CN111511915A - 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
CN111511915A
CN111511915A CN201980006746.4A CN201980006746A CN111511915A CN 111511915 A CN111511915 A CN 111511915A CN 201980006746 A CN201980006746 A CN 201980006746A CN 111511915 A CN111511915 A CN 111511915A
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Prior art keywords
oligonucleotide
compound
solution
solvate
acceptable salt
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Pending
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CN201980006746.4A
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English (en)
Chinese (zh)
Inventor
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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Daiichi Sankyo Co Ltd
Kobe Gakuin Educational Foundation
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Daiichi Sankyo Co Ltd
Kobe Gakuin Educational Foundation
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Application filed by Daiichi Sankyo Co Ltd, Kobe Gakuin Educational Foundation filed Critical Daiichi Sankyo Co Ltd
Priority to CN202310034281.1A priority Critical patent/CN115976028B/zh
Publication of CN111511915A publication Critical patent/CN111511915A/zh
Pending legal-status Critical Current

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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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    • 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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    • 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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    • 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
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    • C12N2310/30—Chemical structure
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    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/30—Chemical structure
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2320/33—Alteration of splicing
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CN201980006746.4A 2018-03-09 2019-03-05 糖原病Ia型治疗药 Pending CN111511915A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202310034281.1A CN115976028B (zh) 2018-03-09 2019-03-05 糖原病Ia型治疗药

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2018043524 2018-03-09
JP2018-043524 2018-03-09
JP2018-128015 2018-07-05
JP2018128015 2018-07-05
PCT/JP2019/008713 WO2019172286A1 (ja) 2018-03-09 2019-03-05 糖原病Ｉａ型治療薬

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CN111511915A true CN111511915A (zh) 2020-08-07

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CN202310034281.1A Active CN115976028B (zh) 2018-03-09 2019-03-05 糖原病Ia型治疗药

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US (1) US11958878B2 (https=)
EP (1) EP3763815A4 (https=)
JP (3) JP6884268B2 (https=)
KR (2) KR20210130854A (https=)
CN (2) CN111511915A (https=)
TW (3) TWI760600B (https=)
WO (1) WO2019172286A1 (https=)

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