Trial Sites
• Moorfields Eye Hospital
• Aberdeen Royal Infirmary
• 1st SMD patient treated, about to treat patients 2 & 3
ClinicalTrials.gov
US: NCT01345006, NCT01344993
UK: NCTO1469832

16. Surgical Overview
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Drs. Steven Schwartz and Robert Lanza
• Subretinal injection of 50,000 RPE cells in a volume of
150µl delivered into a pre-selected area of the
pericentral macula
• Procedure:
• 25 Gauge Pars Plana Vitrectomy
• Posterior Vitreous Separation (PVD Induction)
• Subretinal hESC-derived RPE cells injection
• Bleb Confirmation
• Air Fluid Exchange
Straight-forward surgery that is performed
on outpatient basis

17. Preliminary Results
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• Structural evidence confirmed cells had
attached and persisted
• No signs of hyperproliferation,
abnormal growth, or rejection
• Anatomical evidence of hESC-RPE
survival and engraftment.
• Clinically increased pigmentation
within the bed of the transplant

18. Preliminary Results
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Recorded functional visual improvements in both patients.
• SMD Patient: BCVA improved from hand motions to 20/800 and improved from 0 to 5
letters on the ETDRS visual acuity chart
• Dry AMD Patient: Vision improved in the patient with dry age-related macular
degeneration (21 ETDRS letters to 28)
• Nine Month Follow-up:
• Visual acuity gains remain stable for both patients; SMD Patient continues to
show improvement.
• Similar trends observed for latest AMD and SMD patients
• Update on U.K. SMD01 Patient (3 month follow-up)
• ETDRS: Improved from 5 letters to 10 letters
• Subjective: Reports significantly improved ability to read text on TV

19. Images of hESC-RPE transplantation site in SMD Patient
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pre-transplant 1wk post-op 6wk post-op
Color fundus photographs
Clinically increased pigmentation within the bed of the transplant

20. Images of hESC-RPE transplantation site in SMD Patient
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SD-OCT image collected at month 3 show survival and engraftment of RPE
 Migration of the transplanted cells to the desired anatomical location
3mo post-op

21. Phase II/III Design
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• Design of future studies dependent upon information gathered throughout PI/II
study
• Efficacy
• Patient population less VA impact 20/200?
• Multiple Injections
• Sub macular Injections
• Further evaluation of I/E criteria
• Potentially less immunosuppression
• Other considerations of efficacy:
• New or more sensitive technologies
• Possible saline placebo injection (same eye)
Working with our
experts/investigators in
design of studies

22. RPE Cells – Additional Indications
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• Myopic Macular Dystrophy (MMD)
• Retinopathy of Prematurity
• Angioid Streaks
• Retinitis Pigmentosa
• Bests Disease (vitelliform macular dystrophy)
• Multifocal Choroidopathy Syndromes
Combination Products
• Combined with other cell types (photoreceptor progenitors)
• Combined with anti-angiogenic agents, neuroprotective agents, etc.

23. Therapeutic Pipeline -
Ocular Programs

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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

25. ACT MSC Program

26. Ocular Program –
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Proprietary scaled
manufacturing for generating
“young” MSCs from hESC
and iPS lines
• hESC-and IPS-MSCs are expanded to large numbers in vitro
• Avoid senescence problem of “old” MSC’s
• Renewable cell source and “Off-The-Shelf” therapy
• hESC-derived MSCs are HLA I+, HLA II-
• Higher immunosuppressive potency relative to adult MSC
• MSCs can migrate to injury sites in eye, exert local
immunosuppressive effects, and repair damaged tissue
Ocular Products in Development
▫ Inflammatory diseases of the eye
▫ Photoreceptor/neuron-protective activity
▫ Tolerance to ocular grafts and devices
▫ Delivering therapeutic proteins to the eye

27. Mesenchymal SCs – Hemangioblast Derived
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33,000 units MSCs from hESC-MSCs
1 unit MSCs
from adult BM
Compared to Adult MSC
• Less labor-intensive
• Single Bank Regulatory Process
No need to derive new banks
Quality controls are easier to manage
• Larger yield of MSCs
Compared to hESC-direct
• Less labor-intensive
• Larger yield of MSCs
• Faster acquisition of markers
hESC Direct HB Method
Start 350,000 ESC 200,000 EB’s
Collect 48 days 44 days
Yield 4 Million 85 Million
Strongly advantageous to derive MSCs
from our hESC/HB method vs adult or
hESC-direct MSC

28. ACT Management Team
Highly Experienced and Tightly Integrated Management Team
Gary Rabin – Chairman & CEO
Dr. Robert Lanza, M.D. – Chief Scientific Officer
Edmund Mickunas – Vice President of Regulatory Affairs
Kathy Singh - Controller
Rita Parker – Director of Operations
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
Bill Douglass – Director of Corporate Communications & Social Media
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29. Thank you
For more information, visit www.advancedcell.com