JP7687682B2 - 筋疾患治療用医薬組成物 - Google Patents

筋疾患治療用医薬組成物 Download PDF

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JP7687682B2
JP7687682B2 JP2021513667A JP2021513667A JP7687682B2 JP 7687682 B2 JP7687682 B2 JP 7687682B2 JP 2021513667 A JP2021513667 A JP 2021513667A JP 2021513667 A JP2021513667 A JP 2021513667A JP 7687682 B2 JP7687682 B2 JP 7687682B2
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nucleic acid
double
stranded nucleic
acid complex
acid strand
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JPWO2020209285A5 (https=
JPWO2020209285A1 (https=
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隆徳 横田
哲也 永田
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Tokyo Institute of Technology NUC
Institute of Science Tokyo
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Tokyo Institute of Technology NUC
Institute of Science Tokyo
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    • 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
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    • A61K47/554—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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C12N2310/10—Type of nucleic acid
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021513667A 2019-04-08 2020-04-08 筋疾患治療用医薬組成物 Active JP7687682B2 (ja)

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JP2019073832 2019-04-08
JP2019073832 2019-04-08
PCT/JP2020/015818 WO2020209285A1 (ja) 2019-04-08 2020-04-08 筋疾患治療用医薬組成物

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US (1) US20220175817A1 (https=)
EP (1) EP3954395A4 (https=)
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US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
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US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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