1. Annual
Meeting
LEADING REGENERATIVE MEDICINE
Tuesday, Oct. 22, 2013
Palm Springs, CA

2. Cautionary Statement Concerning Forward-Looking Statements
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
2

3. Gary Rabin
Chief Executive Officer, Chairman
CEO UPDATE
3

4. ACT Overview
• We continue to appreciate your support through this period where we
are working to finally put the past to rest. We share your frustration with
how long it has taken to put the last matters to closure
• We concluded highly successful meeting with our OAB
• We have worked through a 2014 goal-driven plan with our senior
management team to advance both our clinical RPE program and our
other ophthalmic activities, as well as moving our MSC activities
toward the clinic
• We are at the beginnings of seeing a real opportunity in our preclinical animal studies and translating that into a veterinary plan
• We are starting to engage fully in our up-listing and institutional capital
raising strategy, and are optimistic that we will finally achieve our
goals.
4

5. Robust Development Pipeline Provides Multiple Opportunities to
Commercialize and Partner
Ophthalmology Programs
Pre-clinical/
in vitro
Dry AMD
SMD
MMD
Photoreceptors
Ganglion
Neurons
Cornea
Platelets
Mesenchymal
Stem Cells
5
POC –
Animal Studies
IND Approved
Phase I
Phase II
Phase III
Approval

6. Robust Development Pipeline Provides Multiple Opportunities to
Commercialize and Partner
Ophthalmology Programs
Pre-clinical/
in vitro
Dry AMD
SMD
MMD
Photoreceptors
Ganglion
Neurons
Cornea
Platelets
Mesenchymal
Stem Cells
6
POC –
Animal Studies
IND Approved
Phase I
Phase II
Phase III
Approval
First Priority Based
On Current
Funding
Advance into
Phase I and
Partner
Potential Gov’t Funding
Based on POC results,
pursue appropriate
funding and collaborations

7. Several Important clinical milestones Q4 2013, 2014
Phase I
150K
Dry AMD
& SMD Trials
200K ?
Cohort 2a
100K
Phase II
4Q 2013
MMD Trial
7
Patient follow-up
Patient Treatment
PII design
1Q 2014
Phase I
3Q 2014
Patient Treatment
Jan 2015

8. BY PRODUCING THE
HIGHEST DEGREE OF QUALITY
VETERINARY THERAPEUTICS
WE INTEND TO BE
A LEADER IN
VETERINARY
REGENERATIVE MEDICINE
8
September 19, 2013 –
“ACT Files Investigational New Animal
Drug (INAD) Application with FDA to Treat
10 Different Disease Indications Using
Pluripotent Stem Cells”

9. Ophthalmic Advisory Group Summary
• Extremely successful and productive meeting
• Complete unanimity among company, investigators, and
independent board members
• Plan agreed on Phase 2 trial design including endpoints and
termination strategy for current trials
• Currently developing materials to discuss strategy with FDA
• Data review and results showing surprising strong statistical success.
All board members impressed by aggregate data showing acuity
improvements as well as differential between treated and untreated
eyes
• Working on beginning to write paper for submission to top peer
reviewed medical journals
9

10. Dr. Robert Lanza
Chief Scientific Officer
SCIENCE UPDATE
10

11. Research & Development Programs
 Treatment of eye diseases
• Retinal pigment epithelium (macular degeneration)
•
•
•
•
Retinal ganglion progenitors (glaucoma)
Photoreceptor progenitors (advanced blindness)
Mesenchymal stem cells (uveitis)
Corneal endothelium (corneal repair)
 Single-blastomere technology
• Master Cell Bank (ethically-compliant/xeno-free) for future clinical trials
 Induced pluripotent stem (iPS) cell technology
• Episomal, mRNA, microRNA vs. other reprogramming methods
• Master Cell Bank for Phase I/II clinical trials
 Megakaryocytes/platelets
• Preclinical studies safety & efficacy studies
 Mesenchymal stem cells
• Manufacturing under GMP conditions (for pre-clinical/clinical studies)
• Rodent studies (multiple sclerosis, lupus, pain, ALS, Alzheimer's)
• Large-animal studies (hepatitis, glomerulonephritis, osteoarthritis, Crohn’s disease, IBS,
spinal cord/disc disease, meningoencephalitis, hemolytic anemia, pancreatitis, sepsis)
11

12. Ocular Programs
Retinal ganglion progenitors
 Glaucoma
Retinal pigment epithelium
 Macular degeneration (AMD, Stargardt’s)
 Myopia
Corneal endothelium
 Corneal disease
light
Retina
Mesenchymal stem cells
 Uveitis, glaucoma
12
Retinal neural progenitors
 Photoreceptor replacement
 Neuroprotection

13. RPE Clinical Trials
 US Clinical Trials
•
Jules Stein Eye Institute (UCLA)
•
Wills Eye Institute
•
Bascom Palmer Eye Institute
•
Mass Eye & Ear
Dry AMD
Twelve patients treated
- 3 patients (50K cells)
- 6 patients (100K cells)
(includes 3 better vision)
- 3 patients (150K cells)
Stargardt’s Disease
Nine patients treated
- 3 patients (50K cells)
- 4 patients (100K cells)
(includes 1 better vision)
- 2 patients (150K cells)
13
ClinicalTrials.gov
US: NCT01345006, NCT01344993
UK: NCTO1469832
July 12, 2011: First patient in both US trials were treated
at UCLA by Steven Schwartz, M.D.

