JP6546161B2 - Ｂ型肝炎ウイルスを治療するための有機化合物 - Google Patents

Ｂ型肝炎ウイルスを治療するための有機化合物 Download PDF

Info

Publication number
JP6546161B2
JP6546161B2 JP2016519839A JP2016519839A JP6546161B2 JP 6546161 B2 JP6546161 B2 JP 6546161B2 JP 2016519839 A JP2016519839 A JP 2016519839A JP 2016519839 A JP2016519839 A JP 2016519839A JP 6546161 B2 JP6546161 B2 JP 6546161B2
Authority
JP
Japan
Prior art keywords
strand
phosphate
end cap
mer
rnai agent
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2016519839A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016534031A5 (enExample
JP2016534031A (ja
Inventor
リー バリザ，ジェレミー
リー バリザ，ジェレミー
ブロマーズ，マルセル
フェルナンデス，セザール
ゲノ，エリン
ゴセルト，アルバール
グリニッジ，ポレット
ヒュースケン，ディーター
フンツィカー，ユルク
ナット，フランソワ，ジャン−シャルル
パトナイク，アヌップ
パターソン，アンドリュー
ロンド，ジャン−ミシェル，レーヌ
バイラー，ヤン
チュー，メイチョン
ホルドルフ，メーガン
Original Assignee
ノバルティス アーゲー
ノバルティス アーゲー
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ノバルティス アーゲー, ノバルティス アーゲー filed Critical ノバルティス アーゲー
Publication of JP2016534031A publication Critical patent/JP2016534031A/ja
Publication of JP2016534031A5 publication Critical patent/JP2016534031A5/ja
Application granted granted Critical
Publication of JP6546161B2 publication Critical patent/JP6546161B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • C12N15/1131—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 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
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • C12N2310/14—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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (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)
JP2016519839A 2013-10-04 2014-09-30 Ｂ型肝炎ウイルスを治療するための有機化合物 Active JP6546161B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361886760P 2013-10-04 2013-10-04
US61/886,760 2013-10-04
PCT/US2014/058314 WO2015050871A2 (en) 2013-10-04 2014-09-30 Organic compounds to treat hepatitis b virus

Publications (3)

Publication Number Publication Date
JP2016534031A JP2016534031A (ja) 2016-11-04
JP2016534031A5 JP2016534031A5 (enExample) 2017-11-09
JP6546161B2 true JP6546161B2 (ja) 2019-07-17

Family

ID=51842794

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2016519839A Active JP6546161B2 (ja) 2013-10-04 2014-09-30 Ｂ型肝炎ウイルスを治療するための有機化合物

Country Status (6)

Country Link
US (1) US10519446B2 (enExample)
EP (1) EP3052107B1 (enExample)
JP (1) JP6546161B2 (enExample)
CN (1) CN105792832B (enExample)
HK (1) HK1221912A1 (enExample)
WO (1) WO2015050871A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019135236A (ja) * 2013-10-04 2019-08-15 ノバルティス アーゲー ＲＮＡ干渉に使用するためのＲＮＡｉ剤用の３’末端キャップ

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106434665B (zh) 2011-06-30 2021-06-08 箭头药业股份有限公司 用于抑制乙型肝炎病毒的基因表达的组合物和方法
EP3052107B1 (en) 2013-10-04 2018-05-02 Novartis AG Organic compounds to treat hepatitis b virus
WO2015051366A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
AU2016296592B2 (en) * 2015-07-17 2021-08-19 Arcturus Therapeutics, Inc. Compositions and agents against Hepatitis B virus and uses thereof
US20170137821A1 (en) 2015-07-17 2017-05-18 Arcturus Therapeutics, Inc. Molecules and agents for treating hepatitis b virus
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법
JOP20170161A1 (ar) * 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
CN110446259B (zh) * 2018-05-04 2022-04-15 大唐移动通信设备有限公司 一种寻呼机会的位置确定方法及通信设备
EP4200419A2 (en) * 2020-08-21 2023-06-28 F. Hoffmann-La Roche AG Use of a1cf inhibitors for treating hepatitis b virus infection

