GENReports: Market & Tech Analysis

Stem Cells Market 
Trends based on Primary 
Industry Analysis

> Enal Razvi, Ph.D. > Gary Oosta, Ph.D.

Biotechnology Analyst, Biotechnology Analyst,
Managing Director SELECTBIO US
SELECTBIO US
enal@selectbio.us
GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013
 Topic Introduction and Scope

• The focus of this GEN Market & Tech Analysis Report is to

characterize the stem cells marketplace based on the following
classes of data which we have been collecting as part of our
industry analysis of this field:

– Industry SWOT
– Primary Market Data of Markers Used to Characterize Different
Cellular Classes
– Publications Landscape
• Taken together, these data provide a backdrop for understanding
the stem cells marketplace as it exists today

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

 Stem Cells Market Segmentation
and Industry Structure

Tools Space Cellular Therapeutics

Space
Autologous Allogeneic
Basic Research into Cell Therapy Cell Therapy
Stem cell Biology,
Function
[Life Science Research]
¾ 100% Adult Stem ¾ 100% Adult Stem Cells
Toxicity Testing, Screening Cells Today
in Drug Discovery and ¾ HSCs, MSCs, SVF,.. ¾ Primarily HSC and MSC
Development ¾ PBL, BM, Cord Blood, ¾ Clinical Trials of hESC-
Disease Modeling Adipose,.. derived
[Pharma, Biotech Focus] in progress
¾ iPSC Clinical Trials Soon
[ACT, Kobe, et al.]
GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013
 Autologous versus Allogeneic Business
Models in the Cellular Therapy Space:
Industry SWOT
Autologous
ƒ Can be Profitable but is Not
Scalable
ƒ Creates Strong Relationships
Strength between Caregivers and Patients
ƒ No Immunological Rejection
ƒ Small Patient Populations
Weakness ƒ Not Scalable
ƒ Personalized Medicine
ƒ Patients Need to be in Proximity to
Research Hospital/Provider
Opportunity ƒ Specialty Service Business
ƒ Target Disease Areas for which
Personalized Care is Appropriate—
such as Cancers
ƒ High Potential Barriers to Entry
GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013
ƒ iPSC-based Cellular Therapy is a
Threat
Disruptive Technology/Approach
Allogeneic
ƒ Scalable Business Model in Terms of
Patient Populations
ƒ Lower COGS—Cost Structure is
Scalable
ƒ Can Compete against Biologics and
Possibly Small Molecules
ƒ Long Development Times
ƒ Production Must be Scalable—ie.,
Starting Cell Populations Must
Support Massive Expansion
ƒ Immunological Rejection is an
Issue
ƒ Cost Structure Spread Across
Multiple Therapeutic
Targets/Programs
ƒ Develop for Diseases where Point-
of-Care Therapy is Required [such
as AMI, Trauma]
ƒ iPSC-based Cellular Therapy is a
Disruptive Technology/Approach
 Current State of the Stem Cells
Marketplace

• >$1 Billion in Product Sales Currently in 
the Broader Cellular Therapy Marketplace 
Currently
• 30 percent compound annual growth rate
• Product Classes Breakout
– Dermatologic/Wound Care
– Musculo‐skeletal
– Other Classes
GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013
Stem Cell Research Trends based on
Primary Market Research performed
by SELECTBIO
 Market Penetrance of Various Vendors into
the Different Segments of the Stem Cells
Research Marketplace

100
Hepatocytes for Toxicity
90 Screening

Cardiomyocytes for Toxicity

80
Screening
70 Reagents for Stem Cell
Ordinate Represents the Penetrance of a Given Vendor into a (eg.,
Characterization
60
Particular Stem Cell Research Market Segment
antibodies)
Cell Separation Products
50

40 Small Molecules for

Differentiation
30
Genetic Constructs (vectors,
20
reporter constructs, promoter
constructs)
Plasticware
10

0 Cell Culture Supplements

Cell Culture Growth

Factors/Cytokines

Cell Culture Media

Other Stem Cell Types (Cells)

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013
 Markers [Surface or Intracellular] Used to
Characterize Pluripotent Cells (ESCs, iPSCs)

90%

80%

70%

60%

50%
Height of Each Bar is Proportional to the Penetrance of the Given Marker
40% for Characterization of the Cell Type.
For Pluripotent Cells, the Most Penetrant Markers are Oct4 & Nanog
30%

20%

10%

0%

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

 Markers [Surface or Intracellular] Used
to Characterize Mesenchymal Stromal
Cells (MSCs)
60%

50%

40%

30%

20%

10%

0%

Height of Each Bar is Proportional to the Penetrance of the Given Marker for
Characterization of the Cell Type.
For MSCs, the Most-Penetrant Markers are: STRO-1, CD44, CD73, and CD105
GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013
 Markers [Surface or Intracellular] Used
to Characterize Neural Stem Cells
(NSCs)
80%

70%

60%

50%

40%

30%

20%

10%

0%

Height of Each Bar is Proportional to the Penetrance of the Given Marker for
GENengnews.com
Characterization of the Cell Type.
GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

For NSCs, the Most-Penetrant Markers are: Nestin and Sox-2

 Markers [Surface or Intracellular] Used
to Characterize Hematopoietic Stem
Cells (HSCs)
90%

80%

70%

60%

50%

40%

30%

20%

10%

0%
CD34 CD45 CD59 CD133 Thy-1 CD38 c-Kit Lin ABCG2 Sca-1

Height of Each Bar is Proportional to the Penetrance of the Given Marker for
GENengnews.com
Characterization of the Cell Type.
GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

For HSCs, the Most-Penetrant Markers are: CD34 and CD45

Stem Cells Publications Analysis en
bloc performed by SELECTBIO
 Stem Cells En Bloc Publications Hot
Spot Map
gives an immediate picture of what’s big, small and not there at all

Stem Cell  Cancer Autoimmune Neurological Transplant

Types Types

Clin. Heart
Epigenetics More
Trials Diseases

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

 Observations

ƒ The hotspot map (stem cells vs disease type) shows

that
ƒ Transplant dominates publication activity in stem cells
ƒ Assay Methods and Bioreactors are significant themes
ƒ Culture methods and types are an important theme
ƒ Epigenetics is well represented in stem cell research
ƒ Histone, methylation, phosphorylation and even
some sumoylation appear to be connected to stem
cells
ƒ The hotspot map shows many areas not yet explored

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

 Key Takeaways from the Data

• Expansion of the Cellular Therapy Space
– CAGR @ 30%
• Increasing Number of Clinical Trials in the US and 
EU
– 415 cellular therapy product trials under ATMP 
guidelines in the EU
• Stem Cells Publications Space being Populated 
Across Disease Classes
• Products for Stem Cell Research from Research 
Product Vendors Proliferating Æ This space is 
very fragmented and is ripe for consolidation

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013

 SELECTBIO Clinical Translation
of Stem Cells 2014 Conference

• This conference brings together researchers as well as clinicians involved in cellular 
therapy
• Keynotes by CIRM and Clinician‐Scientists Æ Provide Trajectory of the Field, Regulatory 
Trends, Opportunities for Entry, Status of Grants
• Focus on Adipose‐derived Stem Cells, Cord blood‐derived Stem Cells, Adult Stem Cells
• Clinical Practicum Presents Harvest of Adipose‐derived Stem Cells and SVF Preparation
• For more information to attend, exhibit or sponsor, please e‐mail: enal@selectbio.us

GENengnews.com GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. © 2013