CN117597133A - 具有模式化化学修饰的双链siRNA - Google Patents

具有模式化化学修饰的双链siRNA Download PDF

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Publication number
CN117597133A
CN117597133A CN202280037397.4A CN202280037397A CN117597133A CN 117597133 A CN117597133 A CN 117597133A CN 202280037397 A CN202280037397 A CN 202280037397A CN 117597133 A CN117597133 A CN 117597133A
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China
Prior art keywords
formula
sirna molecule
nucleotides
length
sirna
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Pending
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CN202280037397.4A
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Chinese (zh)
Inventor
M·哈斯勒
D·库蒂斯
B·M·D·C·戈丁霍
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Atalanta Treatment Co
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Atalanta Treatment Co
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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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
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • 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
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Silicon Polymers (AREA)
  • Chemically Coating (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280037397.4A 2021-03-24 2022-03-24 具有模式化化学修饰的双链siRNA Pending CN117597133A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163165543P 2021-03-24 2021-03-24
US63/165,543 2021-03-24
PCT/US2022/021790 WO2022204430A1 (en) 2021-03-24 2022-03-24 Double-stranded sirna having patterned chemical modifications

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CN117597133A true CN117597133A (zh) 2024-02-23

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US (1) US20240182892A1 (https=)
EP (1) EP4313075A4 (https=)
JP (1) JP2024511473A (https=)
CN (1) CN117597133A (https=)
AU (1) AU2022242899A1 (https=)
CA (1) CA3214660A1 (https=)
WO (1) WO2022204430A1 (https=)

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Publication number Priority date Publication date Assignee Title
IL319764A (en) * 2022-09-28 2025-05-01 Atalanta Therapeutics Inc Compositions and methods for treating Huntington's disease
WO2024169770A1 (zh) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 一种抑制SCN9A基因表达的siRNA、其药物组合物及用途
WO2025085593A1 (en) * 2023-10-18 2025-04-24 University Of Massachusetts Oligonucleotides with ethylene glycol modification
WO2025108284A1 (en) * 2023-11-20 2025-05-30 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of sodium voltage-gated channel alpha subunit 9 (scn9a)
TW202602463A (zh) 2024-03-22 2026-01-16 日商武田藥品工業股份有限公司 用於抑制細胞色素ｐ４５０家族７次家族ａ成員１（ｃｙｐ７ａ１）表現之組成物及方法

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US9181551B2 (en) * 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10125369B2 (en) * 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
EP3277811B1 (en) * 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
EA201792263A1 (ru) * 2015-04-13 2018-08-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ПРОТИВ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
CN108138182B (zh) * 2015-07-31 2022-08-19 阿尔尼拉姆医药品有限公司 甲状腺素运载蛋白(TTR)iRNA组合物及其治疗或预防TTR相关疾病的使用方法
JP7155239B2 (ja) * 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
JP7406793B2 (ja) * 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
IL274611B2 (en) * 2017-11-13 2024-10-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸
US20200385737A1 (en) * 2019-03-29 2020-12-10 University Of Massachusetts OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72
JP2022545118A (ja) * 2019-08-23 2022-10-25 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド

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US20240182892A1 (en) 2024-06-06
EP4313075A1 (en) 2024-02-07
JP2024511473A (ja) 2024-03-13
CA3214660A1 (en) 2022-09-29
WO2022204430A1 (en) 2022-09-29
AU2022242899A1 (en) 2023-11-09
EP4313075A4 (en) 2025-06-18

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