Lung diseases can be debilitating, making both medical leaders and patients interested in exploring stem cell treatments for lungs. Find out what you need to know about stem cell therapy for lungs, including risks, benefits, clinical trial progress and more. [Read more…]
Academic and Government Organizations Developing iPSC Therapies
Today, there are a growing number of both commercial and non-commercial organizations advancing the development of induced pluripotent stem cell (iPSC) therapies. In addition to the commercial sector, government-funded research institutions and universities continue to play a significant role in the therapeutic applications of iPSCs.
Notable institutions involved in iPSC research and therapy development include:
- RIKEN Center in Kobe, Japan
- Kyoto University in Kyoto, Japan
- California Institute for Regenerative Medicine (CIRM) in California, USA
Each of these organizations has made key contributions to the progress of iPSC therapies. Below is an updated look at their involvement.
RIKEN
RIKEN Center, based in Kobe, Japan, has been a leader in pioneering iPSC-based clinical trials. The first-ever clinical trial involving iPSCs, led by Dr. Masayo Takahashi, started in 2013 with the goal of treating patients with wet-type age-related macular degeneration (AMD) using iPSC-derived retinal tissue.
In 2024, RIKEN and its associated hospitals continued to refine and expand their research in iPSC-based treatments for AMD. RIKEN’s work is particularly groundbreaking in the use of patient-specific iPSCs, generated from skin cells, to create retinal tissue for transplants. While results from the initial trials showed promising safety data, long-term efficacy is still being carefully monitored, with the team progressing toward larger patient cohorts and refining the production processes for these cell therapies.
RIKEN also works closely with the Kobe City Medical Center General Hospital and other local institutions to improve the clinical applications of iPSC-derived therapies, continuing to set standards in stem cell-based regenerative medicine.
Kyoto University
Kyoto University, led by Nobel Laureate Dr. Shinya Yamanaka, continues to be a leader in iPSC research. Dr. Yamanaka’s lab was the first to successfully generate iPSCs from mouse cells in 2006, and since then, the university has expanded its focus to therapeutic applications.
Currently, Kyoto University is involved in several high-profile research projects using iPSCs. For instance, their work on differentiating iPSCs into dopamine-producing neurons for Parkinson’s disease patients has entered clinical trial stages, with studies focusing on safety and efficacy. The trials, which started in the early 2020s, are part of a broader effort to combat neurodegenerative diseases using iPSCs. These trials are still in the early phases of patient testing, but initial results have demonstrated safety and early signs of clinical benefit.
Kyoto University’s iPSC-based platelet production project has also gained attention, as it could help address blood disorders, and is expected to see clinical trials in the coming years.
Dr. Yamanaka’s leadership continues to drive the Kyoto University iPSC Research Center, which collaborates with medical institutions worldwide to advance therapeutic strategies. The center is also engaged in cutting-edge research aimed at improving the scalability and quality of iPSC manufacturing for clinical applications.
California Institute for Regenerative Medicine (CIRM)
CIRM, established in 2004, has been instrumental in funding iPSC research in California, contributing significantly to the translation of iPSC science into therapies. With an initial investment of $3 billion in taxpayer funds, CIRM has remained a key player in supporting stem cell research and its clinical applications.
Over the last few years, CIRM has focused on advancing iPSC-based clinical trials and building infrastructure for large-scale iPSC production. Notably, CIRM continues to fund research into iPSCs for cardiovascular diseases, neurodegenerative diseases, and genetic disorders.
In 2024, CIRM awarded significant grants to various institutions for iPSC-related projects, including a $10 million grant to Stanford University for advancing iPSC-based cardiac cell therapies. This research is focused on better understanding the mechanisms of heart disease and testing personalized treatments derived from patient-specific iPSCs.
CIRM has also been funding efforts to create a comprehensive iPSC biobank that includes both healthy and diseased human tissues. These biobanks support ongoing research on personalized medicine and the development of targeted therapies for conditions such as Alzheimer’s, autism spectrum disorders, and neurodegenerative diseases.
In 2025, Cellular Dynamics International (CDI), now part of Fujifilm Holdings, continues to lead in iPSC production and biobanking efforts. CDI’s collaboration with the Coriell Institute has helped establish one of the world’s largest human iPSC biobanks, which is crucial for advancing iPSC-based research in precision medicine.
CIRM’s ongoing funding has accelerated the translation of iPSC technology from the laboratory to real-world clinical settings, with several trials underway targeting a range of conditions from Parkinson’s disease to liver failure.
