JP7125940B2 - 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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JP7125940B2
JP7125940B2 JP2019531379A JP2019531379A JP7125940B2 JP 7125940 B2 JP7125940 B2 JP 7125940B2 JP 2019531379 A JP2019531379 A JP 2019531379A JP 2019531379 A JP2019531379 A JP 2019531379A JP 7125940 B2 JP7125940 B2 JP 7125940B2
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dystrophin
exon
antisense oligomer
antisense
skipping
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JP2020503010A5 (https=
JP2020503010A (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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Priority to JP2024095058A priority patent/JP7728923B2/ja
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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
    • A61K47/54—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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N2310/10—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
JP2019531379A 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 JP7125940B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2022075930A JP2022101695A (ja) 2016-12-19 2022-05-02 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
JP2024095058A JP7728923B2 (ja) 2016-12-19 2024-06-12 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
JP2025134907A JP2025166161A (ja) 2016-12-19 2025-08-13 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

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662436199P 2016-12-19 2016-12-19
US62/436,199 2016-12-19
US201762443481P 2017-01-06 2017-01-06
US62/443,481 2017-01-06
US201762479177P 2017-03-30 2017-03-30
US62/479,177 2017-03-30
US201762562119P 2017-09-22 2017-09-22
US62/562,119 2017-09-22
PCT/US2017/066351 WO2018118627A1 (en) 2016-12-19 2017-12-14 Exon skipping oligomer conjugates for muscular dystrophy

Related Child Applications (1)

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JP2022075930A Division JP2022101695A (ja) 2016-12-19 2022-05-02 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 JP2020503010A (ja) 2020-01-30
JP2020503010A5 JP2020503010A5 (https=) 2021-01-28
JP7125940B2 true JP7125940B2 (ja) 2022-08-25

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JP2019531379A Active JP7125940B2 (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
JP2022075930A Withdrawn JP2022101695A (ja) 2016-12-19 2022-05-02 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
JP2024095058A Active JP7728923B2 (ja) 2016-12-19 2024-06-12 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
JP2025134907A Pending JP2025166161A (ja) 2016-12-19 2025-08-13 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

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JP2024095058A Active JP7728923B2 (ja) 2016-12-19 2024-06-12 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
JP2025134907A Pending JP2025166161A (ja) 2016-12-19 2025-08-13 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

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US (2) US11382981B2 (https=)
EP (3) EP4122497B1 (https=)
JP (4) JP7125940B2 (https=)
KR (2) KR102639633B1 (https=)
CN (2) CN110337308B (https=)
AU (1) AU2017382741B2 (https=)
BR (1) BR112019012651A2 (https=)
CA (1) CA3046801A1 (https=)
CO (1) CO2019007398A2 (https=)
DK (2) DK3554554T3 (https=)
ES (2) ES2929498T3 (https=)
FI (1) FI4122497T3 (https=)
HR (2) HRP20240705T1 (https=)
HU (2) HUE060065T2 (https=)
IL (2) IL267245B2 (https=)
LT (2) LT3554554T (https=)
MA (1) MA65895B1 (https=)
MD (2) MD3554554T2 (https=)
MX (1) MX2019006989A (https=)
MY (1) MY205600A (https=)
NZ (1) NZ755422A (https=)
PL (2) PL3554554T3 (https=)
PT (2) PT3554554T (https=)
RS (2) RS65583B1 (https=)
SA (1) SA519402143B1 (https=)
SG (1) SG10202100491QA (https=)
SI (2) SI3554554T1 (https=)
SM (2) SMT202400222T1 (https=)
TW (1) TWI780095B (https=)
WO (1) WO2018118627A1 (https=)
ZA (1) ZA201903940B (https=)

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