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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JPWO2020004675A5 (OSRAM
JPWO2020004675A1 (ja
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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)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • 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)
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  • 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 (OSRAM) 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

Country Status (20)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
KR20240004609A (ko) 2021-04-30 2024-01-11 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증에 대한 치료 방법
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
AU2024222908A1 (en) * 2023-02-14 2025-09-04 Stoke Therapeutics, Inc. Antisense oligomer formulations
US20240368596A1 (en) * 2023-05-05 2024-11-07 Biomarin Pharmaceutical Inc. Dystrophin exon skipping oligonucleotides

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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Publication number Priority date Publication date Assignee Title
EP0506830B1 (en) 1989-12-20 2005-12-14 Avi Biopharma, Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
CA3001404C (en) 2002-11-25 2020-07-28 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
EP1811024A1 (en) 2004-10-05 2007-07-25 Nippon Shinyaku Co., Ltd. Oligo double-stranded rna and medicinal composition
US20090069260A1 (en) 2005-05-30 2009-03-12 Nippon Shinyaku Co., Ltd Method for producing a nucleic-acid-containing complex preparation
CA2704261C (en) 2007-11-15 2015-05-26 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
SMT201900159T1 (it) 2008-10-24 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto di esone per dmd
BR112014018427B1 (pt) * 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
KR20240094032A (ko) * 2013-03-14 2024-06-24 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
JP6477464B2 (ja) * 2013-05-24 2019-03-06 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
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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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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Also Published As

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

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