1/8/2013

1. January 2013
LEADING
REGENERATIVE
MEDICINE

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. ACT is a publicly-traded biotechnology company
• Develop cellular therapies for the treatment of diseases and conditions
that impact hundreds of millions of people worldwide
• Premier scientific and research development team
• Cell therapies are traditionally derided for being high cost, high touch
and having lack of scalability. Not our approach at all
>> Centralized, Scalable, Cost Effective Cell Therapy Products <<
About ACT:
– Ticker – ACTC
– Market Capitalization: $160 million
– Principal lab and GMP facility: Marlborough, MA
– Corporate HQ: Santa Monica, CA
– Only hESC-derived tissue trials ongoing worldwide
3
Company Overview

4. Multiple Pluripotent Cell Platforms
Single Blastomere-derived
Embryonic Stem Cell Lines
Generating hESC lines
WITHOUT DESTRUCTION OF EMBRYO
Utilizes a
SINGLE CELL BIOPSY
ACT never needs to make another hESC line again
4
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

5. Multiple Pluripotent Cell Platforms
Induced Pluripotency Stem Cells (iPS)
• Early Innovator in Pluripotency
(before iPS was even a term!)
• Controlling Patent Filings (earliest priority date)
to use of OCT4 for inducing pluripotency
• Science is moving quickly, but
Regulatory Agencies (FDA, EMA)
proceeding carefully
• ACT pursuing platelets as first
iPS-derived product
(cell fragments – no nucleus,
not mitotically active)
5

6. RPE Clinical Program

7. 7
Structure of Retina
The Retina the light-sensitive
tissue lining the inner surface of
the eye
Retina

8. 8
Life Support to Photoreceptors
Provides nutrients and growth factors
• photoreceptors see no blood
Recycles Vitamin A
• maintains photoreceptor excitability
Detoxifies photoreceptor layer
Maintains Bruch’s Membrane
• natural antiangiogenic barrier
• immune privilege of retina
Absorbs stray light / protects from UV
RPE Layer has
multiple
critical roles
in the
health and
function
of photoreceptors and
the retina as a whole.

9. 9
Life Support to Photoreceptors
Loss of RPE cells
Build up of toxic waste
Loss of photoreceptors
Dry AMD
Bruch’s Mem. dehiscence
Choroidal neovascularization
Wet AMD

10. 10
RPE Therapy- Rationale
Dry AMD
represents more than
90%
of all cases of AMD
North America & Europe
alone have more than 30 million dry AMD patients
who should be eligible for our RPE cell therapy
What if we could
replace
missing RPE cells?

11. RPE cell therapy may impact
over 200 retinal diseases
11
RPE Therapy- Rationale
• Massive unmet medical need
• Unique measuring and observation environment
• Easy to identify – aids manufacturing
• Small dosage size – less than 200K cells
• Immune-privileged site - minimal/no immunosuppression
• Ease of administration - no separate device approval

12. 12
RPE Therapy- Rationale
U.S. PATIENT POPULATION
EARLY STAGE
(10-15M)
INTERMEDIATE
(5-8M)
LATE STAGE
(1.75M)
ACT’S RPE Cell Therapy
should address the full range
of dry AMD patients:
Halt progression of vision loss in early
stage patients
Restore some visual acuity in later
stage patients

13. GMP process for differentiation and purification of RPE
– Virtually unlimited supply from stem cell source
– Optimized for manufacturing
Ideal Cell Therapy Product
– Centralized Manufacturing
– Small Doses
– Easily Frozen and Shipped
– Simple Handling by Doctor
GMP Manufacturing
13
Product Cold Chain is Easily Scaled for Global Sales

14. Preclinical - Examples
14
control treated
Injected human RPE cells
repair monolayer structure in
eye
Photoreceptor
layer
photoreceptor layer
is only 0 to 1 cell
thick without
treatment

