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Global Cell Therapy Market—Companies to Action, 2018
A Collaborative Business Model With Focus on Stem Cells, Chimeric

Antigen Receptor T (CAR-T) Cells and Combination Therapies Will
Propel Market Growth
Global Transformational Health Research Team at Frost & Sullivan
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March 2019
Contents
Section Slide Number

 Methodology 7
Executive Summary 8
 Key Findings—Analysis of Companies to Action 9
 Scope and Segmentation 11
 Key Questions Addressed in this Study 12
Companies to Action Overview 13
 Companies to Action Value Creators 14
 Opportunities and Threats 15
Cell Therapy Market Overview 16
 Market Definitions 17
 Market Segmentation 18
 Cell Therapy Value Chain 19
 Vendor Ecosystem 20
 Impact of Key Market Influencers 21
 Top 3 Trends and Vendor Strategies 22
 Key Market Participants Aligned with Top 3 Trends 23
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Contents (continued)

 Cell Therapy—Vendor Landscape 24
C2A Profiles—Tier I 25
 Juno Therapeutics—Overview 26
 Juno Therapeutics—Technology Platforms 27
 Juno Therapeutics—Major Partnerships and Agreements 28
 bluebird bio—Business Overview 29
 bluebird bio—Technology Platform 30
 bluebird bio—Major Partnerships and Agreements 31
 Kite Pharma—Overview 32
 Kite Pharma—Key Technology/ Product/Services 33
 Kite Pharma—Case Example and Recent Developments 34
 Adaptimmune—Overview 35
 Adaptimmune—Technology Platforms 36
 Adaptimmune—Case Example and Recent Developments 37
 Oxford BioMedica—Overview 38
 Oxford BioMedica—Technology Platform 39
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 Oxford BioMedica—Case Example and Recent Developments 40
 JCR Pharmaceuticals—Overview 41
 JCR Pharmaceuticals—Technology Platforms 42
 JCR Pharmaceuticals—Case Example and Recent Developments 43
C2A Profiles—Tier II 44
 Sangamo Therapeutics—Company Overview 45
 Sangamo Therapeutics—Value Proposition 46
 DiscGenics—Company Overview 47
 DiscGenics—Value Proposition 48
 TiGenix—Company Overview 49
 TiGenix—Value Proposition 50
 Bone Therapeutics—Company Overview 51
 Bone Therapeutics—Value Proposition 52
 Cell Medica—Company Overview 53
 Cell Medica—Value Proposition 54
 Mesoblast—Company Overview 55
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 Mesoblast—Value Proposition 56
 Cynata Therapeutics—Company Overview 57
 Cynata Therapeutics—Value Proposition 58
 Medipost—Company Overview 59
 Medipost—Value Proposition 60
 Anterogen—Company Overview 61
 Anterogen—Value Proposition 62
 Pharmicell—Company Overview 63
 Pharmicell—Value Proposition 64
C2A Profiles—Tier III 65
 Short Profiles of Digital Pathology Companies 66
Growth Opportunities and Companies to Action 70
 Transformation in the Industry Ecosystem 71
 Five Major Growth Opportunities 72
 Strategic Imperatives for Growth 73
The Last Word 74
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 Conclusion—Key Takeaways 75
 Legal Disclaimer 76
Appendix 77
 Abbreviation List 78
 List of Exhibits 79
The Frost & Sullivan Story 81
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Methodology
Research Goals, 360o Research

Objectives, and Scope
Research Approach Company

Identification
• Frost Sullivan 360o Companies to Action (C2A) research Profiling and

establishes the foundation for this project by detailing the current Benchmarking
market dynamics, key market participants, customer segments,
and evolving business opportunities.
Detailed Analysis and
• As a part of the C2A research process, Frost & Sullivan analysts Insights
review thousands of companies each year. Among these, they
identify market disruptors, including both start-ups and established
enterprises, that are capable of shaping the future through Strategic Review
implementation of best practices, visionary leadership, and
innovation in business models.
Quality Check
• The following research spotlights promising companies across the Release
stated transformational health market with an in-depth analysis of
their success stories and future growth potential.
Source: Frost & Sullivan
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Executive Summary
Return to contents
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Key Findings—Analysis of Companies to Action
• Globally, cell therapy is one of the leading segments of regenerative medicine, which, in turn,
is emerging as the most profitable segment of the biologics industry. Across cell therapy, stem
cell therapy and Chimeric Antigen Receptor T cell (CAR-T cell) therapies are among the most
promising segments.
• The recent launch of Novartis‘ Kymriah and Gilead‘s Yescarta is a case in point for the rising
research activities around novel cell therapies. Following their footsteps, bluebird bio (Bluebird)
is showing positive results for its upcoming gene therapy LentiGlobin™, which recently
received accelerated assessment by the Committee for Medicinal Products for Human Use
(CHMP) and the European Medicines Agency (EMA) for sickle cell disease.
• The cell therapy market is presently in a nascent stage and hence, is highly fragmented, with
over 500 small-to-mid segment companies across the market. Most of these participants focus
on specific therapeutic segments and resort to collaborative business models to support the
development and manufacture of their cutting-edge therapies. The larger participants on the
other hand, resort to acquiring these smaller niche companies, thereby gaining therapeutic and
technological expertise.
o Juno‘s acquisition by Celgene not only provides the latter with access to Juno‘s CAR-T cell
therapy but also puts Juno in a position to capture a third of the market share in CAR-T
drugs that target the CD19 protein.
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Key Findings—Analysis of Companies to Action (continued)
• Especially for cancer treatment, companies are focusing on the development of cell-gene
therapy combinations. With the success of Kymriah and Yescarta, market participants are
conducting several research studies so as to develop more of these combination therapies.
• Globally, North America (NA) is the leading market for cell therapy, owing to the first launch of
Kymriah and Yescarta in this region. Europe closely follows it, with the recent marketing
authorization of these therapies by EMA.
• However, Asia-Pacific (APAC) is slowly emerging as the key market, with countries such as
Japan and China making specific regulatory changes to support R&D across this segment.
Japan‘s recent introduction of Pharmaceutical and Medical Devices Act (PMD) act and Safety of
Regenerative Medicine Act (SRM) Act is a step toward establishing the country as a hotspot for
cell therapy research by attracting western companies to the region.
• Additionally, the country has also made changes in the approval process for these innovative
drugs by including the conditional approval process, which not only speeds up the time to
market, but also caters to the critical unmet medical needs in this segment.
• For the purpose of this study, Frost & Sullivan has identified some game-changing participants
across this highly dynamic cell therapy market. Some of the key therapeutic areas being
targeted include, musculoskeletal diseases, neurological conditions, cardiovascular diseases,
dermatology, and so on alongside oncology.
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Scope and Segmentation
Scope
Geographic Coverage Global
Study Period 2017–2025
Base Year 2017
Monetary Unit US Dollars
This research service provides comprehensive profiles of leading cell therapy participants, including a
brief overview of their value proposition and services portfolio. The study also emphasizes key strategic
collaborations across the cell therapy market, with a focus on their unique value proposition and product
portfolio and technology platforms.
The profiles are segmented into:
• Company overview
• Company value proposition
• Mergers and acquisitions and partnerships

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Key Questions Addressed in this Study
What are the key market opportunities in the global cell therapy market?
What is the impact of external industry environment on market growth?
Which are some of the unique participants that are introducing cutting-edge therapies in this
segment?
What are the strategic imperatives to be adopted by these participants to bring about this market
transformation?
What is the growth potential of this rapidly expanding cell therapy market?
What are some key success factors in the global cell therapy market?
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Companies to Action Overview
Return to contents
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Companies to Action Value Creators
Frost & Sullivan companies to action have the potential to disrupt current cell therapy market paradigms.
Frost & Sullivan Companies to Action (C2A) research identifies companies bringing disruptive
and innovative approaches to the market based on comprehensive market and technology
understanding.
C2A candidates stand apart from the crowd by Total Cell Therapy Market: C2A Value Creators,
introducing novel products, solutions, and/or Global, 2017
services that are well-recognized by the industry.
More importantly, they create extraordinary value
for their internal and external stakeholders by:
• Showcasing Visionary Leadership: A

powerful team of Thought Leaders with a
transformational vision of the future.
• Implementation of Best Practices: World-

class business processes with evidence of
positive outcomes for the company.
• Business Model Innovation: A revolutionary Business Model

combination of market offering, value Innovation
proposition, target audience, and revenue
stream that is sure to generate sustainable
business growth.
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Opportunities and Threats
Opportunities Threats
• Rising prevalence of • Lack of regulations supporting
chronic diseases such as timely approval of these
diabetes as well as cancer innovative therapies could
calls for innovative hamper market growth.
therapeutic development, • With most participants in the
thus propelling research small-to-mid sized biopharma
across cell therapy. segment, lack of specific
• Transition toward development and
personalized medicine is manufacturing expertise could
driving the growth of the impact the development of
cell therapy market. these novel therapies.
As a result, companies need to identify

industry partners that offer:
Revenue Growth Market Share Technology A Sustainable

Growth Innovation Business Model
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Cell Therapy Market Overview
Return to contents
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Market Definitions
Regenerative Medicine: It is a branch of translational research that focuses on the

replacement, engineering, and growing of regenerating cells, tissues, or organs using
biological processes similar to those normally used in the mammalian body.
Regenerative medicine also involves growing biological cells, tissues, and organs in the
laboratory; this is followed by the transplantation of these cells into the human body.
Cell Therapy (CT)

Cell therapy is the usage of living cells to replace or augment damaged or diseased cells and
tissues, induce an endogenous immune response, or deliver genetic and molecular therapies to
targets. Cells are also used for drug discovery, toxicity testing, and as biomarkers.
Gene Therapy (GT)

Gene therapy is the process of altering a genetic code to treat a diseased state.
Tissue Engineering (TE)

Tissue engineering involves the production and medical application of synthetic materials,
biomaterials, and scaffolds.
Small Molecules and Biologics

These are chemicals and cellular components that stimulate dormant or endogenous cells to recover
regenerative properties.
Image Source: Thinkstock Source: Frost & Sullivan
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Market Segmentation
Stem cell, cell, and gene therapies are being combined with immunotherapy to treat patients.
Total Regenerative Medicine Market: Market Segmentation, Global, 2017
Traditional Regenerative Medicine Market
Small Molecule
Cell Therapy Tissue Engineering Gene Therapy
Biologics
Used mainly for the

treatment of cancer: Highly
Cell-based specific T cells are created Immuno Cell- An increasing number
Immunotherapy that target tumor cells Gene Therapy of clinical trials are
using a cell-based
Combination therapy utilizing combination
antigenic approach
utilizing genetically therapies. The results
modified CAR-T cells are showing enhanced
Novel therapy that curative potential.
Stem cell utilizes stem cells from Stem Cell-
Therapy various sources to Gene Therapy
regenerate specific cell Genetically
or tissue types manipulated stem cells
to aid in targeted
therapy
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Cell Therapy Value Chain
Total Cell Therapy Market: Stakeholder Ecosystem, Global, 2017
Development
Sample Collection Transit and Transit Infusion
Manufacturing
Key Stakeholders
• CDMO/CRO
• Government • CDMOs* • CDMO • Patients
• Government
hospitals • CROs** • CROs • Government
agencies
• Clinical labs • Cold chain • Cold chain hospitals
• Private equity
• Private clinics suppliers suppliers • Clinical labs
• Venture capitalists
• Private clinics
• Academic institutes
• Cold chain • Research • Temperature • Controlled thawing

handling funding • Therapy
Key Services
control
• Diagnosis • Real-time • Scheduling, at -196ºC administration
• Treatment monitoring of modeling • Real-time • Electronic Data
• Monitoring post sample transit • Drug product monitoring of cell Capture (EDC) on
treatment • Automated order manufacturing therapy transport treatment outcome
updates and pick- • Storage and • Receipt and
up time stock monitoring inventory
• Data integration
Image Source: Thinkstockpictures.in
*CDMO—Contract Development & Manufacturing Organization; **CRO—Contract Research Organization Source: Frost & Sullivan
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Vendor Ecosystem
For the purpose of this study, Frost & Sullivan has profiled some of the game-changing biopharma companies
contributing majorly to this market.
Biopharma Research CDMOs/CROs Hospitals/Clinical Cold Chain

