Boston, MA20 September 2012

Boston, MA20 September 2012

LEADINGREGENERATIVEMEDICINE

Stem Cells USA & Regenerative Medicine Congress

This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced CellTechnology Inc”, or “the Company”) salient business characteristics.

The information herein contains “forward‐looking statements” as defined under the federalsecurities laws. Actual results could vary materially. Factors that could cause actual resultsto 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 fromtime to time with the Securities and Exchange Commission. The risks identified therein, aswell as others not identified by the Company, could cause the Company’s actual results todiffer materially from those expressed in any forward‐looking statements. Ropes Gray

Cautionary Statement Concerning Forward‐Looking Statements

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Multiple Pluripotent Cell Platforms

Single Blastomere-derived Embryonic Stem Cell Lines

Generating hESC linesWITHOUT DESTRUCTION OF EMBRYOUtilizes a

SINGLE CELL BIOPSY

Induced Pluripotency Stem Cells (iPS)• Early Innovator in Pluripotency (before iPS was even a term!)• 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

RPE Clinical Program

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retina

Life Support to Photoreceptors

Rod outer segmentsCone outer segments

RPEBruch’s membraneChoroidal vessels

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Life Support to Photoreceptors

Detoxifies photoreceptor layerMaintains Bruch’s Membrane

• natural antiangiogenic barrier• immune privilege of retina

Absorbs stray light / protects from UV

Provides critical nutrients, growth factors, ions and water

• photoreceptors see no blood

Recycles Vitamin A • maintains photoreceptor

excitability

Function of RPE Layer

Rod outer segments

Cone outer segments

RPE

Bruch’s membraneChoroidal vessels

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Life Support to Photoreceptors

Loss of RPE cellsBuild up of toxic waste Loss of photoreceptors

Dry AMD

Bruch’s Mem. dehiscence Choroidal neovascularization

Wet AMD

• 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

RPE cell therapy may impact over 200 retinal diseases

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RPE Therapy- Rationale

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RPE Therapy- Rationale

Early Stage AMD (10-15M)

Intermediate AMD (5-8M)

Late Stage AMD (1.75M)

U.S. Patient Population 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

Dry AMD represents more than 90 percent of all cases of AMD

North America and Europe alone have more than 30 Million dry AMD patients who should be eligible for our RPE cell therapy.

• 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

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Product Cold Chain is Easily Scaled for Global Sales

Characterizing Clinical RPE Lots

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• RPE cells are derived from an extensively tested hES MCB. • Entire process is aspetic; no antibiotics used (~110 days).• Cryopreserved bulk product is extensively tested prior to release.• Bulk product is thawed and formulated for therapeutic on the day of use.

• Some unique quality tests include:• Screening for the absence of hES cells (IFA)• Assessing the extent of differentiation by:

• gene expression (q-RT-PCR)• protein deposition (IFA staining)• morphological evaluation• extent of pigmentation (melanin)• potency by phagocytosis assays (FACS)

In-Process Quality Testing• Frequent Morphological

Assessments (1-2days)• Periodic Sterility Testing• Regular Karyotyping• Immunohistochemical Staining

for RPE Markers

Characterizing Clinical RPE Lots

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Quantitative Potency Assay

RPE cell potency of each lot is assessed by phagocytosis

4°C 37°C

Effects of Pigmentation

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Use melanin content to determine 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 100120

Abso

rban

ce at

475n

m

µg/mL Melanin

Quantitative Pigmentation Assay

Preclinical - Examples

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control treated

Injected human RPE cells repair monolayer structure in eye

Photoreceptorlayer

photoreceptor layer is only 0 to 1 cell

thick without treatment

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 / trialascending 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

Phase I – SMD endpoints

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PRIMARY ENDPOINTS: ASSESSMENT OF

SAFETY

The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of: Any grade 2 (NCI grading system) or greater adverse event related to the cell product Any evidence that the cells are contaminated with an infectious agent Any evidence that the cells show tumorigenic potential

SECONDARY ENDPOINTS

Evidence of successful engraftment will consist of: Structural evidence (OCT, fluorescein angiography, autofluorescense photography, slit-lampexamination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location

Evidence of rejection will consist of: Structural (imaging) evidence that implanted MA09-hRPE cells are no longer in the correctlocation or the presence of vascular leakage. If enhanced electroretinographic activity is observed after the transplantation, subsequentelectroretinographic evidence that activity has returned to pre-transplant conditions may be anindication of graft rejection

CONFIDENTIAL

Phase I – Dry AMD endpoints

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PRIMARY ENDPOINTS: ASSESSMENT OF

SAFETY

The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of: Any grade 2 (NCI grading system) or greater adverse event related to the cell product Any evidence that the cells are contaminated with an infectious agent Any evidence that the cells show tumorigenic potential

SECONDARY ENDPOINTS

Evidence of successful engraftment will consist of: Structural evidence (OCT, fluorescein angiography, autofluorescense photography, slit-lampexamination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location

Evidence of rejection will consist of: Structural (imaging) evidence that implanted MA09-hRPE cells are no longer in the correctlocation or the presence of vascular leakage. If enhanced electroretinographic activity is observed after the transplantation, subsequentelectroretinographic evidence that activity has returned to pre-transplant conditions may be anindication of graft rejection

Additional secondary endpoints will be

evaluated as exploratory evaluations for potential

efficacy endpoints.

CONFIDENTIAL

Participating Clinical Sites

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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

• European Clinical Trial Sites• Moorfields Eye Hospital• Edinburgh Royal Infirmary

ClinicalTrials.govUS: NCT01345006, NCT01344993UK: NCTO1469832

Surgical Overview

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Procedure:• 25 Gauge Pars Plana

Vitrectomy• Posterior Vitreous Separation

(PVD Induction)• Subretinal hESC-derived RPE

cells injection• Bleb Confirmation• Air Fluid Exchange

Preliminary Results

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No Adverse EventsNo signs of hyperproliferation, abnormal growth, rejection or retinal detachment.