Family Cites Families (106)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
FR2288732A1 (fr) 1974-10-22 1976-05-21 Merck Patent Gmbh Nouveaux esters biphenyliques, procede pour leur preparation et application comme constituants de cristaux liquides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
DE3851889T2 (de) 1987-06-24 1995-04-13 Florey Howard Inst Nukleosid-derivate.
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
DE69033495T2 (de) 1989-10-24 2000-07-20 Isis Pharmaceuticals, Inc. 2'-modifizierte nukleotide
DE69031305T2 (de) 1989-11-03 1998-03-26 Univ Vanderbilt Verfahren zur in vivo-verabreichung von funktionsfähigen fremden genen
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5212295A (en) 1990-01-11 1993-05-18 Isis Pharmaceuticals Monomers for preparation of oligonucleotides having chiral phosphorus linkages
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5506351A (en) 1992-07-23 1996-04-09 Isis Pharmaceuticals Process for the preparation of 2'-O-alkyl guanosine and related compounds
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US6262241B1 (en) 1990-08-13 2001-07-17 Isis Pharmaceuticals, Inc. Compound for detecting and modulating RNA activity and gene expression
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
ATE239484T1 (de) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
WO1994022891A1 (en) 1993-03-31 1994-10-13 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
US5428037A (en) 1993-04-09 1995-06-27 Syntex Pharmaceuticals, Ltd. Heterocyclic derivatives in the treatment of Ischaemia and related diseases
KR970004292B1 (ko) 1993-06-17 1997-03-26 엘지전자 주식회사 브이씨알(vcr)의 수명체크장치
US5571902A (en) 1993-07-29 1996-11-05 Isis Pharmaceuticals, Inc. Synthesis of oligonucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5554746A (en) 1994-05-16 1996-09-10 Isis Pharmaceuticals, Inc. Lactam nucleic acids
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
US6127533A (en) 1997-02-14 2000-10-03 Isis Pharmaceuticals, Inc. 2'-O-aminooxy-modified oligonucleotides
DE69839305T2 (de) 1997-02-27 2009-04-09 Takeda Pharmaceutical Co. Ltd. Aminderivate, ihre herstellung und verwendung als inhibitoren der produktion von amyloid-beta
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6271358B1 (en) 1998-07-27 2001-08-07 Isis Pharmaceuticals, Inc. RNA targeted 2′-modified oligonucleotides that are conformationally preorganized
KR20010099682A (ko) 1998-10-09 2001-11-09 추후보충 단일가닥 ｄｎａ의 효소 합성
US5977341A (en) 1998-11-20 1999-11-02 Isis Pharmaceuticals Inc. Antisense modulation of inhibitor-kappa B kinase-beta expression
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
HK1047109A1 (zh) 1999-10-15 2003-02-07 University Of Massachusetts 作为指定基因干预工具的rna干预轨迹基因
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US20050032733A1 (en) 2001-05-18 2005-02-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
DE10015866A1 (de) 2000-03-30 2001-10-11 Bayer Ag Aryl- und Heteroarylsulfonate
RU2322500C2 (ru) 2000-12-01 2008-04-20 Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. Малые молекулы рнк, опосредующие интерференцию рнк
US6745920B2 (en) 2001-07-23 2004-06-08 Pradeep Yohanne Gupta Piston for dispensing device, dispensing device, product containing dispensing device, method of filling, and method of dispensing
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
US6635428B2 (en) 2001-12-04 2003-10-21 Quest Diagnostics Investments Incorporated Oligonucleotides and methods for detecting hepatitis B viral nucleic acids
GB0212303D0 (en) * 2002-05-28 2002-07-10 Isis Innovation Molecular targetting of IGF-1 receptor
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3＇修饰的干扰rna双链体
WO2005060697A2 (en) 2003-12-19 2005-07-07 Chiron Corporation Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
AU2005203822A1 (en) 2004-01-12 2005-07-21 Lorus Therapeutics Inc. Antisense oligonucleotides directed to ribonucleotide reductase R2 and uses thereof in combination therapies for the treatment of cancer
AU2005248147A1 (en) 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
ATE518954T1 (de) 2004-08-18 2011-08-15 Lorus Therapeutics Inc Kleine interferierende rna-moleküle gegen ribonukleotidreduktase und ihre verwendungen
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
CN101426912A (zh) * 2005-08-17 2009-05-06 瑟纳治疗公司 介导rna干扰的化学修饰短干扰核酸分子
WO2007107162A2 (en) 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
FR2909555B1 (fr) 2006-12-11 2009-01-30 Oreal Emulsion e/h a effet correcteur pour la peau
US8012547B2 (en) 2007-04-24 2011-09-06 Merch Patent Gmbh Pyridine compounds for liquid-crystalline mixtures
BRPI0811170B8 (pt) 2007-05-22 2021-05-25 Arcturus Therapeutics Inc oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
DE102008037790A1 (de) 2008-08-14 2010-02-18 Merck Patent Gmbh Bicyclische Triazolderivate
EP2334636A2 (en) 2008-09-19 2011-06-22 Pfizer Inc. Hydroxamic acid derivatives useful as antibacterial agents
US8772308B2 (en) 2009-01-16 2014-07-08 Virginia Commonwealth University Non-peptidyl, potent, and selective mu opioid receptor antagonists
US8207138B2 (en) 2009-05-19 2012-06-26 Medtronic, Inc. Methods and devices for improved efficiency of RNA delivery to cells
WO2011003780A1 (en) 2009-07-06 2011-01-13 F. Hoffmann-La Roche Ag Bi-specific digoxigenin binding antibodies
AR078173A1 (es) 2009-09-15 2011-10-19 Sanofi Aventis Bifeniloximetil dihidro oxazolopirimidinonas sustituidas, su preparacion y su uso
EP3721943A1 (en) 2009-12-23 2020-10-14 Novartis AG Lipids, lipid compositions and methods of using them
JP6106085B2 (ja) 2010-08-24 2017-03-29 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 内部非核酸スペーサーを含む一本鎖ＲＮＡｉ剤
CN106434665B (zh) * 2011-06-30 2021-06-08 箭头药业股份有限公司 用于抑制乙型肝炎病毒的基因表达的组合物和方法
CN103333890B (zh) * 2012-12-21 2015-04-15 厦门成坤生物技术有限公司 治疗乙型病毒性肝炎的rna干扰制剂
US9504747B2 (en) 2013-03-08 2016-11-29 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
JP6390617B2 (ja) 2013-07-12 2018-09-19 住友化学株式会社 テトラゾリノン化合物及びその用途
WO2015051366A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
CN105683163B (zh) 2013-10-04 2018-11-09 诺华股份有限公司 RNA干扰中使用的RNAi剂的3’端帽
WO2015051135A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compositions to treat hepcidin-related diseases
EP3052107B1 (en) 2013-10-04 2018-05-02 Novartis AG Organic compounds to treat hepatitis b virus
WO2015051044A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
PT3083556T (pt) 2013-12-19 2020-03-05 Novartis Ag Lípidos e composições lipídicas para a entrega de agentes ativos
WO2015095346A1 (en) 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
WO2016010840A1 (en) 2014-07-16 2016-01-21 Novartis Ag Method of encapsulating a nucleic acid in a lipid nanoparticle host
JP6731912B2 (ja) 2014-09-05 2020-07-29 ノバルティス アーゲー 活性物質の送達用の脂質および脂質組成物

