ORIZURU THERAPEUTICS INC.
Regenerative Medicine and Drug Discovery using iPS cell technology

The ground-breaking discovery of induced pluripotent stem (iPS) cells in 2006 at Kyoto University opened numerous opportunities for innovative changes in the drug discovery and regenerative medicine development concerning stem cell research. This is because iPS cells are less prone to restriction than traditional embryonic cells such as ethic hurdle.

To create clinical applications for iPS cells, the Center for iPS Cell Research and Application (CiRA) at Kyoto University and Takeda Pharmaceutical Company Limited joined hands to create T-CiRA in 2015 as co-research program led by Prof. Shinya Yamanaka, Kyoto University. As some outputs moving out of discovery phase, Orizuru Therapeutics was established in 2021 to take over those outputs for translational R&D purpose from discovery to early clinical development.

Orizuru Therapeutics focuses on developing regenerative medicine products including cell transplantation, and regenerative medicine research support using the iPS cells technology.

The iPS technology sustains advantages against the traditional fetal, embryonic, and adult stem cells. The ethical controversy arising from embryonic stem cell (ESC) collection is eliminated as cells are harvested from a willing adult without harming them, avoiding embryo destruction involving ESCs. Further, unlike ESCs, using HLA-homozygous iPS cells or patient-derived iPS cells offer an opportunity to minimize the risk of immunorejection.

“Our competitive advantage is the intellectual property that created our core research programs and the brilliant people who work in them,” says Kenji Nonaka, CEO of Orizuru Therapeutics.

From T-CiRA, Orizuru Therapeutics took over two core research programs; iPS cell-derived cardiomyocytes (iCM) and iPS cell-derived pancreatic islet cells (iPIC). The iCM project was initiated to develop a definitive therapy for severe heart failure patients to address donor shortage for heart transplants. The iPIC project was started to formulate a replacement therapy for brittle type 1 diabetes patients to address donor shortage for islet transplantation and/or pancreas organ transplantation treatments.

Various techniques are used to generate a substantial amount of cells for patients under the iCM project. Methods used by Orizuru Therapeutics accomplish high-purity cardiomyocytes, making them safer options against traditional treatment modalities. Transplanting iCMs into a patient’s heart is predicted to restore the lost cardiomyocytes. This project aims to promote remuscularization and improve the quality of life. In treating brittle type 1 diabetes patients, it has established the unique implantation method that accommodates the long-term engraftment of iPIC. Collaborations with expert equipment manufacturers allow Orizuru Therapeutics to acquire billions of cells in one batch using a scalable culture system optimized for iPIC use.

For patients to access its regenerative medicine and cell transplantation treatments, Orizuru Therapeutics starts with preclinical research. From there, it moves to clinical development, follows the procedure to add a new drug application, and receives regulatory approval to launch its products in the market. It currently has over 100 patents including application. Orizuru Therapeutics will take ongoing GLP tox study forward to clinical development soon.

With treatments and products coming out, some drugs may cost an impractical amount of money. The pharmaceutical industry is looking to be cost-conscious, and Orizuru Therapeutics’ possesses the technology to make it happen with its skilled unit.

Orizuru Therapeutics’ dedicated team and talented researchers and technicians enable its scientific innovations to reach new heights, helping the world unleash the power of iPS-based cell therapy. In its mission to discover the clinical application of iPS cells and deliver cell therapy to patients and its goal to bring the world better health with the limitless power of science, Orizuru Therapeutics continues innovating to keep its vow of shaping a better tomorrow.