SlideShare a Scribd company logo
1 of 30
Download to read offline
Leading Regenerative Medicine
September 2011
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
At the Forefront of Regenerative Medicine
• Patented Technology for Producing human Embryonic Stem Cells (“hESCs”)
without Harm to Embryo
• Working with Roslin Cells to create GMP-compliant hESC bank
• 2 Human Clinical Trials utilizing hESC-derived Retinal Pigment Epithelial Cells
• First Patients Treated on July 12, 2011
• Stargardt’s Disease, aka Stargardt’s Macular Dystrophy (SMD)
• Dry AMD – (Dry Age-Related Macular Degeneration)
• Expecting Preliminary Safety and Engraftment Data by Year-End
• Commencing European Trials – estimated first half 2012
• Front-of-the-eye programs: Generating hESC-derived corneal tissues
• Finalizing preclinical work for blood product IND from hemangioblast program
• Generation of off-the-shelf hESC-derived mesenchymal stem cells (MSC) products
• Myoblast program for heart failure approved for Phase II
3
ACT Ocular Programs
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 may be part of mechanism underlying
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
• Ease-of-Handling by Doctor
7
Differentiation Media is Not Permissive for hESCs
Harvest for
Cryopreservation
ES Cells
EB
Formation RPE isolation P1 P2
0 5 weeks 12 weeks 15 weeks 18 weeks
Up to 10 percent hESC spiked
No hESC’s in harvested cells
Limit of Detection for hESC
1:20,000,000 cells
RPE differentiation is the default pathway under
ACT’s Patented Culture Conditions
3 weeks
EB
Outgrowth
30 Weeks
8
RPE Animal Safety Studies
Extensive Safety Studies Shows
Lack of Tumorigenicity
Long-term data
(spanning the life of
the animals) revealed
no evidence of
teratoma formation
after subretinal hESC-
RPE transplantation.
9
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
10
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
11
• Stargardt’s (SMD) Disease
• IND approved in November 2010
• European CTA filed
• 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)
12
• Also referred to as “Juvenile Macular Degeneration”
– Causes progressive vision loss beginning in childhood.
– Stargardt’s Disease is the most common hereditary macular dystrophy.
– Prevalence rate of about 1-in-10,000.
– Usually diagnosed in individuals under the age of twenty.
• ACT has obtained Orphan Drug Designation in United States and Europe
– 7 - 10 Years of Market Exclusivity for using RPE cells to treat Stargardt’s Disease.
Stargardt’s Macular Dystophy
Orphan Drug Opportunity with Reimbursement
80,000-100,000 patients in North America and Europe
13
• AMD - estimate over 30 Million patients in North America and Europe
– The prevalence of AMD in North America in the population aged 40 to 79 years is 8.8%
– The prevalence of AMD in China in the population aged 40 to 79 years is 6.8%
• Approximately 10% of people ages 66 to 74 have symptoms of macular degeneration
• Prevalence increases to 30% in patients 75 to 85 years of age.
Dry AMD (non-exudative)
– The most common form of AMD (estimates as high as 90 percent)
– No Effective Therapy Currently Available
– Estimated $20-30 Billion market
Age-Related Macular Degeneration
Potential for
Blockbuster Status
14
• 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 will be monitored weekly - including high definition imaging of retina
High Definition Spectral Domain Optical Coherence Tomography (SD-OCT)
Retinal Autofluorescence
Adaptive Optics Scanning Laser Ophthalmoscopy (AOSLO)
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
15
Phase I - Clinical Trial Update
• Prospective clinical studies to determine the safety and
tolerability of sub-retinal transplantation of hESC-derived RPE
cells.
• Vitrectomy including surgical induction of posterior vitreous
separation from the optic nerve was carried out
• Submacular injection of 50,000 hESC-derived RPE cells in a
volume of 150µl was delivered into a pre-selected area of the
pericentral macula
• Patients are monitored for systemic safety signals.
• Pre- and weekly postoperative ophthalmic examinations.
Visual acuity, fluorescein angiography, optical coherence tomography
(OCT), autofluorescence imaging and visual field testing
• DSMB Review Underway
More to come….
Early clinical and laboratory
findings with respect to safety,
tolerability and engraftment
to be made available
Drs. Steven Schwartz and Robert Lanza
Straight forward surgical approach
16
Next 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 (DSAEK).
hESC-derived corneal
endothelium resembles
normal human corneal
endothelium
17
ACT Hemangioblast
Program
Hemangioblast Program – JV Update
• Stem Cell & Regenerative Medicine International (SCRMI).
– ACT and CHA agree to restructure their joint venture.
– SCRMI exclusively licensed the rights to hemangioblast program to ACT for North America and to CHA Biotech for Korea
and Japan.
– SCRMI scientists reassigned to ACT to continue research and product development efforts as ACT employees
– Both companies will work to develop clinical therapies based on the joint venture's proprietary hemangioblast cell
technology.
• Products Opportunities include:
– Universal Blood Components, such as Red Blood Cells and Platelets
– Meschenchymal Stem Cells
• Products for treating inflammatory diseases, promoting tolerance to grafts,
repairing connective tissues, delivering therapeutic proteins, etc.
– Revascularization Therapies for treating ischemic injuries
• September 13, 2011: U.S. Patent 8,017,393 broadly covers ACT’s proprietary method for
deriving hemangioblast cells from embryonic stem cells.
19
Robust
Product
Pipeline
Hemangioblast Program: Overview
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.
• Differentiate into both hematopoietic and
endothelial cell lineages – and newly
discovered by ACT, mesenchymal stem
cells (MSC).
• Be used to generate pathogen-free blood
components
• Be used to achieve vascular repair
20
Revascularization Blood Products
Hemangioblasts RBCs
Generation of Blood Products - RBC
Hemangioblasts Enucleated
RBC’s
Capable of generating large quantities of
enucleated red blood cells.
21
- Blood Replacement Products
- Triage and battlefield applications
- Systemic Delivery System
- Blood cells are great for transporting
materials through the body as the
entire circulatory system evolved to
facilitate their movement
- Examples: Delivery of drugs or
imaging agents
Generation of Blood Products - Platelets
• Scalable: Generation of large quantities of Platelets from hESC and iPS sources
• Off-the-shelf allogeneic products: Short in vivo half-life (7 days) and allotolerance for acute uses
• Tractable regulatory approval process
• Therapeutic products for accelerating soft and hard tissue healing.
– Platelets improve the process of tissue repair – release agents involved in inflammation, angiogenesis and extracellular
matrix synthesis – all involved in wound repair
• Product Opportunties: bone fractures and bony defects; laminectomy procedures; lateral epicondylitis (i.e., tennis
elbow); total joint arthroplasty procedures (i.e., knee, hip, shoulder); plantar fasciitis; shoulder arthroscopy and
distal clavicle resection; and spinal fusion.
• Extensive Cosmetic Uses
– Wrinkle and Lift Procedures: Platelets are loaded with growth factors for skin and blood vessels and produce new
collagen without the wounds of a laser and the risk of scarring
22
 Efficiently generate functional megakaryocytes & platelets.
• ES-derived platelets participate in clot formation.
• ES-derived platelets incorporate into mouse thrombus at site of laser-
induced arteriolar injury
Repair of Vascular Damage
Hemangioblasts were tested in animal models of diabetic retinopathy,
heart disease and peripheral vascular damage
Results from treatment with
hemangioblast cells
• Restoration of blood flow to
ischemic limbs.
• Survival after myocardial infarction.
• Revascularizes ischemic retinas
Potential Impact on a Large Number of Vascular Diseases
myocardial infarction, vascular ischemic damage, ischemia-reperfusion
injury, diabetic vascular disease and peripheral artery disease (PAD) that are
leading causes of death and/or disability worldwide.
Hemangioblasts promoted repair in peripheral vascular damage
23
Mesenchymal Stem Cells
hESC-MSCs can be
obtained using the
hemangioblast method
• hESC-MSCs are easy to derive and
can be expanded to large numbers
in vitro
• Quality controls are easier to
manage for a renewable cell source
• Can serve as an “off the shelf”
therapy, available for immediate use
• Products for treating inflammatory
diseases, promoting tolerance to
grafts, repairing connective tissues,
delivering therapeutic proteins, etc.
24
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.
26
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.
27
Financial Update – Strong Balance Sheet
28
Most Stable Financial Situation In Company History
• The Company ended 2011 Q2 with $16.1 million cash on hand
• $17 million more equity available
• Virtually debt-free
• Able to self-fund both U.S. clinical trials and EP clinical trial
• Significantly deepened management team (and on-going)
• Gary Rabin appointed CEO (change from interim status)
• Exciting new Board of Director members to announce
• Unqualified audit opinion
Entering 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. Matthew Vincent, Ph.D. – Director of Business Development
Edmund Mickunas – Vice President of Regulatory Affairs
Bill Douglass – Director of Corporate Communications & Social Media
Stephen Price – Interim SVP – Corporate Development
Gary Rabin – Chairman and CEO
Kathy Singh - Controller
Rita Parker – Director of Operations
29
Thank you for Attending.
For more information, visit www.advancedcell.com

