Department of Molecular Therapy, National Institute of Neuroscience, National Center for Neurology and Phychiatry

Welcome to Department of Molecular Therapy, a major basic-science department at National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo.

Our department is dedicated to elucidating the molecular pathogenesis of neuromuscular disorders, especially muscular dystrophy, and the development of advanced gene- and stem cell-based therapeutics for these diseases. For this purpose, we integrate molecular, pharmacologic, proteomic, and genomic methodologies using various types of unique animal models, including dystrophic dogs, mice, rats, and common marmosets. We are combating these diseases through research aimed at implementing promising novel therapeutic strategies, including oligonucleotide-based exon-skipping therapy and induced pluripotent stem cell (iPSC)-based cell therapy, into clinical practice. One of the significant achievements of our department is the approval of our antisense oligonucleotide-based exon 53-skipping drug, viltolarsen (NS-065/NCNP-01), for muscular dystrophy in Japan, following a successful investigator-initiated trial of NS-065/NCNP-01 (Sci Transl Med. 2018;10).


eSkip-Finder is launched ⇒

It is a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.

Marketing authorization of NS-065/NCNP-01, viltolarsen, for the treatment of Duchenne muscular dystrophy

Viltolarsen (VILTEPSO® Intravenous Infusion 250 mg) has been approved in Japan for the treatment of patients with Duchenne amenable to exon 53 skipping therapy. The drug was granted priority review in the USA with a decision expected in the third quarter of 2020.



After a refreshing season and past the rainy season,

The outside of the lab is turning out to be like being in an oven.

The inside of the lab is air-conditioned and comfortable.

August 1, 2021 by R.T