- Language: English
- 959 Pages
- Published: April 2015
- Region: Global
Complete 2015-16 Induced Pluripotent Stem Cell (iPSC) Industry Report
- ID: 3163771
- April 2015
- Region: Global
- 175 Pages
Stem cell research and experimentation have been in process for well over five decades, as stem cells have the unique ability to divide and replicate repeatedly. In addition, their “unspecialized” nature allows them to differentiate into a wide variety of specialized cell types. The possibilities arising from these characteristics have resulted in great commercial interest, with potential applications ranging from the use of stem cells in reversal and treatment of disease, to targeted cell therapy, tissue regeneration, pharmacological testing on cell-specific tissues, and more. Conditions such as Huntington’s disease, Parkinson’s disease, and spinal cord injuries are examples of clinical applications in which stem cells could offer benefits in halting or even reversing damage.
Traditionally, scientists have worked with both embryonic and adult stem cells for research tools, as well as for cellular therapy. While the appeal of embryonic cells has been their ability to differentiate into any type of cell, there has been significant ethical, moral, and spiritual controversy surrounding their use. Although some adult stem cells do have differentiation capacity, it is often limited in nature, which results in fewer options for use. Thus, induced pluripotent stem cells represent a promising combination of adult and embryonic stem cell characteristics.
Discovery of Induced Pluripotent Stem Cells
Groundbreaking experimentation in 2006 led to the introduction of induced pluripotent stem cells (iPSCs). These are adult cells which are isolated and then transformed into embryonic-like stem cells through the manipulation of gene expression, as well as other methods. Research and experimentation using mouse cells by Shinya Yamanaka’s lab at Kyoto University in Japan was the first instance in which there was successful generation of iPSCs. In 2007, a series of follow-up experiments was done at Kyoto University in which human adult cells were transformed into iPSC cells. Nearly simultaneously, a research group led by James Thomson at the University of Wisconsin-Madison accomplished the same feat of deriving iPSC lines from human somatic cells.
Continued research and experimentation have resulted in numerous advances over the last few years. For example, several independent research groups have announced that they have derived human cardiomyocytes from iPSCs. These cells could be used in a laboratory context to test drugs that treat arrhythmia and other cardiac conditions, and in a clinical context they could potentially be implanted into patients with heart disorders.
Similar advances will continue to be perfected for use of reprogrammed adult cells in the treatment of other diseases and disorders. Original techniques for iPSC production, such as viral induced transcription processes, are being replaced with newer technologies as private industry joined with the scientific community to develop safer and more efficient methods of iPSC production. As innovation around methods of iPSC production continues, clinical grade production of industrial quantities of iPSCs is now becoming possible due to sustained research and experimentation.
Therapeutic Applications of Induced Pluripotent Stem Cells
While there has been continued excitement at the prospect of what such artificially re-manufactured cells could contribute to medical advances, there have also been setbacks along the way. By 2010, there were a number of private companies that were ready to capitalize on the breakthrough technology that iPSCs represent. One such company, Advanced Cell Technology (ACT) in Marlborough, Massachusetts, discovered several problematic issues while conducting experiments for the purpose of applying for U.S. Food and Drug Administration approval to use iPSCs in therapeutic applications. Concerns such as premature cell death, mutation into cancer cells, and low proliferation rates were some of the problems that surfaced.
As a result, ACT (now named “Ocata Therapeutics”) has shifted its induced pluripotent stem cell approach to producing iPSC derived human platelets. One of the benefits of a platelet-based product is that platelets do not contain nuclei, and therefore, cannot divide or carry genetic information. While nothing is completely safe, iPSC derived platelets are likely to be substantially safer than other currently available iPSC therapies, in which uncontrolled proliferation may be a concern. It also shifted to using embryonic stem cells for clinical trial purposes, despite the ethical concerns surrounding this cell type.
Over the next few years, iPSC research advances accelerated exponentially, with perhaps the most momentous milestone being the launch of the first clinical research trial ever involving the transplant of autologous iPSCs into humans (“autologous” meaning the cells are both derived and implanted into the same patient). Previously, all clinical trials using iPSCs involved only the creation of iPSC lines from specific patient populations and subsequent evaluation of these lines for determining whether they could represent a good “model” for a disease of interest within that population.