15. Phase I - Clinical Trial Design
15
SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K.
12 Patients / trial
ascending dosages of 50K, 100K, 150K and 200K cells.
Regular Monitoring - including high definition imaging of retina
50K Cells 100K Cells 150K Cells 200K Cells
Patient 1 Patients 2/3
DSMB Review DSMB Review

16. Participating Retinal Surgeons
16
Steven D. Schwartz, MD
Chief, Retina Division
Ahmanson Professor of Ophthalmology
Director, Diabetic Eye Disease and Retinal Vascular Center
Director, Ophthalmic Photography Clinical Laboratory
Jules Stein Eye Institute
Wills Eye Institute
Carl D. Regillo, MD
Director, Retina Service
Professor of Ophthalmology, Jefferson Medical College
Byron L. Lam, MD
Director, Clinical Visual Physiology
Professor of Ophthalmology
Bascom Palmer Eye Institute

17. Participating Retinal Surgeons
17
Dean Eliott, MD
Associate Director, Retina Service
Professor, Harvard Medical School
Massachusetts Eye and Ear Infirmary
Moorfields Eye Hospital
James Bainbridge, MA MB BChir PhD FRCOphth
Professor of Retinal Studies, UCL Institute of Ophthalmology
Philip J. Rosenfeld, MD PhD
Professor of Ophthalmology
Bascom Palmer Eye Institute
Edinburgh Royal Infirmary
Baljean Dhillon BMed Sci, BM BS, FRCS
Surgeon, Edinburgh Royal Infirmary
Surgeon, Lothian University Hospitals NHS Trust
Professor of Ophthalmology, University of Edinburgh
Professor of Visual Impairment Studies, Heriot Watt University

18. Surgical Overview
18
Procedure:
• 25 Gauge Pars Plana
Vitrectomy
• Posterior Vitreous Separation
(PVD Induction)
• Subretinal hESC-derived RPE
cells injection
• Bleb Confirmation
• Air Fluid Exchange

19. Preliminary Results
19
No Adverse Events
No signs of hyperproliferation,
abnormal growth, rejection or retinal
detachment.
Persistence of cells
Anatomical evidence of hESC-RPE
survival and engraftment.
Increased pigmentation within the bed
of the transplant.
Impact on Acuity
Recorded functional visual
improvements in both patients.

20. 20
Engraftment and Survival: SD-OCT image collected at month 3
show survival and engraftment of RPE
SMD001
3mo post-op
Preliminary Results – Structural

21. 21
Baseline
Injection site
Month 1 Month 2
SMD PATIENT UK04

22. 22
Baseline
Injection site
Month 1 Month 3
SMD PATIENT UK03

23. 23
Baseline 2 months
DRY AMD PATIENT 204

24. Preliminary Results – Functional
24
Visual Acuity Measurements
• 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)
14 month Follow-up:
• Visual acuity gains remain relatively stable for both patients
• SMD Patient continues to show improvement.
U.K. SMD01 Patient (at 9 month follow-up)
• ETDRS: Improved from 5 letters to 10 letters, and stable
• Subjective: Reports significantly improved ability to read text on TV

25. Current Safety Profile – Stargardt’s Trial
25
12 SMD Patients Treated
3 patients (50K cells cohort) treated – US Trial > Cohort Complete
3 patients (50K cells cohort) treated – UK Trial > Cohort Complete
3 patient (100K cells cohort) treated – US Trial > Cohort Complete
3 patient (100K cells cohort) treated – UK Trial > Cohort Complete
No reports of any adverse events or complications due to
cells per se
• No evidence of inflammation or infiltration
• No evidence of ectopic tissue formation
• No evidence of retinal detachment

26. Current Safety Profile – Dry AMD Trial
26
6 dry AMD Patients Treated
3 patients (50K cells cohort) treated > Cohort Complete
3 patient (100K cells cohort) treated > Cohort Complete
No reports of any adverse events or complications due to
cells per se
• No evidence of inflammation or infiltration
• No evidence of ectopic tissue formation
• No evidence of retinal detachment

