JPWO2021030778A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2021030778A5
JPWO2021030778A5 JP2022509588A JP2022509588A JPWO2021030778A5 JP WO2021030778 A5 JPWO2021030778 A5 JP WO2021030778A5 JP 2022509588 A JP2022509588 A JP 2022509588A JP 2022509588 A JP2022509588 A JP 2022509588A JP WO2021030778 A5 JPWO2021030778 A5 JP WO2021030778A5
Authority
JP
Japan
Prior art keywords
oligomeric compound
substituted
region
compound according
alkyl
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.)
Pending
Application number
JP2022509588A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022544587A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/046561 external-priority patent/WO2021030778A1/fr
Publication of JP2022544587A publication Critical patent/JP2022544587A/ja
Publication of JPWO2021030778A5 publication Critical patent/JPWO2021030778A5/ja
Pending legal-status Critical Current

Links

JP2022509588A 2019-08-15 2020-08-14 結合修飾オリゴマー化合物及びその使用 Pending JP2022544587A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962887525P 2019-08-15 2019-08-15
US62/887,525 2019-08-15
US201962953121P 2019-12-23 2019-12-23
US62/953,121 2019-12-23
US202062989442P 2020-03-13 2020-03-13
US62/989,442 2020-03-13
US202063050042P 2020-07-09 2020-07-09
US63/050,042 2020-07-09
PCT/US2020/046561 WO2021030778A1 (fr) 2019-08-15 2020-08-14 Composés oligomères modifiés par liaison et leurs utilisations

Publications (2)

Publication Number Publication Date
JP2022544587A JP2022544587A (ja) 2022-10-19
JPWO2021030778A5 true JPWO2021030778A5 (fr) 2023-08-16

Family

ID=74570461

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022509588A Pending JP2022544587A (ja) 2019-08-15 2020-08-14 結合修飾オリゴマー化合物及びその使用

Country Status (6)

Country Link
US (2) US11629348B2 (fr)
EP (1) EP4013767A4 (fr)
JP (1) JP2022544587A (fr)
KR (1) KR20220062517A (fr)
CN (1) CN114555621A (fr)
WO (1) WO2021030778A1 (fr)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021030778A1 (fr) 2019-08-15 2021-02-18 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés par liaison et leurs utilisations
CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
CN111744019B (zh) 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
WO2023278589A1 (fr) * 2021-06-30 2023-01-05 Ionis Pharmaceuticals, Inc. Procédé de synthèse de composés oligomères modifiés par liaison
WO2023023550A1 (fr) * 2021-08-18 2023-02-23 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés par liaison et leurs utilisations
WO2023076710A1 (fr) * 2021-11-01 2023-05-04 A2Tbio Llc Agents d'arn stabilisés
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023149563A1 (fr) * 2022-02-04 2023-08-10 日東電工株式会社 Procédé de fabrication d'oligonucléotide
TW202400786A (zh) * 2022-03-16 2024-01-01 日商第一三共股份有限公司 具有RNAi活性的化學修飾寡核苷酸
WO2023220744A2 (fr) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Oligonucléotides à boucle simple brin
WO2023245060A2 (fr) 2022-06-15 2023-12-21 Arrowhead Pharmaceuticals, Inc. Agents arni pour inhiber l'expression de la superoxyde dismutase 1 (sod1), compositions associées et procédés d'utilisation
WO2023242324A1 (fr) * 2022-06-17 2023-12-21 F. Hoffmann-La Roche Ag Oligonucléotides antisens pour cibler la progranuline
WO2024019137A1 (fr) * 2022-07-22 2024-01-25 住友化学株式会社 Procédé de production d'oligonucléotide
CN116949044B (zh) * 2023-09-20 2023-12-12 北京炫景瑞医药科技有限公司 一种双链寡链核苷酸、包含该双链寡核苷酸的缀合物以及它们的用途

Family Cites Families (159)

