JP2016516066A - 筋ジストロフィを処置するための改善された組成物 - Google Patents

筋ジストロフィを処置するための改善された組成物 Download PDF

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Publication number
JP2016516066A
JP2016516066A JP2016503163A JP2016503163A JP2016516066A JP 2016516066 A JP2016516066 A JP 2016516066A JP 2016503163 A JP2016503163 A JP 2016503163A JP 2016503163 A JP2016503163 A JP 2016503163A JP 2016516066 A JP2016516066 A JP 2016516066A
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eteprilsen
patient
dystrophin
antisense
treatment
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JP2016516066A5 (enExample
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エドワード エム． ケイ，
エドワード エム． ケイ，
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サレプタ セラピューティクス， インコーポレイテッド
サレプタ セラピューティクス， インコーポレイテッド
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    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2016503163A 2013-03-15 2014-03-14 筋ジストロフィを処置するための改善された組成物 Withdrawn JP2016516066A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361793463P 2013-03-15 2013-03-15
US61/793,463 2013-03-15
PCT/US2014/029610 WO2014144978A2 (en) 2013-03-15 2014-03-14 Improved compositions for treating muscular dystrophy

Related Child Applications (1)

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JP2019030772A Division JP2019108349A (ja) 2013-03-15 2019-02-22 筋ジストロフィを処置するための改善された組成物

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JP2016516066A true JP2016516066A (ja) 2016-06-02
JP2016516066A5 JP2016516066A5 (enExample) 2017-04-20

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JP2016503163A Withdrawn JP2016516066A (ja) 2013-03-15 2014-03-14 筋ジストロフィを処置するための改善された組成物
JP2019030772A Pending JP2019108349A (ja) 2013-03-15 2019-02-22 筋ジストロフィを処置するための改善された組成物

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US (10) US9506058B2 (enExample)
EP (2) EP3662912B1 (enExample)
JP (2) JP2016516066A (enExample)
KR (2) KR20200139271A (enExample)
CN (2) CN113633787A (enExample)
AU (3) AU2014233456B2 (enExample)
BR (1) BR112015022998A2 (enExample)
CA (1) CA2906812A1 (enExample)
EA (1) EA201591792A1 (enExample)
ES (1) ES3063113T3 (enExample)
HK (1) HK1220154A1 (enExample)
IL (3) IL241558B (enExample)
MX (2) MX373959B (enExample)
NZ (2) NZ631289A (enExample)
WO (1) WO2014144978A2 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016517279A (ja) * 2013-03-14 2016-06-16 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物
JP2020503009A (ja) * 2016-12-19 2020-01-30 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2020503010A (ja) * 2016-12-19 2020-01-30 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2021526017A (ja) * 2018-05-29 2021-09-30 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

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JP2020503010A (ja) * 2016-12-19 2020-01-30 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2023156512A (ja) * 2016-12-19 2023-10-24 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
US12377150B2 (en) 2016-12-19 2025-08-05 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP2024113187A (ja) * 2016-12-19 2024-08-21 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP7691462B2 (ja) 2016-12-19 2025-06-11 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP7370344B2 (ja) 2018-05-29 2023-10-27 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2021526017A (ja) * 2018-05-29 2021-09-30 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

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