Therefore, 2013 was the first time in which clinical research involving transplant of iPSCs into humans was initiated, led by Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan. Dr. Takahashi and her team are investigating the safety of iPSC-derived cell sheets in patients with wet-type age-related macular degeneration. While the trial was initiated in 2013 and production of iPSCs from patients began at that time, it was not until August of 2014 that the first patient, a Japanese woman, was implanted with retinal tissue generated using iPSCs derived from her own skin cells. A team of three eye specialists, led by Yasuo Kurimoto of the Kobe City Medical Center General Hospital, implanted a 1.3 by 3.0mm sheet of iPSC-derived retinal pigment epithelium cells into the patient’s retina. Preliminary results are indicating positive results for the participants in this iPSC clinical trial.
Interestingly, Ocata Therapeutics (previously ACT) is conducting similar research, but the company has moved away from its initial focus on iPSCs and has instead chosen to focus on human embryonic stem cells (hESCs). It is currently conducting clinical trials using retinal pigmented epithelial cells produced from hESCs for purposes of treating several types of macular degeneration. The company reported positive preliminary results, which were published in the Lancet in October 2014.
Also of great significance, Kyoto University Hospital in Kobe, Japan announced in February of 2015 that it will be opening an iPSC therapy center in 2019, for purposes of conducting clinical studies on iPSC therapies. The announcement has further positioned Japan as the leading nation committed to bringing iPSC therapies to clinic. Officials for Kyoto Hospital said it will open a 30-bed ward to test the efficacy and safety of the therapies on volunteer patients, with the hospital aiming to initiate construction at the site in February of 2016 and complete construction by September 2019. Current research with iPSCs underway at Kyoto University includes differentiation of iPSCs into dopamine-releasing neurons for transplantation into patients who are afflicted with Parkinson’s disease. University researchers are also working on generating a formulation of platelets that will assist with blood clotting. Dr. Shinya Yamanaka, who is credited with discovering iPSCs in 2006 and who shared the 2012 Nobel Prize in Medicine for the discovery, leads the existing iPSC research center at Kyoto University.
As such, the two founders of iPSC technology (Dr. Shinya Yamanaka and Dr. James Thomson) remain two of the most significant influencers in the iPSC sector. Recall that Dr. Shinya Yamanaka, who will be operating the iPSC therapy center scheduled to open in Japan in 2019, created the first successful generation of iPSCs in 2006, and in 2007, performed follow-up experiments in which his team transformed human adult cells into iPSC cells. Nearly simultaneously, a research group led by James Thomson at the University of Wisconsin-Madison accomplished the same feat of deriving iPSC lines from human somatic cells. James Thomson is both the Founder and current Chief Scientific Officer of Cellular Dynamics International (CDI), a leading supplier of human iPSC lines for purposes that include drug discovery, safety, stem cell banking, cellular safety, and more.
Landmark Events Create Market Opportunities
In 2009 ReproCELL, a company established as a venture company originating from the University of Tokyo and Kyoto University, was the first to make iPSC product commercially available with the launch of human iPSC-derived cardiomyocytes, which it called “ReproCario.” Other stem cell derived cardiomyocytes are now available commercially from Cellular Dynamics International, GE Healthcare, Cellectis, and others.
ReproCELL’s innovation in the area of iPSC commercialization has been driven in part by joint research relationships it established in 2003 with Tokyo University and in 2004 with Kyoto University, the eventual site of iPSC discovery in 2006. Since 2009, ReproCELL has expanded its line of iPSC reagents and iPSC-derived cell lines to include heart, liver, and nerve cells. The company primarily sells these products as research tools, although they also have the potential for use in toxicology and drug discovery applications. Currently, ReproCELL offers the following iPSC products:
• Research reagents optimized for human iPSC culture
• Human iPSC-derived cardiomyocytes, which launched in April of 2009 (the first iPSC product to be sold commercially)
• Human iPSC-derived neurons, launched in October of 2010
• Human iPSC-derived hepatocytes, launched in May of 2012
• Disease model cell generation using human iPS cell technologies
To date, ReproCELL has furthered its dominance in the area of iPSC products through a series of strategic acquisitions, including acquisition of Reinnervate, Stemgent, and BioServe Biotechnologies, all occurring in 2014.
Cellular Dynamics International (CDI) is another major market player in the iPSC sector. Similar to ReproCELL, CDI established its “foothold” on the iPSC industry early, being founded in 2004 by Dr. James Thomson at the University of Wisconsin-Madison, who in 2007 subsequently derived iPSC lines from human somatic cells for the first time ever (although the feat was also accomplished simultaneously by Dr. Shinya Yamanaka’s lab in Japan). CDI currently holds more than 800 patents, which gives it a strong competitive position within the marketplace.
