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 get informed about rapidly-evolving market conditions and identify key industry trends that will give you an advantage over your competition?
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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.
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.
Over the next few years, iPSC research advances accelerated exponentially. 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 was implanted with retinal tissue generated using iPSCs derived from her own skin cells. Also, 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.
Landmark Events Create Market Opportunities for iPSCs
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.” ReproCELL has since furthered its dominance in the area of iPSC products through a series of strategic acquisitions, including acquisition of Reinnervate, Stemgent, and BioServe Biotechnologies.
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, and 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.
Finally, Cellular Dynamics International (CDI) and the Coriell Institute for Medical Research received multi-million dollar grants ($16 million total) from the California Institute for Regenerative Medicine to create 3,000 iPSC lines from healthy and diseased donors, a result will create the world’s largest human iPSC bank.
In aggregate, there is incredible enthusiasm and investment driving growth of the iPSC sector, with recent years seeing major advances in clinical research applications, production and differentiation technologies, and biobanking of iPSCs. There have 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.
Four Primary Areas of Commercialization
There are currently four major areas of commercialization for induced pluripotent stem cells:
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.
While competition is fierce in each of these areas, with the market leaders for each category are shown below:
End-User Survey of iPSC Researchers
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.
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:
In summary, induced pluripotent stem cells represent a promising tool for use in the reversal and repair of many previously incurable diseases. 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: