Stem Cells Market Trends based on Primary Industry Analysis

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1 GENReports: Market & Tech Analysis Stem Cells Market Trends based on Primary Industry Analysis > Enal Razvi, Ph.D. Biotechnology Analyst, Managing Director SELECTBIO US > Gary Oosta, Ph.D. Biotechnology Analyst, SELECTBIO US

2 Topic Introduction and Scope The focus of this GEN Market & Tech Analysis Report is to characterizethestemcellsmarketplacebasedonthefollowing 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 thestemcellsmarketplaceasitexiststoday

3 Stem Cells Market Segmentation and Industry Structure Tools Space Basic Research into Stem cell Biology, Function [Life Science Research] Toxicity Testing, Screening in Drug Discovery and Development Disease Modeling [Pharma, Biotech Focus] Cellular Therapeutics Space Autologous Cell Therapy 100% Adult Stem Cells HSCs, MSCs, SVF,.. PBL, BM, Cord Blood, Adipose,.. Allogeneic Cell Therapy 100% Adult Stem Cells Today Primarily HSC and MSC Clinical Trials of hescderived in progress ipsc Clinical Trials Soon [ACT, Kobe, et al.]

4 Autologous versus Allogeneic Business Models in the Cellular Therapy Space: Industry SWOT Strength Autologous Can be Profitable but is Not Scalable Creates Strong Relationships between Caregivers and Patients No Immunological Rejection Allogeneic Scalable Business Model in Terms of Patient Populations Lower COGS Cost Structure is Scalable Can Compete against Biologics and Possibly Small Molecules Weakness Opportunity Threat Small Patient Populations Not Scalable Personalized Medicine Patients Need to be in Proximity to Research Hospital/Provider Specialty Service Business Target Disease Areas for which Personalized Care is Appropriate such as Cancers High Potential Barriers to Entry ipsc-based Cellular Therapy is a Disruptive Technology/Approach Long Development Times Production Must be Scalable ie., Starting Cell Populations Must Support Massive Expansion Immunological Rejection is an Issue Cost Structure Spread Across Multiple Therapeutic Targets/Programs Develop for Diseases where Pointof-Care Therapy is Required [such as AMI, Trauma] ipsc-based Cellular Therapy is a Disruptive Technology/Approach

5 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

6 Stem Cell Research Trends based on Primary Market Research performed by SELECTBIO

7 Market Penetrance of Various Vendors into the Different Segments of the Stem Cells Research Marketplace Hepatocytes for Toxicity Screening Cardiomyocytes for Toxicity Screening Ordinate Represents the Penetrance of a Given Vendor into a Particular Stem Cell Research Market Segment Reagents for Stem Cell Characterization (eg., antibodies) Cell Separation Products Small Molecules for Differentiation Genetic Constructs (vectors, reporter constructs, promoter constructs) Plasticware Cell Culture Supplements Cell Culture Growth Factors/Cytokines Cell Culture Media Other Stem Cell Types (Cells)

8 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. 30% For Pluripotent Cells, the Most Penetrant Markers are Oct4 & Nanog 20% 10% 0%

9 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

10 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 Characterization of the Cell Type. For NSCs, the Most-Penetrant Markers are: Nestin and Sox-2

11 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 Characterization of the Cell Type. For HSCs, the Most-Penetrant Markers are: CD34 and CD45

12 Stem Cells Publications Analysis en bloc performed by SELECTBIO

13 Stem Cell Types Stem Cells En Bloc Publications Hot Spot Map gives an immediate picture of what s big, small and not there at all Clin. Trials Cancer Types Autoimmune Heart Epigenetics Neurological Diseases Transplant More

14 Observations The hotspot map (stem cells vs disease type) shows that Transplant dominates publication activity in stem cells Assay Methods and Bioreactors are significant themes Culture 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

15 Key Takeaways from the Data Expansion of the Cellular Therapy Space 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

16 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: [email protected] GENReports: Market & Tech Analysis, Produced by Enal Razvi, Ph.D. 2013