European Clinical Trial Site
•
Moorfields Eye Hospital
Stargardt’s Disease
Nine patients treated
- 3 patients (50K cells)
- 3 patients (100K cells)
- 3 patients (150K cells)

14. OVERALL RESULTS:
 No major safety issues related to stem cell treatment
 Clear signs of long-term engraftment & survival
DAY 1
2 MONTHS
6 MONTHS
 During the one-year follow-up period, patients in both the
SMD and dry-AMD clinical trials have shown significant
improvement in visual acuity in the RPE-treated eyes
• vision in one patient improved from
20/400 to 20/40 in first month
 By contrast, the fellow (untreated) eyes remained
unchanged or continued to show decline in visual acuity
during the same time period
14

15. Next Steps – RPE Program

Complete and publish results of Phase I/II Clinical Trials

Preparation and design of Phase II Clinical Trials

NED-7 GMP Master Cell Bank (MCB)
–
hESCs derived using single-blastomere technology (no embryos destroyed)
– No mouse feeders (no longer a xeno product)
– MCB (~500 vials generated)
» Working Cell Bank (~260 vials generated)
» ~800 vials of RPE (made from 2 vials of WCB)
(enough to treat ~800 patients, although could have generated more)
* waiting for manufacturing and regulatory sign-offs

15
Begin Phase I Clinical Trial (UCLA) to treat myopia

16. Megakaryocytes & Platelets
• hES/iPSC-platelets participate in clot formation
• Incorporate into mouse thrombus
(laser-induced arteriolar injury)
Both hES and iPS-MKs produce pure platelets
Normal blood platelets
16
iPS-derived platelets

17. Microtubules
Morphology/ultrastructure of iPS-platelets
identical to normal blood platelets
Glycogen
Granules
Dense Tubular
System
iPS-PLT
Multivesicular
Body
Mitochondria
Alpha-Granule
Glycogen
Granules
blood-PLT
Open
Canalicular
System
Microtubules
17
Alpha-Granule
Open
Canalicular
System
Dense Tubular
System
Mitochondria

18. NEXT STEPS / PLATELET PROGRAM
Achieve
1 Million Dose Scale
 Optimize MK progenitor/platelet production &
functionality in vitro
 Scale-up using microfluidics and bioreactor system
 Continue preclinical testing in animals (efficacy,
biodistribution, safety/tox tumorigenicity)
 Complete transfer of technology to
GMP/manufacturing (includes assay development and
process validation)
 Complete clinical regulatory process/file IND
NOTE: Strategic decision made to fast-track MSC program into clinic
first (potentially greater financial and medical impact in the near term )
1yr
18
2yr
3yr
4yr
5yr
10yr

19. Corneal repair: corneal endothelium derived from hESCs
• 10 million people with corneal blindness
• Cornea the most transplanted organ (1/3 due to endothelial failure)
• Solutions: Tx of whole cornea “Penetrating Keratoplasty” (PKP)
– More popular: Tx corneal endothelium & Descemet’s membrane (DSEK)
hESC-derived cells resemble normal human corneal endothelium
Optimized
cryopreservation
(CECs can now be
sent world-wide)
19
Global gene analysis shows that hESCCECs & adult-CECs are nearly identical

20. Next Steps
 Preclinical Studies
CEC
Lens
Cornea
20
Iris
Continue testing hESC-derived corneal endothelium
in in vivo rabbit cornea model
• Model: In vivo edema injection in rabbit
(inject dissociated cells to replace native
CECs)
• Increase purity of CECs to 99%
• Design and test hydrogel sheet in vitro (needs
to be transparent, flexible and biodegradable)
• Design and test CEC/hydrogel sheet in vivo
(test sheet to mimic clinical DSEK in rabbit
model)

21. Other Retinal Cell Types
RNP
PDE6a/DAPI
Pluripotent
Stem Cell
Eye Field
Stem Cell
Photoreceptor
Opsin (green/red)
PDE6a/DAPI
Math5/DAPI
Ganglion
Rhodopsin/Recoverin
21

