We are the first company in Japan to be approved by the Cabinet Office and the Ministry of Health, Labor and Welfare for the commercialization of the drug discovery using iPS cells derived from human blood under the National Strategic Special Zones Law. We are conducting drug discovery research to elucidate the cause and progression of diseases (elucidate the pathophysiology) and search for therapeutic drug candidates using iPS cell lines (and differentiated cells produced from them) generated from somatic cells of patients with intractable diseases. In the field of neuromuscular diseases, where there are many inherited intractable diseases, disease-specific iPS cell lines are particularly important because they can show abnormalities from the causative gene of the disease and other background factors. In addition, we are accepting requests of the establishment of new iPS cell lines or joint research and contract studies using our disease-specific iPS cell lines.
Establishment of iPS cell lines
We will establish iPS cell lines from peripheral blood mononuclear cells and develop human disease model cells. We mainly establish cell lines under the following conditions.
Initialization method: Episomal method
Serving form: 3 clones x 3 vials (2 x 106 cells/vial)
Test items: Undifferentiated pluripotency markers, chromosomal analysis, mycoplasma, virus test
Colony of iPS cell lines derived from a healthy donor, generated by our company
Charcot-Marie-Tooth disease (CMT) model
We are working on the development of a disease model for Charcot-Marie-Tooth disease in collaboration with Kyoto Prefectural University of Medicine. Charcot-Marie-Tooth disease is an inherited neuromuscular disease in which motor and sensory impairments are caused by damage to peripheral nerves. It has the largest number of patients among hereditary peripheral neuropathy and is designated as one of the intractable diseases by the Ministry of Health, Labor and Welfare, but there are no approved drugs that have been recognized for their therapeutic effect. Therefore, development of effective therapeutic drugs is desperately needed. We have established disease-specific iPS cell lines using blood samples from patients with Charcot-Marie-Tooth disease, and are conducting research and development aimed at the development of disease models and drug discovery applications through the generation of neural cells and the reproduction of pathological conditions. We have confirmed higher expression of the PMP22 gene in Schwann progenitor cells produced from CMT1A patient-derived iPS cell lines. In addition, motor neurons produced from CMT2A patient-derived iPS cell lines are currently being utilized for drug discovery research.
<External link> Interview with the Company’s Representative Director by Kyoto University Innovation Capital Co., Ltd.
