JP2024528659A - 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物 - Google Patents

非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物 Download PDF

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JP2024528659A
JP2024528659A JP2024503387A JP2024503387A JP2024528659A JP 2024528659 A JP2024528659 A JP 2024528659A JP 2024503387 A JP2024503387 A JP 2024503387A JP 2024503387 A JP2024503387 A JP 2024503387A JP 2024528659 A JP2024528659 A JP 2024528659A
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nucleotides
nucleotide
antisense strand
nucleotide sequence
phosphate
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エム ガンスナー，ジョン
アーブ，デイビッド
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Alnylam Pharmaceuticals Inc
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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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    • C12Y101/03—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
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    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Life Sciences & Earth Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024503387A 2021-07-19 2022-07-18 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物 Pending JP2024528659A (ja)

Applications Claiming Priority (3)

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US202163223278P 2021-07-19 2021-07-19
US63/223,278 2021-07-19
PCT/US2022/037453 WO2023003805A1 (en) 2021-07-19 2022-07-18 Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder

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JP2024528659A true JP2024528659A (ja) 2024-07-30
JP2024528659A5 JP2024528659A5 (https=) 2025-08-19

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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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