CN112423795A - 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 - Google Patents

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 Download PDF

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CN112423795A
CN112423795A CN201980046893.4A CN201980046893A CN112423795A CN 112423795 A CN112423795 A CN 112423795A CN 201980046893 A CN201980046893 A CN 201980046893A CN 112423795 A CN112423795 A CN 112423795A
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seq
sirna
nucleotide
antisense strand
nucleotide sequence
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张鸿雁
高山
康代武
孔丽娜
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Suzhou Ruibo Biotechnology Co Ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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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Abstract

本公开提供了一种抑制血管生成素样蛋白3基因表达的siRNA，含有该siRNA的药物组合物和缀合物。所述siRNA中的每个核苷酸各自独立地为修饰或未修饰的核苷酸，该siRNA含有正义链和反义链，所述正义链含有核苷酸序列I，所述核苷酸序列I与SEQ ID NO:1所示的核苷酸序列长度相等，且不多于3个核苷酸差异，所述反义链含有核苷酸序列II，所述核苷酸序列II与SEQ ID NO:2所示的核苷酸序列长度相等，且不多于3个核苷酸差异。本公开提供的siRNA及其药物组合物和缀合物，可以有效治疗和/或预防血脂异常。

Description

PCT国内申请，说明书已公开。

Claims (75)

  1. PCT国内申请，权利要求书已公开。
CN201980046893.4A 2018-12-28 2019-12-26 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 Pending CN112423795A (zh)

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CN2018116213966 2018-12-28
CN201811621396 2018-12-28
PCT/CN2019/128686 WO2020135581A1 (zh) 2018-12-28 2019-12-26 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

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CN112423795A true CN112423795A (zh) 2021-02-26

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Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11492620B2 (en) 2017-12-01 2022-11-08 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
US11633482B2 (en) 2017-12-29 2023-04-25 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
US11660347B2 (en) 2017-12-01 2023-05-30 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing same, preparation method, and use thereof
WO2023116607A1 (zh) * 2021-12-21 2023-06-29 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
WO2023227070A1 (zh) * 2022-05-27 2023-11-30 北京福元医药股份有限公司 用于抑制c3基因表达的双链核糖核酸及其修饰物、缀合物和用途
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
CN117568350A (zh) * 2024-01-15 2024-02-20 苏州时安生物技术有限公司 一种用于调节血管紧张素原基因表达的双链rna、其缀合物、药物组合物及用途
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
WO2024125404A1 (zh) * 2022-12-12 2024-06-20 北京福元医药股份有限公司 用于抑制pnpla3基因表达的双链核糖核酸及其修饰物、缀合物和用途
US12084661B2 (en) 2017-12-01 2024-09-10 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
US12083142B2 (en) 2017-12-01 2024-09-10 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof

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WO2022068923A1 (zh) * 2020-09-30 2022-04-07 纳肽得(青岛)生物医药有限公司 血管生成素样3(ANGPTL3)的siRNA及其用途
CN118401665A (zh) * 2021-12-03 2024-07-26 中天(上海)生物科技有限公司 具有高稳定性和基因沉默活性的小干扰rna分子的修饰模式

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Cited By (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11492620B2 (en) 2017-12-01 2022-11-08 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
US11660347B2 (en) 2017-12-01 2023-05-30 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing same, preparation method, and use thereof
US12084661B2 (en) 2017-12-01 2024-09-10 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
US12083142B2 (en) 2017-12-01 2024-09-10 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
US11633482B2 (en) 2017-12-29 2023-04-25 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
WO2023116607A1 (zh) * 2021-12-21 2023-06-29 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
WO2023227070A1 (zh) * 2022-05-27 2023-11-30 北京福元医药股份有限公司 用于抑制c3基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024125404A1 (zh) * 2022-12-12 2024-06-20 北京福元医药股份有限公司 用于抑制pnpla3基因表达的双链核糖核酸及其修饰物、缀合物和用途
CN117568350A (zh) * 2024-01-15 2024-02-20 苏州时安生物技术有限公司 一种用于调节血管紧张素原基因表达的双链rna、其缀合物、药物组合物及用途
CN117568350B (zh) * 2024-01-15 2024-04-30 苏州时安生物技术有限公司 一种用于调节血管紧张素原基因表达的双链rna、其缀合物、药物组合物及用途

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