CDI has been promoting adoption of iPSC technology by adapting its methods to fit into standard clinical practice through the creation of individual stem cell lines from a standard blood draw. In a landmark event, the company went public in July 2013 with a public offering that raised $43 million dollars, securing its position as the global leader in producing high-quality human iPSCs and differentiated cells in industrial quantities.
Then, in March of 2013, Cellular Dynamics International (CDI) and the Coriell Institute for Medical Research announced receipt of multi-million dollar grants from the California Institute for Regenerative Medicine (CIRM) for the creation of iPSC lines from 3,000 healthy and diseased donors. CIRM awarded CDI $16 million to create three iPSC lines for each of 3,000 healthy and diseased donors and awarded the Coriell Institute $10 million to set up and biobank the iPSC lines. The result will be the creation of the world’s largest human iPSC bank, an incredible feat.
Not surprisingly, Cellular Dynamics International has continued its innovation, announcing in February of 2015 that it would be manufacturing cGMP HLA “Superdonor” stem cell lines that will support cellular therapy applications through genetic matching. Currently, CDI has two HLA superdonor cell lines that provide a partial HLA match to approximately 19% of the population within the U.S., and it aims to expand its master stem cell bank by collecting more donor cell lines that will cover 95% of the U.S. population. The HLA superdonor cell lines were manufactured using blood samples, and used to produce pluripotent iPSC lines, giving the cells the capacity to differentiate into nearly any cell within the human body.
CDI also leads the iPSC market in terms of supporting drug development and discovery. For example, CDI’s “MyCell” products are created using custom iPSC reprogramming and differentiation methods, thereby providing biologically relevant human cells from patients with unique disease-associated genotypes and phenotypes. The company’s iCell and MyCell cells can also be adapted to screening platforms and are matched to function with common readout technologies. CDI’s products are also used for high-throughput screening, and have been used as supporting data for Investigational New Drug (IND) applications submitted to the Federal Drug Administration (FDA). While there are other companies participating in this area – including ReproCELL, Cellectis, ArunA Biomedical, and others – competitors are limited and CDI currently leads the way.
The California Institute for Regenerative Medicine (CIRM), an organization tasked with deploying $3 billion dollars in California tax money to support the translation of stem cell research into clinical therapies, has increasingly been favorable toward funding iPSC research projects with a clinical (“translational”) focus. In one example, the Parkinson’s Institute was awarded $6.5 million to support four separate research projects focusing on development of patient-specific iPSCs from individuals with Parkinson’s disease. Within a brief period of time, CIRM awarded $3 million to the Cedar-Sinai Medical Center for derivation of iPSCs from patients with inherited nerve disease and for research into the feasibility of transplanting these cells back into patients after genetic corrections have been applied. $1.3 million was next awarded to Stanford University to allow for creation of cardiomyocytes from iPSCs that can be used to explore causes of cardiovascular disease. Clearly, CIRM’s favorability toward funding iPSC research is gathering momentum.
Taken in aggregate, the iPSC market forces described above are creating incredible enthusiasm and commercial interest in the iPSC sector. Never before has there been such a rare combination of landmark events supporting development of iPSC tools, technologies, and importantly, therapies. Indeed, recent years have seen major advances in clinical research applications, production and differentiation technologies, and biobanking of iPSCs. There have also been major funding awards, large initial public offerings (IPOs), significant patent challenges, and more. For companies and investors competing within the iPSC marketplace, it is critical to understand these major market events and how they are shifting industry dynamics.
Induced Pluripotent Stem Cell Patent Challenges
The patent environment for iPSCs is complicated, and there are a number of restrictions on how the cells can be used for commercial purposes. 2015 has witnessed a historic patent challenge, one that is challenging the way that iPSCs are derived and utilized for commercial purposes. The 2015 patent challenge could rival in importance the infamous Wisconsin Alumni Research Foundation (WARF) dispute that pertained to patents surrounding the derivation of human embryonic stem cells (hESCs). Jeanne Loring of Scripps Research Institute, and two public interest groups, Consumer Watchdog and the Public Patent Foundation, challenged the WARF patents in 2006. To date, the WARF patent dispute has been the most defining patent dispute within the stem cell sector, although it was settled in U.S. courts between 2008 and 2010, appeals have continued through February of 2015.
In 2006, Drs. Yamanaka and Takahashi filed a U.S. patent claiming a method for creating iPSCs titled “Oct3/4, Klf4, c-Myc and Sox2 produce induced pluripotent stem cells,” which was issued as U.S. Patent No. 8,058,065 on November 15, 2011. While there have been other patents issued pertaining to the cellular reprogramming and creation of iPSCs, this patent is the dominant one within the iPSC sector and the most common one that limits commercial development of iPSC products.
