Leading Regenerative Medicine

Regenerative Medicine Insight Track

~ Biotech Showcase - January 2012 ~

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

ACT Ocular Programs

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

RP

E la

yer

Pho

tore

cept

ors

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 5

Retinal Pigment Epithelial Cells - Rationale

RPE cell as Target

• 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 6

Retinal Pigment Epithelial Cells - Rationale

• 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

7

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

8

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

9

• 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

10

• 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)

11

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

12

Surgical Overview

13

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

Surgical Overview

14

Surgical Overview

15

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

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

16

Ocular Program – Hemangioblasts

17

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.

Ocular Program – Hemangioblasts

18

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

• 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

19

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

Ocular Program – Mesenchymal Stromal Cells

20

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.

Platform Technology for Generating

Robust Human Embryonic Stem Cells

Without the Need to Destroy Embryos

Single Blastomere Technology

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.

22

Single Blastomere Technology

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.

23

Financial Update – Strong Balance Sheet

24

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

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

25

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