JP7160807B2 - 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 - Google Patents

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 Download PDF

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JP7160807B2
JP7160807B2 JP2019531380A JP2019531380A JP7160807B2 JP 7160807 B2 JP7160807 B2 JP 7160807B2 JP 2019531380 A JP2019531380 A JP 2019531380A JP 2019531380 A JP2019531380 A JP 2019531380A JP 7160807 B2 JP7160807 B2 JP 7160807B2
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dystrophin
exon
antisense oligomer
antisense
skipping
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JP2020511943A5 (enExample
JP2020511943A (ja
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マルコ エー． パッシーニ，
ガンナー ジェイ． ハンソン，
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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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Genetics & Genomics (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
JP2019531380A 2016-12-19 2017-12-14 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 Active JP7160807B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022164800A JP7465320B2 (ja) 2016-12-19 2022-10-13 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
JP2023174130A JP2023171961A (ja) 2016-12-19 2023-10-06 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

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662436223P 2016-12-19 2016-12-19
US62/436,223 2016-12-19
US201762443484P 2017-01-06 2017-01-06
US62/443,484 2017-01-06
US201762479178P 2017-03-30 2017-03-30
US62/479,178 2017-03-30
US201762562146P 2017-09-22 2017-09-22
US62/562,146 2017-09-22
PCT/US2017/066509 WO2018118662A1 (en) 2016-12-19 2017-12-14 Exon skipping oligomer conjugates for muscular dystrophy

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JP2020511943A JP2020511943A (ja) 2020-04-23
JP2020511943A5 JP2020511943A5 (enExample) 2021-01-21
JP7160807B2 true JP7160807B2 (ja) 2022-10-25

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JP2019531380A Active JP7160807B2 (ja) 2016-12-19 2017-12-14 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
JP2022164800A Active JP7465320B2 (ja) 2016-12-19 2022-10-13 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
JP2023174130A Withdrawn JP2023171961A (ja) 2016-12-19 2023-10-06 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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JP2023174130A Withdrawn JP2023171961A (ja) 2016-12-19 2023-10-06 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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US (3) US11000600B2 (enExample)
EP (2) EP3554552B1 (enExample)
JP (3) JP7160807B2 (enExample)
KR (2) KR20240006057A (enExample)
CN (1) CN110636866B (enExample)
AU (1) AU2017382773B2 (enExample)
BR (1) BR112019012664A2 (enExample)
CA (1) CA3047010A1 (enExample)
CO (1) CO2019007399A2 (enExample)
DK (1) DK3554552T3 (enExample)
ES (1) ES2928486T3 (enExample)
HR (1) HRP20221155T1 (enExample)
HU (1) HUE059905T2 (enExample)
IL (2) IL297528A (enExample)
LT (1) LT3554552T (enExample)
MD (1) MD3554552T2 (enExample)
MX (1) MX2019006879A (enExample)
MY (1) MY205772A (enExample)
NZ (1) NZ755438A (enExample)
PL (1) PL3554552T3 (enExample)
PT (1) PT3554552T (enExample)
RS (1) RS63621B1 (enExample)
SA (1) SA519402226B1 (enExample)
SG (1) SG10202100557UA (enExample)
SM (1) SMT202200403T1 (enExample)
TW (2) TWI794197B (enExample)
WO (1) WO2018118662A1 (enExample)

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TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
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MD3464305T2 (ro) 2016-05-24 2025-01-31 Sarepta Therapeutics Inc Procedee de preparare de oligomeri
DK3554552T3 (da) 2016-12-19 2022-10-24 Sarepta Therapeutics Inc Exon-skipping-oligomerkonjugater mod muskeldystrofi
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EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
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US20230174979A1 (en) * 2019-10-09 2023-06-08 Children’S Medical Center Corporation Methods and compositions for treating ataxia telangiectasia
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CN118201606A (zh) 2021-07-08 2024-06-14 日本新药株式会社 肾毒性减轻剂
WO2023282345A1 (ja) 2021-07-08 2023-01-12 日本新薬株式会社 腎毒性軽減剤
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