Since the discovery of induced pluripotent stem cell (iPSC) technology 15 years ago, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated.
Today, methods of commercializing iPSCs include:
Cell 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.
Proliferating Applications for iPSCs
Other applications of iPSCs include their use as research products, as well as their integration into 3D bioprinting, tissue engineering, and increasingly, clean meat production. Plus, technology allowing for the mass-production and differentiation of iPSCs in industrial-scale bioreactors is advancing at breakneck speed, with companies like TreeFrog Therapeutics leading the charge.
In recent years, iPSC-derived cells have increasingly been used within preclinical testing and early stage-stage trials. The first clinical trial using iPSCs started in 2008, and today, that number has surpassed 100 worldwide. Most of these clinical trials 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.
However, there are companies forging forward with clinical trials involving iPSC-derived cell therapeutics, such as Cynata Therapeutics (and its partner Fujifilm), Healios K.K. (in collaboration with Sumitomo Dainippon Pharma), and Fate Therapeutics.
Other companies involved with the commercialization of iPSC-derived cell therapeutics include:
- Aspen Neuroscience
- Avery Therapeutics (in collaboration with I Peace, Inc.)
- Bluerock Therapeutics (acquired by Bayer in 2019)
- Century Therapeutics
- Citius Pharmaceuticals
- Heartseed Inc.
- Hopstem Biotechnology
- I Peace Inc.
- Neurophth Biotechnology Ltd.
- Novo Nordisk
- Platelet BioGenesis
- RheinCell Therapeutics
- Semma Therapeutics
- Shoreline Biosciences
- Stemson Therapeutics
- U.S. NIH
- Vita Therapeutics
Of course, there are numerous physician-led studies employing human iPSC-derived cell types that have been undertaken as well, including ones by Keio University, Kyoto University Hospital, Osaka University, and RIKEN.
Within the research products realm, the number of market competitors has surged in recent years, although the following industry stalwarts continue to defend their position as iPSC market leaders: Fujifilm Cellular Dynamics, Inc.(USA/Japan), REPROCELL (Japan), Evotec (Europe), Ncardia (Europe), and Axol Bioscience (Europe).
Finally, the world’s largest research supply companies are also commercializing a plethora of iPSC-related products. Examples of these market leaders include Lonza, BD Biosciences, Thermo Fisher Scientific, Merck, Takara Bio, and others.