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019135236A (ja) * 2013-10-04 2019-08-15 ノバルティス アーゲー ＲＮＡ干渉に使用するためのＲＮＡｉ剤用の３’末端キャップ
US11008570B2 (en) 2013-10-04 2021-05-18 Novartis Ag 3′ end caps for RNAi agents for use in RNA interference

Also Published As

Publication number Publication date
US10519446B2 (en) 2019-12-31
WO2015050871A2 (en) 2015-04-09
US20160215288A1 (en) 2016-07-28
WO2015050871A3 (en) 2015-07-02
EP3052107B1 (en) 2018-05-02
EP3052107A2 (en) 2016-08-10
JP2016534031A (ja) 2016-11-04
CN105792832A (zh) 2016-07-20
HK1221912A1 (zh) 2017-06-16
CN105792832B (zh) 2021-03-23

Similar Documents

Publication Publication Date Title
JP6546161B2 (ja) Ｂ型肝炎ウイルスを治療するための有機化合物
EP3052627B1 (en) Novel formats for organic compounds for use in rna interference
JP7662687B2 (ja) キラルデザイン
JP7625544B2 (ja) キラル制御
JP6841859B2 (ja) ＲＮＡ干渉に使用するためのＲＮＡｉ剤用の３’末端キャップ
BR112020007417A2 (pt) molécula de ácido nucleico para redução de papd5 e papd7 de mrna para o tratamento de infecção hepatite b
WO2015051044A2 (en) Novel formats for organic compounds for use in rna interference
WO2025063238A1 (ja) ホスホランバンの発現を抑制するｓｉＲＮＡ
HK1210595B (en) Method for making chiral oligonucleotides

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170928

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20170928

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180724

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20181023

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20181225

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20190528

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20190620

R150 Certificate of patent or registration of utility model

Ref document number: 6546161

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250