Often called the “Yamanaka Patent,” this famous patent is being challenged by a group called “BioGatekeeper.” If the “Yamanaka Patent” challenge is successful, it could spur incredible innovation within the stem cell sector by allowing for lower cost creation of iPSC products, technologies, and therapies. Currently, most companies are forced to pay licensing fees to use the methodology described in the “Yamanaka Patent,” fees which are often prohibitively expensive.
In February of 2015, a long series of appeals to WARF’s embryonic stem cell patents ended with the U.S. Supreme Court decision to not hear appeals to the case being brought by Consumer Watchdog of Santa Monica, California, and Jeanne Loring, head of the stem cell program at Scripps Research Institute. The decision means that WARF will get to keep its patent rights for embryonic stem cells, discovered in 1998 by James Thompson, founder of CDI. Nonetheless, the challengers succeeded in preventing WARF from gaining rights over induced pluripotent stem cells, which would have given WARF nearly impenetrable control over pluripotent stem cells, as embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSC) are two of the most versatile stem cell types.= SHOW LESS READ MORE >
1. EXECUTIVE SUMMARY
1.1. Report Overview
1.2. Key Report Findings
2. PURPOSE OF REPORT
2.1. To Survey Recent iPSC Advancements
2.2. To Provide a Snapshot” of the Global iPSC Market
2.3. To Provide Market Analysis in the Context of Future Applications
2.4. To Assess Opportunities for Commercialization
2.5. To Identify Major Market Players and Assess the Competitive Environment
2.6. To Identify Existing and Emerging Trends
2.7. To Identify Critical Opportunities and Threats within the iPSC Market
3. TARGET DEMOGRAPHIC
4. SCOPE OF iPSC MARKET
4.5. Adjunct Products and Services
5. RESEARCH METHODOLOGY
5.1. Input Sources
5.2. Research & Analysis Methodologies
5.3. Criteria for Identifying iPSC Market Leaders
5.3.1. Size of Company
5.3.2. Type of Product/Services
5.3.3. Degree of Specialization
5.3.4. Patent Positions
5.3.5. Business Reputation
5.3.6. Financial Strength
6. TECHNICAL OVERVIEW OF iPSCs
6.2. Discovery of iPSCs
6.3. Advantages & Disadvantages of iPSCs
7. TIMELINE OF KEY iPSC EVENTS
7.1. Production of iPSCs (Key Events)
- (TIMELINE) Production of Induced Pluripotent Stem Cells
7.2. Full Timeline of Events
- (TABLE) Key Events in the Discovery and Evolution of iPSC Products
8. LEVELS OF REPAIR INDUCIBLE WITH iPSCs
- (TABLE) Four Levels of Repair Inducible with iPSCs
8.1. Gene-Level Repair (Gene Therapy)
- (TABLE) Examples of Gene-Level Repair with iPSCs
8.2. Cellular-Level Repair (Cell Replacement Therapy)
8.3. iPSC-derived Blood Products
8.4. Whole Tissue Repair (Organ Replacement Therapy)
9. iPSC INDUSTRY CONDITIONS
9.1. Origin of the iPSC Market
9.2. Therapeutic Applications of Induced Pluripotent Stem Cells
9.3. Landmark Events Create Market Opportunities
9.4. iPSC Patent Challenges
9.5. Four Primary Areas of Commercialization
- (IMAGE) Four Primary Areas of Commercialization for iPSCs
9.6. Rapidly-Evolving Market Opportunities for iPSCs
9.7. Market Leaders by Area of Commercialization
9.8. End-User Perspective of iPSC Scientists
9.9. Summary of iPSC Industry Findings
10. RESEARCH APPLICATIONS FOR iPSCs
10.1. Drug Development & Discovery
10.1.1. Traditional Drug Development & Discovery
10.1.2. Disease Modeling
- (IMAGE) Disease Modeling with Induced Pluripotent Stem Cells
10.2. Cell Therapy
10.3. Toxicology Screening
10.4. Stem Cell Biobanking
10.5. Breakdown of iPSC Research, By Applied Research Application