Companies Organizations/ Labs/Medical Suppliers
Academic Institutes Centers
Image Source: Company Web sites Source: Frost & Sullivan
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Impact of Key Market Influencers
Total Cell Therapy Market: Impact of Key Market Influencers, Global, 2018
Lack of Sufficient Funding
for Non-cancer Therapies Movement Toward Value-
Growing Market
based Payment Models
Consolidation Cancer accounts for almost 50%
of the global cell therapy trials, As healthcare systems are
With the growing number which renders the other looking for better treatment
of small to mid-segment segments short of capital value, cell therapy
participants, larger participants investment. As a result, less than manufacturers are adopting
aim at acquiring their 200 trials are active for other innovative payment models with
therapeutic and technology areas of cell therapy. insurance companies to offer
expertise, hence, paving way
an efficient and effective
for advanced novel cellular
payment model.
therapies.
Lack of Sufficient
Regulatory Support
Allogeneic Stem Cell
Therapies Being a nascent market,
emerging countries such as India
Companies across the globe and China lack the required
are focusing on adoption of regulatory support for propelling
allogeneic stem cells to Research and Development
address variability issues of (R&D). Also, developed countries
autologous cells, thus, have stringent policies for
supporting the development Positive impact on Negative impact on assessment of biologics, which
of stem cell based therapies. market revenue market revenue impacts the time to market.
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Top 3 Trends and Vendor Strategies
Industry Needs Vendor Strategy

• Although cancer is among the leading areas • Focus on development of combination therapies (gene
for cell therapy research, there is a general modified cell therapies) especially focusing on CTLA-4
rise in certain genetic and lifestyle-related antagonists with PD-1 blockers combination.
Expanding
conditions. • Participants such as Sanofi are investing in devising cell
Therapeutic
• The prevalence of diabetes type I is rising. therapies using human beta cells for treating type I
Portfolio Additionally, there is a surge in heart diabetes.
conditions such as heart failure, which is • Big pharma is looking at acquiring small-to-mid segment
propelling research across cell therapy. participants to gain therapeutic expertise in specific areas.
• Issues pertaining to lack of sufficient stem • Use of allogenic stem cells, as opposed to autologous
cell donors is impacting stem cell research. stem cells for haemopoitic stem cell transplantation and
Manufacturing • With the focus now shifting toward precision other stem cell based drug development, for better
and Technology medicine, there is an ardent need for scalability is imperative.
Innovation advanced small-scale manufacturing • Use single-use disposable bioreactors for improving
techniques for better efficacy and quicker manufacturing outcomes and improve the time to market
turnaround. of these therapies.
• A collaborative drug development approach • Undertake joint R&D strategies to develop innovative
Mergers & is required for risk sharing through co- T-cell therapies and stem cell based therapies.
Acquisitions development. • Leverage on state-of-the-art drug development and
(M&A), • Expanding funding opportunities for the cell manufacturing facilities and technology platforms of
Collaborations therapy market by means of venture development partners such as CDMOs.
& Partnerships capitalist funding or in-house funding is also • Lonza launched the world‘s largest cell and gene therapy
necessary to support ongoing R&D activities. manufacturing facility in 2017.
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Key Market Participants Aligned with Top 3 Trends
Total Cell Therapy Market: Key Market Participants Aligned with Top 3 Trends, Global, 2017
Single-use Manufacturing
Techniques Kymriah Approval for
Adult B-cell Lymphoma
Expanding
Manufacturing Facility Therapeutic
Expansions Portfolio Type I Diabetes
Manufacturing &
Technology
innovation
Cell and Gene Therapy Heart Failure

Manufacturing Facility
Mergers &
Acquisitions
(M&A),
Collaborations
& Partnership
One-time Gene
Replacement Therapy
Expertise in CAR-T cell Academic Collaborations Outsourcing Services

Therapy
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Cell Therapy—Vendor Landscape
While most small to mid segment players have a wide spread focus across therapy areas, the industry leaders
are observed to primarily focus on Oncology and musculoskeletal diseases.
Total Cell Therapy Market: Vendor Landscape, Global, 2018

Others
Cardiovascular and
Hematology
Rohto
Pharmaceuticals
Educell
Renova Neurological
Viacyte, Gamida
Therapeutics Disorders
Inc. Cell
Anterogen Cytopeutics
Healios JCR Pharma BioCardia
Reneuron Japan Cellectis
Athersys Regenerative
Promethera Athersys Medicine
Enzyvant Biosciences Corestem
Cynata Pharmicell ExcellThera Oncology
Mesoblast BrainStorm Cell
Therapeutics
Pluristem Therapeutics Takara Bio
Tigenix Therapeutics Athersys CellSeed
bluebird Atara
bio CreaGene
Cell
K-Stem Cell Medica
Kite TxCell
bluebird Pharma
bio Cyfuse
Juno Adaptimmune
Bone Biomedical Musculoskeletal
CBMG
Novartis Therapeutics Therapeutics
DiscGenics
Novartis Histogenics Regeneus
Tier I Tier II Tier III

Key: The list of companies is indicative and not exhaustive Source: Frost & Sullivan
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C2A Profiles—Tier I
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Juno Therapeutics—Overview
The company develops cell-based cancer immunotherapies, leveraging on its CAR-T cell and TCR-based
technology platforms.
Overview:
• The company works on developing innovative immunotherapies against several cancer types based on CAR-T cell and
TCR technology.
• Engineered T-cells are under investigation with an objective to overcome problems associated with resistance and immune
system evasion of tumor cells when subjected to conventional chemotherapeutic procedures.
Differentiators:
• The company‘s JCAR017 is a potentially best-in-class CD-19 targeted CAR-T therapy that is currently in pivotal trials
against relapsed/refractory Diffuse Large B-cell Lymphoma (DLBCL).
• Juno‘s recent acquisition by Celgene will provide it more financial and technological liberty to advance its current product
pipeline.
• Its collaboration with Thermo Fisher will also support Juno in attaining manufacturing capabilities for best-in-class CAR-T
therapies using Thermo Fisher‘s Cell Therapy Systems‘ (CTS‘) activation reagents.
Total Cell Therapy Market: Juno Therapeutics—Product Pipeline, Global, 2018
CD19: JCAR017 Key Focus Areas

CD19: JCAR014
Immunotherapy Non-small cell lung cancer
Technology Mesothelioma
WT1: JTCR016
Platform: Acute Myeloid Leukemia (AML)
CAR-T cell CD22: JCAR018 Acute Lymphoblastic Leukemia (ALL)
Diffuse Large B-cell Lymphoma (DLBCL)
TCR
MUC16: JCAR020 Non- Hodgkin's Lymphoma (NHL)
Ovarian cancer
ROR1: JCAR024
Key: TCR—T-cell Receptor technology Source: Juno Therapeutics Web site; Frost & Sullivan
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Juno Therapeutics—Technology Platforms
CAR-T cell Technology TCR Technology
These technologies
• CAR-T cells merge the • The technology makes use of
together provide
specificity of an antibody with genetically modified T cells to
Juno with a strong
the cytotoxic and memory enhance the tumor-killing activity of T
therapeutic
functions of T cells. cells.
expertise in a
• CAR-T cells are able to • TCRs are engineered to recognize a
majority of B-cell
recognize the proteins tumor-specific protein fragment/major
malignancies
expressed on the surface of histocompatibility complex (MHC)
including, ALL,
tumor cells. combination. The TCR technology
CLL*, and DLBCL;
• JCAR024, JCAR020, JCAR023, allows T cells to recognize tumor-
thereby, positioning
JCAR018, JCAR017, and specific proteins inside the cells.
it as a disruptive
JCAR014 are the key • JTCR016 is the leading product
participant in the
investigational drugs currently in candidate based on TCR technology.
market.
early-to-late stage development. It is undergoing clinical trials against
mesothelioma and AML among other
cancer types.
• After acquisition by Celgene, Juno Therapeutics‘ stock price surged by nearly 50% due to anticipation of strong growth in the future.
• The acquisition would benefit Celgene in rebuilding a strong immuno-oncology pipeline with 11 molecules from Juno in early-stage
development. In Q1 2018, Celgene invested $2.2 billion, an increase of 54% in R&D investment compared to the same period in 2017,
to support this newly acquired pipeline.
• Further, both companies are expected to mutually benefit from the deal, placing them in a strong position in the highly dynamic cancer
immunotherapy market, further enhancing the industry focus on precision medicine.
Image Source: Juno Therapeutics Web site Key: CLL—Chronic lymphocytic Leukaemia Source: Juno Therapeutics Web site; Investopedia; Frost & Sullivan
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Juno Therapeutics—Major Partnerships and Agreements
Total Cell Therapy Market: Case Study— Celgene’s Acquisition of Juno Therapeutics, Global, 2018
Deal
• Celgene acquired Juno Therapeutics in March 2018 at a value of $9 billion in one of the largest deals in this segment ever.
Value
• This acquisition positions Celgene at the forefront of cellular Immunotherapy, with Juno Therapeutics‘ lead candidate
JCAR017, which is expected to show potential peak sales of $3 billion post launch. Celgene also gains access to advanced
Benefits cellular manufacturing capabilities from Juno. With Celgene‘s products such as Revlimid set to lose market share, the
addition of Juno‘s pipeline is expected to accelerate Celgene‘s revenue diversification in 2020 and beyond.
• On the other hand, Juno can compete with industry giants such as Novartis and Gilead with support from Celgene.
• This acquisition is being considered as a ‗smart‘ acquisition by industry experts and the deal is expected to revolutionize
cancer treatment while promising huge profits for both companies.
• While competitors have been venturing into this arena for a while, Juno‘s wide spread focus on B-cell lymphoma and other
Future cancers, including leukemia, places it in a stronger market position.
Potential • Industry experts estimate that Juno will capture a third of the market share in CAR-T drugs that target the CD19 protein
across several types of cancers such as DLBCL, primary mediastinal B-cell lymphoma, mantle cell lymphoma, chronic
lymphocytic leukemia, and acute lymphoblastic leukemia, thereby positioning it in line with other market leaders in this
segment.
Total Cell Therapy Market: Juno Therapeutics—Major Partnerships and Agreements, Global, 2018
Partner Date Description
Eli Lilly, December Juno and Eli Lilly entered into a licensing agreement for a program in multiple myeloma using Gamma Secretase
FHCRC and 2017 Inhibitors (GSIs) combined with B-cell maturation antigen (BCMA) – directed CAR-T Cells. Through this agreement,
OncoTracker Juno will acquire a license to the therapy LY3039478 (GSI) which has been studied in 411 patients and healthy
volunteers. The licensing agreement between the parties provides Juno with exclusive rights to intellectual property
within the field of combinations of GSIs and BCMA-directed engineered T cells.
Thermo Fisher December This is a seven-year non-exclusive licensing and supply agreement with Thermo Fisher, allowing access to the latter‘s
2017 CTS activation reagents, to support the manufacturing of CAR-T cell therapies.
Key: FHCRC—Fred Hutchinson Cancer Source:https://www.investors.com/news/technology/why-celgenes-rumored-acquisition-of-juno-actually-makes-sense/;
Research Centre http://fortune.com/2018/01/22/celgene-juno-deal-analysts-respond/;Juno‘s press releases; PR newswire; Frost & Sullivan
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bluebird bio—Business Overview
The company develops transformative gene therapies, targeting several genetic disorders and cancer types.
Overview:
• The company enjoys a broad therapeutic potential by leveraging on its lentiviral-based gene therapy, gene editing
capabilities, and T cell engineering (CAR-T and TCR) and aims at building an integrated product platform.
• With operations in the US, the UK, and Switzerland, the company generated over $35.4 million in revenue from licensing,
royalties, and collaboration in 2017.
Differentiators:
• Its lead product bb2121, is considered as one of the most promising therapies for multiple myeloma with a high response
rate and safety profile.
• The grant of breakthrough therapy designation for Lenti-D for the treatment of Cerebral Adrenoleukodystrophy (CALD) by
the Food and Drug Administration (FDA) in early 2018 has provided a strong boost to the company‘s pipeline.
• The use of unparalleled sequence targeting and gene editing efficiencies through homing endonuclease and megaTAL-
based gene editing technologies stands out in comparison with other competing products.
Total Cell Therapy Market: bluebird bio—Technology With Product Pipeline, Global, 2018
Lenti-D Drug Product