27. RPE Program Milestone Objectives
27
Key upcoming milestones
• Continue to treat and review patient data
• Define efficacy endpoints and targeted patient visual
criteria
• Treat earlier stage disease to determine curative
power of dissociated cell injections
• Simplify shipping and cell-prep to enhance scaled
distribution platform

28. Intellectual Property – RPE Program
Treatment Dominant Patent Position for Treating Retinal
Degeneration
Manufacturing Broad Coverage for Manufacturing RPE Cells from hESC
Preparations Claims directed to pharmaceutical preparations of RPE
Cells from hESC, including both cell suspensions and
scaffolded RPE layers.
Sources Issued patents cover RPE Cells derived from other
pluripotent stem cells (including iPS cells)
Vigilance Regularly Filing on Improvements
• Extend patent life cycle, with significance to commercialization
• Include composition-of-matter claims (cell preparations,
pharmaceutical preparations, etc.)
28

29. Price Justification
29
Unmet Therapeutic Need
Efficacy
Patient Prevalence
Pharmacoeconomics
Patient Advocacy
Pricing Justification
across all categories
of consideration

30. RPE Program - Investment Thesis
30
• Immense unmet medical need
• Small Doses
• Immunoprivileged – permits central (allogeneic) source of cells
• Noninvasive monitoring of retina
Market potential: More than 50 million patients in major markets.
1% market penetration may represent $5-10B market opportunity.
Orphan indications are meaningful: Estimating a 10% market penetration
with reoccurring treatments every 3-5 years, Stargardt’s disease can be a
$100+ million/year product.

31. Therapeutic Pipeline -
Ocular Programs

32. Retinal Pigment Epithelial Cells
 Macular Degeneration - dry AMD
 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
32
Retina

33. Mesenchymal Stem Cell
Program
33

34. Mesenchymal Stem Cells in Therapy
34
Mesenchymal stem cells (MSCs) regulate immune responses
provide therapeutic potential for treating autoimmune or
inflammatory diseases.
• Allogeneic - without HLA matching.
• Track Record - Adult-derived MSCs already in 200+ clinical trials.
Derived from Bone Marrow and Adipost Tissue
However, allogeneic adult-derived MSC’s are limited
by replicative senescence, and as it turns out, a relative
lack of potency

35. hESC- and iPS – derived MSC
35
ACT Proprietary Process
• hESC- and iPS-derived MSCs
• virtually inexhaustible source of starting material
• replicative capacity is more than 33,000 times greater
Advantages for Manufacturing
• Use Single Master Cell Bank
• Simplifies FDA/regulatory process
• No need for continually finding and qualifying
donors
• Less labor-intensive

36. Potential applications
36
• >100 autoimmune diseases
• Multiple Sclerosis
• Osteoarthritis
• Crohn’s Disease/IBS
• Aplastic Anemia
• Chronic Pain
• Limb Ischemia
• Heart Failure/MI
• Stroke
• Graft-versus-host Disease
• Spinal Cord Injury
• Parkinson’s Disease
• Liver Cirrhosis
• Emphysema/Pulmonary Diseases
• Wound healing
(ulcers/decubitus/burns)
• HSC engraftment/irradiated
cancer patients
• Eye diseases (uveitis, retinal
degeneration, glaucoma)

37. ACT
Blood Components
Program

38. Hemangioblast Program: Overview
38
Hemangioblast: common precursor to hematopoietic and endothelial cells.
Multipotent cell produces all
cell types in the circulatory and
vascular systems

39. Generation of Blood Products
39
Hemangioblasts RBCsHemangioblasts Enucleated
RBC’s
Process generates large
quantities of functional
red blood cells
and
megakaryocytes &
platelets

40. The Case for Platelets
40
Platelets are key elements of
hemostasis and thrombosis as well as
tissue regeneration after injury or surgery.
• Maintain vascular integrity and play vital roles in wound repair
• Thrombocytopenia is a major cause of morbidity in sepsis,
cancer and preeclampsia
• Used regularly in soft- and hard-tissue applications in most fields
of surgery.
.
Beyond Donor Supply: Estimate
demand for additional 1-2M units/year

41. The Case for Platelets
41
Donor-derived platelets raise concerns:
• variability of quality and quantity
• risk of infectious transmission
• short lifespan of stored platelets
• bacterial contamination during storage
• development of alloantibodies in multi-transfused patients
Platelets are the blood product most difficult to
maintain without unnecessary wastage.
• Platelets do not tolerate refrigeration.
• Storage at room temperature is limited to 5–7 days
Need for new strategies
to generate platelets
for infusion therapy.