More Related Content

What's hot

Clinical Outlooks for Regenerative Medicine, Boston. June 2012
Clinical Outlooks for Regenerative Medicine, Boston. June 2012Clinical Outlooks for Regenerative Medicine, Boston. June 2012
Clinical Outlooks for Regenerative Medicine, Boston. June 2012Advanced Cell Technology, Inc.
 
2013 Aegis Capital Healthcare Conference, Sept 26th 2013
2013 Aegis Capital Healthcare Conference, Sept 26th 20132013 Aegis Capital Healthcare Conference, Sept 26th 2013
2013 Aegis Capital Healthcare Conference, Sept 26th 2013Advanced Cell Technology, Inc.
 
Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...
Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...
Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...Advanced Cell Technology, Inc.
 
Corporate presentation 20141112 (1)
Corporate presentation 20141112 (1)Corporate presentation 20141112 (1)
Corporate presentation 20141112 (1)John Redaelli
 
INNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-A
INNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-AINNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-A
INNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-AHealthegy
 
Act corporate presentation may 2014 - print version
Act corporate presentation   may 2014 - print versionAct corporate presentation   may 2014 - print version
Act corporate presentation may 2014 - print versionJohn Redaelli
 
DIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg Engineering
DIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg EngineeringDIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg Engineering
DIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg EngineeringHealthegy
 
Non-viral ocular delivery
Non-viral ocular deliveryNon-viral ocular delivery
Non-viral ocular deliverytacomeau
 
Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear...
 Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear... Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear...
Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear...Andrew Stephens
 

What's hot (18)

Clinical Outlooks for Regenerative Medicine, Boston. June 2012
Clinical Outlooks for Regenerative Medicine, Boston. June 2012Clinical Outlooks for Regenerative Medicine, Boston. June 2012
Clinical Outlooks for Regenerative Medicine, Boston. June 2012
 
Rodman Conference, September 2012
Rodman Conference, September 2012Rodman Conference, September 2012
Rodman Conference, September 2012
 
2013 Aegis Capital Healthcare Conference, Sept 26th 2013
2013 Aegis Capital Healthcare Conference, Sept 26th 20132013 Aegis Capital Healthcare Conference, Sept 26th 2013
2013 Aegis Capital Healthcare Conference, Sept 26th 2013
 
Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...
Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...
Terrapinn's Stem Cells USA & Regenerative Medicine Congress, Boston, Mass., S...
 
