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~ Induced Pluripotent Stem Cells, iPSC's ~
2015 Global Strategic Report

Therapeutic Applications of Induced Pluripotent Stem Cells (iPSCs)

While there has been continued excitement at the prospect of what 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 (now Ocata Therapeutics, a wholly owned subsidiary of Astellas Pharma) discovered several problematic issues while conducting experiments for the purpose of applying for U.S. FDA approval to use iPSCs for therapeutic applications. Concerns such as premature cell death, mutation into cancer cells, and low proliferation rates were some of the problems that surfaced.

Over the next few years, iPSC research advances accelerated exponentially, with a momentous milestone being achieved when iPSC-derived cells were first tested in humans during a clinical trial in Japan. 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. While the trial was later suspended due to safety concerns, RIKEN announced in June 2016 that the trial will be resumed.

Additionally, 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.

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 products commercially available with the launch of its human iPSC-derived cardiomyocytes, which it called “ReproCario. Other stem cell derived cardiomyocytes are now available commercially from Cellular Dynamics International, Axiogenesis, GE Healthcare, Cellectis, and range of 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.

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. The result will be the creation of the world’s largest human iPSC bank, an incredible feat.

A European leader within the iPSC market is Axiogenesis. Founded in 2001 and headquartered in Cologne, Germany, Axiogenesis initially focused on generating mouse embryonic stem cell derived cells and assays. After Yamanaka’s groundbreaking iPSC technology became available, Axiogenesis was the first European company to license and adopt Yamanaka’s iPSC technology in 2010. Now Axiogenesis specializes in human induced pluripotent stem cell products, including in vitromodels of healthy and diseased cell types and tissues. In 2014 Axiogenesis founded its American subsidiary in Philadelphia, PA.

Axiogenesis’ current focus lies on preclinical drug discovery and drug safety through the development of functional assays using human neuronal and cardiac cells, although it is expanding into new areas. Its flagship offering is its Cor.4U human cardiomyocyte product family, including cardiac fibroblasts. Cor.4U cells are used in applications for single cell analysis to high-throughput screening (HTS) in early cardiac safety and safety assessment, as well as in cardiovascular drug development.

Of course, there are other companies participating in this area too, including Axol Bioscience, ReproCELL, Cellectis, ArunA Biomedical, and many others.

Four Primary Areas of Induced Pluripontent Stem Cell (iPSC) Commercialization

There are 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 are being explored in cellular therapy applications for purposes of reversing 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:  iPSC repositories provide researchers with the opportunity to investigate a diverse range of conditions using iPSC-derived cell types produced from both healthy and diseased donors.

Rapidly-Evolving Market Opportunities for Induced Pluripotent Stem Cells (iPSCs)

Since the discovery of iPSCs a large and thriving research product market has grown into existence, largely because the cells are 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.) However, the first clinical study involving transplant of iPSCs into humans was initiated in August 2013, as mentioned above.

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