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).


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.



Spring came in a hurry this year.
The atmosphere of early summer is already in the air.

This is the season when the trees, which have been quiet during the winter, are sprouting.
The Institute is located at the end of a tunnel of fresh greenery.

The gradation of the double-layered cherry blossoms may soon be at an end.

The sink in the discussion room has also been renewed.
It's the beginning of the new fiscal year with a fresh heart.

April 1, 2020