- (TABLE) Percent of iPSC Research, By Applied Research Application
11. CHARACTERIZATION OF iPSC RESEARCH ADVANCES
11.1. Methods of Making iPSCs – Comparison of Methods by Downstream Application
- (TABLE) Methods for Making iPSCs - Comparison of Methods by Downstream Application
11.2. Summary of iPSC Derivation Methods, by Cell Type
- (TABLE) Summary of iPSC Derivation Methods, by Cell Type
11.3. Diseases in Which iPSCs Have Been Derived from Patients
- (TABLE) Diseases in which iPSCs have Been Derived from Patients
12. COMPANIES SELLING iPSC RESEARCH PRODUCTS
12.1. List of Companies
- (TABLE) List of Companies Offering iPSC Research Products
12.2. Rate of Entrants, by Year
- (TABLE) Number of Companies Offering iPSC Research Products, by Year
- (GRAPH) Number of Companies Offering iPSC Research Products, by Year
13. iPSC RESEARCH PRODUCTS MARKET
13.1. iPSC Research Product Categories
- (TABLE) Induced Pluripotent Stem Cell (iPSC) Product Categories
13.2. Functionally-Defined Categories
- (TABLE) Functionally-Defined Induced Pluripotent Stem Cell (iPSC) Product Categories
13.3. iPSC Market Share, by Functional Product Category
- (TABLE) iPSC Market Share Breakdown, by Functional Product Category
- (CHART) iPSC Market Share Breakdown, by Functional Product Category
14. COMPANIES DEVELOPING iPSC THERAPIES
14.1. Commercial Entities
14.1.1. Cellular Dynamics International, Owned by FujiFilm Holdings (Industrial-Scale Production of iPSCs)
14.1.3. Fate Therapeutic
14.1.5. Ocata Therapeutics (Previously “Advanced Cell Technology”)
14.2. Non-Commercial Organizations Developing/Supporting iPSC Therapy Development
14.2.1. RIKEN Center
14.2.2. Kyoto University
14.2.3. California Institute for Regenerative Medicine (CIRM)
- (TABLE) Amount of Grant Funding by CIRM for iPSC Production and Biobanking
15. STRATEGIC COLLABORATIONS FOR DEVELOPMENT OF iPSC PRODUCTS
15.1. Life Technologies, Owned by Thermo Fisher Scientific
15.2. Lonza Group AG
15.3. EMD Millipore
15.4. Sigma Aldrich
15.5. Roslin Cells, Ltd.
15.6. ArunA Biomedical
15.7. CeeTox and Cellular Dynamics International (Owned by Fujifilm Holdings)
16. MARKET SIZE DETERMINATIONS & PROJECTIONS
16.1. Definition: Induced Pluripotent Stem Cell (iPSC) Market
16.2. Experimental Approach
16.3. Induced Pluripotent Stem Cell Market – Annual Market Size
16.4. Market Size Determination, by Product Area: iPSC Products, Services, Technologies, Therapies, and Adjunct Products and Services
- (TABLE) iPSC Market Share Breakdown, by Product Area
- (GRAPH) iPSC Market Share Breakdown, by Product Area
16.5. Market Size Projections
16.5.1. Product Life Cycle Stage
- (TABLE) Stem Cell Product Life Cycle Chart
16.5.2. Five-Year Market Size Projections: iPSC Products, Services, & Technologies Market (2015-2019)
- (TABLE) Induced Pluripotent Stem Cell (iPSC) Market, 5-Year Projections
- (CHART) Induced Pluripotent Stem Cell (iPSC) Market, 5-Year Projections
17. MARKET TREND ANALYSIS
17.1. Clinical Trial Rate Analysis
17.1.1. Clinical Trial Rate, by Year
- (TABLE) Number of iPSC Clinical Trials, by Year
- (GRAPH) Number of iPSC Clinical Trials, by Year
17.1.2. Clinical Trial Rate, by Region
- (TABLE) iPSC Clinical Trial, by Region of the World
- (IMAGE) iPSC Clinical Trials, by Region of the World
17.1.3. iPSC Clinical Trial Sponsors
- (TABLE) Top iPSC Clinical Trial Sponsors
17.1.4. “Pretrial Clinical Research” in Japan (First Celular Therapy Study)
17.2. Grant Rate Analysis
- (TABLE) Number of iPSC Grant Rates, by Year (REPORTer Analysis)
- (IMAGE) Number of iPSC Grant Rates, by Year
17.3. iPSC Patent Rate Analysis
17.3.1. Role of Patent Analysis for Competitive Intelligence