22. Retinal Neural Progenitors
hESC- RNP cells reversed the progression of
photoreceptor degeneration
b-wave
(post-synaptic retinal cells)
(cones & rods)
RNP, 2 mo
ONL
RNP, 2 mo
RNP, 1 mo
RNP, 1 mo
PBS, 1 mo
PBS, 2 mo
22
} ONL
}
PBS, 1 mo
PBS, 2 mo
Thickness of ONL (µm)
a-wave
Increase in retinal (ONL)
thickness by OCT after 2 mos
60
40
20
0
Control
No Injection Cell treatment

23. Identification of endocrine/neuroprotective factors
Genomic DNA Q-PCR analysis
of whole retina
mouse specific probe
human specific probe
Proteins screened using antibody arrays
23

24. Preservation of Outer Segments (OS) of Rod Photoreceptors in RCS rats
Tail vein injection
PBS
Rod OS in cell-treated
rat retina shown by
rhodopsin staining
Missing OS
24
Intravitreal injection

25. Subretinal transplantation
Integration of Photoreceptor PROGENITORS
1 week after transplantation
3 week after transplantation
HNA antibody/transplanted human cells
Human cells migrating into ONL
25

26. Next Steps

Continue testing RNPs in in vitro and in animal models
 Collaborations underway with several leading groups studying various retinal/ocular disease
models
 Continue identification of paracrine/neuroprotective factors (proteomics, antibody arrays, 2D
gel analysis, and mass spec)
 Test ability of cell-free lysates (or specific identified factors) to prevent or delay a range of
ocular & non-ocular degenerative diseases
 Photoreceptor Progenitors
 Complete studies of recovery of host visual function and retinal structure
 Continue in vitro neuroprotection studies using conditioned medium
 In vitro and in vivo functional tests of secreted factors
 Ganglion Progenitors
 Continue animal experiments/show long term survival of transplanted cell
 Integration of transplanted cells
 Protection/replacement of host retinal ganglion cells
 Optic nerve regeneration
26

27. Mesenchymal Stem Cells (MSCs)




27
Found in bone marrow, adipose, umbilical
cord, tooth buds
Can differentiate into fat, bone, cartilage
MSCs are immunoprivileged and can:
- migrate to injury site
- exert immunosuppressive effects
- facilitate tissue repair
Over 200 clinical trials
(www.clinicaltrials.gov)
- wide range of clinical indications being
pursued
- companies such as Pluristem,
Mesoblast, Neostem are developing
MSC-based therapies using BM or
other primary sources of MSCs

28. hESC-MSCs versus adult derived MSCs
●
●
●
●
●
●
“Off the shelf” therapy available for immediate use
Unlimited cell source
Easy to derive
Can be expanded to large numbers in vitro
More youthful and live much longer
Potentially greater efficacy
(older MSCs often serve as negative controls)
● Hemangioblast-derived hESC-MSCs exponentially
greater yields than other reported hESC methods
A sampling of their in vivo efficacy
will be given in the following slides
28
>30,000 X more units from hESC-MSCs
than from adult bone marrow MSCs

29. Treatment of Multiple Sclerosis in Mice (EAE model)
 hESC-MSCs dramatically reduce clinical
symptoms of EAE
 both prophylactic and therapeutic
inhibition
 In vitro inhibition of T-cell function
 Differential cytokine expression (hESCMSCs vs. BM-MSCs)
 Differential ability to migrate into damaged
tissues (hESC-MSCs vs.
BM-MSCs)
Untreated
Clinical Score: 2-4
(partial to complete
hind/front leg paralysis)
hESC-MSC Treated*
 Additional follow-up studies being
completed for scientific publication (based
on reviewer suggestions)
Clinical Score: <1
(no leg paralysis)
 Multiple sclerosis is a leading candidate for
clinical translation
*BM-MSCs had minimal impact (animals still showed paralysis)
29

30. hESC-MSC treatment for Uveitis
Untreated EAU mice
 An inflammatory/auto-immune condition of the uvea
 Accounts for 10% of blindness in US (mostly 20-40
year olds)
 Experimental autoimmune uveitis (EAU) in mice
resembles
that of human uveitis
 Disease state can be assessed using funduscope
imaging/histology
30
PMN and lymphocytes
Preliminary studies show
that hESC-MSC treatment
is effective in treating EAU
uveitis

31. hESC-MSC treatment for SLE/Lupus Nephritis
 Systemic lupus erythematosus (SLE) or lupus is a systemic autoimmune disease that can
effect virtually any organ or system in the body
 There is no cure for SLE
 SLE most often harms the heart, joints, skin, lungs, blood vessels, liver, kidneys, and nervous
system
 50% of SLE patients get lupus nephritis (LN)
 Immune complex deposition in kidney glomeruli leads to kidney dysfunction/failure
 NZB/NZW mice spontaneously develop lupus nephritis (very similar to human SLE)
NZB/NZW lupus model
 hESC-MSCs have a dramatic effect on
morbidity (including proteinuria/kidney
function
 Protect from death (show marked
improvement in survival)
 Lead candidate for clinical translation
31

