Viltolarsen sodium  (Synonyms: NS-065/NCNP-01 sodium)

Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium 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 sodium has the potential for Duchenne muscular dystrophy (DMD) research^[1][2].

Viltolarsen (NS-065/NCNP-01；2 天) 在删除了外显子 45-52 的 DMD 模型细胞中诱导外显子 53 跳跃，EC₅₀ 值为 0.63 μM；在删除了外显子 48-52 的DMD 模型细胞中， EC₅₀ 值为 2.3 μM^[2]。
Viltolarsen (10 μM；3 天) 导致 DMD 模型细胞中表达肌营养不良蛋白^[2]。

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

7386.42

C₂₄₄H₃₆₀N₁₁₃Na₂₁O₈₈P₂₀

固体

Room temperature in continental US; may vary elsewhere.

-20°C, stored under nitrogen, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)

• [1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031.  [Content Brief]

  [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.  [Content Brief]