Looking Ahead
As of 2025, iPSC therapies are advancing steadily, but the field is still in the clinical trial phase for many indications. The next decade holds great promise, as major institutions like RIKEN, Kyoto University, and CIRM continue to lead the charge in developing scalable, safe, and effective iPSC therapies. Successes in early-phase clinical trials are providing a foundation for larger-scale applications, and the coming years are expected to see iPSC-based therapies for a wider array of diseases.
The challenge ahead remains the need for standardized methods for iPSC production, as well as addressing the long-term safety and efficacy of these treatments. However, with continued investment and research, iPSC-based therapies are likely to become an integral part of the future of regenerative medicine.
To learn more about this exciting market, view the “Global Induced Pluripotent Stem Cell (iPSC) Industry Report – Market Size, Trends, and Forecasts, 2025.”
iPSC Culture and Differentiation: The Future of Disease Modeling and Drug Discovery
The generation of induced pluripotent stem cells (iPSCs) involves technologies such as reprogramming, culturing of iPS cells, differentiation of cell types from iPSCs, cell analysis, cell engineering, and beyond. Although several firms are engaged in providing iPSC tools and technologies—such as reprogramming vectors, transfection kits, maintenance and differentiation media, immunocytochemistry, and live staining kits—it is still difficult for researchers to produce required quantities of iPSCs.
Because of this, many researchers prefer to outsource reprogramming or acquire commercial iPSC-derived cell types from companies such as FUJIFILM CDI, Ncardia, Axol Bioscience, ReproCELL, Evotec, and others. These companies streamline the process, offering ready-to-use, fully characterized iPSC lines, as well as specialized tissue-specific cells that can be immediately used in experiments or therapeutic applications.
iPSC Cultures
iPSC cultures are often grown in 2D cell culture plates, dishes, or flasks. Unfortunately, 2D cell cultures induce the formation of undesired gradients, including media components, metabolic waste products, paracrine factors, and gases. Consequently, 2D iPSC culture techniques necessitate daily media changes, which is quite cumbersome. This limitation can impact the scalability and reproducibility of experiments, particularly when large numbers of cells are required for high-throughput screening or clinical applications.
To produce large quantities of cells, ‘scale-out’ of the 2D approach is often resorted to by multiplying culture dishes or by using multi-layered flasks. However, recently launched small benchtop 3D suspension bioreactor systems, such as ABLE® Biott®’s bioreactors, can ‘scale-up’ without ‘scale out’. Such systems can provide mass stem cell culture with fewer plates and culture media, without the need for external matrix supplementation and frequent feeding. These bioreactors offer a more efficient approach to culturing iPSCs in a more physiologically relevant environment, mimicking the 3D architecture of tissues in vivo, which helps enhance the functionality of the derived cell types.
Differentiation of iPSCs into Mature Cell Types
Once the iPSCs are created, they are directed to differentiate into the tissue cells of interest, resulting in iPSC-derived mature cell types. Companies such as FUJIFILM CDI, Ncardia, Evotec, and others have invested significantly in developing and optimizing the reprogramming process of iPSC to enable the generation of industrial quantities of differentiated tissue cells that can recapitulate relevant donor disease biology in the laboratory. These advancements in differentiation protocols are vital for studying complex diseases and developing new therapies, as they allow researchers to model diseases at a cellular level using patient-specific iPSCs.
The ready availability of adequate quantities of cryopreserved iPSC-derived cell aliquots is already significantly impacting drug discovery, with an expectation that it will reduce the risk of late-stage attrition during drug development. Commercial suppliers are mainly focusing on cardiac cells, hepatic cells, neuronal cells, and pancreatic cells. These cell types are crucial for drug testing and toxicity screening, as they replicate specific organ functions and disease states. By providing access to reliable, scalable, and reproducible iPSC-derived cell types, these suppliers are helping to bridge the gap between basic research and clinical application.
Advancements in iPSC Applications for Disease Modeling and Drug Testing
Beyond drug discovery, iPSCs are revolutionizing the way we study complex diseases. By generating patient-specific iPSCs, researchers can create personalized disease models that mimic the genetic and environmental factors contributing to the disease. This approach enables the study of disease progression at the cellular and molecular levels, offering deeper insights into underlying mechanisms and facilitating the identification of novel therapeutic targets. Furthermore, these models enable the testing of drug responses, providing a more accurate prediction of how a patient may respond to a given treatment, thus paving the way for personalized medicine.