* 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
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
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
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
DE3329892A1 (de) 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg Verfahren zur herstellung von oligonucleotiden
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
USRE34036E (en) 1984-06-06 1992-08-18 National Research Development Corporation Data transmission using a transparent tone-in band system
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
DE3788914T2 (de) 1986-09-08 1994-08-25 Ajinomoto Kk Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere.
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
US5276019A (en) 1987-03-25 1994-01-04 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
AU598946B2 (en) 1987-06-24 1990-07-05 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
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
WO1989005358A1 (fr) 1987-11-30 1989-06-15 University Of Iowa Research Foundation Molecules d'adn et d'arn stabilisees par modifications de la liaison de phosphodiester de la terminaison 3', et leur utilisation en tant que sondes d'acide nucleique et en tant qu'agents therapeutiques afin de bloquer l'expression de genes specifiquement cibles
US5403711A (en) 1987-11-30 1995-04-04 University Of Iowa Research Foundation Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved
EP0406309A4 (en) 1988-03-25 1992-08-19 The University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
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
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5194599A (en) 1988-09-23 1993-03-16 Gilead Sciences, Inc. Hydrogen phosphonodithioate compositions
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
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
US5721218A (en) 1989-10-23 1998-02-24 Gilead Sciences, Inc. Oligonucleotides with inverted polarity
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
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
WO1991006556A1 (fr) 1989-10-24 1991-05-16 Gilead Sciences, Inc. Oligonucleotides modifies en position 2'
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5220007A (en) 1990-02-15 1993-06-15 The Worcester Foundation For Experimental Biology Method of site-specific alteration of RNA and production of encoded polypeptides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
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
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
ATE154246T1 (de) 1990-07-27 1997-06-15 Isis Pharmaceuticals Inc Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
HU217036B (hu) 1990-08-03 1999-11-29 Sanofi Eljárás génexpresszió gátlására alkalmas vegyületek előállítására
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
EP0549686A4 (en) 1990-09-20 1995-01-18 Gilead Sciences Inc Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5672697A (en) 1991-02-08 1997-09-30 Gilead Sciences, Inc. Nucleoside 5'-methylene phosphonates
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
DE69232816T2 (de) 1991-11-26 2003-06-18 Isis Pharmaceuticals Inc Gesteigerte bildung von triple- und doppelhelices aus oligomeren mit modifizierten pyrimidinen
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5792608A (en) 1991-12-12 1998-08-11 Gilead Sciences, Inc. Nuclease stable and binding competent oligomers and methods for their use
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5700922A (en) 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
EP0577558A2 (fr) 1992-07-01 1994-01-05 Ciba-Geigy Ag Nucléosides carbocycliques contenant des noyaux bicycliques, oligonucléotides en dérivant, procédé pour leur préparation, leur application et des intermédiaires
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
RU95104940A (ru) 1992-07-27 1997-01-10 Хайбрайдон Способ введения в олигонуклеотид алкилфосфонотиоатной или арилфосфонотиоатной межнуклеотидной связи, способ получения олигонуклеотида, олигонуклеотиды, способ ингибирования генной экспрессии, способ лечения
CA2154578A1 (fr) 1993-01-25 1994-08-04 Ekambar R. Kandimalla Alkylphosphonates et alkylphosphonothioates d'oligonucleotides
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
AU6449394A (en) 1993-03-30 1994-10-24 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
HU9501974D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc Oligonucleotides with amide linkages replacing phosphodiester linkages
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
CN1048254C (zh) 1993-12-09 2000-01-12 托马斯杰弗逊大学 用于将预定的改变引入靶基因中的化合物
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
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
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
ES2203635T3 (es) 1994-04-27 2004-04-16 Novartis Ag Nucleosidos y oligonucleotidos con grupos 2'-eter.
US5646269A (en) 1994-04-28 1997-07-08 Gilead Sciences, Inc. Method for oligonucleotide analog synthesis
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
EP2341058A3 (fr) 1997-09-12 2011-11-23 Exiqon A/S Analogues d'oligonucléotides
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
ATE332969T1 (de) * 1998-10-26 2006-08-15 Avi Biopharma Inc Auf morpholin basierendes p53-antisense- oligonucleotid und dessen verwendungen
NZ513402A (en) 1999-02-12 2003-06-30 Sankyo Co Novel nucleosides and oligonucleotide analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
PT1178999E (pt) 1999-05-04 2007-06-26 Santaris Pharma As Análogos de l-ribo-lna
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
AU2001282522A1 (en) 2000-08-29 2002-03-13 Takeshi Imanishi Novel nucleoside analogs and oligonucleotide derivatives containing these analogs
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
US6426220B1 (en) 2000-10-30 2002-07-30 Isis Pharmaceuticals, Inc. Antisense modulation of calreticulin expression
WO2003002587A2 (fr) 2001-06-29 2003-01-09 Micrologix Biotech Inc. Composes a base d'acides nucleiques
JP2005514005A (ja) 2001-09-04 2005-05-19 エクシコン エ／エス 新規のｌｎａ組成物およびその使用
WO2004041889A2 (fr) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Composes oligomeres renfermant un substitut de sucre polycyclique et compositions intervenant dans la modulation genique
ATE555118T1 (de) 2003-08-28 2012-05-15 Takeshi Imanishi Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ， インコーポレーテッド ４’−チオヌクレオシドおよびオリゴマー化合物
EP1765415A4 (fr) 2004-06-03 2010-03-24 Isis Pharmaceuticals Inc Composes oligomeres facilitant la charge "risc"
EP1801114B8 (fr) * 2005-11-23 2009-12-02 Roche Diagnostics GmbH Polynucléotides contenant des phosphates mimétiques
JP5342881B2 (ja) 2006-01-27 2013-11-13 アイシス ファーマシューティカルズ， インコーポレーテッド ６−修飾された二環式核酸類似体
CN103554205A (zh) 2006-05-05 2014-02-05 Isis制药公司 调节gccr的表达的化合物和方法
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
WO2008109080A2 (fr) 2007-03-01 2008-09-12 Siemens Medical Solutions Usa, Inc. Marqueurs d'imagerie de la prolifération à base de nucléosides
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (fr) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Analogues d'acide nucléique bicyclique carbocylique
CN101796062B (zh) 2007-07-05 2014-07-30 Isis制药公司 6-双取代双环核酸类似物
WO2009067647A1 (fr) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Analogues d'acide nucléique alpha-l-bicyclique carbocyclique
WO2010048585A2 (fr) * 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Composés oligomères et méthodes
WO2011139699A2 (fr) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Nucléosides modifiés en 5' et composés oligomères préparés à partir de ceux-ci
EP2601204B1 (fr) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Nucléosides modifiés et composés oligomères préparés à partir de ceux-ci
EP2742136B1 (fr) * 2011-08-11 2017-09-27 Ionis Pharmaceuticals, Inc. Composés oligomères à brèche comprenant des désoxyribonucléosides modifiés en 5' dans la brèche, et leurs utilisations
KR102095699B1 (ko) 2011-11-18 2020-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴（ＴＴＲ） 관련 질병을 치료하기 위한 ＲＮＡｉ 제제, 조성 및 그의 사용방법
EP2992009B1 (fr) * 2013-05-01 2020-06-24 Ionis Pharmaceuticals, Inc. Compositions et procedes de modulation de l'expression de l'apolipoproteine (a)
AU2014284152B2 (en) * 2013-06-21 2020-01-23 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
SG10201913791QA (en) 2014-08-20 2020-03-30 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
WO2018098328A1 (fr) 2016-11-23 2018-05-31 Alnylam Pharmaceuticals, Inc. Agents arn modifiés à effet hors cible réduit
RU2740501C2 (ru) * 2017-02-21 2021-01-14 Федеральное государственное бюджетное учреждение науки Институт химической биологии и фундаментальной медицины Сибирского отделения Российской академии наук (ИХБФМ СО РАН) МОДИФИЦИРОВАННЫЕ ОЛИГОНУКЛЕОТИДЫ, АКТИВИРУЮЩИЕ РНКазу Н
WO2019073018A1 (fr) * 2017-10-13 2019-04-18 Roche Innovation Center Copenhagen A/S Procédés d'identification de variants d'oligonucléotides phosphorothioate stéréodéfinis améliorés d'oligonucléotides antisens mettant en œuvre des sous-bibliothèques d'oligonucléotides partiellement stéréodéfinis
EP3752612A4 (fr) 2018-02-12 2021-11-10 Ionis Pharmaceuticals, Inc. Composés modifiés et leurs utilisations
CA3096667A1 (fr) 2018-04-12 2019-10-17 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs procedes d'utilisation
KR20210018267A (ko) 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 간외 전달
WO2020072991A1 (fr) 2018-10-05 2020-04-09 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés et leurs utilisations
UY38562A (es) 2019-01-29 2020-08-31 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
EP4013428A4 (fr) 2019-08-15 2023-08-09 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés et leurs utilisations
WO2021030778A1 (fr) 2019-08-15 2021-02-18 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés par liaison et leurs utilisations

