Recent months have seen the first iPSC clinical trial in humans, creation of the world’s largest iPSC biobank, major funding awards, a historic challenge to the “Yamanaka Patent”, a Supreme Court ruling affecting industry patent rights, announcement of an iPSC cellular therapy clinic scheduled to open in 2019, and much more. Furthermore, iPSC patent dominance continues to cluster in specific geographic regions, while clinical trial and scientific publication trends give clear indicators of what may happen in the industry in 2015 and beyond.
Is it worth it to you to get informed about rapidly evolving market conditions and identify key industry trends that will give you an advantage over your competition?
This global strategic report is produced for:
- Management of Stem Cell Product Companies
- Management of Stem Cell Therapy Companies
- Stem Cell Industry Investors
It is designed to increase your efficiency and effectiveness in:
- Commercializing iPSC products, technologies, and therapies
- Making intelligent investment decisions
- Launching high-demand products
- Selling effectively to your client base
- Increasing revenue
- Taking market share from your competition
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
In recent 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.
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 thecreation 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.
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.
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.
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