Nippon Shinyaku had developed long-strand DNA synthesis in the late 1990ies and started collaboration with the National Center for Mental and Neurological Medicine to develop an antisense RNA (NS-065/NCNP-01) with exon skipping action against DMD. The synthetic double-stranded RNA polyI: polyC is prepared using a novel RNA synthesis method (2-cyanoethoxymethyl or CEM method) and delivered with cationic liposomes. Physician-initiated trials are underway in Japan and the USA.
Nippon Shinyaku report 2017
Nippon Shinyaku develops antisense drug against Duchenne and Fukuyama muscular dystrophy