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 (https=
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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    • C12N15/113—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
    • C12N2310/30—Chemical structure
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
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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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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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JP2022553348A JP2022553348A (ja) 2022-12-22
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JP7676377B2 true JP7676377B2 (ja) 2025-05-14

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

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JP6868561B2 (ja) 2015-01-28 2021-05-12 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欠損を有する被験者を治療する方法
SG11202002940QA (en) 2017-11-01 2020-04-29 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
US12268733B2 (en) 2018-08-10 2025-04-08 Alexion Pharmaceuticals, Inc. Methods of treating neurofibromatosis type 1 and related conditions with alkaline phosphatase
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
WO2021081026A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
AU2021337652B2 (en) 2020-09-04 2025-01-30 Alexion Pharmaceuticals, Inc. Methods for treating bone mineralization disorders
WO2022226127A1 (en) * 2021-04-20 2022-10-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement component 3 expression
EP4346843A4 (en) * 2021-05-27 2025-05-21 Apellis Pharmaceuticals, Inc. GENOME 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
CN118302525A (zh) 2021-10-29 2024-07-05 阿尔尼拉姆医药品有限公司 补体因子B（CFB）iRNA组合物及其使用方法
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
TW202405170A (zh) * 2022-04-02 2024-02-01 大陸商上海舶望製藥有限公司 用於抑制補體成分c3蛋白表達的組合物和方法
KR20250088573A (ko) * 2022-10-14 2025-06-17 사네진 바이오 유에스에이 인크. C3을 표적화하는 소형 간섭 rna 및 그의 용도
WO2024092105A2 (en) 2022-10-27 2024-05-02 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
WO2024189348A1 (en) 2023-03-14 2024-09-19 Argonaute RNA Limited Conjugate comprising a double stranded rna molecule linked to a single stranded dna molecule
CN121175419A (zh) * 2023-04-14 2025-12-19 苏州炫景生物科技有限公司 抑制c3基因表达的化合物、药物组合物及其用途
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
TW202516005A (zh) * 2023-06-21 2025-04-16 美商翰森生物有限責任公司 Ｓｉｒｎａ，含有ｓｉｒｎａ的綴合物，醫藥組成物及其用途
CN121488036A (zh) * 2023-07-21 2026-02-06 上海舶望制药有限公司 用于抑制补体成分3(c3)表达的组合物和方法
AU2023463068A1 (en) * 2023-09-01 2026-04-16 Rigerna Therapeutics Co., Ltd. Double-stranded oligonucleotide and conjugate for inhibiting expression of c3 genes, and uses thereof
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