stem cells market trends based on primary industry – primary market data of markers used to...

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

> Enal Razvi, Ph.D.Biotechnology Analyst,Managing DirectorSELECTBIO USenal@selectbio.us

GENReports: Market & Tech Analysis

Stem Cells Market Trends based on Primary 

Industry Analysis

> Gary Oosta, Ph.D.Biotechnology Analyst,SELECTBIO US

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

• The focus of this GEN Market & Tech Analysis Report is tocharacterize the stem cells marketplace based on the followingclasses of data which we have been collecting as part of ourindustry 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 understandingthe stem cells marketplace as it exists today

Topic Introduction and Scope

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

Stem Cells Market Segmentation and Industry Structure

Toxicity Testing, Screeningin Drug Discovery and

DevelopmentDisease Modeling

[Pharma, Biotech Focus]

AutologousCell Therapy

AllogeneicCell Therapy

100% Adult Stem Cells Today

Primarily HSC and MSCClinical Trials of hESC-

derivedin progressiPSC Clinical Trials Soon[ACT, Kobe, et al.]

Tools Space Cellular Therapeutics Space

100% Adult Stem Cells

HSCs, MSCs, SVF,..PBL, BM, Cord Blood,Adipose,..

Basic Research intoStem cell Biology,

Function[Life Science Research]

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

Autologous versus Allogeneic Business Models in the Cellular Therapy Space: Industry SWOT

Autologous AllogeneicCan be Profitable but is Not ScalableCreates Strong Relationships between Caregivers and PatientsNo Immunological Rejection

Scalable Business Model in Terms of Patient PopulationsLower COGS—Cost Structure is ScalableCan Compete against Biologics and Possibly Small Molecules

Small Patient PopulationsNot ScalablePersonalized MedicinePatients Need to be in Proximity to Research Hospital/Provider

Long Development TimesProduction Must be Scalable—ie., Starting Cell Populations Must Support Massive ExpansionImmunological Rejection is anIssue

Specialty Service BusinessTarget Disease Areas for which Personalized Care is Appropriate—such as CancersHigh Potential Barriers to Entry

Cost Structure Spread Across Multiple TherapeuticTargets/ProgramsDevelop for Diseases where Point-of-Care Therapy is Required [such as AMI, Trauma]

iPSC-based Cellular Therapy is a Disruptive Technology/Approach

iPSC-based Cellular Therapy is a Disruptive Technology/Approach

Strength

Weakness

Opportunity

Threat

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

• >$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

Current State of the Stem CellsMarketplace

Stem Cell Research Trends based on Primary Market Research performed

by SELECTBIO

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

Market Penetrance of Various Vendors into the Different Segments of the Stem Cells Research Marketplace

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100Hepatocytes for ToxicityScreening

Cardiomyocytes for ToxicityScreening

Reagents for Stem CellCharacterization (eg.,antibodies)Cell Separation Products

Small Molecules forDifferentiation

Genetic Constructs (vectors,reporter constructs, promoterconstructs)Plasticware

Cell Culture Supplements

Cell Culture GrowthFactors/Cytokines

Cell Culture Media

Other Stem Cell Types (Cells)

Ordinate Represents the Penetrance of a Given Vendor into aParticular Stem Cell Research Market Segment

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

Markers [Surface or Intracellular] Used to Characterize Pluripotent Cells (ESCs, iPSCs)

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Height of Each Bar is Proportional to the Penetrance of the Given Marker for Characterization of the Cell Type.

For Pluripotent Cells, the Most Penetrant Markers are Oct4 & Nanog

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

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

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

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

Markers [Surface or Intracellular] Used to Characterize Neural Stem Cells (NSCs)

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Height of Each Bar is Proportional to the Penetrance of the Given Marker for Characterization of the Cell Type.

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

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

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

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

Height of Each Bar is Proportional to the Penetrance of the Given Marker for Characterization of the Cell Type.

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

Stem Cells Publications Analysis en bloc performed by SELECTBIO

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

Stem Cells En Bloc Publications Hot Spot Mapgives an immediate picture of what’s big, small and not there at all

Stem Cell Types

Clin.Trials

CancerTypes

Autoimmune

HeartEpigenetics

Neurological

Diseases

Transplant

More

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

Observations

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

Transplant dominates publication activity in stem cellsAssay Methods and Bioreactors are significant themesCulture methods and types are an important theme

Epigenetics is well represented in stem cell research Histone, methylation, phosphorylation and even some sumoylation appear to be connected to stem cells

The hotspot map shows many areas not yet explored

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

• 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  This space is very fragmented and is ripe for consolidation

Key Takeaways from the Data

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

• This conference brings together researchers as well as clinicians involved in cellular therapy

• Keynotes by CIRM and Clinician‐Scientists  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

SELECTBIO Clinical Translationof Stem Cells 2014 Conference