NS-051

NS-051 (also known as NCNP-04) is an antisense oligonucleotide therapy being developed by NS Pharma, a subsidiary of Nippon Shinyaku, in collaboration with the National Center of Neurology and Psychiatry (NCNP). It is designed for the treatment of Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 51 skipping. By binding to the dystrophin pre-mRNA, NS-051 induces the skipping of exon 51 during splicing, which restores the open reading frame and enables the production of a truncated but functional dystrophin protein. This mechanism is intended to stabilize muscle function and slow disease progression. As of 2025, the drug is in preclinical development and has received FDA Orphan Drug and Rare Pediatric Disease designations.

Other names

NCNP-04-NSRANCNP04-NSRANCNP 04-NSRA