JP2024535850A - 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法 - Google Patents

補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法 Download PDF

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JP2024535850A
JP2024535850A JP2024516854A JP2024516854A JP2024535850A JP 2024535850 A JP2024535850 A JP 2024535850A JP 2024516854 A JP2024516854 A JP 2024516854A JP 2024516854 A JP2024516854 A JP 2024516854A JP 2024535850 A JP2024535850 A JP 2024535850A
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nucleotides
ome
nucleotide
antisense strand
strand
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JPWO2023044370A5 (https=
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バリー，ジョセフ
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Health & Medical Sciences (AREA)
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  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2024516854A 2021-09-17 2022-09-15 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法 Pending JP2024535850A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163245260P 2021-09-17 2021-09-17
US63/245,260 2021-09-17
PCT/US2022/076464 WO2023044370A2 (en) 2021-09-17 2022-09-15 Irna compositions and methods for silencing complement component 3 (c3)

Publications (3)

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JP2024535850A true JP2024535850A (ja) 2024-10-02
JPWO2023044370A5 JPWO2023044370A5 (https=) 2025-09-25
JP2024535850A5 JP2024535850A5 (https=) 2025-09-25

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JP2024516854A Pending JP2024535850A (ja) 2021-09-17 2022-09-15 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法

Country Status (4)

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US (1) US20250019698A1 (https=)
EP (1) EP4401742A2 (https=)
JP (1) JP2024535850A (https=)
WO (1) WO2023044370A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202134435A (zh) 2019-10-22 2021-09-16 美商阿尼拉製藥公司 補體成分ｃ３ ｉｒｎａ組成物及其使用方法
CN120265773A (zh) * 2022-10-14 2025-07-04 美国圣因生物股份有限公司 靶向c3的小干扰rna及其用途
AU2023367106A1 (en) 2022-10-27 2025-05-01 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement component c3 (c3), pharmaceutical compositions thereof, and methods of use
WO2026052021A1 (zh) * 2024-09-07 2026-03-12 苏州瑞博生物技术股份有限公司 寡核苷酸、寡核苷酸缀合物及组合物和用途

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