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1. WO2020004675 - COMPOSITION COMPRISING ANTISENSE OLIGONUCLEOTIDE AND USE THEREOF FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Publication Number WO/2020/004675
Publication Date 02.01.2020
International Application No. PCT/JP2019/026393
International Filing Date 26.06.2019
IPC
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21
Drugs for disorders of the muscular or neuromuscular system
04
for myasthenia gravis
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
12
Genes encoding animal proteins
A61K 31/7088 (2006.01)
A61P 21/04 (2006.01)
C12N 15/113 (2010.01)
C12N 15/12 (2006.01)
CPC
A61K 31/7088
A61P 21/04
C12N 15/113
Applicants
  • 日本新薬株式会社 NIPPON SHINYAKU CO., LTD. [JP/JP]; 京都府京都市南区吉祥院西ノ庄門口町14番地 14, Kisshoin Nishinosho Monguchicho, Minami-ku, Kyoto-shi, Kyoto 6018550, JP
Inventors
  • 宇野 朋典 UNO, Tomonori; JP
  • 夏川 隆資 NATSUKAWA, Takashi; JP
  • 江川 洋一 EGAWA, Youichi; JP
  • 佐藤 洋平 SATOU, Youhei; US
Agents
  • 小林 浩 KOBAYASHI, Hiroshi; 東京都中央区八重洲二丁目8番7号 福岡ビル9階 阿部・井窪・片山法律事務所 ABE, IKUBO & KATAYAMA, Fukuoka Bldg. 9th Fl., 8-7, Yaesu 2-chome, Chuo-ku, Tokyo 1040028, JP
Priority Data
62/690,27026.06.2018US
62/739,38601.10.2018US
Publication Language Japanese (JA)
Filing Language Japanese (JA)
Designated States
Title
(EN) COMPOSITION COMPRISING ANTISENSE OLIGONUCLEOTIDE AND USE THEREOF FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
(FR) COMPOSITION COMPRENANT UN OLIGONUCLÉOTIDE ANTISENS ET SON UTILISATION POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE
(JA) アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用
Abstract
(EN)
The present invention relates to a composition containing an antisense oligonucleotide and a use thereof for the treatment of Duchenne muscular dystrophy. The present invention, specifically, relates to the composition effective for the treatment of Duchenne muscular dystrophy when administered at a treatment dose, and a use thereof.
(FR)
La présente invention concerne une composition contenant un oligonucléotide antisens et son utilisation pour le traitement de la dystrophie musculaire de Duchenne. La présente invention, spécifiquement, concerne la composition efficace pour le traitement de la dystrophie musculaire de Duchenne lorsqu’elle est administrée à une dose de traitement, et son utilisation.
(JA)
本発明は、アンチオリゴヌクレオチドを含有する組成物及びデュシェンヌ型筋ジストロフィーを治療するためのその使用に関する。本発明は、特に、処置の投与量で投与した場合にデュシェンヌ型筋ジストロフィーの治療に有効な上記組成物およびその使用に関する。
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