TW202035692A - 含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途 - Google Patents

含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途 Download PDF

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TW202035692A
TW202035692A TW108122381A TW108122381A TW202035692A TW 202035692 A TW202035692 A TW 202035692A TW 108122381 A TW108122381 A TW 108122381A TW 108122381 A TW108122381 A TW 108122381A TW 202035692 A TW202035692 A TW 202035692A
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treatment
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patients
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TW108122381A
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宇野朋典
夏川資
江川洋一
佐藤洋平
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日商日本新藥股份有限公司
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special 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)
  • Microbiology (AREA)
  • 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)
TW108122381A 2018-06-26 2019-06-26 含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途 TW202035692A (zh)

Applications Claiming Priority (4)

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

Publications (1)

Publication Number Publication Date
TW202035692A true TW202035692A (zh) 2020-10-01

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TW108122381A TW202035692A (zh) 2018-06-26 2019-06-26 含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途

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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
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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
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
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
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
JP2018529715A (ja) * 2015-09-30 2018-10-11 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための方法
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine

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Publication number Publication date
PH12020552078A1 (en) 2021-05-31
SG11202011554PA (en) 2020-12-30
CN112399849A (zh) 2021-02-23
JP7345466B2 (ja) 2023-09-15
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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