Researchers are also exploring the use of iPSCs for cell-based therapies, such as tissue regeneration and transplantation. With iPSCs, it is possible to generate autologous cells (cells derived from the same patient), which reduces the risk of immune rejection and offers the potential for treating diseases where tissue damage has occurred, such as in neurodegenerative disorders, heart disease, and diabetes. However, this area still faces significant challenges, including ensuring the safety and stability of the differentiated cells, preventing tumorigenesis, and refining protocols for large-scale production.
Cell Sorting and Analysis in iPSC Research
Cell analysis involves the use of flow cytometry and other techniques to assess the quality and purity of iPSC-derived cells. Cell sorting is also required soon after somatic cell reprogramming to separate the reprogrammed cells from partly reprogrammed cells. For example, BD offers its BD Stemflow Human iPSC Sorting and Analysis Kit. The kit contains pre-titrated antibodies for the identification of iPSCs, instrument setup reagents, isotype controls, and a protocol for consistent results. This step is crucial for ensuring that only fully reprogrammed iPSCs are used in experiments, minimizing the risk of genetic abnormalities or incomplete reprogramming that could affect the outcomes.
Moreover, the integration of high-throughput screening techniques with iPSC technologies is speeding up the drug discovery process. Automated platforms enable researchers to analyze large numbers of compounds and evaluate their effects on iPSC-derived tissues. By using these systems, drug candidates can be screened more efficiently, providing critical insights into their efficacy and toxicity profiles. This combination of advanced technologies is driving the development of more effective treatments for a wide range of diseases.
Looking Toward the Future of iPSC Research
As iPSC technologies continue to evolve, the ability to produce high-quality, disease-specific cell models at scale will open new avenues for both basic research and clinical applications. The ongoing advancements in gene editing, such as CRISPR-Cas9, allow for precise modifications of iPSCs, further enhancing their potential for disease modeling and therapeutic development. In the future, iPSCs may be instrumental in creating organoids or even entire organs for transplantation, offering a potential solution to the global organ shortage.
Furthermore, the integration of iPSCs into precision medicine could drastically reduce the time and cost required to develop new drugs. By incorporating patient-derived iPSCs into drug discovery pipelines, pharmaceutical companies could more accurately predict how a patient will respond to a particular drug, leading to more targeted and effective treatments. With these developments, iPSCs are poised to play a critical role in shaping the future of medicine.
Are you working with iPSCs? If so, share your experiences and approaches in comments below.
To learn more about this expanding market, view the “Global Induced Pluripotent Stem Cell (iPSC) Industry Report – Market Size, Trends, and Forecasts, 2025.”
Innovative Functional Peptides Successfully Co-Developed as Alternatives to Growth Factors Used in the Manufacturing of Regenerative Medicine Products
Orizuru Therapeutics, Inc. (OZTx), a Japanese biotechnology company focusing on the research and development of iPSC-derived regenerative medicine, and PeptiGrowth Inc. (PeptiGrowth), a Japanese biotechnology company specializing in the development of synthetic peptide based growth factors, are pleased to announce the successful completion of the joint development project (first announced on August 23, 2023) for chemically synthesized peptide as an alternative to specific recombinant growth factors used in the manufacturing of regenerative medicine products developed by OZTx. The developed peptide was confirmed to bind to cellular receptors in a manner similar to the respective recombinant growth factor, inducing phosphorylation and signaling within the cells, thereby triggering differentiation and proliferation of iPSCs. [Read more…]
Eterna Therapeutics Releases Positive Preclinical Results for ERNA-101, an Allogenic iMSC Therapeutic
ERNA-101 is a cell therapy that utilizes iPSCs to create induced allogenic mesenchymal stem cells (iMSCs) that secrete interleukins IL-7 and IL-15.
Proof-of-concept study provides roadmap for treatment of ovarian cancer.
CAMBRIDGE, Mass., Jan. 14, 2025 — Eterna Therapeutics (Nasdaq: ERNA), a leader in cell therapies for the treatment of advanced solid tumors, announced positive results from a preclinical study on the company’s lead cell therapy product, ERNA-101, which is designed to activate and regulate the immune system’s response to recognize and attack ovarian cancer cells. This successful proof-of-concept study demonstrated massive T cell infiltration after only one dose of ERNA-101, reduced tumor burden, and extended survival in mice with ovarian cancer. [Read more…]
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