BioEurope Presentation, March 2010
BioEurope Presentation, March 2010BioEurope Presentation, March 2010
BioEurope Presentation, March 2010
 
Corporate presentation 20141112 (1)
Corporate presentation 20141112 (1)Corporate presentation 20141112 (1)
Corporate presentation 20141112 (1)
 
INNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-A
INNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-AINNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-A
INNOVATION IN RETINAL IMAGING: OPPORTUNITIES & CHALLENGES - History of OCT-A
 
2012 BIO International Convention, Boston
2012 BIO International Convention, Boston2012 BIO International Convention, Boston
2012 BIO International Convention, Boston
 
Act corporate presentation may 2014 - print version
Act corporate presentation   may 2014 - print versionAct corporate presentation   may 2014 - print version
Act corporate presentation may 2014 - print version
 
DIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg Engineering
DIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg EngineeringDIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg Engineering
DIAGNOSTICS-IMPACT ON THE PREMIUM CHANNEL - Heidelberg Engineering
 
OCT ANGIOGRAPHY
OCT ANGIOGRAPHYOCT ANGIOGRAPHY
OCT ANGIOGRAPHY
 
OCT ANGIOGRAPHY
OCT ANGIOGRAPHYOCT ANGIOGRAPHY
OCT ANGIOGRAPHY
 
Coding Yesterday's Nomenclature Today
Coding Yesterday's Nomenclature TodayCoding Yesterday's Nomenclature Today
Coding Yesterday's Nomenclature Today
 
Non-viral ocular delivery
Non-viral ocular deliveryNon-viral ocular delivery
Non-viral ocular delivery
 
Rodman Conference Presentation: William Caldwell
Rodman Conference Presentation: William CaldwellRodman Conference Presentation: William Caldwell
Rodman Conference Presentation: William Caldwell
 
Solix brochure
Solix brochureSolix brochure
Solix brochure
 
Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear...
 Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear... Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear...
Piramal Imaging to Present New Research in PET Imaging at Society of Nuclear...
 
PIPEs Conference Presentations, November 2006
PIPEs Conference Presentations, November 2006PIPEs Conference Presentations, November 2006
PIPEs Conference Presentations, November 2006
 

Viewers also liked

Viewers also liked (6)

Ancillary task research ROMANCE
Ancillary task research ROMANCEAncillary task research ROMANCE
Ancillary task research ROMANCE
 
Buildium Story and Culture
Buildium Story and CultureBuildium Story and Culture
Buildium Story and Culture
 
2014 BIO CEO Corporate Presentation, NY, NY Feb 11, 2014
2014 BIO CEO Corporate Presentation, NY, NY Feb 11, 20142014 BIO CEO Corporate Presentation, NY, NY Feb 11, 2014
2014 BIO CEO Corporate Presentation, NY, NY Feb 11, 2014
 
Internship Report
Internship  ReportInternship  Report
Internship Report
 
Versetamide 129009-83-2-api
Versetamide 129009-83-2-apiVersetamide 129009-83-2-api
Versetamide 129009-83-2-api
 
Prueba martes
Prueba martesPrueba martes
Prueba martes
 

Similar to Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011

Act corporate-presentationfeb13
Act corporate-presentationfeb13Act corporate-presentationfeb13
Act corporate-presentationfeb13Company Spotlight
 
2012 World Stem Cell & Regenerative Medicine Congress, London
2012 World Stem Cell & Regenerative Medicine Congress, London2012 World Stem Cell & Regenerative Medicine Congress, London
2012 World Stem Cell & Regenerative Medicine Congress, LondonAdvanced Cell Technology, Inc.
 
Act corporate-presentation jan13
Act corporate-presentation jan13Act corporate-presentation jan13
Act corporate-presentation jan13Company Spotlight
 
Act corporate-presentation14
Act corporate-presentation14Act corporate-presentation14
Act corporate-presentation14Company Spotlight
 
Act corporate presentation bioceo - february 2014- print version
Act corporate presentation   bioceo - february 2014- print versionAct corporate presentation   bioceo - february 2014- print version
Act corporate presentation bioceo - february 2014- print versionJohn Redaelli
 
Corporate presentation-20141120
Corporate presentation-20141120Corporate presentation-20141120
Corporate presentation-20141120John Redaelli
 
Act april13regen-med-investor-day
Act april13regen-med-investor-dayAct april13regen-med-investor-day
Act april13regen-med-investor-dayCompany Spotlight
 
ARM's Regen Med Investor Day, New York City, April 17, 2013
ARM's Regen Med Investor Day, New York City, April 17, 2013ARM's Regen Med Investor Day, New York City, April 17, 2013
ARM's Regen Med Investor Day, New York City, April 17, 2013Advanced Cell Technology, Inc.
 