Homing
Endo- Key Focus Areas
nucleases LentiGlobin Drug Product • Cerebral Adrenoleukodystrophy
Technology
Hybrid (ALD)
Platform for bb2121
megaTALs • Transfusion dependent
Gene
β-thalassemia
Editing TAL
bb21217
Arrays • Severe sickle cell disease
• multiple myeloma
BCL11a shRNA(miR)*
Source: bluebird bio Web site; Frost & Sullivan
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bluebird bio—Technology Platform
Gene Editing Technology

Academic collaborations
for discovery and
CRISPR/Cas9/ development of advanced
Talen/ gene therapy products
CAR – T Cell/
Zinc Finger TCR
Homing Homing Nucleases
Endonucleases Technology
Endonucleases
megaTAL
Homing
Endonucleases Lentiviral CAR-T cell therapies
• These are compact Vectors
proteins that effectively with higher specificity
wrap around and ‗saddle‘ in anti-tumor activity
the required • Fusion proteins are created
Deoxyribonucleic acid with by combining homing
TAL Arrays endonucleases with modular
(DNA) molecule with
correct identification and • These can be DNA binding domains of
paired with Therapy bb2121, targeting B cell
cleavage. TALENs and TAL arrays.
maturation antigen, showed
• Application: DNA homing • Characteristics: Advanced
endonucleases to sustained promise for multiple
recognition chemistries DNA recognition is possible
create hybrid myeloma
can be effectively with extreme precision and
reprogrammed to work on megaTALs. efficiency.
the required DNA target • These are the most efficient
sequences. genome editing ‗machines‘,
Image Source: bluebird bio Web site created so far.
TALEN—Transcription Activator-like Effector Nuclease Source: bluebird bio Web site; Frost & Sullivan
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bluebird bio—Major Partnerships and Agreements
Total Cell Therapy Market: Case Study—Celgene’s Partnership With Bluebird, Global, 2018
• It is a co-development and co-promotion partnership for bb2121, the lead oncology candidate for relapsed and
Deal refractory multiple myeloma.
Value • As per the deal, Bluebird is set to receive up to $70 million in milestone payments for the first indication, with
additional payments for the second indication alongside royalty payments.
• As a part of the deal, Bluebird will receive milestones and royalties for the product on ex-US sales.
• Additionally, this collaboration takes Bluebird a step ahead in building a strong and integrated oncology franchise
Benefits by acquiring extensive research and development capabilities from Celgene.
• Additionally, Bluebird is investing in in-house manufacturing facility expansions alongside external deals with
Brammer Bio, Novasep, and so on to support product portfolio expansion.
• As per the deal, both the companies will equally share the costs and profits gained from bb2121, across the US
market. After the deal Bluebird experienced a surge of 2.6% in pre-market trade, reflecting a positive impact of the
Future deal.
Potential • With bb2121 receiving a breakthrough therapy designation from the FDA and PRIME designation from the European
Medicines Agency (EMA), it has established a strong potential as a cellular immunotherapy for multiple myeloma,
which is among the incurable diseases, currently. with limited therapeutic options.
Total Cell Therapy Market: Bluebird—Major Partnerships and Agreements, Global, 2018
TC Pharma December Bluebird and TC BioPharm collaborated to develop gamma delta CAR-T cell product candidates for cancer
2017 immunotherapy. This deal combines C BioPharm‘s leading gamma delta T cell capabilities with Bluebird‘s therapy
expertise across all stages of CAR-T and gene therapy product development, in order to develop next-generation liquid
and solid tumor product candidates.
Duke March 2017 The company collaborated with Duke University‘s Robert J. Margolis, MD, Center for Health Policy, with an aim to develop
University strategies for a value-based payment approach for its transformative gene therapies.
Apceth December This is a strategic manufacturing agreement for the commercial production of Lenti-D product candidate for Transfusion
Biopharma 2016 Dependent β-Thalassemia (TDT).
Source: bluebird bio Web site; Frost & Sullivan
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Kite Pharma—Overview
It is a clinical-stage biopharmaceutical company involved in the development and commercialization of novel
cancer immunotherapy products.
Overview:
• The company is developing engineered autologous cell therapy product candidates, leveraging on its TCR and CAR-T cell
technology platforms against solid and hematological tumors.
• Its recent acquisition by Gilead Sciences underlines the prospects its technology and product pipeline and also strengthens
the financial and technological support for its product pipeline.
Differentiators:
• Kite Pharma‘s YescartaTM is the first CAR-T cell therapy approved by the FDA for the treatment of adults with refractory
large B-cell lymphoma post two or more lines of systemic therapy. Yescarta‘s comparatively lower pricing will provide a
competitive edge against Novartis‘ Kymriah (against the same indication in pediatric patients).
• It has a strong collaborative business approach and its partners include Fosun Pharma, Daiichi-Sankyo, Amgen,
Genentech, Cell Design Labs, Adimab, and bluebird bio, among others.
Total Cell Therapy Market: Kite Pharma: Product/ Technology Depiction, Global, 2018
Axicabtagene ciloleucel Key Therapy Focus

Immunotherapy
• Lymphoma:
Technology KTE-C19 o Diffuse Large B-Cell Lymphoma (DLBCL),
Platform: Primary Mediastinal B-cell Lymphoma (PMBCL)
CAR-T cell KITE-585 (anti-BCMA) o Transformed Follicular Lymphoma (TFL)
o Indolent Non-Hodgkin‘s Lymphoma (NHL)
TCR o Mantle Cell Lymphoma (MCL)
KITE-718 (MAGE A3/A6)
• Solid tumors
• Adult and pediatric ALL
• Multiple myeloma
Source: Kite Pharma Web site; Frost & Sullivan
PA16-52 32
Kite Pharma—Key Technology/ Product/Services
The company leverages on its two main technological platforms: CAR-T and TCR to develop innovative
therapies against several cancer types.
Therapy Development Procedure

White blood cells collection—T cell isolation and activation—T cell engineering— T cell expansion—T cell
infusion
CAR-T Cell Therapy TCR Cell Therapy TCR Technology:

• Makes use of genetically modified T
• CAR-T cell therapy is composed of two distinct • TCR-engineered T cells can identify
cells and
to enhance bind to
tumor-killing
functional components: first component has an cancer-associated peptides on the
activity of T surface
cells of tumor
antibody fragment (allows target recognition on cells and provide subsequent signals for cytokine
• TCRs are engineered to recognize a
cancer cells); second component provides signals release, proliferation, and tumor-specific
cytolytic activity.
protein
for T cell activation fragment/MHC combination. Allows
• The company strengthened its TCR
T cells product
to recognize platform
tumor-specific
• Kite Pharma is leveraging on the CAR-T platform to by acquiring T-Cell Factory B.V. (private
proteins Dutch
on the inside of cells.
develop lead product candidates, which includes company) in 2015. The acquisition
• JTCR016 is the leadingKite
enabled to
product
axicabtagene ciloleucel and KTE-C19. leverage on its TCR-GENErator TM
candidate discovery
based on TCR platform
technology
undergoing
for rapid and high-throughput clinical trialsof
identification against
• The company has strengthened the strategic mesothelioma and AML among
TCR-based product candidates.
support to its CAR-T cell platform through multiple other cancer types.
partnerships with Amgen, Genentech (axicabtagene • To further advance the TCR product pipeline, the
ciloleucel and atezolizumab), Cell Design Labs company has signed strategic partnership
(acquired in 2017), Adimab, and Tel-Aviv Sourasky agreements with a number of companies, including
Medical Center. Daiichi-Sankyo (MAGE-A3/A6), Fosun Pharma, and
bluebird bio among others.
PA16-52 33
Kite Pharma—Case Example and Recent Developments
Total Cell Therapy Market: Case Study—Gilead’s Acquisition of Kite Pharma, Global, 2018
• Gilead sciences acquired Kite Pharma at a huge sum of $11.9 billion, in one of the biggest deals across the global
Deal
biopharmaceutical industry.
Value
• As a part of the deal, Kite Pharma became a wholly owned subsidiary of Gilead Sciences.
• Gilead gains access to Kite‘s cutting-edge cell therapy, Yescarta, thereby, establishing itself as a leader across this market
and competing directly with Novartis‘ Kymriah.
Benefits
• The deal enables Kite to leverage on Gilead‘s expertise in shortening drug development time and simplifying complicated
manufacturing processes for CAR-T cell therapies.
• Gilead (Kite Pharma) enjoys a strong competitive advantage with its therapy, Yescarta, in terms of cost. Yescarta‘s per
Future therapy cost of $373,000 is relatively cheaper than Kymriah, which costs around $475,000 per therapy.
Potential • This deal will provide a greater market reach for Kite Pharma by utilizing Gilead‘s vast presence in the global
biopharmaceutical market.
Total Cell Therapy Market: Kite Pharma—Major Partnerships and Agreements, Global, 2018
National May 2018 Kite Pharma leased a 117,000 square-foot site in Hoofddorp, Amsterdam, from National Cancer Institute to support the
Cancer development of cell therapies in Europe. It will enable Kite to efficiently manufacture and improve the accessibility of cell
Institute therapies in Europe.
Sangamo February It is a worldwide collaboration to use Sangamo‘s Zinc Finger Nuclease (ZFN) technology platform to support Kite Pharma
Therapeutics 2018 in the development of next-generation ex-vivo cell therapies for autologous and allogenic use in oncology.
Cell Design December Kite Pharma, along with its parent company Gilead Sciences, entered into a definitive agreement to acquire Cell Design
Labs Inc. 2017 Labs Inc., in a deal approximately valued at $567 million.
Fosun January A joint venture (Fosunkite) has been established to develop, manufacture, and commercialize axicabtagene ciloleucel in
Pharma 2017 China, with further prospects of including additional products (including two TCR product candidates). Kite Pharma set to
receive $40 million upfront payment, in addition to milestone payments, profit shares, and royalties upon the
commercialization of acicabtagene ciloleucel.
PA16-52 34
Adaptimmune—Overview
It focuses on the development and commercialization of multiple T cell therapies against several cancer
types.
Overview:
• The company makes use of its proprietary SPEAR T-cell platform that enables identification of cancer targets, genetic
engineering of T-cell receptors, and subsequent development of therapeutic candidates.
• The company has achieved major manufacturing milestones and is on track to become the first fully integrated TCR T cell
therapy company.
Differentiators:
• Its NY-ESO SPEAR T-cell therapy for soft tissue sarcoma received breakthrough therapy and orphan drug designation by
the FDA. Additionally, the therapy was also granted PRIME designation by the EMA for synovial sarcoma, thus, facilitating
robust data collection, high-quality market authorization, and speeding up of the medicine evaluation procedure.
• Adaptimmune‘s recent collaboration with Catapult allows it to receive a consistent vector supply beyond 2020.
• Currently, its manufacturing facility is equipped to deliver cell therapies to about 300 patients annually and shows potential
for expansion to meet the therapeutic needs of over 1,000 patients per year.
Total Cell Therapy Market: Adaptimmune—Product Pipeline, Global, 2018
Key Focus Areas

MAGE-A10 TCR
Immunotherapy Non small cell lung cancer
Technology AFP TCR Melanoma and bladder cancer
Platform: Hepatocellular cancer
SPEAR T-cell MAGE-A4 TCR Synovial sarcoma
therapy Myxoid round cell liposarcoma
NY-ESO TCR Ovarian cancer
Multiple myeloma
Key: SPEAR—Specific Peptide Enhanced Affinity Receptor Source: Adaptimmune Web site, Frost & Sullivan
PA16-52 35
Adaptimmune—Technology Platforms
SPEAR T-cell Technology
Identify and Validate Identify Preclinical Safety and Clinical

