TWI760600B - 糖原病Ia型治療藥 - Google Patents

糖原病Ia型治療藥 Download PDF

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TWI760600B
TWI760600B TW108107814A TW108107814A TWI760600B TW I760600 B TWI760600 B TW I760600B TW 108107814 A TW108107814 A TW 108107814A TW 108107814 A TW108107814 A TW 108107814A TW I760600 B TWI760600 B TW I760600B
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Taiwan
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compound
mmol
g6pc
synthesis
variant
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TW108107814A
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Chinese (zh)
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TW202003847A (zh
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小泉誠
大西朗之
益田剛
岩本充広
関口幸子
伊藤健太郎
辻真之介
松尾雅文
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日商第一三共股份有限公司
學校法人神戶學院
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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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
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    • 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
    • C12N15/1137—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 enzymes
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    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2320/00—Applications; Uses
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    • C12N2320/33—Alteration of splicing
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)

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TW108107814A 2018-03-09 2019-03-08 糖原病Ia型治療藥 TWI760600B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
JP2018-043524 2018-03-09
JP2018043524 2018-03-09
JP2018-128015 2018-07-05
JP2018128015 2018-07-05

Publications (2)

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TW202003847A TW202003847A (zh) 2020-01-16
TWI760600B true TWI760600B (zh) 2022-04-11

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TW108107814A TWI760600B (zh) 2018-03-09 2019-03-08 糖原病Ia型治療藥
TW112150508A TW202417628A (zh) 2018-03-09 2019-03-08 糖原病Ia型治療藥
TW110137469A TW202202622A (zh) 2018-03-09 2019-03-08 糖原病Ia型治療藥

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TW112150508A TW202417628A (zh) 2018-03-09 2019-03-08 糖原病Ia型治療藥
TW110137469A TW202202622A (zh) 2018-03-09 2019-03-08 糖原病Ia型治療藥

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US (1) US11958878B2 (enExample)
EP (1) EP3763815A4 (enExample)
JP (3) JP6884268B2 (enExample)
KR (2) KR20210130854A (enExample)
CN (2) CN115976028B (enExample)
TW (3) TWI760600B (enExample)
WO (1) WO2019172286A1 (enExample)

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KR20210130854A (ko) 2021-11-01
CN115976028A (zh) 2023-04-18
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