iXgene Co., Ltd. (Headquarters: Tokyo, Representative Director: Shunji Furukawa, hereinafter referred to as “iXgene”, Keio University School of Medicine (Dean of Medicine: Takanori Kanai) and Sumitomo Pharma Co., Ltd. (Headquarters: Osaka City, Representative Director and President: Hiroshi Nomura, hereinafter “ Sumitomo Pharma”) hereby announces that they have concluded a three-way collaborative research agreement for the practical application of a malignant brain tumor treatment using genome-edited iPS cell-derived neural stem cells.Based on

　this joint research agreement , The three parties will jointly conduct a non-clinical study or a preliminary study on the efficacy and safety of iPS cell-derived neural stem cells into which therapeutic genes have been introduced by editing, against malignant brain tumors.Malignant brain tumors are a disease with an extremely

　poor prognosis. Even with surgery, chemotherapy, and radiotherapy, complete cure is difficult, and there is no proven effective treatment for recurrence.As a new treatment for this intractable disease, the Department of Neurosurgery, Keio University School of Medicine, We will apply genome-edited iPS cell-derived neural stem cells developed by Professor Masahiro Toda.These therapeutic cells are created by introducing therapeutic genes into iPS cells through genome editing and inducing differentiation into neural stem cells. As a therapeutic gene, we have selected the fusion suicide gene yeast cytosine deaminase–uracil phosphoribosyl transferase (yCD-UPRT). This is the gene for the enzyme that converts the drug to 5-Fluorouracil (5-FU).When this therapeutic cell is administered to a patient with a malignant brain tumor, and after a certain period of time, the prodrug 5-FC is administered, the molecules that have migrated around the tumor cells are The suicide gene introduced into cells activates and locally produces high concentrations of the anticancer drug 5-FU, killing tumor cells.In
　this joint research, Keio University With the technical cooperation of the School of Medicine and Sumitomo Pharma, iXgene will primarily conduct non-clinical studies related to safety and efficacy, as well as preliminary tests.

[About genome editing]
Genome editing technology: Can cut any base sequence It is possible to cut the target sequence on genomic DNA using a DNA cutting enzyme and knock out or knock in genes using the cell’s DNA repair mechanism.

[About iXgene]
iXgene was established at Keio University School of Medicine in 2020 . It was founded as a startup. Using genome-edited iPS cells, which combine iPS cell technology and genome editing technology, we aim to develop treatments for intractable diseases for which there are no existing treatments.