Engineering
Targets TCR Efficacy Testing Testing
• Identification and • Isolation of T cells and TCRs • Minimizes cross reactivity or potential off-target
assessment of targets • TCRs engineered to enhance binding
• Mass spectrometry cell affinity • Additionally, supports human clinical testing by
confirms surface • Generation of a T-Cell therapy, assessing if the affinity-enhanced engineered TCR is
expression and by engineering sequences able to bind to normal cells and whole blood samples
expression on tumor within a T-Cell, to target
cells cancer by functioning with the
• Low-risk targets patient‘s own immune system
selected for TCR
therapy
• So far, the technology platform has helped identify over 25 intracellular target peptides in cancer cells,
while studies across 12 more such peptides are ongoing.
• Owing to its strong therapeutic focus, the company reported a revenue increase of 166%, reaching
$37.8 million in 2017. Additionally, Q1 2018 revenues were recorded at $8.2 million.
• The company continues to make robust investments in R&D activities, with Q1 2018 R&D expenses
reaching $25.7 million.
Source: Adaptimmune Web site; Investopedia; Frost & Sullivan
PA16-52 36
Adaptimmune—Case Example and Recent Developments
Total Cell Therapy Market: Case Study—Collaboration With GSK, Global, 2018
• It has entered into a strategic collaboration and license agreement for the development, manufacture, and commercialization
Deal of TCR therapeutic candidates.
• A highly versatile program (and platform)—NY-ESO SPEAR T-cell program—targets six different cancer types.
• TCR therapy targets the NY-ESO peptide, present across several cancer types.
Solution
Approach • The deal aims to engineer TCR to produce high-affinity T cells that are resistant to suppression and can break cancer
immune tolerance.
• US FDA has awarded it an orphan drug designation and breakthrough therapy designation against soft tissue sarcoma and
EMA has given it PRIME regulatory access for the program against synovial sarcoma in phase 1/2 clinical trials against
Current multiple indications.
Progress • Durable antitumor responses have been observed in patients with synovial sarcoma during clinical trials.
• As of 31 December 2017, Adaptimmune has received development milestones of $49.3 million as part of its agreement for
the product development program. Initially, it received an upfront payment of $42.1 million in June 2014.
Total Cell Therapy Market: Adaptimmune—Major Partnerships and Agreements, Global, 2018
Cell and Gene February Agreement with Cell and Gene Therapy Catapult for production of vectors in the UK. The agreement
Therapy 2018 enables the company to ensure vector supply for its product pipeline beyond 2019, with the initiation of its
Catapult own vector manufacturing capability at Catapult‘s facility.
MD Anderson September Adaptimmune entered into a multi-year strategic alliance to advance the development of immunotherapies
2016 (adoptive T cell therapies) against multiple types of cancer.
Bellicum December This is a strategic collaboration to evaluate, develop, and commercialize next-generation T cell therapies.
Pharmaceuticals 2016 It also involves the evaluation of Bellicum‘s GoTCR technology with Adaptimmune‘s affinity-optimized
SPEAR T-cells to create enhanced TCR product candidates.
Source: Adaptimmune Web site, Frost & Sullivan
PA16-52 37
Oxford BioMedica—Overview
It is focused on developing lentiviral vectors to support cell therapy bioprocessing and R&D activities.
Overview:
• With a broad-ranging therapeutic potential, Oxford BioMedica is a pioneer in cell and gene therapy development. It has a robust
pipeline of products, including both proprietary products as well as those developed under collaborations with other industry leaders.
• The company makes use of its proprietary LentiVector platform (gene delivery system) that enables the development of in vivo and ex
vivo products targeting a wide range of serious diseases with unmet medical needs.
Differentiators:
• Oxford BioMedica‘s proprietary LentiVector® platform exhibits superior abilities over earlier generation vectors in being able to
overcome safety and delivery challenges. The platform finds applications as a research tool for transgenesis, stem cell manipulation,
and gene discovery, among others.
• The company is the sole supplier of lentiviral vectors for Novartis‘ Kymriah, making it the first lentiviral-enabled product approved in the
US and awaiting EU approval.
• It has received seven regulatory approvals for its pipeline products for clinical studies in the US and for ocular indications and
Parkinson‘s disease in Europe.
• Additionally, it also possesses a Manufacturer/Importer license granted by the UK MHRA for commercial production and supply of
lentiviral vectors.
Total Cell Therapy Market: Oxford BioMedica—Product Pipeline, Global, 2018
OXB-202
Key Focus Areas
Lentiviral- OXB-302
based Gene Corneal graft rejection
Delivery OXB-201 Multiple cancer
System: Ophthalmology (Wet AMD)
LentiVector® AXO-LENTI-PD Central Nervous System (CNS) (Parkinson’s
disease)
Platform SAR422459 Ophthalmology (Stargardt disease)
Usher syndrome type 1B
SAR421869
Source: Oxford BioMedica Web site; Frost & Sullivan
PA16-52 38
Oxford BioMedica—Technology Platform
LentiVector® Platform
In vivo program • A lentiviral-based gene delivery system Ex vivo program
• Direct administration of vectors that resolves safety and delivery • Produce lentiviral vector
into target organ in vivo challenges in novel therapy development encoding target gene
• Preference for lentiviral vectors • T cell isolation followed by
• Capable of taking up large therapeutic
over Adeno associated virus transduction
(AAV) vectors
payloads of up to 9kb • Infusion back to patients
• Potential for single-application • Supports vector administration and • T cell multiplication and cancer
treatment that gives permanent generation on a large scale cells targeted and destroyed
efficiency • Also helps make permanent modifications • E.g., Kymriah
• E.g., OXB-102 in dividing and non-dividing cells
Impact
• The company anticipates a revenue potential of over $100 million through its supply agreement with Novartis and an additional
$105 million through the collaboration and licensing agreement with Bioverativ for its hemophilia gene therapies.
• With its ongoing collaborative business model, the company showed a gross income increase of 28% in 2017 over 2016 to reach
$52.08 million.
• In order to support its ongoing collaborations, the company has a full suite of laboratories and bioprocessing units. Its recent
collaborations with Novartis and Orchard Therapeutics largely contributed to the growth in gross income.
Image Source: Oxford BioMedica Web site Source: Frost & Sullivan
PA16-52 39
Oxford BioMedica—Case Example and Recent Developments
Total Cell Therapy Market: Case Study—Supply Agreement With Novartis, Global, 2018
• Collaborated with Novartis for the commercial and clinical supply of lentiviral vector for its lead product Kymriah
Deal
and other undisclosed CAR-T products.
Value
• The company is set to receive an amount of over $100 million over the next three years.
Deal • The deal provides sole ownership to Oxford BioMedica for the manufacturing of the lentiviral vector for the
Details product and also gives eligibility for sales-based royalties for Kymriah.
• The deal gives Oxford BioMedica an opportunity for the commercial application of its LentiVector platform.
Benefits • This has in turn paved the way for multiple partnering opportunities for Oxford BioMedica‘s platform through
several high-value deals with Bioverative, Axovant Sciences, and so on.
Total Cell Therapy Market: Oxford BioMedica—Major Partnerships and Agreements, Global, 2018
Axovant June It got into an exclusive worldwide licensing agreement to develop and commercialize OXB-102 for Parkinson‘s disease by
Sciences 2018 making use of the LentiVector platform, for a value of $842.5 million.
Bioverativ February The two companies agreed to a collaboration and licensing agreement for the development and manufacturing of lentiviral
2018 vectors for the treatment of hemophilia. The deal provides Bioverativ with a license to use Oxford BioMedica‘s LentiVector-
enabled technology and provides access to its industrial-scale manufacturing technology.
Multiple October Oxford BioMedica has entered into a two-year agreement worth $2.7 million for a collaborative project to explore and apply
Partners 2017 novel advanced technologies to expand its LentiVector platform to deliver high-quality vectors for clinical and commercial use.
These partners include Cell and Gene Therapy Catapult, Stratophase Ltd, and Synthace Ltd, co-funded by Innovate UK.
Innovate June The agreement is for the commercial and clinical supply of lentiviral vectors in the development of Novartis‘ Kymriah. It
UK 2017 includes $10 million as upfront payment and potential payment of $100 million to Oxford BioMedica over three years.
Source: Oxford BioMedica Web site; Frost & Sullivan
PA16-52 40
JCR Pharmaceuticals—Overview
It is focused on the discovery, development, and manufacturing of allogeneic stem cell therapies.
Overview:
• Founded in 1975, the company aims to strengthen its position in the regenerative medicine market, building on the success
of TEMCELL; highlighted recently by plans to advance the DPC platform in collaboration with Teinjen.
• It is engaged in the R&D, manufacturing and sales of biotherapeutics and regenerative medicines, primarily in Japan. Its
major products include GROWJECT (recombinant human growth hormone) and TEMCELL (MSC* therapy).
Differentiators:
• TEMCELL, licensed by the company for Japan, is the country‘s first allogeneic stem cell therapy and the world‘s first human
MSC-based product against acute Graft vs. Host Disease (GvHD). The product generated sales of over $1.9 million in the
last two fiscal years.
• Cell Processing Center (CPC) is an exclusive facility (200.71 m2 floor space) dedicated to the manufacturing of preclinical
and clinical trial material for regenerative medicinal products. It is a large-scale production facility supported by an ultra-low-
temperature logistics system. The company possesses the largest cell culture facility in Japan, in Seishin.
• SDDU (built in collaboration with MEDIPAL) for transportation and storage (for more than 10 days) is capable of maintaining
ultra-low temperatures (< -150 oC), which ensures stable quality and on-time delivery of the product to the clinical site.
Total Cell Therapy Market: JCR Pharmaceuticals—Product Pipeline, Global, 2018
Stem Cell
Therapy: JR-031 Key Focus Areas
Allogeneic
Acute GvHD
stem cell
Epidermolysis bullosa
therapy JTR-161/JR-161 Acute Cerebral Infarction
Key: SDDU—Specialty Drug Distribution Unit; DPC—Dental Pulp Stem Cells, MSC—Mesenchymal Stem Cells Source:JCR Pharma Web site, Frost & Sullivan
PA16-52 41
JCR Pharmaceuticals—Technology Platforms
MSC Therapy Platform

MSC manufacturing process TEMCELL
MSC—Migration Ability product in focus: TEMCELL Functional Properties
Bone Product Low immunogenicity
MSC activation by Marrow Dose
Isolation Verification • Universal administration
secretions at the site
• Evasion of patient‘s
of inflammation within
allogeneic immune response
blood vessels
Donor
Cell
Bank Immuno-modulation
MSC migration and Donor Screening
• Suppression and T-
accumulation at the site (acceptance test lymphocyte induction
and process-
of inflammation control tests) • Secretion of anti-inflammatory
molecules
Intermediate
Tests (safety Homing to the sites of
Therapeutic effect— tests)
inflammation
secretion of various • Migration to inflammation site
anti-inflammatory
substances Release
Tests
• The company is set to construct a second Clinical Trial Material Manufacturing Center, owing to its expected increase in the number
of drug candidates and subsequent Active Pharmaceutical Ingredient (API) demand. Subsequently they aim at future global
expansion which will be contributed by their objectives of expanding target indications for its DPC platform.
• Revenue from TEMCELL increased by a staggering 124% in FY2017 compared to FY2016.
• TEMCELL might face competition from Novartis‘ Ruxolitinib (in phase III trial currently) in the future
Image Source: Company Web site Source: Company Web site; Frost & Sullivan
PA16-52 42
JCR Pharmaceuticals—Case Example and Recent Developments
Total Cell Therapy Market: Case Study—Codevelopment Agreement With Teijin, Global, 2018
• JCR has entered into a co-development and licensing agreement for JTR-161/JR-161 to treat acute cerebral
Program
infarction (stroke).
• It is an allogeneic regenerative medicine product and makes use of dental pulp stem cells (DPSC).
Solution
• Mass culturing methods are used for DPSCs.
Approach
• Intravenous injection is the Intended mode of administration.
• JCR will undertake manufacturing of the product for clinical trials. Upon approval, it will oversee the manufacturing
and supply of the commercial product.
Program
• Teijin will be responsible for the conduction of clinical and non-clinical studies and filing of the marketing approval
Highlights
application. It will also be responsible for handling product sales in Japan after approval.
• Clinical trials are scheduled to start in 2018.
Total Cell Therapy Market: JCR Pharmaceuticals—Major Partnerships and Agreements, Global, 2018

