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Since the discovery of induced pluripotent stem cells (iPSCs) in 2006, a large and thriving research products market has emerged, largely because the cells are non-controversial and can be generated directly from adult cells. It is clear that iPSCs represent a lucrative market segment, because methods for commercializing this cell type are expanding every year and clinical studies investigating iPSCs are swelling in number.
Therapeutic applications of iPSCs are also emerging. In 2013, RIKEN launched the world’s first study of an iPSC-derived cell therapy product, treating the first patient in 2014 with iPS cell-derived retinal sheets. By 2016, Cynata Therapeutics launched the world’s first formal trial of an allogeneic iPSC-derived cell product (CYP-001) for the treatment of GvHD. Cynata is now testing these cells in the treatment of GvHD, critical limb ischemia (CLI) and osteoarthritis (OA).
Within United States, the NIH has undertaken the first U.S. clinical trial of an iPSC-derived therapeutic. This Phase I/IIa clinical trial will involve 12 patients with advanced-stage geographic atrophy of the eye. Riding the momentum within the immunotherapy field, Fate Therapeutics is developing iPSC-derived NK and CAR-T cell product candidates.
Numerous studies are also underway in Japan, with iPSC-derived cell therapeutics being used for the treatment of Parkinson’s disease, heart disease, spinal cord injury, and platelet production.
You can see a full list of biotech and pharma companies who are developing iPSC-derived cellular therapeutics at this link.
iPSC Cell Applications in 2024
While the therapeutic progress is exciting, other methods of commercializing iPS cells have also expanded exponentially. Today, there are at least 13 distinct iPS cell applications, which include:
1. Research Products
Dozens of market competitors provide iPSC specific tools to scientists worldwide, including human iPSC lines and differentiated cells types, as well as optimized reagents, protocols, differentiation kits and more.
2. 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.
3. Cellular Therapy
iPSCs are being explored in a diverse range of cell therapy applications for the purpose of reversing injury or disease.
4. 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.
5. 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 has added a new dimension to personalized medicine.
6. 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.”
7. Stem Cell Banking
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.
8. Tissue Engineering
iPSCs can be seeded onto scaffolds made from biocompatible materials. These scaffolds mimic the structure and properties of the target tissue and can provide a supportive environment for cell growth and differentiation.
9. Organoid Production
iPS cells can be coaxed to self-organize into three-dimensional structures called organoids, which mimic the structure and function of organs. Organoids can be used for studying organ development, modeling diseases, and testing drugs.
10. Gene Editing
iPS cells can be genetically modified using techniques like CRISPR-Cas9 to correct disease-causing mutations or introduce specific genetic changes. These edited iPS cells can then be differentiated into the desired cell type for transplantation or disease modeling.
11. Cultured (“Clean”) Meat Production
iPSCs are being utilized in clean meat production by serving as the cellular foundation for the creation of lab-grown meat. Specifically, iPSCs derived from animals can be differentiated into various cell types, including muscle cells (myocytes) and fat cells (adipocytes). Scientists use these cells to grow cultured meat products without the need to raise and slaughter animals. For example, Meatable is an startup company that uses porcine and bovine iPSCs to create lab-grown meat products.
12. 3D Bioprinting
iPSCs can be directed to differentiate into specific cell types of interest, such as skin, heart, or liver cells, which can then incorporated into bioinks for 3D printing applications. In particular, iPSC-derived skin cells are being explored for a applications related to wound closure and treatment.
13. Wildlife Conservation and De-extinction Projects
Other novel applications for iPSCs include wildlife conservation and de-extinction projects. For example, the company Colossal Biosciences is using iPSC technology to explore the possibility of performing woolly mammoth de-extinction. Their long-term goal is to accomplish wholly mammoth ex utero (outside the uterus) gestation. Currently, the company is working on the generation of elephant-derived iPSCs are a “proof of concept”.
What makes iPSC more attractive than other cell types?
There are several compelling reasons that now make iPSCs more attractive than other cell types. These advantages include that iPSCs:
- Can be produced in nearly infinite quantities
- Have the potential to be produced in uniform batches, from a single donor, and at low cost
- Are non-controversial
- Can be originated from any adult donor
- Have a diverse range of applications including cell therapy applications, personalized medicine, drug development and discovery, toxicology testing, disease modelling, cultured meat production, and beyond.
Interestingly, technology allowing for the mass-production and differentiation of iPSCs in industrial-scale bioreactors has also advanced rapidly in recent years, with companies like TreeFrog Therapeutics and Cynata Therapeutics emerging as pioneers in this area.
Why do pharma and biotech companies use iPSCs?
As mentioned above, cell therapy developers use iPSCs in their research because iPSC-derived cell types can be manufactured in unlimited quantities, in uniform batches, from a single donor, and at low cost. This is a powerful and rare combination of advantages that will potentially permit cell therapy developers to leverage economies of scale.
Companies who are using iPSC-derived cell types for therapeutics applications include Cynata Therapeutics, Fate Therapeutics, Bayer (who acquired Bluerock Therapeutics in 2019), Century Therapeutics, and over 60 others, which you can see listed here.
With the pharmaceutical world, drug development companies use iPSCs to screen potential drug compounds for efficacy at a large-scale and low cost. They also use iPSC-derived cell types to detect drug toxicities and cellular side affects at an early stage.
Additionally, pharma companies use iPSC-derived cell types to keep costs down compared to animal studies. Thankfully, the U.S. FDA Modernization Act 2.0, passed December 29, 2022, now allows pharma companies to replace certain animal models with human cells, organoids, and AI-assisted approaches. As a result, many of the major drug developers now use iPSC-derived cell types for diverse range of drug development applications. Pharma companies who are public about their use of iPSC-derived cell types for drug development applications include Bayer, Evotec, and Takeda Pharma, and others.
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