32. hESC-MSC treatment for Pain
 PAIN behavior can be
measured in mice using
operant equipment (using a
reward/conflict paradigm)
 hESC-MSCs significantly
reduce chemotherapyinduced neuropathy in
response to taxol
 Also a candidate for clinical
translation
32

33. Potential therapeutic applications for MSCs

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













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33
>100 autoimmune diseases
Multiple Sclerosis
Osteoarthritis
Lupus
Aplastic Anemia
 No need for immunosuppression
Crohn’s Disease/IBS
Chronic Pain
 Persist transiently
Limb Ischemia
Heart Failure/MI
 Can be irradiated
Stroke
Graft-versus-host Disease
Spinal Cord Injury
Liver Disease
Kidney Disease
Emphysema/Pulmonary Diseases
Wound healing (ulcers/decubitus/burns)
HSC engraftment/irradiated cancer patients
Eye diseases (uveitis, retinal degeneration, glaucoma)
hES/hiPS-MSCs are
Ideal for Clinical Translation

34. Canine Indications for Human MSC Therapy
Intervertebral disk disease
Orthopedic
Osteoarthritis
Sepsis
MSC Therapy
Inflammatory
Acute pancreatitis
Granulomatous
meningoencephalitis
Immune-mediated
Inflammatory bowel
disease
Chronic hepatitis
Glomerulonephritis
Anal furunculosis
Immune-mediated
hemolytic anemia
34
 Filed Investigational New Animal Drug (INAD)
application with the FDA to treat 10 different
disease indications
 Naturally-occurring diseases in large animals,
such as dogs, provide an excellent model of
human conditions. May provide a more robust
assessment of safety and therapeutic endpoints
than what can be obtained from inbred rodent
models
 Initiated collaboration with Tufts University
School of Veterinary Medicine, which has a large
population of suitable patients, and world-experts
in regenerative medicine
 Have already received IACUC approval to begin
several of the studies
 In additional to the obvious veterinary
applications, the results may help inform and
optimize human clinical trials

35. Ted Myles
Chief Financial Officer & EVP of Corporate Development
CORPORATE UPDATE
35

36. Improving the Balance Sheet, and therefore the Company to Position a 2014 up-listing
ACT - Current
~$4m - $5m cash on hand and funding
itself through bi-daily issuances of
~2.5m shares of common stock
The Company draws from the market
slightly less than it spends, in order to
build its cash balance, albeit slowly
This funding vehicle puts continuous
downward pressure on stock and is not
enabling of scale up as we prepare for
larger clinical trials
ACT – end of 2013/early 2014
Raising a tranche of funds ($10m - $15m) in
the near-term will improve the balance
sheet, and therefore the stability of the
company
Will also decrease our reliance on the
frequent draws, therefore reducing the
constant downward pressure on the stock
Having ~12 months of cash in the bank will
allow the Company to be more strategic as
it pursues clinical expansion, corporate
development and Nasdaq listing
ACT has never had real balance sheet strength and has therefore
been forced to make decisions from a “less than optimal” position
36

37. Why Up-List to Nasdaq?
“Over-the-Counter”
OTC companies are sometimes
associated as being of “poor quality”
Naked shorting can damage
shareholder value
Trading/liquidity is less efficient
Equity research analysts want to cover
and support companies on national
exchanges
Institutional investors who can invest
substantial capital are less likely to
invest in “penny stocks” no matter how
great the company
Nasdaq Listed
“Class effect”, being a Nasdaq-listed
company carries with it a cache of quality,
based partly on perception and largely on the
strict listing requirements
Increased shareholder protection due to
stringent listing requirements
Institutional investors have expressed
interest in investing substantial capital into
ACT, after up-listing
Building a syndicate of equity research
analysts who cover the Company can create
additional demand for the shares
Management
believes that
a Nasdaq uplisting is in
the best
interest of all
stakeholders
Completing a fully marketed financing deal that includes a broad syndicate of
bankers and analysts could capitalize the Company through Phase II, and create
global awareness of the ACT story
37

38. Clinical and Corporate Development Support Up-listing,
and Growth in Shareholder Value
AMD Trials
Phase 1
Pre-clinical
Clean up
Corporate
Development
MMD Trial
MSC and
other
R&D
Progress
SEC, etc.
Non-dilutive
funding
Financial
38
Phase 2
Phase 1
All components
of the
Company
complement
one another. A
transformative
process for
ACT
Partnering
Reverse split
Nasdaq
“re-IPO”

39. Thank you
For more information, visit www.advancedcell.com