Teijin July 2017 JCR and Teijin entered into a co-development and licensing agreement for JTR-161, an allogeneic regenerative
medical product that makes use of DPSC for the treatment of acute cerebral infarction (stroke) in Japan. JCR is
set to receive upfront payments with potential milestone payments and royalties as a part of the deal, with clinical
development set to start in 2018.
Source: Company Web site; Frost & Sullivan
PA16-52 43
C2A Profiles—Tier II
Return to contents
PA16-52 44
Sangamo Therapeutics—Company Overview
It is focused on the development of genomic therapies by making use of technology platforms that include
genome editing, gene therapy, gene regulation, and cell therapy.
Company Snapshot Business Profile
Business
Gene and cell therapeutics
Ownership Public Segments
Year Founded 1995

Efficient multiplatform approach to
Core Business
2017 Revenue gene therapeutics supported by
36.6 Model
($ Million) strategic partnerships and financing
Sandy Macrae
(President & CEO) Notable Pfizer, Bioverativ, Shire, Kite
Partners Pharma
Edward Conner
Key People
(SVP & CMO)
• Raised over $12 million in grants
and collaborations in the first
Kathy Yi
quarter of 2018 to support its
―Advancing (SVP & CFO)
product pipeline
genomic Market • Additionally, a sum of $230
Number of
therapies to Employees
182 Position million raised through public
offering, mostly to support its
transform individual and collaborative
Headquarters California, US
patients’ lives‖ therapeutic pipeline
Geographic
US, EU
Presence
Source: Sangamo Web site; Frost & Sullivan
PA16-52 45
Sangamo Therapeutics—Value Proposition
ZFN Gene Editing Technology Value Proposition Target Therapy Segment

• Makes use of Zinc Finger Nuclease (ZFN) gene editing • A highly efficient multi-platform • Inherited metabolic disorders
technology to knockout or insert therapeutic genes into approach to gene-based • CNS disorders
genomes therapeutics that helps address
• The company shows capabilities across gene therapy and • Oncology
unmet medical needs in • Immunology
genome editing, which support production of liver cells
derived therapeutic proteins for developing new type of monogenic diseases
cell –based therapies
Key Differentiators
• It has received an ODD and rare pediatric disease designation from the FDA for its SB-318 in vivo genome editing program for MPS I.
The FDA designation speeds up the medicine evaluation procedure and positions the product for cost-effective commercialization in
the US.
• Its acquisition of TxCell has further fortified its position in the immunotherapy segment with respect to CAR-T cell therapies.
• It has a robust therapeutic and non-therapeutic partnership profile, with collaborative programs in development with key industry
participants such as Pfizer (hemophilia A), Kite Pharma (oncology research), and Bioverativ (inherited blood disorders).
Business Model Future Growth Strategy

• The company focuses on developing innovative therapies for rare disorders • With the acquisition of TxCell, Sangamo has positioned itself at
by leveraging on its multiple technology platforms in gene and cell the forefront of immunotherapy research.
therapeutics. • The synergy between the company‘s gene editing capabilities
• Its strong collaborative business model with pharma leaders and strong and TxCell‘s Treg biology expertise, is set to unlock innovative
financing supports the expansion and advancement of its product portfolio. therapies for autoimmune diseases, with the first CAR-Treg trial
• Partners: It has collaborated with Pfizer to develop the ZFP gene therapy for expected to start in 2019, followed by ZFN-edited CAR-Treg
Amyotrophic Lateral Sclerosis (ALS) 7 Frontotemporal Lobar Degeneration programs.
(FTLD). Sangamo received a $12 million upfront payment with eligibility to • Furthermore, Sangamo expects to be the first company to
milestone payments up to $150 million and other royalties from sales.as a develop and commercialize a CAR-Treg candidate and is
part of the deal. planning to expand its manufacturing capabilities by
constructing a new state-of-the-art cGMP manufacturing facility
in Brisbane, CA to support these products.
Key: ZFP—Zinc Finger DNA-binding Proteins; MPS I—Mucopolysaccharidosis I; ODD—Orphan Drug Designation;
Treg—Regulatory T cells Source: Sangamo Web site; Frost & Sullivan
PA16-52 46
DiscGenics—Company Overview
It has developed a unique technology platform for stem cell therapy development against disc degenerative
disorders.

Business
Degenerative spinal disorders
Ownership Private Segments
Strong patent profile with financial

Year Founded 2007
Core Business and technical backing through
Model strategic collaborations for private
2017 Revenue funding
-
($ Million)
Flagg Flanagan Notable

(Chairman and CEO) CTI, CCRM, GE Healthcare
Partners
Bob Wynalek
(President, COO and • Recently received an amount
Key People
Director) of $14 million in a series B
Kevin T. Foley Financing from a new investment
(Chief Medical partner Mitsubishi UFJ Capital
―Improving Officer, Director) co. Ltd., alongside existing
Market funding partners
lives through Number of
11–20 Position • In April 2018, announced the
Employees
innovation‖ successful treatment of first
Salt Lake City, Utah, patient in US clinical trials of
Headquarters
US injectable Discogenic Cell
Geographic Therapy (IDCT) for degenerative
US disc disease
Presence
Source: DiscGenics Web site; Frost & Sullivan
PA16-52 47
DiscGenics—Value Proposition
Patented Allogenic Cell Culture Manufacturing Platform Value Proposition Target Therapy segment
• Hypothesised discogenic cells manufactured using the • Focus on addressing unmet • Degenerative Spinal Disorders
patented manufacturing technology; posses key needs of curative treatments in (DDD)
regenerative and anti-inflammatory properties of MSCs spine disorders while developing
alongside the ability to produce molecules that support permanent and cost-effective
structure of the disc solutions to lower back pain
• Possible to dose manufacture in thousands from a single related to Degenerative Disc
donor using this technology Disease (DDD)
Key Differentiators
• The company has a patented an allogeneic cell culture manufacturing technology, which is used to isolate cells from donated adult
human intervertebral disc tissue that are then converted into discogenic cells.
• These do not require adaptational tests in the disc environment compared to other unspecialized cells, while having the regenerative,
anti-inflammatory properties and molecule production for disc support and structure.
• With over 7 million patients suffering from DDD in the US alone, IDCT is considered as an extremely viable alternative to existing DDD
treatments, which include opioids and surgery, with a significant market opportunity.

• The company holds a strong patent profile with 24 patents • The company is looking to expedite its product development
registered globally and has been able to garner strong financial program and market expansion through strong strategic support
backing from investors by raising a total of $21.7 million from and multiple collaborations.
private funding for its IDCT program. • In early 2018, it hired Cryoport as its logistics partner for IDCT
therapy.
• Partners: It has collaborated with CCRM and GE Healthcare to • It also has an agreement with GE Healthcare and CCRM that
support its objectives of scaling up and optimization of IDCT provide manufacturing support for its IDCT program.
manufacturing. • DiscGenics has joined MedCision‘s ThawSTAR Clinical Early
Adopter program to support cell handling risks during thawing
procedures for its IDCT therapies.
Source: DiscGenics Web site; Frost & Sullivan
PA16-52 48
TiGenix—Company Overview
It leverages on its adipose-derived stem cell platform to develop novel therapies against inflammatory and
autoimmune disorders.

Business
Ownership Private Regenerative biologics
Segments
Year Founded 2000 Robust technology platform allows

novel therapy development; strong
2017 Revenue Core Business Intellectual Property (IP) protection
1.03 Model through global patent licensing;
($ Million)
strong strategic partnerships and
Dr. Claudia market expansion
D’Augusta
Notable
(CFO & General Takeda, Mesoblast, Lonza
Partners
Manager)
Dr. Wilfried
Dalemans
Key People • Was recently acquired by
(Chief Technical
Takeda, making it a wholly
Officer)
owned subsidiary of Takeda
Dr. Mary Carmen
―Turning hope Diez • Acquisition also extends their
(VP, Medical Affairs & Market existing collaboration to develop
into reality‖ Commercialisation) Position and commercialize Cx601for
Number of perianal fistulas
89
Employees • After acquisition, product Alofisel
recently received commercial
Headquarters Leuven, Belgium manufacturing license in the EU
Geographic
US, EU
Presence
Source: Tigenix Web site; Frost & Sullivan
PA16-52 49
TiGenix—Value Proposition
ASC and CSC Platforms Value Proposition Target Therapy Segment
• ASCs: Show strong immuno-modulatory • Use of allogeneic adipose- • Autoimmune disorders

characteristics with a strong cell stability during derived stem cells allows • Inflammatory disorders
expansion capitalization of their benefits • Cardiovascular disorders
• CSCs: Derived from cardiac stem cells; portray without any associated ethical
robust cardio-protective and immune-regulatory issues
properties
Key Differentiators
• The use of adipose-derived stem cells allows easy isolation from donor, a rich supply, and stable cell lines, while technology platforms
allow commercial-level scalability.
• The lead product Alofisel (previously Cx601; against complex perianal fistulas) is the first allogeneic stem cell therapy to receive
Market Authorization (MA) approval in Europe. This approval positions Alofisel as a lead product in the European market along
with its competitive edge in terms of efficacy and disease relapse, compared to existing treatments (infliximab, surgery).

• The company aims at meeting unmet medical needs in • Tigenix aims at leveraging on its strong product pipeline to
autoimmune disorders through advancement of its two core expand its geographic presence in the US and Europe.
technology platforms. • The company also intends to expand R&D collaborations and
• With expertise in stem cell manufacturing and development, professional service contracts in the US, fuelled by its lead
product receiving US FDA orphan drug designation in late
the company exploits its strong distribution network, which is
2017.
further bolstered by multiple strategic partnerships with key
• Presently, it is looking to advance its pipeline product candidate
industry participants. SEPCELL which is in Ib/IIa. The product is currently being
• Partners: Apart from the acquisition by Takeda, Tigenix developed by SEPCELL consortium, which includes research
collaborated with Mesoblast in December 2017, with the institutes and medical and biotech entities with a funding from
latter granting Tigenix an exclusive global patent license to the European Union‘s Horizon 2020 Research and Innovation
use ADSCs in the local treatment of fistulae. Programme.
Key: ADSC—Adipose Derived Stem Cells; CSC—Cardiac Stem Cells Source: Tigenix Web site; Frost & Sullivan
PA16-52 50
Bone Therapeutics—Company Overview
It specializes in the development of cell therapy products for orthopedics and bone diseases.
Business
Ownership Private Orthopedics and bone diseases
Segments
Year Founded 2006

Unique cell therapy platform;
Core Business extensive cell therapy expertise and
2017 Revenue Model a strong intellectual property
4.8
($ Million) portfolio
Thomas Lienard
(CEO)
Notable
Jean-Luc AsahiKASEI, Fujifilm, Kasios SPRL
Partners
Vandebroek (CFO)
Key People
Benoit Champluvier
(Chief Technology & • Recently raised $24.1 million to
Manufacturing
―Innovation to Officer)
support its cell therapy pipeline
• Product candidate ALLOB
address unmet Market (Phase IIA) targets 715,000
Number of
needs in Employees
>100 Position potential DUF patients
• Overall, 1.5 million potential
orthopaedics & patients targeted in the EU, the
Headquarters Gosselies, Belgium
bone diseases‖ US, Japan
Geographic
US, EU, Japan
Presence
Key: DUF—Delayed Union Fracture Source: Bone Therapeutics Web site; Frost & Sullivan
PA16-52 51
Bone Therapeutics—Value Proposition
Cell Therapy Platform Value Proposition Target Therapy Segment
• Allogeneic platform through the company‘s ALLOB • Unique osteoblastic cell based • Musculoskeletal:
program technology platform that o Osteonecrosis of the hip
• Autologous cell therapy development through the enables therapy development o DUF
PREOB program through both autologous and o Degenerative spine
allogeneic stem cells disorders
Key Differentiators
• It uses a minimally invasive approach for therapy administration by implanting cells through a needle (or trephine) at the bone defect
site, thus, eliminating painful invasive surgeries.
• It is the only company that develops bone cell products from differentiated bone cells for the treatment of DUF. Its product candidate
ALLOB is in Phase IIA clinical trial for the treatment of DUF. The company is targeting 715,000 patients in Europe and Asia
• The company‘s product PREOB (phase III trials) has received orphan drug designation for osteonecrosis from the EMA and FDA
which positions the product for cost-effective commercialization in the US and Europe.