17.3.2. Induced Pluripotent Stem Cell (iPSC) Patent Landscape
17.3.3. Methodology of Induced Pluripotent Stem Cell (iPSC) Patent Analysis
17.3.4. Current iPSC Patents (Including Inventor, Assignee, Assignee Location, and Date)
- (TABLE) iPSC Patents in U.S. Patent and Trademark Office (USPTO) Full-Text & Image Database
17.3.5. Breakdown of iPSC Patents, by Inventor
- (TABLE) Breakdown of iPSC Patents, by Inventor
- (GRAPH) Breakdown of iPSC Patents, by Inventor
- (TABLE) Full List of iPSC Patent Inventors
17.3.6. Breakdown of iPSC Patents, by Assignee
- (TABLE) Breakdown of iPSC Patents, by Assignee
- (GRAPH) Breakdown of iPSC Patents, by Assignee
- (TABLE) Full List of iPSC Patent Assignees
17.3.7. Breakdown of iPSC Patents, by Assignee Location
- (TABLE) Breakdown of iPSC Patents, by Assignee Location
- (GRAPH) Breakdown of iPSC Patents, by Assignee Location
- (TABLE) Full List of iPSC Patent Assignee Locations
- (TABLE) Breakdown of iPSC Patents, by Country of Assignee
- (GRAPH) Breakdown of iPSC Patents, by Country of Assignee
17.4. Scientific Publication Rate Analysis
- (TABLE) Analysis of iPSC Publications, by Year
- (GRAPH) Analysis of iPSC Publications, by Year
- (TABLE) Recent Year-over-Year Growth Rates in iPSC Scientific Publications
18. MARKETING ANALYSIS: PRICE-PER-CLICK FOR iPSC-RELATED SEARCH TERMS
18.1. Purpose of Analysis
18.2. Exact iPSC Search Terms
18.2.1. U.S. Analysis
- (IMAGE) Google Adwords Price-Per-Click iPSC Terms (Geography: USA Only)
18.2.2. Global Analysis
- (IMAGE) Google Adwords Price-Per-Click iPSC Terms (Geography: Worldwide)
18.3. Derivative Search Terms
18.3.1. U.S. Analysis
- (IMAGE) Search Terms Containing iPSC Search Phrases (Geography: USA Only)
18.3.2. Global Analysis
- (IMAGE) Search Terms Containing iPSC Search Phrases (Geography: Worldwide)
18.4. Summary of Findings
- (TABLE) Google Adwords Pay-Per-Click Prices for iPSC Related Search Terms
19. SCIENTIST PANEL: DETAILED END-USER SURVEY
19.1. Survey Overview
19.2. Characterization of Market Survey Respondents
19.2.1. Geographic Distribution of Respondents
- (GRAPH) Geographic Distribution of Respondents
19.2.2. Respondent Breakdown by Industry Affiliation
- (GRAPH) Respondent Breakdown by Industry Affiliation
19.2.3. Breakdown of Respondents by Duration of iPSC Research Activity
- (GRAPH) Analysis of Time Duration for iPSC Research
19.3. Survey Findings
19.3.1. General Scope
Question 1 – Do you perform basic or applied research?
Question 2 – What is your preferred species source for iPSCs utilized in your research?
Question 3 – What kind of culture do you use for your iPSC research (feeder-dependent vs. feeder-free culture)?
Question 4 – How often do you purchase iPSC products?
Question 5 – How much do you spend annually on iPSC products?
Question 6 - How important is price on your iPSC purchasing decisions?
Question 7 – What is the primary source of your iPSC research funding?
19.3.2. Comparison of Providers / Brand Preferences
Question 1 – What is most influential in your choice to purchase from a specific provider of iPSC products?
Question 2 – What is your primary method for making iPSC product purchasing decisions?
Question 3 – What is the number of different suppliers from whom you have bought iPSC products?
19.3.3. Marketing Assessment
Question 1 – What is your preferred provider for purchasing iPSC products?
Question 2 – To what types of iPSC advertising have you been exposed?
Question 3 – When purchasing iPSC products, have you ever responded to the following?
Question 4 – Which of the following phrases would be “most influential” to cause you to buy?
