JP7676377B2 - 補体成分Ｃ３ ｉＲＮＡ組成物およびその使用方法 - Google Patents

補体成分Ｃ３ ｉＲＮＡ組成物およびその使用方法 Download PDF

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JP7676377B2
JP7676377B2 JP2022523709A JP2022523709A JP7676377B2 JP 7676377 B2 JP7676377 B2 JP 7676377B2 JP 2022523709 A JP2022523709 A JP 2022523709A JP 2022523709 A JP2022523709 A JP 2022523709A JP 7676377 B2 JP7676377 B2 JP 7676377B2
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nucleotides
antisense strand
salt
nucleotide
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JP2022553348A5 (enExample
JP2022553348A (ja
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キーティング，マーク
ディ マキニンチ，ジェイムズ
ユシアス，クリスティーナ
カイタニス，チャラランボス
カストレノ，アダム
ソロモン，サラ
フィシュレビッチ，エラーネ
ケイ シュリーゲル，マーク
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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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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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
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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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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JP2022523709A 2019-10-22 2020-10-21 補体成分Ｃ３ ｉＲＮＡ組成物およびその使用方法 Active JP7676377B2 (ja)

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US201962924210P 2019-10-22 2019-10-22
US62/924,210 2019-10-22
PCT/US2020/056563 WO2021081026A1 (en) 2019-10-22 2020-10-21 Complement component c3 irna compositions and methods of use thereof

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US (3) US12365896B2 (enExample)
EP (1) EP4048793A1 (enExample)
JP (2) JP7676377B2 (enExample)
KR (1) KR20220084399A (enExample)
CN (1) CN115176004A (enExample)
AR (1) AR120267A1 (enExample)
AU (1) AU2020369515A1 (enExample)
BR (1) BR112022007540A2 (enExample)
CA (1) CA3158320A1 (enExample)
IL (1) IL292360A (enExample)
MX (1) MX2022004726A (enExample)
TW (1) TW202134435A (enExample)
WO (1) WO2021081026A1 (enExample)

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US10603361B2 (en) 2015-01-28 2020-03-31 Alexion Pharmaceuticals, Inc. Methods of treating a subject with an alkaline phosphatase deficiency
US11866701B2 (en) 2017-11-01 2024-01-09 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
US11913039B2 (en) 2018-03-30 2024-02-27 Alexion Pharmaceuticals, Inc. Method for producing recombinant alkaline phosphatase
JP7820148B2 (ja) 2018-08-10 2026-02-25 アレクシオン ファーマシューティカルズ， インコーポレイテッド アルカリホスファターゼを用いて神経線維腫症１型および関連状態を治療する方法
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
BR112022007540A2 (pt) 2019-10-22 2022-07-12 Alnylam Pharmaceuticals Inc Composições de irna de componente complementar c3 e métodos de uso das mesmas
MX2023002673A (es) 2020-09-04 2023-05-12 Alexion Pharma Inc Métodos para el tratamiento de trastornos de mineralización ósea.
IL307721A (en) * 2021-04-20 2023-12-01 Astrazeneca Ireland Ltd Compounds and methods for inhibiting expression of component 3
WO2022251409A2 (en) * 2021-05-27 2022-12-01 Apellis Pharmaceuticals, Inc. Genomic editing of complement
JP2024535850A (ja) * 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 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
CA3234636A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023088455A1 (zh) * 2021-11-22 2023-05-25 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
CN119546763A (zh) * 2022-04-02 2025-02-28 上海舶望制药有限公司 用于抑制补体成分c3蛋白表达的组合物和方法
IL320218A (en) * 2022-10-14 2025-06-01 Sanegene Bio Usa Inc Small interfering RNA targeting C3 and its uses
US12110492B2 (en) * 2022-10-27 2024-10-08 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of complement component C3 (C3), pharmaceutical compositions thereof, and methods of use
WO2024169907A1 (zh) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 一种调节补体C3表达的siRNA、其缀合物和药物组合物及用途
EP4680740A1 (en) 2023-03-14 2026-01-21 Argonaute Rna Limited Conjugate comprising a double stranded rna molecule linked to a single stranded dna molecule
EP4600358A4 (en) * 2023-04-14 2026-05-20 Rigerna Therapeutics Suzhou Co Ltd COMPOUND TO INHIBIT C3 GENE EXPRESSION, PHARMACEUTICAL COMPOSITION AND ASSOCIATED USES
WO2024217504A1 (zh) * 2023-04-18 2024-10-24 深圳扬厉医药技术有限公司 缀合物及其应用和制备
CN121568699A (zh) * 2023-06-21 2026-02-24 翰森生物有限责任公司 Sirna，含有sirna的缀合物，药物组合物及其用途
KR20260041829A (ko) * 2023-07-21 2026-03-27 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 보체 성분 3의 발현을 억제하기 위한 조성물 및 방법
IL326612A (en) * 2023-09-01 2026-04-01 Rigerna Therapeutics Co Ltd Double-stranded oligonucleotide and its conjugate for inhibiting expression of C3 genes and uses thereof
WO2025168134A1 (zh) * 2024-02-08 2025-08-14 北京安龙生物医药有限公司 靶向补体成分c3基因的寡核苷酸及其用途
WO2025252184A1 (en) * 2024-06-07 2025-12-11 Qilu Pharmaceutical Co., Ltd. iRNA COMPOSITION AND METHOD OF USING SAME
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