• Its business model revolves around its expertise in cell therapy for • The company looks to exploit different segments of the
bone diseases through its proprietary bone regeneration approach. global orthobiologics market, targeting nearly 1.5 million
• It has a strong intellectual property portfolio, comprising 9 patent patients in Europe, the USA and Japan for the three main
families, covering various bone-related disorders targeting the global indications entailed by its product pipeline.
market. • The ―add on therapy‖ nature of its products eliminates a
• Partners: It entered into an exclusive license agreement with Asahi lot of competition within the orthobiologics market and
Kasei Corporation in late 2017, for the development and hence, it looks to create a niche for its products by
commercialization of PREOB in Japan and other Asian countries, accelerating its product development pipeline.
with an upfront payment of over $2 million.
Source: Bone Therapeutics Web site; Frost & Sullivan
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Cell Medica—Company Overview
The company specializes in the development of cellular immuno-therapeutics against cancer and infectious
diseases.

Business Cellular immunotherapeutics for
Ownership Private Segments oncology
Year Founded 2005 Advanced technological platform

Core Business (CAR-T and TCR) along with strong
Model financial backing and strategic
2017 Revenue
- partnerships
($ Million)
Catapult CGT, Baylor College of

Gregg Sando Notable
Medicine, University College
(CEO & director) Partners
London
Kevin S. Boyle Sr.
Key People
(CFO)
Karen Hodgkin • Over $180 million raised till date,
(COO) by means of external financing
―Transforming for advancing its oncology
lives through Number of
product pipeline
69 Market
cellular Employees Position o $74 million raised in 2017, to
advance its 3 proprietary
immunotherapy‖ Headquarters
St. Pancras Way, platforms, including its lead
London, UK oncology product CMD-003, in
Geographic CITADEL Phase 2 clinical trial
EU, NA
Presence
Source: Cell Medica Web site; Frost & Sullivan
PA16-52 53
Cell Medica—Value Proposition
PENTRA Antibody Technology Value Proposition Target Therapy Segment
• Used to generate CARs and immune-modulatory • Diverse technology platforms • Oncology

molecules for expression on CAR/TCR engineered that provide versatility in • Hematological malignancies
immune cells oncology therapy development,
• Improves potency of immune cells by helping them to backed by strong financing
overcome the suppressive microenvironment of solid and manufacturing capabilities
tumors
Key Differentiators
• It has a strong product pipeline, with 7 products in preclinical and early clinical trials, covering a vast range of cancer types.
• It displays a robust financial backing, with a total of $180 million being raised so far, owing to its promising technology and product
pipeline.
• It has 4 advanced technology platforms (Dominant TCR, PENTRA Antibody, NKT cell, and CAR-T) that help target almost all cancer
antigens (intracellular and cell-surface proteins via CAR-T and TCR approach), thereby, providing therapeutic versatility and efficacy.

• The company‘s business model revolves around the application • With the acquisition of Catapult Therapy TCR Ltd.,
of its proprietary technology platform incorporating CAR and the company aims to integrate its dominant TCR
engineered TCR to open up possibilities in the field of oncology. platform with the WT1-TCR therapy, thus enhancing
• Backed by strong strategic partnerships and external financing, the product efficacy for the treatment of solid tumors.
company has been bolstering its manufacturing capabilities to • In addition, the company will initiate early trials for its
realize the objectives of commercial production of immunotherapy innovative CAR-NKT product, targeting GD2 in 2018,
products in the field of oncology while focusing on the expansion of its existing product
• Partners: It acquired WT1 cancer immunotherapy from CGT pipeline, most of which includes preclinical and early
Catapult and has further collaborated to establish cell therapy clinical trial molecules.
manufacturing operations for the company at the GMP production
facility of CGT Catapult.
Source: Cell Medica Web site; Frost & Sullivan
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Mesoblast—Company Overview
The company develops cellular medicines targeting a wide range of life-threatening diseases.

Business • Regenerative and therapeutic
Segments biologics
Ownership Private
• Disruptive cellular technology

platform to create clinically
Year Founded 2001 Core Business
differentiated products
Model
• Targeting serious/life threatening
2018 Revenue conditions with unmet need
17.3
($ Million)
Notable
JCR Pharmaceuticals
Silviu Itescu Partners
(CEO and MD)
Josh Muntner
Key People (CFO) • Significant increase in revenues
Donna Skerrett in 2018 owing to strong
―Disruptive (Chief Medical performance of TEMCELL
Officer) Market • Product grew by 152% in Japan
cellular Position alone which was solely
medicine to Melbourne, Victoria, responsible for the robust growth
Headquarters in revenue
overcome Australia
unmet Geographic
APAC
healthcare Presence
needs‖
Source: Mesoblast Web site; Frost & Sullivan
PA16-52 55
Mesoblast—Value Proposition
MLC Technology Platform Value Proposition Target Therapeutic Segment
• Proprietary MLCs enable the development of a • Disruptive MLC cellular technology • Spine and orthopedics
broad product range that is the progeny of the platform, deep intellectual property diseases
earliest precursors of MSC lineage in adults portfolio, late-phase clinical assets, and • Acute GvHD
• Promote tissue repair and modulate immune industrialized manufacturing capabilities • Heart diseases
• Also, improved efficacy of MLC-based
responses • Inflammatory diseases
products; immune privileged avoiding
• Positions the company as a global leader in • Oncology
immune responses in patients
cellular medicines • Hematology
Key Differentiators
• It has three products in phase III trial, which, if approved, have the potential of generating newer market opportunities across
cardiovascular diseases and the GVHD segment.
• The company recently received an FDA Regenerative Medicine Advanced Therapy Designation for its heart failure cell therapy under
the 21st Century Cures Act, which aims to expedite the development of regenerative medicines for the treatment of life-threatening
diseases.
• TEMCELL, the first allogeneic cellular medicine that received full approval in Japan, is expected to provide a consistent revenue
stream. With over 800 patents across 69 patent families, the company has a strong scientific and R&D presence.

• The company is applying its proprietary cell technology • It plans to explore CAR-T therapies across a wide range of
platform to build an advanced cell-based product portfolio. therapeutic conditions. Its recent partnership with Cartherics to
• The company‘s ‗off-the-shelf‘ products are allogenic in develop CAR-T cell therapies against relapsed ovarian and gastric
nature, thereby increasing the scope for stem cell cancer is a case in point.
transplantation. • It wants to focus on partnerships to speed up product development
• Partners: it has a licensing agreement with JCR and approval.
pharmaceuticals for the distribution of TEMCELL against • It aims to enter the US and Europe with its product MSC-100-IV,
GvHD in Japan. which is likely to be approved in the US for treatment of acute GVHD.
Key: MPC—Mesenchymal Precursor Cells; MLC—Mesenchymal Lineage Cells Source: Mesoblast Web site; Frost & Sullivan
PA16-52 56
Cynata Therapeutics—Company Overview
The company develops MSCs from iPSCs for various indications using its proprietary Cymerus technology
platform.

Business
Spinal Therapeutics
Segments
Ownership Private
Unique technology platform,
CymerusTM for commercially scalable
2003 (formerly Eco production of MSCs from induced
Year Founded Core Business
Quest Limited) Pluripotent Stem Cells (iPSCs) with
Model
strong strategic and financial
2018 Revenue collaboration with multiple industry
1.51
($ Million) players
Ross Alexander Notable

Celularity, Fujifilm, Apceth
MacDonald – (MD, Partners
CEO & Executive
Key People Director) • Currently has a strong pipeline
Dr. Killian Kelly - across the orphan drug segment
(VP of Product • Lead MSC product candidate
―Innovation in Development) CYP-001, received the FDA
Orphan Drug Designation in June
regenerative Number of
- Market Position 2018 for the treatment of GvHD
Employees
and therapeutic • Strong clinical data could compel
biologics‖ Headquarters Carlton, Australia partner Fujifilm to exercise a
worldwide license to market
Geographic CYP-001 to prevent and treat
South Asia GvHD
Presence
Source: Cynata Therapeutics Web site; Frost & Sullivan
PA16-52 57
Cynata Therapeutics—Value Proposition
Cymerus™ Platform Technology Value Proposition Target Therapy Segment
• Platform allows economic manufacturing of • Technology aims to address conventional • Acute GvHD
cell therapy products, including MSCs at a hurdles in MSC production such as donor • Cardiovascular diseases
commercial scale dependence and variability, contamination • Respiratory and pulmonary
• Utilizes iPSCs and a newly identified precursor with non-target cells, limited scalability and diseases
cell called as Mesenchymoangioblast (MCA) so on and deriving multiple cell therapeutic
platforms at a considerably lower cost
Key Differentiators
• The proprietary technology has the advantage of versatility (manufacture of a wide range of cell types), manufacturing scalability
(iPSC use allows infinite expansion capacity), cost and complexity optimization (eliminates the need for multiple donors), consistency
(requiring a single donor), and safety (immune privileged) compared to other technology platforms for MSC production.
• The lead product, CYP-001 received Orphan Drug Designation from the FDA to treat GvHD which positions the product for cost-
effective commercialization in the US. This establishes the product as a promising alternative to existing therapies by reducing the
overdependence on steroids.

• Cynata‘s business model revolves around the advancement of its • The company looks to leverage on its unique technology
technology platform to create a consistent supply of MSCs for multiple platform to advance its product pipeline, especially
therapeutic usages. its first MSC product, CYP-001, which has shown
• It holds multiple patents and filings for its technology, accompanied by promising results against GvHD in early-stage clinical
considerable progress of CYP-001 trials. The company is backed by trials.
several financial and strategic agreements with industry participants, • With strong financial support, including the ~$10 million
which allows continuous expansion of its product portfolio. capital raised in 2017, from its agreement with Fujifilm
• Partners: Recent, strategic partnership and license agreement worth and Apceth, Cynata is looking to expand its product
$60 million (with Fujifilm with potential milestone payments and pipeline for further indications in the field of
royalties on eventual product sales, including lead product candidate cardiovascular, pulmonary diseases, and oncology.
CYP-001) with a $3 million upfront payment.
• Other partners include Celularity and Apceth. Source: Cynata Therapeutics Web site; Frost & Sullivan
PA16-52 58
Medipost—Company Overview
It develops stem cell therapies for severe indications and cosmetics based on umbilical cord blood derived
stem cells.

Business Allogenic mesenchymal stem cells
Segments (cord blood cells)
Ownership Private
Core Business Robust R&D, with large technical

Year Founded 2000
Model and financial investment
2017 Revenue
~37.09
($ Million)
Notable
CCRM, Inter Stemma
Yang, Yoon Sun Partners
(CEO)
Dong Jin Hwang
Key People
(President, Director)
Jang Yeong Lee • Over 7,000 vial sales of
(MD) CARTISTEM by the end of 2017
―Life changing Number of Market
since its approval in 2012
192 • Total sales growth of over 47% in
innovations for Employees Position
2017, compared to 2016
a healthy • Over $10 million revenue from
Headquarters South Korea
world‖ sales in the first quarter of 2018
Geographic
APAC, EU, NA
Presence
Source: Medipost Web site; Frost & Sullivan
PA16-52 59
Medipost—Value Proposition
CELLTREE – Umbilical Cord Blood Bank Value Proposition Target Therapy Segments
• Serves the extraction, storage, and transplantation of • First to commercialize an • Musculoskeletal: osteoarthritis
umbilical cord blood derived stem cells to facilitate allogenic stem cell drug • Pulmonary diseases:
production of HSCs and MSCs CARTISTEM for the treatment of Bronchopulmonary Dysplasia
• Follows a penta-safety process that entails extraction of knee cartilage defects in (BPD)
cord blood up to transplantation osteoarthritis patients • Neurological disorders:
• Product CARTISTEM®—an allogenic cord blood derived Alzheimer‘s disease
MSC developed using the CELLTREE platform
Key Differentiators
• The company has a wide product portfolio, spanning different therapy areas.
o CARTISTEM: World‘s first allogeneic stem cell drug approved for commercial use by the Ministry of Food & Drug Safety in Korea.
o PNEUMOSTEM: Granted orphan drug designation in the US and the EU, alongside its approval by FDA for clinical trials; has successfully
completed phase II trials in South Korea.
o NEUROSTEM: Presently in early-stage development for Alzheimer‘s disease.
• Medipost‘s CELLTREE is South Korea‘s leading cord blood bank that occupies the highest market share in umbilical cord blood (UCB)
storage and is recognized as the country‘s largest HSC transplant program provider.

