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.
Viltolarsen (NS-065/NCNP-01; 2 days) sodium induces exon 53 skipping in cells from a DMD model with a deletion of exons 45-52 with an EC50 value of 0.63 μM and in cells with a deletion of exons 48-52 with an EC50 value of 2.3 μM[2].
Viltolarsen (10 μM; 3 days) sodium causes the dystrophin protein expression in cells from a DMD model[2].
References:
[1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031.
[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.