2 2. hyung min chung ppt
2 2. hyung min chung ppt2 2. hyung min chung ppt
2 2. hyung min chung pptJohn Redaelli
 
Dr Gerry Chader - AGM 2011 - Research Advancements
Dr Gerry Chader - AGM 2011 - Research AdvancementsDr Gerry Chader - AGM 2011 - Research Advancements
Dr Gerry Chader - AGM 2011 - Research AdvancementsFighting Blindness
 
World stem cells & regenerative medicine congress, london, may 22, 2013
World stem cells & regenerative medicine congress, london, may 22, 2013World stem cells & regenerative medicine congress, london, may 22, 2013
World stem cells & regenerative medicine congress, london, may 22, 2013Advanced Cell Technology, Inc.
 
Act corporate-presentation---may-2013---wscrm---print-version
Act corporate-presentation---may-2013---wscrm---print-versionAct corporate-presentation---may-2013---wscrm---print-version
Act corporate-presentation---may-2013---wscrm---print-versionJohn Redaelli
 
15.15 matthew vincent
15.15 matthew vincent15.15 matthew vincent
15.15 matthew vincentJohn Redaelli
 
Act annual-shareholders'-meeting-10.22.13
Act annual-shareholders'-meeting-10.22.13Act annual-shareholders'-meeting-10.22.13
Act annual-shareholders'-meeting-10.22.13John Redaelli
 
Presentation at BIO CEO & Investor Conference, New York City. February 2011
Presentation at BIO CEO & Investor Conference, New York City. February 2011Presentation at BIO CEO & Investor Conference, New York City. February 2011
Presentation at BIO CEO & Investor Conference, New York City. February 2011Advanced Cell Technology, Inc.
 
CGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptx
CGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptxCGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptx
CGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptxEEPD1
 

Similar to Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011 (20)

Act corporate-presentationfeb13
Act corporate-presentationfeb13Act corporate-presentationfeb13
Act corporate-presentationfeb13
 
2012 World Stem Cell & Regenerative Medicine Congress, London
2012 World Stem Cell & Regenerative Medicine Congress, London2012 World Stem Cell & Regenerative Medicine Congress, London
2012 World Stem Cell & Regenerative Medicine Congress, London
 
Stem Cell Meeting on the Mesa, Oct. 29, 2012
Stem Cell Meeting on the Mesa, Oct. 29, 2012Stem Cell Meeting on the Mesa, Oct. 29, 2012
Stem Cell Meeting on the Mesa, Oct. 29, 2012
 
Act corporate-presentation jan13
Act corporate-presentation jan13Act corporate-presentation jan13
Act corporate-presentation jan13
 
Biotech Showcase, San Francisco, Calif., Jan. 8, 2013
Biotech Showcase, San Francisco, Calif., Jan. 8, 2013Biotech Showcase, San Francisco, Calif., Jan. 8, 2013
Biotech Showcase, San Francisco, Calif., Jan. 8, 2013
 
Act corporate-presentation14
Act corporate-presentation14Act corporate-presentation14
Act corporate-presentation14
 
Act corporate presentation bioceo - february 2014- print version
Act corporate presentation   bioceo - february 2014- print versionAct corporate presentation   bioceo - february 2014- print version
Act corporate presentation bioceo - february 2014- print version
 
Corporate presentation-20141120
Corporate presentation-20141120Corporate presentation-20141120
Corporate presentation-20141120
 
Act april13regen-med-investor-day
Act april13regen-med-investor-dayAct april13regen-med-investor-day
Act april13regen-med-investor-day
 
ARM's Regen Med Investor Day, New York City, April 17, 2013
ARM's Regen Med Investor Day, New York City, April 17, 2013ARM's Regen Med Investor Day, New York City, April 17, 2013
ARM's Regen Med Investor Day, New York City, April 17, 2013
 
2 2. hyung min chung ppt
2 2. hyung min chung ppt2 2. hyung min chung ppt
2 2. hyung min chung ppt
 
Dr Gerry Chader - AGM 2011 - Research Advancements
Dr Gerry Chader - AGM 2011 - Research AdvancementsDr Gerry Chader - AGM 2011 - Research Advancements
Dr Gerry Chader - AGM 2011 - Research Advancements
 
Act corp-pres-may13
Act corp-pres-may13Act corp-pres-may13
Act corp-pres-may13
 
World stem cells & regenerative medicine congress, london, may 22, 2013
World stem cells & regenerative medicine congress, london, may 22, 2013World stem cells & regenerative medicine congress, london, may 22, 2013
World stem cells & regenerative medicine congress, london, may 22, 2013
 
Act corporate-presentation---may-2013---wscrm---print-version
Act corporate-presentation---may-2013---wscrm---print-versionAct corporate-presentation---may-2013---wscrm---print-version
Act corporate-presentation---may-2013---wscrm---print-version
 
15.15 matthew vincent
15.15 matthew vincent15.15 matthew vincent
15.15 matthew vincent
 
ACT Annual Shareholders' Meeting, October 22, 2013
ACT Annual Shareholders' Meeting, October 22, 2013ACT Annual Shareholders' Meeting, October 22, 2013
ACT Annual Shareholders' Meeting, October 22, 2013
 
Act annual-shareholders'-meeting-10.22.13
Act annual-shareholders'-meeting-10.22.13Act annual-shareholders'-meeting-10.22.13
Act annual-shareholders'-meeting-10.22.13
 
Presentation at BIO CEO & Investor Conference, New York City. February 2011
Presentation at BIO CEO & Investor Conference, New York City. February 2011Presentation at BIO CEO & Investor Conference, New York City. February 2011
Presentation at BIO CEO & Investor Conference, New York City. February 2011
 
CGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptx
CGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptxCGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptx
CGR PPT by DrPPS PRABHAKRA SASTRY-10-2023.pptx
 

More from Advanced Cell Technology, Inc.

Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013
Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013
Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013Advanced Cell Technology, Inc.
 
Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011
Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011
Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011Advanced Cell Technology, Inc.
 
Presentation at OneMedForum, San Francisco. January 2011
Presentation at OneMedForum, San Francisco. January 2011Presentation at OneMedForum, San Francisco. January 2011
Presentation at OneMedForum, San Francisco. January 2011Advanced Cell Technology, Inc.
 
Presentation at Bio-Europe Conference, Munich. November 2010
Presentation at Bio-Europe Conference, Munich. November 2010Presentation at Bio-Europe Conference, Munich. November 2010
Presentation at Bio-Europe Conference, Munich. November 2010Advanced Cell Technology, Inc.
 
Presentation at the Proactive Investors United Kingdom One2One Investor Forum
Presentation at the Proactive Investors United Kingdom One2One Investor ForumPresentation at the Proactive Investors United Kingdom One2One Investor Forum
Presentation at the Proactive Investors United Kingdom One2One Investor ForumAdvanced Cell Technology, Inc.
 
Presentation at BioPartnering Europe Conference, London. October 2010
Presentation at BioPartnering Europe Conference, London. October 2010Presentation at BioPartnering Europe Conference, London. October 2010
Presentation at BioPartnering Europe Conference, London. October 2010Advanced Cell Technology, Inc.
 
Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...
Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...
Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...Advanced Cell Technology, Inc.
 
ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...
ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...
ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...Advanced Cell Technology, Inc.
 
ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...
ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...
ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...Advanced Cell Technology, Inc.
 

More from Advanced Cell Technology, Inc. (9)

Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013
Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013
Rodman and Rendshaw 2013 Annual Global Investment Conference, NYC, Sept 2013
 
Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011
Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011
Presentation at Bio-Europe Spring 2011 Conference, Milan, Italy. March 2011
 
Presentation at OneMedForum, San Francisco. January 2011
Presentation at OneMedForum, San Francisco. January 2011Presentation at OneMedForum, San Francisco. January 2011
Presentation at OneMedForum, San Francisco. January 2011
 
Presentation at Bio-Europe Conference, Munich. November 2010
Presentation at Bio-Europe Conference, Munich. November 2010Presentation at Bio-Europe Conference, Munich. November 2010
Presentation at Bio-Europe Conference, Munich. November 2010
 
Presentation at the Proactive Investors United Kingdom One2One Investor Forum
Presentation at the Proactive Investors United Kingdom One2One Investor ForumPresentation at the Proactive Investors United Kingdom One2One Investor Forum
Presentation at the Proactive Investors United Kingdom One2One Investor Forum
 
Presentation at BioPartnering Europe Conference, London. October 2010
Presentation at BioPartnering Europe Conference, London. October 2010Presentation at BioPartnering Europe Conference, London. October 2010
Presentation at BioPartnering Europe Conference, London. October 2010
 
Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...
Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...
Presentation at the 12th Annual Rodman & Renshaw healthcare Conference in New...
 
ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...
ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...
ACT Presentation at the 11th Annual B. Riley Investor Conference in Santa Mon...
 
ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...
ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...
ACT Presentation at Rodman & Renshaw Annual Global Investment Conference, Lon...
 

Recently uploaded

Boost PC performance: How more available memory can improve productivity
Boost PC performance: How more available memory can improve productivityBoost PC performance: How more available memory can improve productivity
Boost PC performance: How more available memory can improve productivityPrincipled Technologies
 
Data Cloud, More than a CDP by Matt Robison
Data Cloud, More than a CDP by Matt RobisonData Cloud, More than a CDP by Matt Robison
Data Cloud, More than a CDP by Matt RobisonAnna Loughnan Colquhoun
 
A Call to Action for Generative AI in 2024
A Call to Action for Generative AI in 2024A Call to Action for Generative AI in 2024
A Call to Action for Generative AI in 2024Results
 
Real Time Object Detection Using Open CV
Real Time Object Detection Using Open CVReal Time Object Detection Using Open CV
Real Time Object Detection Using Open CVKhem
 
Breaking the Kubernetes Kill Chain: Host Path Mount
Breaking the Kubernetes Kill Chain: Host Path MountBreaking the Kubernetes Kill Chain: Host Path Mount
Breaking the Kubernetes Kill Chain: Host Path MountPuma Security, LLC
 
Tata AIG General Insurance Company - Insurer Innovation Award 2024
Tata AIG General Insurance Company - Insurer Innovation Award 2024Tata AIG General Insurance Company - Insurer Innovation Award 2024
Tata AIG General Insurance Company - Insurer Innovation Award 2024The Digital Insurer
 
A Year of the Servo Reboot: Where Are We Now?
A Year of the Servo Reboot: Where Are We Now?A Year of the Servo Reboot: Where Are We Now?
A Year of the Servo Reboot: Where Are We Now?Igalia
 
Factors to Consider When Choosing Accounts Payable Services Providers.pptx
Factors to Consider When Choosing Accounts Payable Services Providers.pptxFactors to Consider When Choosing Accounts Payable Services Providers.pptx
Factors to Consider When Choosing Accounts Payable Services Providers.pptxKatpro Technologies
 
