We are developing a definitive therapy for severe heart failure patients with limited therapeutic options.
iCM is an iPS cell-derived cardiomyocyte created by a T-CiRA’s project led by Dr. Yoshinori Yoshida, Associate Professor at Center for iPS Cell Research and Application(CiRA), Kyoto University. The myocardial differentiation method, which has been studied at CiRA has been further improved towards practical use by using the cardiomyocytes purification method by small molecule compound discovered in the T-CiRA program. By transplanting iCMs, which is considered to be highly engraftable, pure and safe cardiomyocytes, into the patient's heart, it is expected to replenish the cardiomyocytes lost as the disease progresses and promotes remuscularization. Restoring the cardiac function of patients with severe heart failure, which has been considered difficult to treat, can be expected to improve the quality of life (QOL) and prognosis of these patients.
Solution from iCM
Achievement of both high productivity and high purity.
Mass production friendly suspension culture system. Removal of non-cardiomyocytes that could induce adverse effects, such as arrhythmia and cancer risk.
Stable supply to the people who need it when they need it.
Cryopreservable and off-the-shelf cell product
Assisting cardiac function for a long period of time.
High efficacy can be achieved by our highly efficient engraftable cardiomyocytes beyond the current drug therapy
Cardiomyocyte therapy requires the delivery of hundreds of millions of cardiomyocytes or more to patients. In order to provide cell products to patients with severe heart failure who are eagerly awaiting treatment, it is necessary to develop cell manufacturing technologies that can efficiently produce large numbers of cells. One of the most important technologies is a culture technique in which cells are suspended in a medium and cultured. We have been working since the early stage of development on a method for cardiomyocyte differentiation using a suspension culture system, which has been considered difficult to obtain high-quality cardiomyocytes. In addition, we have discovered unique small molecule compounds that efficiently remove non-cardiomyocytes generated during cardiac differentiation. By combining these state-of-art technologies with the differentiation methods for preparing highly engraftable cardiomyocytes found in CiRA, high-performance, safe iCMs can be produced with a simple differentiation process. iCMs are highly effective when administered as single cells, eliminating the need for sheet or aggregate reformation. In addition, iCM is suitable for a wide range of administration methods, including catheter administration, enabling minimally invasive treatment with less burden on the patient.
Our goal is to deliver definitive therapy with iPSC-derived pancreatic islet cells (iPIC) to brittle type 1 diabetes patients as a replacement therapy for islet transplantation
iPICs are human iPS cell-derived pancreatic islet cells expected to be suitable for cell therapy. They were discovered after a five-year optimization study through a joint research program "T-CiRA" between Takeda Pharmaceutical and Center for iPS Cell Research and Application (CiRA), Kyoto University, based on the pancreatic cell differentiation induction method (Stem Cell Res. 2015;14:185-97) discovered by Dr. Taro Toyoda, CiRA Junior Associate Professor. This is an aggregate of high-purity pancreatic endocrine cells that contains both insulin and NKX6.1 positive cells, which is a characteristic of pancreatic β cells. After transplantation in vivo, pancreatic islet structures containing glucagon positive cells, another important endocrine cell, are formed, and physiological insulin-secreting capabilities can be demonstrated in response to glucose loading and hypoglycemia. Orizuru will continue to work with Dr. Toyoda as a scientific advisor towards the clinical application of iPICs as early as possible.
Development of novel cell therapy product utilizing iPIC
To provide the novel treatment option for brittle type 1 diabetes patients, we have established the original subcutaneous implantation method that supports the long-term engraftment of iPIC. We then demonstrated proof-of-concept that implanted iPIC can treat diabetic rodents and pig models so far. High purity of expected endocrine cells, achieved by the combinatory approach of single-cell RNA sequencing-based transcriptomics and original compound-based purification, is one strength of our product. Fruitful collaboration with expertized equipment manufacturers enables us to yield 10^9 cells at one experiment with culture system optimized for iPIC. Now we are developing the product that can be supplied exactly at the surgery date with the freeze-thaw tolerance of iPIC.
Solution from iPIC
islet-like cells (iPIC)
- Scalable culture process
- Homogeneous and stable quality
- High purity
- Pancreas islet-like morphology
- Physiological insulin secretion
- Less invasive and retrievable surgery
- Freedom from hypoglycemic events
- Independence of insulin injection
- Minimization of immunosuppressant use
By utilizing the cutting-edge iPS cell technology and the related technologies which have been cultivated through the T-CiRA program, we will support the creation of innovative pharmaceutical products through new drug discovery approaches and develop infrastructures for regenerative medicine research to provide iPS cell products at a reasonable cost and a stable supply.
Business development of candidate seeds through collaboration and commissioned business with other operating companies and university institutions
Solutions provided by iPS cell utilization technology
Consolidation of human resources who inherit the cutting-edge iPS cell utilization technology cultivated at T-CiRA
Production and analysis technologies for a wide variety of iPS cell-derived cells and tissue organoids
Support for drug discovery research utilizing iPS cell technology and development of regenerative medicine research platform