CN111050806A - 寡核苷酸组合物及其使用方法 - Google Patents

寡核苷酸组合物及其使用方法 Download PDF

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Publication number
CN111050806A
CN111050806A CN201880048436.4A CN201880048436A CN111050806A CN 111050806 A CN111050806 A CN 111050806A CN 201880048436 A CN201880048436 A CN 201880048436A CN 111050806 A CN111050806 A CN 111050806A
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oligonucleotide
composition
oligonucleotides
independently
sequence
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Chinese (zh)
Inventor
C·瓦格斯
N·岩本
D·C·D·巴特勒
S·马拉帕
陆根良
张敬新
V·瓦蒂帕迪卡尔
M·D·弗兰克-卡梅内斯基
L·H·阿波尼
曺永镇
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Priority to CN202510680630.6A priority Critical patent/CN120884604A/zh
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    • 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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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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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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01003—Triacylglycerol lipase (3.1.1.3)

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CN201880048436.4A 2017-06-02 2018-06-01 寡核苷酸组合物及其使用方法 Pending CN111050806A (zh)

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Applications Claiming Priority (11)

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US201762514769P 2017-06-02 2017-06-02
US201762514771P 2017-06-02 2017-06-02
US62/514,769 2017-06-02
US62/514,771 2017-06-02
US201862656949P 2018-04-12 2018-04-12
US62/656,949 2018-04-12
US201862670686P 2018-05-11 2018-05-11
US201862670709P 2018-05-11 2018-05-11
US62/670,709 2018-05-11
US62/670,686 2018-05-11
PCT/US2018/035687 WO2018223056A1 (en) 2017-06-02 2018-06-01 Oligonucleotide compositions and methods of use thereof

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US (2) US11603532B2 (https=)
EP (1) EP3630199A4 (https=)
JP (3) JP2020522510A (https=)
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WO (1) WO2018223056A1 (https=)

Cited By (3)

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CN115916978A (zh) * 2020-05-19 2023-04-04 哈德森医学研究所 寡核苷酸
CN116745306A (zh) * 2020-12-30 2023-09-12 分子渡轮公司 修饰核酸缀合物
WO2025153061A1 (zh) * 2024-01-19 2025-07-24 云合智药（苏州）生物科技有限公司 用于抑制激活素iib型受体基因表达的核酸及其用途

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