NCNP-01 Chemical Properties

storage temp. 

Store at -20°C

form 

Solid

color 

White to off-white

Sequence

RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C)

NCNP-01 Usage And Synthesis

Uses

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research[1][2].

References

[1] Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. DOI:10.1007/s40265-020-01339-3
[2] Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449. DOI:10.1016/j.omtn.2018.09.017