19.4. Terms Used in Online Product Search
- (TABLE) Write-In Responses, Search Terms Used to Find iPSC Products Online
20. STRATEGIES FOR ACCESSING THE iPSC MARKETPLACE
20.1. Awards Given to iPSC Researchers
- (TABLE) Awards Given to iPSC Researchers
20.2. iPSC Industry Influencers
20.2.1. Robert J. Palay, CEO of Cellular Dynamics International (Owned by Fujifilm Holdings Corp.)
20.2.2. Masayo Takahashi of the RIKEN Center for Developmental Biology (Kobe, Japan)
20.2.3. Prof. Jun Takahashi at CiRA for Developmental Biology (Kobe, Japan)
20.2.4. Chikafumi Yokoyam, CEO of ReproCELL, Inc. (Kanagawa, Japan)
20.2.5. Dr. Robert Lanza, CSO of Ocata Therapeutics (previously “Advanced Cell Therapeutics)
20.3. Events of Interest
- (TABLE) Stem Cell Conferences and Events for 2015-16
20.4. iPSC Core Facilities
- (TABLE) iPSC Core Facilities
APPENDIX A – Properties and Characteristics of Induced Pluripotent Stem Cells
APPENDIX B – iPSC Patents Held by Cellular Dyamics International (Owned by Fujifilm Holdings)
APPENDIX C – Current Clinical Trials Involving iPSCs (ClinicalTrials.gov Analysis)
APPENDIX D – Full List of iPSC Clinical Trial Sponsors (ClinicalTrials.gov Analysis)
APPENDIX E – List of Grants that Contain iPSC Search Terms within the Title
(2006 to Present; RePORTer Tool)
APPENDIX F – NIH Center for Regenerative Medicine (CRM) iPSC Stem Cell Line – Control, Reporter, & Differentiated Lines
Four Primary Areas of Commercialization
There are currently four major areas of commercialization for induced pluripotent stem cells, as described below:
1) Drug Development & Discovery: iPSCs have the potential to transform drug discovery by providing physiologically relevant cells for compound identification, target validation, compound screening, and tool discovery.
2) Cellular Therapy: iPSCs can be used for cellular therapy applications, including autologous transplantation and potentially gene therapy. The purpose of cellular therapy is to reverse injury or disease.
3) Toxicology Screening: iPSCs can be used for toxicology screening, which is the use of stem cells or their derivatives (tissue-specific cells) to assess the safety of compounds or drugs within living cells.
4) Stem Cell Biobanking: The goal of stem cell biobanking is to create a repository of stem cell specimens, including source tissue from which iPSCs can be derived, differentiated cell types produced from iPSCs, and disease tissues produced from iPSCs. Large-scale stem cell repositories provide researchers with the opportunity to investigate a diverse range of conditions using iPSC derived cells produced from both healthy and diseased donors. Importantly, these repositories can also greatly expand the capacity for global research and collaboration.
Each of these areas will be vital to the future commercialization of iPSCs. At this time, Cellular Dynamics International (CDI) is best positioned to excel in all areas, largely due to its participation in creating the world’s largest ever iPSC bank (funded through a CIRM grant and in partnership with Coriell Institute for Medical Research), and its ability to produce clinical grade iPSCs in industrial quantities. However, there are other industry competitors that could be well-positioned to control one of these market areas, such as ReproCELL’s strong positioning in the iPSC reagent space. Therefore, smaller industry players may choose to specialize and compete in one of these market areas, with the potential for later expansion into other areas.
Rapidly-Evolving Market Opportunities for iPSCs
Since the discovery of iPSCs a large and thriving research product market has grown into existence, largely because the cells are completely non-controversial and can be generated directly from adult cells. Today, the number of iPSC products sold worldwide is increasing with double-digit growth. In addition, 22% of all stem cell researchers now self-report having used iPSCs within a research project. It is clear that iPSCs represent a lucrative product market, but methods for commercializing this cell type are still being explored, as clinical studies investigating iPSCs continue to increase in number.
Currently, nearly all clinical studies involving iPSCs are for the creation and evaluation of iPSC lines from specific patient populations in order to determine if these cell lines could be a good model for a disease of interest in that patient population. (See ClinicalTrials.gov for a current list of these trials or view the “Clinical Trial Trend Analysis” section of this report for a full discussion of findings.) However, the first clinical study involving transplant of iPSCs into humans was initiated in August 2013.
Market Leaders Have Begun to Emerge in All Areas of iPSC Development
Finally, market leaders have begun to emerge in all areas of iPSC development, including:
- Drug Development and Discovery: Cellular Dynamics International (CDI) in Madison, Wisconsin
- Cellular Therapy (Therapeutic Applications of iPSCs): RIKEN Center, in Kobe, Japan, and Kyoto University in Kyoto, Japan
- Stem Cell Biobanking: Cellular Dynamics International (CDI) in Madison, Wisconsin
- iPSC Research Products: ReproCELL in Kanagawa, Japan; Thermo Fisher Scientific in Rockville, MD; STEMCELL Technologies in Vancouver, Canada; and BD Biosciences in San Jose, California
End-User Survey of iPSC Scientists
A distinctive feature of this report is an end-user survey of 273 researchers (131 U.S. / 143 International) that identify as having induced pluripotent stem cells as a research focus. These survey findings reveal iPSC researcher needs, technical preferences, key factors influencing buying decisions, and more. The findings can be used to make effective product development decisions, create targeted marketing messages, and produce higher prospect-to-client conversion rates.
