Induced pluripotent stem cells (iPS cells or iPSCs) are laboratory-made pluripotent stem cells that are produced using non-controversial adult cells. They are derived from somatic cells through reprogramming and turning on the expression of specific pluripotency genes. This reprogramming can be achieved using different techniques with varying efficiencies.
iPSC technology was first discovered in 2006 by Shinya Yamanaka’s lab in Kyoto, Japan. He and his team introduced four specific genes encoding transcription factors and converted adult cells into pluripotent stem cells. Dr. James Thomson at the University of Wisconsin-Madison derived iPSC lines from human somatic cells for the first time in 2007.
Today, iPSCs are being explored for applications related to basic and applied research, drug screening, toxicological studies, disease modeling, cell therapy, personalized medicine, cultured meat production, and beyond. The somatic cells used for reprogramming commonly include skin cells and blood cells. iPSCs are also leveraged by scientists to learn more about disease onset and progression, as well as to develop and test new drugs and therapies.
Specifically, methods of commercializing induced pluripotent stem cells (iPSCs) include:
- Cellular Therapy: iPSCs are being explored in a diverse range of cell therapy applications for the purpose of reversing injury or disease.
- Disease Modelling: By generating iPSCs from patients with disorders of interest and differentiating them into disease-specific cells, iPSCs can effectively create disease models “in a dish”.
- Drug Development and Discovery:iPSCs have the potential to transform drug discovery by providing physiologically relevant cells for compound identification, target validation, compound screening, and tool discovery.
- Personalized Medicine: The use of techniques such as CRISPR enable precise, directed creation of knock-outs and knock-ins (including single base changes) in many cell types. Pairing iPSCs with genome editing technologies is adding a new dimension to personalized medicine.
- Toxicology Testing: 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.
- Research Tools: iPSCs and iPSC-derived cell types are being widely used within basic and applied research applications.
- Other Applications: Other applications of iPSCs include their integration into 3D bioprinting, tissue engineering, and clean meat production.
iPSC-Derived Cellular Therapeutics
Since the discovery of iPSCs in 2006, it took only seven years for the first iPSC-derived cell product to be transplanted into a human patient in 2013. Since then, iPSC-derived cells have been used within a rapidly growing number of preclinical studies, physician-led studies, and clinical trials worldwide. There are also over 100 clinical trials underway that do not involve the transplant of iPSCs into humans, but rather, the creation and evaluation of iPSC lines for clinical purposes. Within these trials, iPSC lines are created from specific patient populations to determine if these cell lines could be a good model for a disease of interest.
2013 was a landmark year because it saw the first cellular therapy involving the transplant of iPSCs into humans initiated at the RIKEN Center in Kobe, Japan. Led by Dr. Masayo Takahashi, it investigated the safety of iPSC-derived cell sheets in patients with macular degeneration. In another world first, Cynata Therapeutics received approval in 2016 to launch the first formal clinical trial of an allogeneic iPSC-derived cell product (CYP-001) for the treatment of GvHD. CYP-001 is a iPSC-derived MSC product. In this historic trial, CYP-001 met its clinical endpoints and produced positive safety and efficacy data for the treatment of steroid-resistant acute GvHD.
Given this early success, Cynata is has advanced its iPSC-derived MSCs into Phase 2 trials for the severe complications associated with COVID-19, as well as GvHD and critical limb ischemia (CLI). It is also undertaking an impressive Phase 3 trial that will utilize Cynata’s iPSC-derived MSC product, CYP-004, in 440 patients with osteoarthritis (OA). This trial represents the world’s first Phase 3 clinical trial involving an iPSC-derived cell therapeutic product and the largest one ever completed. Not surprisingly, the Japanese behemoth FUJIFILM has been involved with the co-development and commercialization of Cynata’s iPSC-derived MSCs through its 9% ownership stake in the company.
