Department of Molecular Therapy, National Institute of Neuroscience, NCNP

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).

Lab Tour !

WHAT'S NEW

01 Oct. 2025: Method
Ono and colleagues' method article, titled [Neurological Behavioral Assessment of Exon-Skipping Therapy for Neuromuscular Disorders Using Displaced Object Recognition Test, Open Field Test, and Three-Chamber Social Approach Test] has been published in Methods Mol Biol.; Method
Ashida and colleagues' method article, titled [Evaluation for the Efficacy of Exon-Skipping Therapy Using In Vivo Assessment of Skeletal Muscle Function and Fragility] has been published in Methods Mol Biol.; Method
Kunitake and colleagues' method article, titled [Splice Modulation Studies Using Urine-Derived Cells] has been published in Methods Mol Biol.
19 Sep. 2025: MED
[Press release] FDA Grants Orphan Drug Designation to NS-051/NCNP-04 for the Treatment of Duchenne Muscular Dystrophy
03 Sep. 2025: Review
Tominari and colleagues' method article, titled [Stem/progenitor cell-based therapy for Duchenne muscular dystrophy] has been published in Front Cell Dev Biol.
29 Jul. 2025: Method
Sathyaprakash and colleagues' method article, titled [In Vivo Evaluation of Single- and Multi-Exon-Skipping in mdx52 Mice] has been published in Methods Mol Biol.
22 Jul. 2025: Report
RDC Japan symposium report, titled [Report on the rare disease consortium Japan inaugural symposium - July 18, 2023, shonan health innovation park, Japan] has been published in J Neuromuscul Dis.
06 Jun. 2025: Research
Kunitake and colleagues' research article, titled [Protocol for using MYOD1-transduced human urine-derived cells as a predictive platform for exon skipping therapy in Duchenne muscular dystrophy] has been published in STAR Protocols.
24 Apr. 2025: Review
Tominari and Aoki's review, titled [The Pathophysiology of Muscle Atrophy Common to Neuromuscular diseases (Japanese)] has been published in Prec Med.
03 Apr. 2025: Lab
[Member] updated.
25 Mar. 2025: Press Release
[Press Release] Innovatibe mRNA Therapeutics for the treatment of intractable Diseases -Opening New Possibilities for the Treatment of Muscular Dystrophy-（Reference: Adv Sci.）
07 Mar. 2025: Research
Du and colleagues' research, titled [Polyplex Nanomicelle-Mediated Pgc-1𝜶4 mRNA Delivery Via Hydrodynamic Limb Vein Injection Enhances Damage Resistance in Duchenne Muscular Dystrophy Mice], has been published in Adv Sci.
03 Feb. 2025: Review
Aoki and colleagues' research, titled [Duchenne muscular dystrophy: recent insight in brain related comorbidities] has been published in Nat Commun.
23 Jan. 2025: MED
FDA Grants Rare Pediatric Disease Designation to NS-051/NCNP-04 for the Treatment of Duchenne Muscular Dystrophy　【Press release】
21 Jan. 2025: Research
Kunitake and colleagues' research, titled [Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy] has been published in Cell Rep Med.
10 Jan. 2025: MED
Brogidirsen: A Dual-Targeting Exon 44 Skipping Antisense Oligonucleotide Shows Promising Safety and Efficacy in Phase I/II Trial for Duchenne Muscular Dystrophy [Press Release] [Cell Press]
02 Dec. 2024: MED
Senkuteki Iyakuhin (Pioneering Drug) Designation and Orphan Drug Designation granted to NS-089/NCNP-02 for the Treatment of Duchenne Muscular Dystrophy. Press release
2024年11月07日: RDCJ
Survey on the problems of healthcare professionals in rare diseases (in Japanese).
2023年10月24日: MTG
Aoki Han-kaigi annual meeting program is now available (in Japanese).⇒　[Download page]
1 Oct. 2024: RDCJ
The Rare Disease Consortium Japan (RDCJ) is now accepting applications for membership.
25 Sep. 2024: Commentary
A commentary authored by Dr. Aoki was published in Mol Ther Nucleic Acids.PubMed
22 Sep. 2024: Review
A review article authored by Dr. Tominari and Dr. Aoki was published in Neurol Ther.
6 Sep. 2024: MED
FDA grants rare pediatric disease designation to NS-050/NCNP-03 for the treatment of Duchenne muscular dystrophy. Press Release
5 Sep. 2024: Lab
「Member」updated.
31 Jul. 2024: Update
Article on Dr. Shibasaki, an alumnus, is posted in the「Interview」.
22 Jun. 2024: Review
A review article authored by Mr. Okoshi and his colleagues was published in J Clin Exp Med (IGAKU NO AYUMI).
27 May. 2024: Press Release
NS Pharma announced the Preliminary results of viltolarsen (NS-065/NCNP-01) in phase 3 clinical trials (RACER53 study). Press Release
21 May. 2024: Press Release
Dr. Imamura and his colleagues successfully established a DMD-edited microminipig model by genetically modifying pigs, targeting the DMD gene, using the CRISPR/Cas9 system. Press Release
3 May. 2024: Research
A research article authored by Dr. Imamura and his colleagues was published in Commun Biol. PubMed
1 Apr. 2024: Research
A research article authored by Dr. Imamura and his colleagues was published in In Vitro Cell Dev Biol Anim. PubMed
29 Feb. 2024: RDC Japan
RDC Japan has begun activities as a voluntary organization. ⇒　RDC Japan
29 Feb. 2024: Lab
The 45th annual scientific meeting, NIN, NCNP was held on 29 Feb.-1 Mar. 2024.
6 Feb. 2024: RDC Japan
Rare Disease Consortium Japan website is now open. ⇒　RDC Japan
28 Jan. 2024: Review
A review article authored by Dr. Kunitake and his colleagues was published in Sci Rep. PubMed
25 Dec. 2023: Research
A research article authored by Dr. Tominari and his colleagues was published in Int J Mol Sci.TUAT Press Release　Pubmed
21 Dec. 2023: MED
NS-089/NCNP-02 received Orphan Drug Designation form tha European Commission for the treatment of Duchenne muscular dystrophy.Press Release
20 Dec. 2023: MTG
Annual Meeting (Aoki-han) was held on 19-20 Dec. 2023.
29 Nov. 2023: MTG Annual Meeting (Aoki-han) program is now available. [download page]
29 Nov. 2023: LAB The website has been renewed.
On 1st September 2021,: Updated Members.
On 1st August 2021,: Updated Publications.
On 1st August 2021,: Updated column.
On 1st April 2021,: Updated Publications.
On 1st April 2021,: Updated Members.
On 1st April 2021,: Updated column.
On 18th December 2020,: Updated column.
On 22th October 2020,: Updated Work or study with us !
On 6th October 2020,: Updated column.
On 24th September 2020,: The top 1% article of the academic field of Clinical Medicine based on a highly cited threshold for the field and publication year (Web of Science) : A Review of Duchenne Muscular Dystrophy by Dr. Y. Shimizu-Motohashi. J. Pers. Med. 2019, 9(1), 1
On 17th September 2020,: Dr. Hotake Takizawa, a Researcher at Department of Molecular Therapy, received the Best Oral Award in Clinical Category at the 61st Annual Meeting of the Japanese Society of Neurology.
On 24th August 2020,: our website has been totally renewed.