Key Report Findings Include:
- Metrics, Timelines, Tables, and Graphs for the iPSC Industry
- Trend Rate Data for iPSC Grants, Clinical Trials, and Scientific Publications
- Analysis of iPSC Patent Environment, including Key Patents and Patent Trends
- Market Segmentation
- 5-Year Market Size Projections (2015-2019)
- Market Size Estimations, by Market Segment
- Updates on Crucial iPSC Industry and Technology Trends
- Analysis of iPSC Market Leaders, by Market Segment
- Geographical Assessment of iPSC Innovation
- SWOT Analysis for the iPSC Sector (Strengths, Weaknesses, Opportunities, Threats)
- Preferred Species for iPSC Research
- Influential Language for Selling to iPSC Scientists
- Breakdown of the Marketing Methods, including Exposure and Response Rates
- And Much More
Summary of Findings
Induced pluripotent stem cells represent a promising tool for use in the reversal and repair of many previously incurable diseases. The cell type represents one of the most promising advances discovered within the field of stem cell research during the past decade, making this a valuable industry report for both companies and investors to claim in order to optimally position themselves to sell iPSC products. To profit from this lucrative and rapidly expanding market, you need to understand your key strengths relative to the competition, intelligently position your products to fill gaps in the market place, and take advantage of crucial iPSC trends.
The content and statistics contained in this report were compiled using a broad range of sources. These sources include:
- Stem Cell Grant Funding Database (NIH database)
- Stem Cell Patent Database (USPTO)
- Stem Cell Clinical Trial Database (ClinicalTrials.gov)
- Stem Cell Scientific Publication Database (PubMed)
- Stem Cell Product Launch Announcements (Trade Journals, Google News)
- Stem Cell Industry Events (Google News, Google Alerts)
- Stem Cell Company News (SEC Filings, Investor Publications)
- International Surveys (Electronically Distributed End-User Surveys)
- And More
This report employs varied techniques for deriving market research. The following constitute the basis for our Research & Analysis:
- Preliminary Research:Extensive secondary research based on preliminary market evaluations
- Fill-Gap Research:Selectively sampled and focused primary research as a fill-gap strategy
- Historic Analysis – Primary Products:Comprehensive analysis of all data for each primary product market
- Historic Analysis – End-user Markets:Historic analysis of all end-user industries/markets, requiring technology and market evaluations, growth projections, and market size estimation of end-user markets
- Historic Supply Chain/Raw Materials Analysis:Comprehensive analysis of data for each primary market segment
- Data Consolidation:Merging historic end-user market data to yield consolidated primary market data
- Cross Linking:Comparing primary market data (historic) with resulting end-user consolidated market data and calculating the variance in percentages between data sets by year
- Variance Determination:Placing a median figure for each year with a tolerance range equal to twice the variance percentage and recording the resulting numbers
- Projections:Projecting forward end-user markets based upon historic metrics, technology and market trends, and primary research from the marketplace
- Variance Factorization:Consolidation of projected end-user market data to yield derived primary market data. The data is adjusted to the historic variance determinations, as above. The resulting data is verified by confirmatory primary research
- Confirmatory Primary Research:Presenting resulting data from companies or individuals participating as research partners. Variations from derived data are adjusted to reflect primary research based consensus
- Electronically Based End-User Surveys:Distribution of electronically based end-user surveys to a panel of industry representatives working within market segment(s) of interest. Statistical filtering and analysis is performed on collected user-response data
- ArunA Biomedical
- California Institute for Regenerative Medicine (CIRM)
- CeeTox and Cellular Dynamics International (Owned by Fujifilm Holdings)
- Cellular Dynamics International, Owned by FujiFilm Holdings (Industrial-Scale Production of iPSCs)
- EMD Millipore
- Fate Therapeutic
- Kyoto University
- Life Technologies, Owned by Thermo Fisher Scientific
- Lonza Group AG
- Ocata Therapeutics (Previously “Advanced Cell Technology”)
- RIKEN Center
- Roslin Cells, Ltd.
- Sigma Aldrich