42. Platelets 2.0
42
Ability to control platelet manufacturing provides
opportunities to improve storage, fine tune
platelets for use in wound healing applications, as well
as to engineer new roles for platelets beyond
traditional involvement in wound healing.
• Improve cryopreservation of platelets
• Make lyophilization of platelets tractable solution
• Utilize platelets for drug delivery
• Utilize platelets in imaging (load with contrast agents)
CONFIDENTIAL - BUSINESS PROPRIETARY

43. Platelet Production Stages
43
Stem Cell
Promegakaryoblast
megakaryoblast
Promegakaryocyte
PFC: Proplatelet
Formation Cell
Platelets
Stage I Stage II Stage III
Stage IV
CFU-GEMM BFU-MK CFU-MK
endomitosis

44. Size and Polyploidy
44
Average size (diameter) of cells
at the beginning and 6 days after MK culture
DNA ploidy analysis by flow cytometry of CD41a+ cells.
DNA ploidy up to 32N was observed in these cells.

45. Platelets
45
Bone Marrow
Limited By Source Infinite Supply
Cord Blood hESC

46. Representative iPS-platelet prep
46
Blood platelets iPS platelets
CD42b
CD41a

47. Achieving Function and Scale
47
• Ultrastructural and morphological features of SC-derived platelets are
indistinguishable from normal blood platelets.
• SC-derived platelets respond to thrombin stimulation, form
microaggregates, and facilitate clot formation and retraction.
• SC-platelets form lamellipodia and filopodia in response to thrombin
activation, and tethered to each other as observed in normal blood.
• SC-derived platelets contribute to developing thrombi at sites of laser-induced
vascular injury in mice - first evidence for in vivo functionality of SC-
derived platelets.

48. Activation
48
iPS-PLT’s display appropriate
cytoskeletal function
in glass spreading assays.

49. Characterization of Platelets
49
hESC- and iPS-derived platelets participate
in clot formation and retraction

50. Clinical Program Status
50
• Animal model studies show
proper in vivo function
• Achieved clinical dose
scale manufacturing
• Completely feeder-free process
(bioreactor capable!)
• Pre-IND meeting requested
with FDA
Human platelets incorporating into
laser-induced into mouse thrombus

51. ACT Corporate Overview

52. Financial Update – Strong Balance Sheet
52
• Company ended 2012 Q3 with $42 million in cash or
availability of cash through financing commitments
• $16 million annual cash-burn rate
(funded through early 2015)
• Settled nearly all litigation hangover from previous
management

53. 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
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
Kathy Singh - Controller
Rita Parker – Director of Operations
Dr. Matthew Vincent, Ph.D. – Director of Business Development
Bill Douglass – Dir. of Corporate Communications & Social Media
53

54. Dr. Ronald M. Green: Chairman
Dr. Judith Bernstein
Dr. Jeremy B.A. Green
Dr. Robert Kauffman
Dr. Carol A. Tauer
ACT Leadership
Gary Rabin: Chairman & CEO
Dr. Robert S. Langer, ScD: Prolific medical inventor; Chair – ACT SAB
Gregory S. Perry: EVP – Immunogen
Michael Heffernan: CEO – Collegium Pharma
Zohar Loshitzer: CEO Presbia; Founder LifeAlert Medical
Dr. Alan C. Shapiro: Renowned business school professor
54
World Class Board of Directors
Highly-regarded Ethics Advisory Board

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