TOPICS

NS-051/NCNP-04 (exon 51 skipping drug) for the treatment of DMD

FDA Orphan drug designation to NS-051/NCNP-04 for the treatment of Duchenne muscular dystrophy.

【19 Sep. 2025】Designation of Orphan drug to NS-051/NCNP-04

PAST NOTICE

【13 Jan. 2025】Designation of Rare pediatric disease to NS-051/NCNP-04

Brogidirsen, NS-089/NCNP-02 (exon 44 skipping drug) for the treatment of DMD

NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy notice regarding publication (Cell Rep Med) of the results of an investigator-initiated clinical trial (First in human trial)

【10 Jan. 2025】Brogidirsen: A Dual-Targeting Exon 44 Skipping Antisense Oligonucleotide Shows Promising Safety and Efficacy in Phase I/II Trial for Duchenne Muscular Dystrophy

PAST NOTICE

【02 Dec. 2024】Senkuteki Iyakuhin (Pioneering Drug) Designation and Orphan Drug Designation granted to NS-089/NCNP-02 for the Treatment of Duchenne Muscular Dystrophy

【21 Dec. 2023】Orphan Drug Designation form the European Commission

【17 Oct. 2023】Preclinical data published in Mol Ther Nucleic Acids.

【07 Aug. 2023】Designations of Breakthrough therapy, Orphan drug, and Rare pediatric disease to NS-089/NCNP-02.

【14 Apr. 2023】FDA clearance to initiate phase II study for NS-089/NCNP-02.

【17 Mar. 2022】Result of Investigator-Initiated Trials (First In Human study)

NS-050/NCNP-03 (exon 50 skipping drug) for the treatment of DMD

FDA grants rare pediatric disease designation to NS-050/NCNP-03 for the treatment of Duchenne muscular dystrophy.

【6 Sep. 2024】Orphan drug designation

PAST NOTICE