MIJ invests in Gene Therapy Research Institution Co., Ltd.2019.12.24

We are pleased to announce that MIJ has invested in Gene Therapy Research Institution Co., Ltd., a drug development start-up company that carries out R&D on gene therapy technology targeting mainly neurodegenerative diseases. In addition to drug development, the company is also active in the development of “AAV vectors” (gene carriers).

The “AAV vectors” used in the therapies/treatments that Gene Therapy Research Institution Co., Ltd. is developing are characterized by their nonpathogenicity as well as their excellent therapeutic gene expression efficiency, despite the extremely low possibility of therapeutic genes being incorporated into chromosomes. In 2007, a clinical trial was carried out at Jichi Medical University whereby “AAV vectors” expressing AADC were administered to both sides of the putamen in the brains of Parkinson’s disease patients. Not only did assessments after six months find improvement in the patients’ motor symptoms, but also it has been reported that AADC expression is continuing even five years after the gene transfer, and high expectations are held that this will prove to be an unprecedented new therapy.

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