iPSC Market Competitors
Many market competitors are also commercializing iPSC-derived products for use in drug development and discovery, disease modeling, and toxicology testing. Across the broader iPSC sector, FUJIFILM CDI (FCDI) is one of the largest and most dominant players. Cellular Dynamics International (CDI) was founded in 2004 by Dr. James Thomson at the University of Wisconsin-Madison, who in 2007 derived iPSC lines from human somatic cells for the first time. The feat was accomplished simultaneously by Dr. Shinya Yamanaka’s lab in Japan. FUJIFILM acquired CDI in April 2015 for $307 million. Today, the combined company is the world’s largest manufacturer of human cells created from iPSCs for use in research, drug discovery and regenerative medicine applications.
Another iPSC specialist is ReproCELL, a company that was established as a venture company originating from the University of Tokyo and Kyoto University in 2009. It became the first company worldwide to make iPSC products commercially available when it launched its ReproCardio product, which are human iPSC-derived cardiomyocytes. Within the European market, the dominant competitors are Evotec, Ncardia, and Axol Bioscience.
Headquartered in Hamburg, Germany, Evotec is a drug discovery alliance and development partnership company. It is developing an iPSC platform with the goal to industrialize iPSC-based drug screening as it relates to throughput, reproducibility, and robustness. Today, Evotec’s infrastructure represents one of the largest and most advanced iPSC platforms globally.
Ncardia was formed through the merger of Axiogenesis and Pluriomics in 2017. Its predecessor, Axiogenesis, was founded in 2011 with an initial focus on mouse embryonic stem cell-derived cells and assays. When Yamanaka’s iPSC technology became available, Axiogenesis became the first European company to license it in 2010. Today, the combined company (Ncardia) is a global authority in cardiac and neural applications of human iPSCs.
Founded in 2012, Axol Bioscience is a smaller but noteworthy competitor that specializes in iPSC-derived products. Headquartered in Cambridge, UK, it specializes in human cell culture, providing iPSC-derived cells and iPSC-specific cell culture products.
In the realm oforder their mutation of choice in bit.bio’s opti-ox™ powered human-iPSC derived cells, receiving consistent, defined, cryopreserved products that can be incorporated into drug discovery workflows. Importantly, these disease model cells help scientists generate data in a human context, which supports improved translatability in early stage drug discovery. Alongside other iPSC companies, bit.bio has benefited from the FDA Modernisation Act 2.0, which permits cell based assays as an alternative to animal testing for the purposes of drug and biological product applications.
Of course, the world’s largest research supply companies are also commercializing a diverse range of iPSC-derived products and services. Examples of these companies include Lonza, BD Biosciences, Thermo Fisher Scientific, Merck, Takara Bio, and countless others. In total, at least 80 market competitors now offer a diverse range of iPSC products, services, technologies, and therapeutics.
Global iPSC Industry
Recently, BioInformant released a global strategic report that reveals all major iPSC market competitors worldwide, including their core technologies, strategic partnerships, and products under development. It covers the current status of iPSC research, biomedical applications, manufacturing technologies, patents, and funding events, as well as all known trials for the development of iPSC-derived cell therapeutics worldwide.
Importantly, it profiles leading market competitors worldwide and presents a comprehensive market size breakdown for iPSCs by Application, Technology, Cell Type, and Geography (North America, Europe, Asia/Pacific, and Rest of World). It also presents total market size figures with projected growth rates through 2030.
You can view the Executive Summary and Table of Contents here.
Founded in 2006, BioInformant is a U.S. market research firm that attracts an online readership of nearly one million viewers per year. As the first and only market research firm to specialize in the stem cell industry, BioInformant’s research has been cited by the Wall Street Journal, Xconomy, and Vogue Magazine. Headquartered near Washington, DC, BioInformant is strategically positioned to be near the National Institutes of Health (NIH), U.S. FDA, Maryland Biotech Corridor, and policy makers on Capitol Hill.