TOKIWA-Bio has been selected by the Japan Agency for Medical Research and Development (AMED) in FY2018 as “gene / cell therapy research and development infrastructure project”. Research target diseases are: Hunter’s disease, X-SCID, hemophilia A, phenylketonuria and Menkes disease.
By using Stealth RNA Vector (SRV) for diseases with large therapeutic genes, with coenzymes necessary for enzyme activity in addition to the enzyme to be supplemented, etc. TOKIWA-Bio is anticipating some feasible therapeutic strategies that cannot be realized by existing AAV vectors or lentiviral vectors.