• It has a strong R&D-centric business model, with robust technical and • Medipost is targeting both domestic and global expansion
financial support. while increasing its product portfolio with strong R&D
• The company possesses over 68 domestic and international patents, support.
with additional application for over 45 patents and has successfully • The company targets commercialization of CARTISTEM
commissioned 29 national projects. (Phase 1/2a) and PNEUMOSTEM (Phase 1/2) in the US,
• Partners: It has signed an MoU on mutual cooperation and support in alongside establishing joint-ventures in China and Japan.
stem cell research with the Canadian Government funded CCRM. As • Investments in strategic partnerships in Australia, New
per the deal, Medipost will receive support and benefits during trials, Zealand, Canada, and Central America are aimed at
licensing, and registering of its pipeline products for entry into the strengthening its overseas market expansion efforts.
Canadian stem cell drug market.
Key: CCRM—Center for Commercialization of Regenerative Medicine; HSC—Hematopoietic Stem Cells Source: Medipost Web site; Frost & Sullivan
PA16-52 60
Anterogen—Company Overview
It develops stem cell therapy products for skincare and rare indications by making use of adipose-derived
stem cell technology.
Business
Tissue regeneration
Ownership Private Segments
Year Founded 2000 A versatile adipose stem cell

Core Business technology, backed by robust R&D
Model and in-house manufacturing
2017 Revenue
2.4 capabilities
($ Million)
Lee Sung-Koo
(CEO)
Mi-Hyung Kim, PhD
Key People (Senior MD)
Bang-Hee Han • 2018 Q2 sales of around
―Regenerative (Senior MD and
Head Clinic) Market
$8.7 million
therapies for Position • Revenue growth of more than

9% recorded in 2017 compared
better health Headquarters
Geumcheon-gu to the previous year
and happiness‖ Seoul, South Korea
Geographic
APAC
Presence
Source: Anterogen Web site; Frost & Sullivan
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Anterogen—Value Proposition
Adipose Stem Cell Therapy Value Proposition Target Therapy Segment
• Uses mesenchymal stem cells manufactured from • First company to use ADSCs in • Dermatology
donor‘s adipose tissues, hence autologous in nature South Korea to develop stem • Rare diseases
• Better promise in stem cell research, as adipose cell based cell therapy • Diabetic complications
tissue shows more than 10,000 times higher number products, with three products • Musculoskeletal conditions
of stem cells than those isolated from similar amount already approved by KFDA
of bone marrow
Key Differentiators
• The company relies only on ADSC‘s and is the first in South Korea to use them for the development of stem cell based
products. Its key cell therapy approved products include:
o Cupistem® injection: The first ever approved product in the world derived from ADSCs
o Queencell®: Derived from autologous, minimally manipulated cell products using adipose tissues for subcutaneous
adipose tissue
o Adipocell®: Immature adipocyte differentiated from ADSCs

• The company has a strong R&D profile, which includes animal facility • The company looks to expedite its product
for pre-clinical trials as well as certified, in-house cGMP manufacturing development program and is targeting Phase III trials
capabilities. for its therapy program for treatment of Diabetic Foot
• Anterogen also facilitates various analysis services for external clients Ulcer (DFU).
including sample analysis for clinical studies, identification and purity • The success of this program will position Anterogen
testing, potency testing, and virus testing of other biopharmaceuticals, in as one of the leaders in the DFU market.
addition to its existing product portfolio. • The company looks to continue its market expansion,
• It also boasts of a strong patent profile with 24 patents issued and 42 in with additional external financing for stem cell
the application process.
therapy in the coming years.
Source: Anterogen Web site; Frost & Sullivan
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Pharmicell—Company Overview
It develops autologous stem cell therapies for rare indications, biocosmetics, alongside offering services in
technology consulting.
Business Stem cell therapeutics (MSCs and

Ownership Private Segments dendritic stem cells)
Expertise in stem cell R&D,

Year Founded 2002
Core Business supported by in-house
Model manufacturing capabilities, with a
2017 Revenue strong product pipeline
23
($ million)
Kim Hyun Soo Notable University of Utah, Seoul Asan

(CEO) Partners Medical Center
Seong Rae Kim
Key People
(Co-CEO, Director)
Wun Seong Baek
(MD)
―Leading the
Number of 2018 first quarter: total product
way to a better 115 Market
sales of $5.5 million; increase of
Employees Position
future‖ over 7% compared to the same
Gangnam-gu, Seoul, period in 2017
Headquarters
South Korea
Geographic
APAC
Presence
Source: Pharmicell Web site; Frost & Sullivan
PA16-52 63
Pharmicell—Value Proposition
Stem Cell Bank—Twelve Value Proposition Target Therapy Segment

• An integrated stem cell bank that contributes to • Leader in stem cell research • Cardiovascular conditions
therapeutics research in oncology and neurological across the region with the first • Pulmonary disorders
disorders, among others ever approved stem cell drug, • Liver diseases
• Provides a storage capacity/period of 15 to 20 years, Cellgram-AMI in South Korea, • Oncology
with an aim to provide bio-insurance through adult followed by other candidates in
stem cell and cord blood storage approval stage
Key Differentiators
• It is a leader in stem cell therapy with the successful approval of Cellgram-AMI, the company‘s lead product, which is the
world‘s first stem cell drug approved by Korean MFDS in July 2011.
• It maintains an integrated stem cell bank for adults and infants, including Twelve adult and Twelve baby stem cel banks
respectively.
• It has a strong product pipeline, with two drugs in late stage of development and several others in Phase 2 and preclinical
development for the treatment of a wide range of indications.

• The company‘s business is centered around extensive research • The company looks to continue its market expansion and
efforts in stem cell therapy and in-house manufacturing acceleration of product development by gaining market
capabilities certified by the Korean MFDS. approval for Cellgram-LC.
• Through its extensive product pipeline, technology consulting, • If granted, this therapy will be the fifth approved stem cell
and CMO services, the company has succeeded in establishing a therapy in South Korea.
wide market presence in major countries such as China and the • Alongside MSCs, with multiple patents in dendritic cell
US through regulatory approval for its stem cell therapy for CVD. technology, it is also aiming to expedite research on
• Partners: The company is currently in research partnerships with dendritic cell based therapies in oncology, with prospective
the University of Utah and Seoul Asan Medical Center. Investigational New Drug (IND) application in the later stage
of 2018.
Source: Pharmicell Web site; Frost & Sullivan
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C2A Profiles—Tier III
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PA16-52 65
Short Profiles of Digital Pathology Companies
• CellSeed has a strong focus on using cell sheets for several diseases such as cancer of esophagus, corneal
CellSeed regeneration, cardiac patches, cartilage sheets, and so on.
• The company presently has three products in the market: UpCell® for Cell Sheet engineering, RepCell® for
cell collection, and HydorCell® for low cell binding.
• With these products, the company has engaged with the challenges of commercializing regenerative
medicine by developing cell sheets.
• Takara Bio, which started as the biomedical business unit of Takar Shuzo Co. Ltd, is focusing on three
business segments: the bioindustry business, the agribio business, and the gene therapy business. With the
Takara Bio support of the Japanese Government, the company is accelerating the development of new products in the
Inc. field of regenerative medicine, with a key focus on oncology.
• The company works with two different engineered cell therapies. One is siTCR gene therapy, using its
proprietary siTCR vector technique, the clinical trial for which is ongoing in collaboration with Mie University.
• On the other hand, it has begun clinical trials for CD19 CAR gene therapy, as an independent developer, for
adult acute lymphocytic leukemia. It aims to commercialize the CAR gene therapy by 2021.
• It is a Japan-based company, founded in 2011, with the aim of commercializing the research results of Prof.
Mega- Eto‘s research team and Prof. Nakauchi‘s research team, after their recognition as an A-Step Project.
Karyon • The company holds patent rights to use technology for producing platelets from iPSCs (includes techniques
Corporation for producing blood cells, (especially platelets) from ESCs and iPSc; these are aimed at increasing the
production of immortalized megakaryocyte precursor cells that can be cryopreserved).
• In June 2018, the company was selected for the J-Startup program and also raised JPY 3,700 million in
additional funding through private placement of new shares.
Source: CellSeed, Takara, MegaKaryon Web sites; Frost & Sullivan
PA16-52 66
Short Profiles of Digital Pathology Companies (continued)
• It is a Japan-based company working toward developing breakthrough mechanism of actions and new
Cyfuse advanced treatment methods with its proprietary Micro Needle Array Technology platform that provides 3D
Biomedical tissue fabrication with cell only.
• The technology is also capable of making small tissue pieces that can mimic the in vivo pieces for the
purpose of in vitro research.
• It has a strong product pipeline that focuses on bone and cartilage regeneration, vascular regeneration,
neural regeneration, and liver structure.
• It is a US-based gene and peptide therapy company, focusing on developing gene therapy for
Renova cardiometabolic diseases.
Therapeutics • Its lead investigational candidate RT-100 AC6 for CHF is a first-in-class, single-dose gene therapy
candidate. It is presently in Phase III of development and has received the fast track designation from FDA
in December 2017.
• It also has a phase II molecule for diabetes and early-stage molecules for obesity, hypertension, and Non
alcoholic Fatty Liver Diseases (NAFLD).
• Viacyte, Inc. a San Diego based private venture capital backed regenerative-medicine company is set to
develop a first-of-its-kind cell therapy for diabetes type I. The company is using stem cells by differentiation
into pancreatic beta cell precursors (PEC-01™).
Viacyte, • The developed product would be administered using subcutaneous implantation in a retrievable medical
device.
Inc., • The company recently received a grant of $1.4 million by California Institute for Regenerative Medicine
(CIRM) to support the initial development of immune-evasive pluripotent stem cell lines.
• Additionally, the company received the Small Business Innovative Research Phase II Award from National
Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) for its work toward development of
functional human beta cells in vitro.
Source: Cyfuse, Viacyte, Renova Web sites; Frost & Sullivan
PA16-52 67
• It has long-standing expertise in cell handling and manufacturing. It established the Regenerative Medicine
Rohto Research & Planning Division in 2013 and the Regenerative Medicine Research Center in Ryukyu University
Pharmaceutic in Okinawa.
als • Its proprietary automatic cell culture system has been jointly developed with Kyoto‘s Micronics co.
• Its high-purity stem cell manufacturing (serum free) with ADSC therapy development targets liver and skin
diseases, hepatic disorders, immunology, neurology, and oncology.
• It has a Licensing and distribution agreement with Nuo Therapeutics for the rights to develop and
commercialize Nuo‘s Aurix system in Japan.
• It has a wide-ranging therapeutic focus, including CVD, muskuloskeletal- and diabetes-related

complications, and autoimmune diseases, among others for developing novel stem cell based therapies.
Cytopeutics
• The key focus is on MSCs with a technology platform that enables the differentiation of MSCs into
mesangial cells and nerve cells, opening up newer possibilities for therapeutics development. It is one of
the few companies with such capabilities.
• It has developed the first-of-its-kind medical lab with a central monitoring and control system SCADA* with
cGMP certification.
• The company develops allogenic stem cell products, enabling treatment from the same cell bank, off-the-
shelf. Using its unique and scalable stem cell technologies, the company has developed a strong stem cell
therapy focused pipeline.
ReNeuron
• Its lead CTX cell therapy candidate is primarily focused on stroke treatment; simultaneously, the company is
also testing a molecule for critical limb ischemia. These trials use a cryopreserved CTX stem cell line
formulation which is designated CTXcryo.
• The company recently won a grant of GBP 1.5 million from UK‘s innovation agency, Innovate UK, to support
the development of its hRPC stem cell therapy candidate for blindness-causing degenerative diseases of
the retina.
Key: SCADA—Supervisory Control and Data Acquisition Source: Rohto, Cytopeutics, Reneuron Web sites; Frost & Sullivan
PA16-52 68
• The company focuses on the development of gene edited off-the-shelf immunotherapies for cancer
Cellectis treatment.
• Its flagship technology Talen for cell engineering uses allogeneic cells for the development of CAR-T cell
therapies. Owing to its strong focus on oncology, its drug candidate UCART22 was recently granted IND
approval for B-ALL.
• In May 2018, Cellectis collaborated with Harvard‘s Wyss Institute, putting to use Cellectis‘ TALEN
gene-editing technology to create the first virus-resistant human cells for manufacturing therapeutics and
developing new cell-based therapies.
Educell • The company has a strong focus on developing cell therapies for rare diseases and neurological disorders.
• It possess four proprietary technology platforms ImmunoArt™, CrohnArt™, ChondroART 1D, and
ChondroART 2D; each focuses on a different therapy area.
• Another new technology platform, UroART™, focuses on several urological conditions.
Source: Cellectis, Educell Web sites; Frost & Sullivan
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Growth Opportunities and Companies to Action
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Transformation in the Industry Ecosystem
While global cell and gene therapy has garnered interest from several participants, small-to-mid sized
companies have larger contribution to the pipeline, supported by outsourcing partners and academia.
CDMO/CROs Academic Research Partners
Large Size Mid Size Smaller