Similar Documents

Publication Publication Date Title
JP6978099B2 (ja) ４’−リン酸アナログ及びそれを含むオリゴヌクレオチド
JP7249080B2 (ja) 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用
US20230020192A1 (en) Compounds and methods for modulating c90rf72
US11505569B2 (en) GalNAc phosphoramidites, nucleic acid conjugates thereof and their use
JPWO2021030778A5 (fr)
CN105164261B (zh) 用于增强的细胞摄取的化合物和方法
JP2023164585A (ja) 肝臓外送達
JP2022544587A (ja) 結合修飾オリゴマー化合物及びその使用
JP2019513771A (ja) トリチル−モノ−ＧａｌＮＡｃ化合物とその利用
JP2019530656A5 (fr)
WO2014179446A2 (fr) Composés microarn et procédés permettant la modulation de l'activité de mir-122
JP2017514489A5 (fr)
CA3122930A1 (fr) Molecules d'inhibiteur d'acide nucleique double brin contenant une triboucle
US20220177880A1 (en) Double-stranded nucleic acid inhibitor molecules with shortened sense strands
WO2020262555A1 (fr) Agent de réduction des effets secondaires pour médicament à base d'acide nucléique, composition médicamenteuse comprenant un agent de réduction des effets secondaires pour médicament à base d'acide nucléique et procédé de réduction des propriétés d'induction d'effets secondaires d'un médicament à base d'acide nucléique
WO2024073732A1 (fr) Agents arn double brin modifiés