CN119662650A - 用于抑制α-ENaC表达的RNAi剂及使用方法 - Google Patents

用于抑制α-ENaC表达的RNAi剂及使用方法 Download PDF

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Publication number
CN119662650A
CN119662650A CN202411877733.3A CN202411877733A CN119662650A CN 119662650 A CN119662650 A CN 119662650A CN 202411877733 A CN202411877733 A CN 202411877733A CN 119662650 A CN119662650 A CN 119662650A
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China
Prior art keywords
enac
nucleotides
rnai agent
antisense strand
sense strand
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CN202411877733.3A
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English (en)
Chinese (zh)
Inventor
李珍
朱锐
裴涛
A·尼古拉斯
E·W·布什
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Arrowhead Pharmaceuticals Inc
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Arrowhead Pharmaceuticals Inc
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Publication of CN119662650A publication Critical patent/CN119662650A/zh
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/343—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Endocrinology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202411877733.3A 2017-07-06 2018-07-05 用于抑制α-ENaC表达的RNAi剂及使用方法 Pending CN119662650A (zh)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201762529132P 2017-07-06 2017-07-06
US62/529132 2017-07-06
US201862631683P 2018-02-17 2018-02-17
US62/631683 2018-02-17
US201862679549P 2018-06-01 2018-06-01
US62/679549 2018-06-01
CN201880045293.1A CN110832077B (zh) 2017-07-06 2018-07-05 用于抑制α-ENaC表达的RNAi剂及使用方法
PCT/US2018/040874 WO2019010274A1 (en) 2017-07-06 2018-07-05 RNAI AGENTS OF INHIBITION OF ALPHA-ENAC GENE EXPRESSION AND METHODS OF USE

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Publications (1)

Publication Number Publication Date
CN119662650A true CN119662650A (zh) 2025-03-21

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CN202411877733.3A Pending CN119662650A (zh) 2017-07-06 2018-07-05 用于抑制α-ENaC表达的RNAi剂及使用方法

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EP (1) EP3649240A4 (enExample)
JP (2) JP2020526192A (enExample)
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CN (2) CN110832077B (enExample)
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CA (1) CA3061752A1 (enExample)
CL (1) CL2020000019A1 (enExample)
CO (1) CO2019014671A2 (enExample)
CR (1) CR20190572A (enExample)
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UY (1) UY37800A (enExample)
WO (1) WO2019010274A1 (enExample)
ZA (1) ZA202100643B (enExample)

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TW202130809A (zh) * 2019-10-29 2021-08-16 美商愛羅海德製藥公司 用於抑制β-ENaC表現之RNAi藥劑、其組合物及使用方法
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EP4562147A1 (en) * 2022-07-27 2025-06-04 E-Therapeutics plc Nucleic acid compounds
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CN110832077B (zh) 2025-01-03
MX2019014800A (es) 2020-02-10
PE20200746A1 (es) 2020-07-24
US20220090077A1 (en) 2022-03-24
EP3649240A4 (en) 2021-07-07
JP2020526192A (ja) 2020-08-31
JP2023158192A (ja) 2023-10-26
IL271843A (en) 2020-02-27
EP3649240A1 (en) 2020-05-13
TW201919654A (zh) 2019-06-01
US20190010494A1 (en) 2019-01-10
CA3061752A1 (en) 2019-01-10
CN110832077A (zh) 2020-02-21
PH12019502660A1 (en) 2020-06-08
BR112019023650A2 (pt) 2020-06-02
AU2018297262A1 (en) 2020-02-27
JOP20190263A1 (ar) 2019-11-12
US11214802B2 (en) 2022-01-04
TN2019000308A1 (en) 2021-05-07
WO2019010274A1 (en) 2019-01-10
KR20200024793A (ko) 2020-03-09
CR20190572A (es) 2020-05-23
ZA202100643B (en) 2023-03-29
US10590416B2 (en) 2020-03-17
UY37800A (es) 2019-01-02
CO2019014671A2 (es) 2020-01-17
ECSP20000655A (es) 2020-05-29
US20200299691A1 (en) 2020-09-24
CL2020000019A1 (es) 2020-06-12

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