[2024]Digital Global Overview Report 2024 Meltwater.pdf
[2024]Digital Global Overview Report 2024 Meltwater.pdf[2024]Digital Global Overview Report 2024 Meltwater.pdf
[2024]Digital Global Overview Report 2024 Meltwater.pdfhans926745
 
Boost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdfBoost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdfsudhanshuwaghmare1
 
Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...
Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...
Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...Miguel Araújo
 
What Are The Drone Anti-jamming Systems Technology?
What Are The Drone Anti-jamming Systems Technology?What Are The Drone Anti-jamming Systems Technology?
What Are The Drone Anti-jamming Systems Technology?Antenna Manufacturer Coco
 
The Codex of Business Writing Software for Real-World Solutions 2.pptx
The Codex of Business Writing Software for Real-World Solutions 2.pptxThe Codex of Business Writing Software for Real-World Solutions 2.pptx
The Codex of Business Writing Software for Real-World Solutions 2.pptxMalak Abu Hammad
 
Understanding Discord NSFW Servers A Guide for Responsible Users.pdf
Understanding Discord NSFW Servers A Guide for Responsible Users.pdfUnderstanding Discord NSFW Servers A Guide for Responsible Users.pdf
Understanding Discord NSFW Servers A Guide for Responsible Users.pdfUK Journal
 
Artificial Intelligence: Facts and Myths
Artificial Intelligence: Facts and MythsArtificial Intelligence: Facts and Myths
Artificial Intelligence: Facts and MythsJoaquim Jorge
 
EIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptx
EIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptxEIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptx
EIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptxEarley Information Science
 
08448380779 Call Girls In Diplomatic Enclave Women Seeking Men
08448380779 Call Girls In Diplomatic Enclave Women Seeking Men08448380779 Call Girls In Diplomatic Enclave Women Seeking Men
08448380779 Call Girls In Diplomatic Enclave Women Seeking MenDelhi Call girls
 
Driving Behavioral Change for Information Management through Data-Driven Gree...
Driving Behavioral Change for Information Management through Data-Driven Gree...Driving Behavioral Change for Information Management through Data-Driven Gree...
Driving Behavioral Change for Information Management through Data-Driven Gree...Enterprise Knowledge
 
Axa Assurance Maroc - Insurer Innovation Award 2024
Axa Assurance Maroc - Insurer Innovation Award 2024Axa Assurance Maroc - Insurer Innovation Award 2024
Axa Assurance Maroc - Insurer Innovation Award 2024The Digital Insurer
 
Powerful Google developer tools for immediate impact! (2023-24 C)
Powerful Google developer tools for immediate impact! (2023-24 C)Powerful Google developer tools for immediate impact! (2023-24 C)
Powerful Google developer tools for immediate impact! (2023-24 C)wesley chun
 

Recently uploaded (20)

Boost PC performance: How more available memory can improve productivity
Boost PC performance: How more available memory can improve productivityBoost PC performance: How more available memory can improve productivity
Boost PC performance: How more available memory can improve productivity
 
Data Cloud, More than a CDP by Matt Robison
Data Cloud, More than a CDP by Matt RobisonData Cloud, More than a CDP by Matt Robison
Data Cloud, More than a CDP by Matt Robison
 
A Call to Action for Generative AI in 2024
A Call to Action for Generative AI in 2024A Call to Action for Generative AI in 2024
A Call to Action for Generative AI in 2024
 
Real Time Object Detection Using Open CV
Real Time Object Detection Using Open CVReal Time Object Detection Using Open CV
Real Time Object Detection Using Open CV
 
Breaking the Kubernetes Kill Chain: Host Path Mount
Breaking the Kubernetes Kill Chain: Host Path MountBreaking the Kubernetes Kill Chain: Host Path Mount
Breaking the Kubernetes Kill Chain: Host Path Mount
 
Tata AIG General Insurance Company - Insurer Innovation Award 2024
Tata AIG General Insurance Company - Insurer Innovation Award 2024Tata AIG General Insurance Company - Insurer Innovation Award 2024
Tata AIG General Insurance Company - Insurer Innovation Award 2024
 
A Year of the Servo Reboot: Where Are We Now?
A Year of the Servo Reboot: Where Are We Now?A Year of the Servo Reboot: Where Are We Now?
A Year of the Servo Reboot: Where Are We Now?
 
Factors to Consider When Choosing Accounts Payable Services Providers.pptx
Factors to Consider When Choosing Accounts Payable Services Providers.pptxFactors to Consider When Choosing Accounts Payable Services Providers.pptx
Factors to Consider When Choosing Accounts Payable Services Providers.pptx
 
[2024]Digital Global Overview Report 2024 Meltwater.pdf
[2024]Digital Global Overview Report 2024 Meltwater.pdf[2024]Digital Global Overview Report 2024 Meltwater.pdf
[2024]Digital Global Overview Report 2024 Meltwater.pdf
 
Boost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdfBoost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdf
 
Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...
Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...
Mastering MySQL Database Architecture: Deep Dive into MySQL Shell and MySQL R...
 
What Are The Drone Anti-jamming Systems Technology?
What Are The Drone Anti-jamming Systems Technology?What Are The Drone Anti-jamming Systems Technology?
What Are The Drone Anti-jamming Systems Technology?
 
