TW202333748A - 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物 - Google Patents

用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物

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TW202333748A
TW202333748A TW111126889A TW111126889A TW202333748A TW 202333748 A TW202333748 A TW 202333748A TW 111126889 A TW111126889 A TW 111126889A TW 111126889 A TW111126889 A TW 111126889A TW 202333748 A TW202333748 A TW 202333748A
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nucleotides
nucleotide sequence
phosphate
antisense
dsrna
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TW111126889A
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English (en)
Chinese (zh)
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約翰Ｍ 甘斯納
大衛 厄爾貝
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美商艾拉倫製藥股份有限公司
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Publication of TW202333748A publication Critical patent/TW202333748A/zh

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    • 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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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03—Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
    • C12Y101/03015—(S)-2-Hydroxy-acid oxidase (1.1.3.15)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01027—L-Lactate dehydrogenase (1.1.1.27)
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    • C12Y105/00—Oxidoreductases acting on the CH-NH group of donors (1.5)
    • C12Y105/05—Oxidoreductases acting on the CH-NH group of donors (1.5) with a quinone or similar compound as acceptor (1.5.5)

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  • Plant Pathology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW111126889A 2021-07-19 2022-07-18 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物 TW202333748A (zh)

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US202163223278P 2021-07-19 2021-07-19
US63/223,278 2021-07-19

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TW202333748A true TW202333748A (zh) 2023-09-01

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US (1) US20240344070A1 (https=)
EP (1) EP4373934A1 (https=)
JP (1) JP2024528659A (https=)
TW (1) TW202333748A (https=)
WO (1) WO2023003805A1 (https=)

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CN118460650B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团（天津）股份有限公司 一种Nedosiran的制备方法

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