CN111212909A - 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物 - Google Patents

用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物 Download PDF

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CN111212909A
CN111212909A CN201880066319.0A CN201880066319A CN111212909A CN 111212909 A CN111212909 A CN 111212909A CN 201880066319 A CN201880066319 A CN 201880066319A CN 111212909 A CN111212909 A CN 111212909A
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asgr1
rnai agent
sense strand
nucleotide
antisense strand
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CN201880066319.0A
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Chinese (zh)
Inventor
李珍
裴涛
朱锐
B.D.吉文
S.梅尔奎斯特
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Arrowhead Pharmaceuticals Inc
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Arrowhead Pharmaceuticals Inc
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
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  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Dermatology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN201880066319.0A 2017-10-17 2018-10-16 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物 Pending CN111212909A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762573206P 2017-10-17 2017-10-17
US62/573206 2017-10-17
US201762608606P 2017-12-21 2017-12-21
US62/608606 2017-12-21
US201862635277P 2018-02-26 2018-02-26
US62/635277 2018-02-26
PCT/US2018/056077 WO2019079294A1 (en) 2017-10-17 2018-10-16 RNAI-BASED AGENTS AND COMPOSITIONS FOR INHIBITING ASIALOGLYCOPROTEIN RECEPTOR 1 RECEPTOR 1

Publications (1)

Publication Number Publication Date
CN111212909A true CN111212909A (zh) 2020-05-29

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CN201880066319.0A Pending CN111212909A (zh) 2017-10-17 2018-10-16 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物

Country Status (9)

Country Link
US (1) US11492624B2 (enExample)
EP (1) EP3697909A4 (enExample)
JP (2) JP7473472B2 (enExample)
CN (1) CN111212909A (enExample)
AU (1) AU2018352379A1 (enExample)
CA (1) CA3079413A1 (enExample)
TW (1) TW201918555A (enExample)
UY (1) UY37936A (enExample)
WO (1) WO2019079294A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023151624A1 (zh) * 2022-02-11 2023-08-17 武汉大学 Asgr1抑制剂在促进胆固醇外排和治疗非酒精性脂肪性肝病中的应用
WO2024046297A1 (zh) * 2022-09-02 2024-03-07 北京福元医药股份有限公司 抑制去唾液酸糖蛋白受体基因表达的siRNA、其缀合物和药物组合物及用途

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109462981B (zh) * 2016-09-02 2023-07-07 箭头药业股份有限公司 靶向配体

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1587408A (zh) * 2004-08-23 2005-03-02 中国人民解放军军事医学科学院放射医学研究所 靶向去唾液酸糖蛋白受体的抗乙型肝炎病毒反义寡核苷酸的结构和用途
US20110150897A1 (en) * 2006-10-11 2011-06-23 Meyer Thomas F Influenza targets
US20120282674A1 (en) * 2009-09-11 2012-11-08 Maxplanck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Method for the preparation of an influenza virus
US20170096665A1 (en) * 2015-10-01 2017-04-06 Arrowhead Pharmaceuticals, Inc. Compositions and Methods for Inhibiting Gene Expression of LPA
WO2017058944A1 (en) * 2015-09-29 2017-04-06 Amgen Inc. Asgr inhibitors

Family Cites Families (11)

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US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
EP1159441B8 (en) 1999-03-10 2008-10-29 Marie Curie Cancer Care Delivery of nucleic acids and proteins to cells
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
ES2562817T3 (es) 2010-02-24 2016-03-08 Arrowhead Research Corporation Composiciones para el suministro dirigido de ARNip
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
MX2014001971A (es) 2011-08-26 2014-03-31 Arrowhead Res Corp Polimeros de poli(vinilester) para suministro de acido nucleico in vivo.
MX2014001965A (es) 2012-04-18 2014-03-31 Arrowhead Res Corp Polimeros de poli(acrilato) para suministro de acido nucleico in vivo.
CN109069529B (zh) 2016-03-07 2021-08-20 箭头药业股份有限公司 用于治疗性化合物的靶向配体
UY37376A (es) * 2016-08-26 2018-03-23 Amgen Inc Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso
CN109462981B (zh) * 2016-09-02 2023-07-07 箭头药业股份有限公司 靶向配体

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1587408A (zh) * 2004-08-23 2005-03-02 中国人民解放军军事医学科学院放射医学研究所 靶向去唾液酸糖蛋白受体的抗乙型肝炎病毒反义寡核苷酸的结构和用途
US20110150897A1 (en) * 2006-10-11 2011-06-23 Meyer Thomas F Influenza targets
US20120282674A1 (en) * 2009-09-11 2012-11-08 Maxplanck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Method for the preparation of an influenza virus
WO2017058944A1 (en) * 2015-09-29 2017-04-06 Amgen Inc. Asgr inhibitors
US20170096665A1 (en) * 2015-10-01 2017-04-06 Arrowhead Pharmaceuticals, Inc. Compositions and Methods for Inhibiting Gene Expression of LPA

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023151624A1 (zh) * 2022-02-11 2023-08-17 武汉大学 Asgr1抑制剂在促进胆固醇外排和治疗非酒精性脂肪性肝病中的应用
WO2024046297A1 (zh) * 2022-09-02 2024-03-07 北京福元医药股份有限公司 抑制去唾液酸糖蛋白受体基因表达的siRNA、其缀合物和药物组合物及用途

Also Published As

Publication number Publication date
JP2020537653A (ja) 2020-12-24
TW201918555A (zh) 2019-05-16
AU2018352379A1 (en) 2020-06-04
CA3079413A1 (en) 2019-04-25
US11492624B2 (en) 2022-11-08
UY37936A (es) 2019-05-31
JP7473472B2 (ja) 2024-04-23
EP3697909A1 (en) 2020-08-26
WO2019079294A1 (en) 2019-04-25
EP3697909A4 (en) 2021-10-13
JP2024086876A (ja) 2024-06-28
US20200283777A1 (en) 2020-09-10

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Application publication date: 20200529