JP2013541334A - 修飾されたｉＲＮＡ剤 - Google Patents

修飾されたｉＲＮＡ剤 Download PDF

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Publication number
JP2013541334A
JP2013541334A JP2013529298A JP2013529298A JP2013541334A JP 2013541334 A JP2013541334 A JP 2013541334A JP 2013529298 A JP2013529298 A JP 2013529298A JP 2013529298 A JP2013529298 A JP 2013529298A JP 2013541334 A JP2013541334 A JP 2013541334A
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Prior art keywords
irna
irna duplex
independently
occurrence
duplex agent
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JP2013529298A
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Japanese (ja)
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JP2013541334A5 (https=
Inventor
ジー ラジーヴ，カランソッタシル
ジマーマン，トレーシー
マノハラン，ムシィアー
メイアー，マーティン
フィッツゲラルド，ケヴィン
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of JP2013541334A publication Critical patent/JP2013541334A/ja
Publication of JP2013541334A5 publication Critical patent/JP2013541334A5/ja
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • C12N15/09—Recombinant DNA-technology
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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
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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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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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
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2013529298A 2010-09-15 2011-09-14 修飾されたｉＲＮＡ剤 Pending JP2013541334A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US38325010P 2010-09-15 2010-09-15
US61/383,250 2010-09-15
PCT/US2011/051597 WO2012037254A1 (en) 2010-09-15 2011-09-14 MODIFIED iRNA AGENTS

Publications (2)

Publication Number Publication Date
JP2013541334A true JP2013541334A (ja) 2013-11-14
JP2013541334A5 JP2013541334A5 (https=) 2014-10-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
JP2013529298A Pending JP2013541334A (ja) 2010-09-15 2011-09-14 修飾されたｉＲＮＡ剤

Country Status (6)

Country Link
US (1) US9290760B2 (https=)
EP (1) EP2616543A1 (https=)
JP (1) JP2013541334A (https=)
AU (1) AU2011302152B2 (https=)
CA (1) CA2812046A1 (https=)
WO (1) WO2012037254A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
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KR20170002471A (ko) * 2014-05-01 2017-01-06 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
WO2017010575A1 (ja) * 2015-07-16 2017-01-19 協和発酵キリン株式会社 β２ＧＰＩ遺伝子発現抑制核酸複合体
US10487103B2 (en) 2015-03-24 2019-11-26 Gifu University Oligonucleotide derivative, oligonucleotide construct using the same, and methods for producing them
JP2019534005A (ja) * 2016-11-07 2019-11-28 ナノスール エルエルシー 転写後化学修飾二重鎖ｒｎａ

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