NS-050

NS-050 (also known as NS-050/NCNP-03) is an investigational antisense oligonucleotide therapy designed for the treatment of Duchenne muscular dystrophy (DMD) in patients with gene mutations amenable to exon 50 skipping. By binding to pre-messenger RNA, it induces skipping of exon 50 during mRNA splicing, restoring the reading frame and enabling production of a shorter but functional dystrophin protein. This approach aims to slow or suppress muscle function deterioration in DMD patients. The drug is administered intravenously and is currently being evaluated in a Phase 1/2 clinical trial involving ambulant boys aged 4–14 years with confirmed DMD mutations suitable for exon 50 skipping. The program has received Rare Pediatric Disease Designation from the FDA[2][4][5][6][10].

Other names

NCNP-03NCNP03NCNP 03