The Codex of Business Writing Software for Real-World Solutions 2.pptx
The Codex of Business Writing Software for Real-World Solutions 2.pptxThe Codex of Business Writing Software for Real-World Solutions 2.pptx
The Codex of Business Writing Software for Real-World Solutions 2.pptx
 
Understanding Discord NSFW Servers A Guide for Responsible Users.pdf
Understanding Discord NSFW Servers A Guide for Responsible Users.pdfUnderstanding Discord NSFW Servers A Guide for Responsible Users.pdf
Understanding Discord NSFW Servers A Guide for Responsible Users.pdf
 
Artificial Intelligence: Facts and Myths
Artificial Intelligence: Facts and MythsArtificial Intelligence: Facts and Myths
Artificial Intelligence: Facts and Myths
 
EIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptx
EIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptxEIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptx
EIS-Webinar-Prompt-Knowledge-Eng-2024-04-08.pptx
 
08448380779 Call Girls In Diplomatic Enclave Women Seeking Men
08448380779 Call Girls In Diplomatic Enclave Women Seeking Men08448380779 Call Girls In Diplomatic Enclave Women Seeking Men
08448380779 Call Girls In Diplomatic Enclave Women Seeking Men
 
Driving Behavioral Change for Information Management through Data-Driven Gree...
Driving Behavioral Change for Information Management through Data-Driven Gree...Driving Behavioral Change for Information Management through Data-Driven Gree...
Driving Behavioral Change for Information Management through Data-Driven Gree...
 
Axa Assurance Maroc - Insurer Innovation Award 2024
Axa Assurance Maroc - Insurer Innovation Award 2024Axa Assurance Maroc - Insurer Innovation Award 2024
Axa Assurance Maroc - Insurer Innovation Award 2024
 
Powerful Google developer tools for immediate impact! (2023-24 C)
Powerful Google developer tools for immediate impact! (2023-24 C)Powerful Google developer tools for immediate impact! (2023-24 C)
Powerful Google developer tools for immediate impact! (2023-24 C)
 

Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011

  • 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. At the Forefront of Regenerative Medicine • Patented Technology for Producing human Embryonic Stem Cells (“hESCs”) without Harm to Embryo • Working with Roslin Cells to create GMP-compliant hESC bank • 2 Human Clinical Trials utilizing hESC-derived Retinal Pigment Epithelial Cells • First Patients Treated on July 12, 2011 • Stargardt’s Disease, aka Stargardt’s Macular Dystrophy (SMD) • Dry AMD – (Dry Age-Related Macular Degeneration) • Expecting Preliminary Safety and Engraftment Data by Year-End • Commencing European Trials – estimated first half 2012 • Front-of-the-eye programs: Generating hESC-derived corneal tissues • Finalizing preclinical work for blood product IND from hemangioblast program • Generation of off-the-shelf hESC-derived mesenchymal stem cells (MSC) products • Myoblast program for heart failure approved for Phase II 3
  • 5. 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 may be part of mechanism underlying 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
  • 6. • 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
  • 7. • 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 • Ease-of-Handling by Doctor 7
  • 8. Differentiation Media is Not Permissive for hESCs Harvest for Cryopreservation ES Cells EB Formation RPE isolation P1 P2 0 5 weeks 12 weeks 15 weeks 18 weeks Up to 10 percent hESC spiked No hESC’s in harvested cells Limit of Detection for hESC 1:20,000,000 cells RPE differentiation is the default pathway under ACT’s Patented Culture Conditions 3 weeks EB Outgrowth 30 Weeks 8
  • 9. RPE Animal Safety Studies Extensive Safety Studies Shows Lack of Tumorigenicity Long-term data (spanning the life of the animals) revealed no evidence of teratoma formation after subretinal hESC- RPE transplantation. 9
  • 10. 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 10
  • 11. 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 11
  • 12. • Stargardt’s (SMD) Disease • IND approved in November 2010 • European CTA filed • 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) 12
  • 13. • Also referred to as “Juvenile Macular Degeneration” – Causes progressive vision loss beginning in childhood. – Stargardt’s Disease is the most common hereditary macular dystrophy. – Prevalence rate of about 1-in-10,000. – Usually diagnosed in individuals under the age of twenty. • ACT has obtained Orphan Drug Designation in United States and Europe – 7 - 10 Years of Market Exclusivity for using RPE cells to treat Stargardt’s Disease. Stargardt’s Macular Dystophy Orphan Drug Opportunity with Reimbursement 80,000-100,000 patients in North America and Europe 13
  • 14. • AMD - estimate over 30 Million patients in North America and Europe – The prevalence of AMD in North America in the population aged 40 to 79 years is 8.8% – The prevalence of AMD in China in the population aged 40 to 79 years is 6.8% • Approximately 10% of people ages 66 to 74 have symptoms of macular degeneration • Prevalence increases to 30% in patients 75 to 85 years of age. Dry AMD (non-exudative) – The most common form of AMD (estimates as high as 90 percent) – No Effective Therapy Currently Available – Estimated $20-30 Billion market Age-Related Macular Degeneration Potential for Blockbuster Status 14
  • 15. • 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 will be monitored weekly - including high definition imaging of retina High Definition Spectral Domain Optical Coherence Tomography (SD-OCT) Retinal Autofluorescence Adaptive Optics Scanning Laser Ophthalmoscopy (AOSLO) 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 15
  • 16. Phase I - Clinical Trial Update • Prospective clinical studies to determine the safety and tolerability of sub-retinal transplantation of hESC-derived RPE cells. • Vitrectomy including surgical induction of posterior vitreous separation from the optic nerve was carried out • Submacular injection of 50,000 hESC-derived RPE cells in a volume of 150µl was delivered into a pre-selected area of the pericentral macula • Patients are monitored for systemic safety signals. • Pre- and weekly postoperative ophthalmic examinations. Visual acuity, fluorescein angiography, optical coherence tomography (OCT), autofluorescence imaging and visual field testing • DSMB Review Underway More to come…. Early clinical and laboratory findings with respect to safety, tolerability and engraftment to be made available Drs. Steven Schwartz and Robert Lanza Straight forward surgical approach 16
  • 17. Next 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 (DSAEK). hESC-derived corneal endothelium resembles normal human corneal endothelium 17
  • 19. Hemangioblast Program – JV Update • Stem Cell & Regenerative Medicine International (SCRMI). – ACT and CHA agree to restructure their joint venture. – SCRMI exclusively licensed the rights to hemangioblast program to ACT for North America and to CHA Biotech for Korea and Japan. – SCRMI scientists reassigned to ACT to continue research and product development efforts as ACT employees – Both companies will work to develop clinical therapies based on the joint venture's proprietary hemangioblast cell technology. • Products Opportunities include: – Universal Blood Components, such as Red Blood Cells and Platelets – Meschenchymal Stem Cells • Products for treating inflammatory diseases, promoting tolerance to grafts, repairing connective tissues, delivering therapeutic proteins, etc. – Revascularization Therapies for treating ischemic injuries • September 13, 2011: U.S. Patent 8,017,393 broadly covers ACT’s proprietary method for deriving hemangioblast cells from embryonic stem cells. 19 Robust Product Pipeline
  • 20. Hemangioblast Program: Overview 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. • Differentiate into both hematopoietic and endothelial cell lineages – and newly discovered by ACT, mesenchymal stem cells (MSC). • Be used to generate pathogen-free blood components • Be used to achieve vascular repair 20 Revascularization Blood Products
  • 21. Hemangioblasts RBCs Generation of Blood Products - RBC Hemangioblasts Enucleated RBC’s Capable of generating large quantities of enucleated red blood cells. 21 - Blood Replacement Products - Triage and battlefield applications - Systemic Delivery System - Blood cells are great for transporting materials through the body as the entire circulatory system evolved to facilitate their movement - Examples: Delivery of drugs or imaging agents
  • 22. Generation of Blood Products - Platelets • Scalable: Generation of large quantities of Platelets from hESC and iPS sources • Off-the-shelf allogeneic products: Short in vivo half-life (7 days) and allotolerance for acute uses • Tractable regulatory approval process • Therapeutic products for accelerating soft and hard tissue healing. – Platelets improve the process of tissue repair – release agents involved in inflammation, angiogenesis and extracellular matrix synthesis – all involved in wound repair • Product Opportunties: bone fractures and bony defects; laminectomy procedures; lateral epicondylitis (i.e., tennis elbow); total joint arthroplasty procedures (i.e., knee, hip, shoulder); plantar fasciitis; shoulder arthroscopy and distal clavicle resection; and spinal fusion. • Extensive Cosmetic Uses – Wrinkle and Lift Procedures: Platelets are loaded with growth factors for skin and blood vessels and produce new collagen without the wounds of a laser and the risk of scarring 22  Efficiently generate functional megakaryocytes & platelets. • ES-derived platelets participate in clot formation. • ES-derived platelets incorporate into mouse thrombus at site of laser- induced arteriolar injury
  • 23. Repair of Vascular Damage Hemangioblasts were tested in animal models of diabetic retinopathy, heart disease and peripheral vascular damage Results from treatment with hemangioblast cells • Restoration of blood flow to ischemic limbs. • Survival after myocardial infarction. • Revascularizes ischemic retinas Potential Impact on a Large Number of Vascular Diseases myocardial infarction, vascular ischemic damage, ischemia-reperfusion injury, diabetic vascular disease and peripheral artery disease (PAD) that are leading causes of death and/or disability worldwide. Hemangioblasts promoted repair in peripheral vascular damage 23
  • 24. Mesenchymal Stem Cells hESC-MSCs can be obtained using the hemangioblast method • hESC-MSCs are easy to derive and can be expanded to large numbers in vitro • Quality controls are easier to manage for a renewable cell source • Can serve as an “off the shelf” therapy, available for immediate use • Products for treating inflammatory diseases, promoting tolerance to grafts, repairing connective tissues, delivering therapeutic proteins, etc. 24
  • 25. Platform Technology for Generating Robust Human Embryonic Stem Cells Without the Need to Destroy Embryos Single Blastomere Technology
  • 26. 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. 26 Single Blastomere Technology
  • 27. 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. 27
  • 28. Financial Update – Strong Balance Sheet 28 Most Stable Financial Situation In Company History • The Company ended 2011 Q2 with $16.1 million cash on hand • $17 million more equity available • Virtually debt-free • Able to self-fund both U.S. clinical trials and EP clinical trial • Significantly deepened management team (and on-going) • Gary Rabin appointed CEO (change from interim status) • Exciting new Board of Director members to announce • Unqualified audit opinion Entering clinical trials with a strong balance sheet
  • 29. 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. Matthew Vincent, Ph.D. – Director of Business Development Edmund Mickunas – Vice President of Regulatory Affairs Bill Douglass – Director of Corporate Communications & Social Media Stephen Price – Interim SVP – Corporate Development Gary Rabin – Chairman and CEO Kathy Singh - Controller Rita Parker – Director of Operations 29
  • 30. Thank you for Attending. For more information, visit www.advancedcell.com