CN115976028B - 糖原病Ia型治疗药 - Google Patents

糖原病Ia型治疗药 Download PDF

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Publication number
CN115976028B
CN115976028B CN202310034281.1A CN202310034281A CN115976028B CN 115976028 B CN115976028 B CN 115976028B CN 202310034281 A CN202310034281 A CN 202310034281A CN 115976028 B CN115976028 B CN 115976028B
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compound
mmol
added
synthesis
reduced pressure
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CN115976028A (zh
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小泉诚
大西朗之
益田刚
岩本充广
关口幸子
伊藤健太郎
辻真之介
松尾雅文
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Shenda University As Legal Person Of National University
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Daiichi Sankyo Co Ltd
Kobe Gakuin Educational Foundation
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    • A—HUMAN NECESSITIES
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    • 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
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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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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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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
    • 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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    • 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
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    • 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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202310034281.1A 2018-03-09 2019-03-05 糖原病Ia型治疗药 Active CN115976028B (zh)

Applications Claiming Priority (6)

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
CN201980006746.4A CN111511915A (zh) 2018-03-09 2019-03-05 糖原病Ia型治疗药
PCT/JP2019/008713 WO2019172286A1 (ja) 2018-03-09 2019-03-05 糖原病Ｉａ型治療薬

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CN115976028A CN115976028A (zh) 2023-04-18
CN115976028B true CN115976028B (zh) 2025-03-25

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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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