CN117580952A - 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 - Google Patents

双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 Download PDF

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CN117580952A
CN117580952A CN202280046071.8A CN202280046071A CN117580952A CN 117580952 A CN117580952 A CN 117580952A CN 202280046071 A CN202280046071 A CN 202280046071A CN 117580952 A CN117580952 A CN 117580952A
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nucleotide
nucleotide sequence
double
stranded oligonucleotide
nucleotides
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梁子才
张鸿雁
高山
李海涛
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Suzhou Ruibo Biotechnology Co ltd
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Suzhou Ruibo Biotechnology Co ltd
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CN202280046071.8A 2021-07-16 2022-07-13 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 Pending CN117580952A (zh)

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CN2021108074639 2021-07-16
CN202110807463 2021-07-16
PCT/CN2022/105340 WO2023284763A1 (zh) 2021-07-16 2022-07-13 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途

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CN117580952A true CN117580952A (zh) 2024-02-20

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US (1) US20240309378A1 (https=)
EP (1) EP4372086A1 (https=)
JP (1) JP2024528634A (https=)
CN (1) CN117580952A (https=)
TW (1) TW202308665A (https=)
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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118147134A (zh) * 2023-11-06 2024-06-07 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN118516348A (zh) * 2024-07-23 2024-08-20 深川生物科技(山东)股份公司 单链核酸或双链核酸及在制备治疗杜氏肌营养不良的药物中的应用
CN118995718A (zh) * 2024-10-24 2024-11-22 苏州炫景生物科技有限公司 含有末端核苷酸修饰的双链寡核苷酸及其在抑制agt基因表达中的应用
CN119410644A (zh) * 2025-01-08 2025-02-11 北京安龙生物医药有限公司 修饰的双链寡核苷酸及其缀合物和组合物
WO2026026776A1 (zh) * 2024-08-01 2026-02-05 北京尧景基因技术有限公司 抑制APP基因表达的siRNA及其缀合物和应用

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
JP7606758B2 (ja) * 2019-05-22 2024-12-26 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
CN119421952A (zh) * 2022-06-27 2025-02-11 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
WO2024140101A1 (zh) * 2022-12-28 2024-07-04 北京炫景瑞医药科技有限公司 修饰的双链寡核苷酸分子、修饰的双链寡核苷酸缀合物及其用途
TW202508607A (zh) * 2023-05-19 2025-03-01 大陸商北京炫景瑞醫藥科技有限公司 雙股寡核苷酸及其共軛物和用途
TW202509216A (zh) * 2023-07-14 2025-03-01 大陸商北京安龍生物醫藥有限公司 靶向血管收縮素原的寡核苷酸及其用途
CN121548643A (zh) * 2023-08-08 2026-02-17 苏州炫景生物科技有限公司 Fto抑制剂组合物及其应用
CN117384907B (zh) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 抑制PCSK9表达的siRNA分子及其应用
WO2026052079A1 (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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100069461A1 (en) * 2005-11-09 2010-03-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
CA2658183A1 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CN102140458B (zh) 2010-01-29 2013-05-22 苏州瑞博生物技术有限公司 小干扰核酸和药物组合物及其制药应用
CN103380113B (zh) 2010-11-15 2018-03-30 生命科技公司 含胺的转染试剂及其制备和使用方法
AU2013299717B2 (en) 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
WO2015006740A2 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
EP3118315A4 (en) * 2014-03-13 2017-11-15 Kyowa Hakko Kirin Co., Ltd. Nucleic acid that inhibits expression of irf5
EP3719127A4 (en) * 2017-12-01 2021-10-20 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE
CN110944675B9 (zh) * 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CA3083970A1 (en) * 2017-12-01 2019-06-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
DK3719128T3 (da) * 2017-12-01 2025-03-03 Suzhou Ribo Life Science Co Ltd Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf
EP3719126B1 (en) * 2017-12-01 2025-01-01 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
AU2018394875B2 (en) 2017-12-29 2023-08-03 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
WO2020135673A1 (zh) * 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118147134A (zh) * 2023-11-06 2024-06-07 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN118147134B (zh) * 2023-11-06 2025-02-25 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN118516348A (zh) * 2024-07-23 2024-08-20 深川生物科技(山东)股份公司 单链核酸或双链核酸及在制备治疗杜氏肌营养不良的药物中的应用
WO2026026776A1 (zh) * 2024-08-01 2026-02-05 北京尧景基因技术有限公司 抑制APP基因表达的siRNA及其缀合物和应用
CN118995718A (zh) * 2024-10-24 2024-11-22 苏州炫景生物科技有限公司 含有末端核苷酸修饰的双链寡核苷酸及其在抑制agt基因表达中的应用
CN119410644A (zh) * 2025-01-08 2025-02-11 北京安龙生物医药有限公司 修饰的双链寡核苷酸及其缀合物和组合物

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TW202308665A (zh) 2023-03-01
WO2023284763A1 (zh) 2023-01-19
JP2024528634A (ja) 2024-07-30
EP4372086A1 (en) 2024-05-22
US20240309378A1 (en) 2024-09-19

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