JP7345466B2 - 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 - Google Patents

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 Download PDF

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JP7345466B2
JP7345466B2 JP2020527712A JP2020527712A JP7345466B2 JP 7345466 B2 JP7345466 B2 JP 7345466B2 JP 2020527712 A JP2020527712 A JP 2020527712A JP 2020527712 A JP2020527712 A JP 2020527712A JP 7345466 B2 JP7345466 B2 JP 7345466B2
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JPWO2020004675A1 (ja
JPWO2020004675A5 (enExample
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朋典 宇野
隆資 夏川
洋一 江川
洋平 佐藤
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Nippon Shinyaku Co Ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2020527712A 2018-06-26 2019-06-26 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 Active JP7345466B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023143451A JP7595719B2 (ja) 2018-06-26 2023-09-05 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
JP2024205093A JP2025028961A (ja) 2018-06-26 2024-11-26 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

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862690270P 2018-06-26 2018-06-26
US62/690,270 2018-06-26
US201862739386P 2018-10-01 2018-10-01
US62/739,386 2018-10-01
PCT/JP2019/026393 WO2020004675A1 (ja) 2018-06-26 2019-06-26 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

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JP2023143451A Division JP7595719B2 (ja) 2018-06-26 2023-09-05 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

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JPWO2020004675A1 JPWO2020004675A1 (ja) 2021-07-15
JPWO2020004675A5 JPWO2020004675A5 (enExample) 2022-03-03
JP7345466B2 true JP7345466B2 (ja) 2023-09-15

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JP2020527712A Active JP7345466B2 (ja) 2018-06-26 2019-06-26 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
JP2023143451A Active JP7595719B2 (ja) 2018-06-26 2023-09-05 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
JP2024205093A Pending JP2025028961A (ja) 2018-06-26 2024-11-26 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

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JP2023143451A Active JP7595719B2 (ja) 2018-06-26 2023-09-05 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
JP2024205093A Pending JP2025028961A (ja) 2018-06-26 2024-11-26 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

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US (2) US20210261963A1 (enExample)
EP (1) EP3815696A4 (enExample)
JP (3) JP7345466B2 (enExample)
KR (1) KR20210023988A (enExample)
CN (1) CN112399849A (enExample)
AU (1) AU2019293687B2 (enExample)
BR (1) BR112020026542A2 (enExample)
CA (1) CA3101321A1 (enExample)
CL (1) CL2020003367A1 (enExample)
CO (1) CO2020015685A2 (enExample)
EC (1) ECSP20083454A (enExample)
IL (1) IL279692A (enExample)
MX (2) MX2020013880A (enExample)
MY (1) MY209148A (enExample)
PE (1) PE20210630A1 (enExample)
PH (1) PH12020552078A1 (enExample)
SG (1) SG11202011554PA (enExample)
TW (1) TW202035692A (enExample)
WO (1) WO2020004675A1 (enExample)
ZA (1) ZA202007682B (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2022014151A (es) 2020-05-11 2022-11-30 Stoke Therapeutics Inc Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
CN121194802A (zh) * 2023-02-14 2025-12-23 斯托克制药公司 反义寡聚物调配物
TW202448485A (zh) * 2023-05-05 2024-12-16 美商拜奧馬林製藥公司 肌萎縮蛋白外顯子跳躍寡核苷酸

Citations (2)

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Publication number Priority date Publication date Assignee Title
JP2014054250A (ja) 2010-09-01 2014-03-27 Nippon Shinyaku Co Ltd アンチセンス核酸
WO2017059131A1 (en) 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy

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WO1991009033A1 (en) 1989-12-20 1991-06-27 Anti-Gene Development Group Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
CA2942791C (en) 2002-11-25 2019-08-20 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP1811024A1 (en) 2004-10-05 2007-07-25 Nippon Shinyaku Co., Ltd. Oligo double-stranded rna and medicinal composition
WO2006129594A1 (ja) 2005-05-30 2006-12-07 Nippon Shinyaku Co., Ltd. 核酸含有複合体製剤の製造方法
HRP20140646T1 (hr) 2007-11-15 2014-09-26 Sarepta Therapeutics, Inc. Postupak sinteze morfolino oligomera
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
CN111893117B (zh) * 2012-01-27 2024-06-04 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
SMT201900139T1 (it) * 2013-03-14 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto dell'esone per il trattamento della distrofia muscolare
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
EP3015467B1 (en) * 2013-05-24 2024-12-04 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine

Patent Citations (2)

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JP2014054250A (ja) 2010-09-01 2014-03-27 Nippon Shinyaku Co Ltd アンチセンス核酸
WO2017059131A1 (en) 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy

Non-Patent Citations (1)

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KOMAKI H. et al.，Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in p，Science Translational Medicine，2018年04月18日，Vol.10，eaan0713, p.1-11

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Publication number Publication date
AU2019293687B2 (en) 2025-10-02
JP7595719B2 (ja) 2024-12-06
BR112020026542A2 (pt) 2021-04-06
US20210261963A1 (en) 2021-08-26
MX2020013880A (es) 2021-03-09
JPWO2020004675A1 (ja) 2021-07-15
WO2020004675A1 (ja) 2020-01-02
EP3815696A4 (en) 2022-11-30
US20240158792A1 (en) 2024-05-16
CN112399849A (zh) 2021-02-23
CO2020015685A2 (es) 2021-04-30
JP2025028961A (ja) 2025-03-05
ZA202007682B (en) 2024-04-24
ECSP20083454A (es) 2021-01-29
CA3101321A1 (en) 2020-01-02
MY209148A (en) 2025-06-24
IL279692A (en) 2021-03-01
PH12020552078A1 (en) 2021-05-31
TW202035692A (zh) 2020-10-01
AU2019293687A1 (en) 2021-01-07
EP3815696A1 (en) 2021-05-05
SG11202011554PA (en) 2020-12-30
JP2023171731A (ja) 2023-12-05
MX2025001486A (es) 2025-03-07
CL2020003367A1 (es) 2021-05-24
KR20210023988A (ko) 2021-03-04
PE20210630A1 (es) 2021-03-23

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