Biotech Companies Biotech Companies Biotech Companies
Image Source: Company Web sites (Lonza, Medpace, Charles River labs, Spark Therapeutics, Kite Pharma, Juno Therapeutics, Viacyte, Inc.,
Mesoblast, JCR Pharma, Cynata Therapeutics, Cyfuse Medical, DiscGenics, Avexis, Adaptimmune, bluebird bio) Source: Frost & Sullivan
PA16-52 71
Five Major Growth Opportunities
Diverse
Combination Growing M&A Genome Editing Manufacturing
Therapeutic
Therapies Activities - CRISPR/Cas9 Automation
Focus
• Cell-gene therapy • A new wave of • The emergence of cell • As the fastest, • Growing demand for
combinations are therapies such as therapy is attracting simplest, and most cell therapies calls for
rapidly gaining ADCs and bi- bigger pharma and economic gene improvised manufacturing
momentum. They specific antibodies biotech participants to editing tool, CRISPR requirements.
make use of gene is adding to the make investments. is gaining sharp • As a result, the demand
editing tools and precision medicine • These participants look uptake. for process automations
vector delivery landscape of at collaborating with • Mustang Bio recently in the form of digital
systems to devise oncology care. small-to-mid sized collaborated with solutions is on the rise.
innovative, curative• On the other hand, niche participants with Harvard University
therapies. • Promethera Lifesciences
there is an an aim to acquire and Beth Israel is expanding its
• Novartis‘ recently emergence of therapeutic, geographic, Deaconess Medical manufacturing
approved CAR-T IPSCs, MSCs, and and technological Center (BIDMC). capabilities by
cell therapy ADSCs for novel expertise. • This collaboration collaborating with
Kymriah is an therapeutic • Kite Pharma‘s aims to develop Shibuya to develop a
example of a treatments for acquisition by Gilead CRISPR/Cas9- high-throughput,
combination neurological, Sciences provided the enhanced CAR-T commercial-scale, and
therapy for the musculoskeletal, latter with Kite‘s cell therapies for clinical-grade cell therapy
treatment of and dermatological blockbuster CAR-T cell cancer. and regenerative
refractory B-cell conditions, among therapy Yescarta. medicine manufacturing
acute lymphoblastic others. platform.
leukemia.
PA16-52 72
Strategic Imperatives for Growth
New Product Development:

• Most participants such as Sangamo Therapeutics, Mesoblast, DiscGenics, and so on are focusing
on using allogenic stem cells as opposed to traditional autologous cells, so as to provide better
scalability and storage capabilities to stem cells; hence, they are dealing with issues pertaining to
lack of adequate donors.
Research-based Collaborations:
• While collaborating with small-to-mid segment participants for specific therapeutics expertise,
market participants also need to focus on certain academic and research collaborations in order to
support new therapeutic innovations with the help of research partners.
Value-based Pricing:
• Prices of existing cell therapies range from $300,000 to $500,000, and the lack of a specific
Strategic reimbursement model directly impacts their accessibility and uptake.
Imperative for • Novartis and Gilead, the makers of the first two cell-gene therapies, are working toward introducing
Growth in Cell an outpatient value-based payment model for their therapies in collaboration with CMS, which will
significantly reduce the cost burden on payers, providers, and patients.
Therapy
Markets Regulatory Amendments:
• Reducing the duration between drug development and market launch will have a strong impact on
the cell therapy market.
• Implementation of PMD and ASRM acts in Japan are aimed at expediting the regulatory approval
process for regenerative medicine therapies in Japan; thus, propelling market growth.
Manufacturing Automation:
• With the industry shifting toward high-value low-volume manufacturing, there is an ardent
requirement to upgrade manufacturing facilities to easy-to-use and single-use techniques and
real-time monitoring to reduce cost and time to market.
• Companies such as Merck Millipore and GE Healthcare provide cutting-edge single-use
technologies that support novel drug manufacturing.
Key: PMD—Pharmaceutical and Medical Devices
ASRM—Act of the Safety of Regenerative Medicine Source: Frost & Sullivan
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The Last Word
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Conclusion—Key Takeaways
The global cell therapy market is dominated by small-to-mid segment participants,

which is leading to large-scale market consolidation with bigger biopharma
companies vouching for therapeutic and technological expertise.
APAC is emerging as a key market for cell therapy research, with companies
such as Pharmicell, Cynata Therapeutics, Mesoblast, Takara Bio, and so on
developing innovative stem cell based therapies for non-oncology indications.
Apart from stem cell therapies, CAR-T cell therapies are considered as one of
the key segments driving the cell therapy market, with market leaders such as
Novartis, Gilead, bluebird bio, Juno Therapeutics, and so on focusing on advanced
cancer research with their respective molecules.
PA16-52 75
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PA16-52 76
Appendix
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PA16-52 77
Abbreviation List
Abbreviation Full Form Abbreviation Full Form

ADSC Adipose Derived Stem Cell EMA European Medicines Agency
ALL Acute Lymphocytic Leukemia GT Gene Therapy
AML Acute Myeloid Leukemia GVHD Graft Vs. Host Disease
Committee for Medicinal Products for iPSC Induced Pluripotent Stem Cells
CHMP
Human Use
FDA Food & Drug Administration
CALD Cerebral Adrenoleukodystrophy
M&A Mergers & Acquisitions
CAR-T Chimeric Antigen Receptor
PD1 Blocker Programmed Cell Death protein 1
CD19 Cluster of Differentiation
PMD Pharmaceutical and Medical Devices Act
CT Cell Therapy
PRIME Priority Medicine
Contract Development & Manufacturing
CDMO R&D Research & Development
Organization
SRM Safety of Regenerative Medicine Act
CRO Contract Research Organization
TE Tissue Engineering
`CSC Cardiac Stem Cells
TFL Transformed Follicular Lymphoma
Cytotoxic T-lymphocyte–associated
CTLA4
antigen MSC Mesenchymal Stem Cell
DLBCL Diffuse large b-cell lymphoma NHL Non-Hodgkin‘s Lymphoma
DDD Degenerative Disc Disease ZFN Zinc Finger Nuclease
DPSC Dental Pulp Stem Cell
EDC Electronic Data Capture
PA16-52 78
List of Exhibits
Exhibit Slide Number

Total Cell Therapy Market: C2A Value Creators, Global, 2017 14
Total Regenerative Medicine Market: Market Segmentation, Global, 2017 18
Total Cell Therapy Market: Stakeholder Ecosystem, Global, 2017 19
Total Cell Therapy Market: Impact of Key Market Influencers, Global, 2018 21
Total Cell Therapy Market: Key Market Participants Aligned with Top 3 Trends, Global, 2017 23
Total Cell Therapy Market: Vendor Landscape, Global, 2018 24
Total Cell Therapy Market: Juno Therapeutics—Product Pipeline, Global, 2018 26
Total Cell Therapy Market: Juno Therapeutics—Major Partnerships and Agreements, Global, 2018 28
Total Cell Therapy Market: Case Study— Celgene‘s Acquisition of Juno Therapeutics, Global, 2018 28
Total Cell Therapy Market: bluebird bio—Technology With Product Pipeline, Global, 2018 29
Total Cell Therapy Market: Bluebird—Major Partnerships and Agreements, Global, 2018 31
Total Cell Therapy Market: Case Study—Celgene‘s Partnership With Bluebird, Global, 2018 31
Total Cell Therapy Market: Kite Pharma: Product/ Technology Depiction, Global, 2018 32
Total Cell Therapy Market: Case Study—Gilead‘s Acquisition of Kite Pharma, Global, 2018 34
Total Cell Therapy Market: Kite Pharma—Major Partnerships and Agreements, Global, 2018 34
Total Cell Therapy Market: Adaptimmune—Product Pipeline, Global, 2018 35
Total Cell Therapy Market: Case Study—Collaboration With GSK, Global, 2018 37
Total Cell Therapy Market: Adaptimmune—Major Partnerships and Agreements, Global, 2018 37
PA16-52 79
List of Exhibits (continued)
Exhibit Slide Number

Total Cell Therapy Market: Oxford BioMedica—Product Pipeline, Global, 2018 38
Total Cell Therapy Market: Case Study—Supply Agreement With Novartis, Global, 2018 40
Total Cell Therapy Market: Oxford BioMedica—Major Partnerships and Agreements, Global, 2018 40
Total Cell Therapy Market: JCR Pharmaceuticals—Product Pipeline, Global, 2018 41
Total Cell Therapy Market: Case Study—Codevelopment Agreement With Teijin, Global, 2018 43
Total Cell Therapy Market: JCR Pharmaceuticals—Major Partnerships and Agreements, Global, 2018 43
PA16-52 80
The Frost & Sullivan Story
The Journey to Visionary Innovation
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PA16-52 81
The Frost & Sullivan Story
PA16-52 82
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Global Cell Therapy Market—Companies to Action, 2018

A Collaborative Business Model With Focus on Stem Cells, Chimeric

Global Transformational Health Research Team at Frost & Sullivan

Section Slide Number

Section Slide Number

Section Slide Number

Section Slide Number

Section Slide Number

Research Goals, 360o Research

Research Approach Company

• Frost Sullivan 360o Companies to Action (C2A) research Profiling and

Geographic Coverage Global

Study Period 2017–2025

Base Year 2017

Monetary Unit US Dollars

The profiles are segmented into:

• Company value proposition

• Mergers and acquisitions and partnerships

What is the impact of external industry environment on market growth?

Source: Frost & Sullivan

• Showcasing Visionary Leadership: A

• Implementation of Best Practices: World-

• Business Model Innovation: A revolutionary Business Model

As a result, companies need to identify

Revenue Growth Market Share Technology A Sustainable

Regenerative Medicine: It is a branch of translational research that focuses on the

Cell Therapy (CT)

Gene Therapy (GT)

Tissue Engineering (TE)

Small Molecules and Biologics

Total Regenerative Medicine Market: Market Segmentation, Global, 2017

Traditional Regenerative Medicine Market

Used mainly for the

Source: Frost & Sullivan

Total Cell Therapy Market: Stakeholder Ecosystem, Global, 2017

• Cold chain • Research • Temperature • Controlled thawing

Biopharma Research CDMOs/CROs Hospitals/Clinical Cold Chain

Image Source: Company Web sites Source: Frost & Sullivan

Industry Needs Vendor Strategy

Source: Frost & Sullivan

Cell and Gene Therapy Heart Failure

Expertise in CAR-T cell Academic Collaborations Outsourcing Services

Source: Frost & Sullivan

Total Cell Therapy Market: Vendor Landscape, Global, 2018

Tier I Tier II Tier III

Total Cell Therapy Market: Juno Therapeutics—Product Pipeline, Global, 2018

CD19: JCAR017 Key Focus Areas

CAR-T cell Technology TCR Technology

Lenti-D Drug Product

Source: bluebird bio Web site; Frost & Sullivan

Gene Editing Technology

Axicabtagene ciloleucel Key Therapy Focus

Source: Kite Pharma Web site; Frost & Sullivan

Therapy Development Procedure

CAR-T Cell Therapy TCR Cell Therapy TCR Technology:

Source: Kite Pharma Web site; Frost & Sullivan

Total Cell Therapy Market: Adaptimmune—Product Pipeline, Global, 2018

Key Focus Areas

SPEAR T-cell Technology

Identify and Validate Identify Preclinical Safety and Clinical

Source: Adaptimmune Web site; Investopedia; Frost & Sullivan

Source: Oxford BioMedica Web site; Frost & Sullivan

Total Cell Therapy Market: JCR Pharmaceuticals—Product Pipeline, Global, 2018