Persistence of cellsAnatomical evidence of hESC-RPE survival and engraftment.Increased pigmentation within the bed of the transplant.

Impact on AcuityRecorded functional visual improvements in both patients.

Preliminary Results – Initial Patients

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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)One Year Follow-up:

• Visual acuity gains remain relatively stable for both patients• SMD Patient continues to show improvement.

U.K. SMD01 Patient (at 6 month follow-up)• ETDRS: Improved from 5 letters to 10 letters• Subjective: Reports significantly improved ability to read text on TV

Current Safety Profile – Stargardt’s Trial

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7 SMD Patients Treated (as of 7 September 2012)

3 patients (50K cells cohort) treated at UCLA – US Trial3 patients (50K cells cohort) treated at Moorfields Eye – UK Trial1 patient (100K cells cohort) treated at Wills Eye – US Trial

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

Current Safety Profile – Dry AMD Trial

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4 dry AMD Patients Treated (as of 7 September 2012)

3 patients (50K cells cohort) treated at UCLA – US Trial1 patient (100K cells cohort) treated at Wills Eye – US Trial

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

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.)

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Price Justification

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Unmet Therapeutic Need

Efficacy

Patient Prevalence

Pharmacoeconomics

Patient Advocacy

Pricing Justification across all categories of consideration

RPE Program - Investment Thesis

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• 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.

ACT MSC Program

Mesenchymal Stem Cells in Therapy

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Mesenchymal stem cells (MSCs) regulate immune responsesprovide therapeutic potential for treating autoimmune or inflammatory diseases.

• Allogeneic - without HLA matching.

• Potential• Autoimmune conditions, such as MS, lupus, and Crohn's/IBD.• Inflammatory Diseases

• Track Record - Adult-derived MSCs already in 200+ clinical trials.

An "off-the-shelf" cellular drug ready for treatment of a wide range of inflammatory and autoimmune diseases.

Adult Mesenchymal Stem Cells

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Impacts on Cell Banking • Limitation on the number of doses that can be generated from

adult donors • Requires constantly creating and validating MSC banks

from new donors

Impacts on Potency• Passaging reduces immunomodulatory potency of MSC’s.

Replicative Capacity - limits adult sources (bone marrow, fat, etc) for allogeneic MSC therapies.

Substantial need for better MSC products

hESC- and iPS – derived MSC

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ACT Proprietary Process• hESC-derived MSCs can be expanded to large

numbers of cells• Have qualities similar to fetal MSC’s• Avoid replicative capacity problem of “old” adult MSC’s

Advantages for Manufacturing• Use Single Master Cell Bank

• Simplifies FDA/regulatory process• No need for finding donors

• Less labor-intensive

Preliminary Data

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Animal Models testing hESC-derived MSC’s

Substantially decrease and reverse disease conditions in autoimmune models.

• Far more potent than adult (BM) derived MSCs.• Have longer duration of action compared to adult

(BM) derived MSCs.

Potential implications of increased potency and duration…• Broader utility in range of diseases.• Reduced cells per dose – improved safety profile.• Longer duration between injections.

Potential applications

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• >100 autoimmune diseases• Multiple Sclerosis• Osteoarthritis• Aplastic Anemia• Crohn’s Disease/IBS• 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)

ACT Vascular Program

Hemangioblast Program: Overview

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The Hemangioblast cell is a multipotent cell, and a commonprecursor to hematopoietic and endothelial cells.

Hemangioblast cells can be used to produce all cell types in the circulatory and vascular systems

Generation of megakaryoctyes/platelets

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Human ES cells  Hemangioblasts

Megakaryocyte Pro‐plateletsCD41/vWF/DAPI

Characterization of Platelets

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hESC- and iPS-derived platelets participate in clot formation and retraction

Next Steps

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Testing hES/hiPSC-platelets in vitro • Morphology• Biochemical status• Physiological responses

Testing hES/hiPSC-platelets in vivo• Collaborations underway with several leading groups

(including Harvard University, Columbia University,and University of Illinois College of Medicine) in vivo circulation and half-life in vivo function

Financial Update – Strong Balance Sheet

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• Company ended 2012 Q2 with $10 million cash on hand• $35 million more available under equity line• Virtually debt-free• Received shareholder approval for reverse split• Filed application for NASDAQ uplisting and have initial comments

Other 2012 Milestones (so far)• IRB approvals from Wills Eye Institute, Bascom Palmer Eye Hospital and

Massachusetts Eye & Ear• Initiated Europe’s first human ESC-derived transplant at Moorfields Eye Hospital• Published first report of hESC-derived cells transplanted into humans in top

medical journal, The Lancet.• Completed Dose Cohort 1 of patients in both U.S. trials; • Dosed first SMD and dry AMD patients in 100,000 cell cohorts – no AE’s observed.

ACT Management TeamHighly Experienced and Tightly Integrated Management Team

Gary Rabin – Chairman & CEODr. Robert Lanza, M.D. – Chief Scientific OfficerEdmund Mickunas – Vice President of Regulatory AffairsDr. Irina Klimanskaya, Ph.D. – Director of Stem Cell BiologyDr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of ResearchDr. Roger Gay, Ph.D. - Senior Director of ManufacturingKathy Singh - ControllerRita Parker – Director of OperationsDr. Matthew Vincent, Ph.D. – Director of Business DevelopmentBill Douglass – Dir. of Corporate Communications & Social Media

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Thank youFor more information, visit www.advancedcell.com