TW202202622A - 糖原病Ia型治療藥 - Google Patents

糖原病Ia型治療藥 Download PDF

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Publication number
TW202202622A
TW202202622A TW110137469A TW110137469A TW202202622A TW 202202622 A TW202202622 A TW 202202622A TW 110137469 A TW110137469 A TW 110137469A TW 110137469 A TW110137469 A TW 110137469A TW 202202622 A TW202202622 A TW 202202622A
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Taiwan
Prior art keywords
oligonucleotide
compound
solvate
acceptable salt
pharmacologically acceptable
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TW110137469A
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English (en)
Chinese (zh)
Inventor
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
Original Assignee
日商第一三共股份有限公司
學校法人神戶學院
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Publication of TW202202622A publication Critical patent/TW202202622A/zh

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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • 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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    • A—HUMAN NECESSITIES
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    • 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
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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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    • 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
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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
    • A—HUMAN NECESSITIES
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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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    • A01K2227/10—Mammal
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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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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TW110137469A 2018-03-09 2019-03-08 糖原病Ia型治療藥 TW202202622A (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 (1)

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TW202202622A true TW202202622A (zh) 2022-01-16

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

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US (1) US11958878B2 (https=)
EP (1) EP3763815A4 (https=)
JP (3) JP6884268B2 (https=)
KR (2) KR102398295B1 (https=)
CN (2) CN111511915A (https=)
TW (3) TW202202622A (https=)
WO (1) WO2019172286A1 (https=)

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US11958878B2 (en) 2024-04-16
WO2019172286A1 (ja) 2019-09-12
KR20200128513A (ko) 2020-11-13
TWI760600B (zh) 2022-04-11
US20200407394A1 (en) 2020-12-31
JP6884268B2 (ja) 2021-06-09
EP3763815A1 (en) 2021-01-13
TW202003847A (zh) 2020-01-16
CN115976028A (zh) 2023-04-18
JPWO2019172286A1 (ja) 2021-03-18
KR20210130854A (ko) 2021-11-01
JP2023159397A (ja) 2023-10-31
KR102398295B1 (ko) 2022-05-17
EP3763815A4 (en) 2021-12-15
TW202417628A (zh) 2024-05-01
JP2021072800A (ja) 2021-05-13
CN115976028B (zh) 2025-03-25

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