JP2024539629A5 - - Google Patents

Info

Publication number
JP2024539629A5
JP2024539629A5 JP2024522206A JP2024522206A JP2024539629A5 JP 2024539629 A5 JP2024539629 A5 JP 2024539629A5 JP 2024522206 A JP2024522206 A JP 2024522206A JP 2024522206 A JP2024522206 A JP 2024522206A JP 2024539629 A5 JP2024539629 A5 JP 2024539629A5
Authority
JP
Japan
Prior art keywords
dsrna agent
pharmaceutically acceptable
acceptable salt
group
linkers
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
JP2024522206A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024539629A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/046668 external-priority patent/WO2023064530A1/en
Publication of JP2024539629A publication Critical patent/JP2024539629A/ja
Publication of JP2024539629A5 publication Critical patent/JP2024539629A5/ja
Pending legal-status Critical Current

Links

JP2024522206A 2021-10-15 2022-10-14 肝外デリバリーiRNA組成物およびその使用方法 Pending JP2024539629A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163255984P 2021-10-15 2021-10-15
US63/255,984 2021-10-15
PCT/US2022/046668 WO2023064530A1 (en) 2021-10-15 2022-10-14 Extra-hepatic delivery irna compositions and methods of use thereof

Publications (2)

Publication Number Publication Date
JP2024539629A JP2024539629A (ja) 2024-10-29
JP2024539629A5 true JP2024539629A5 (OSRAM) 2025-10-22

Family

ID=84360072

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024522206A Pending JP2024539629A (ja) 2021-10-15 2022-10-14 肝外デリバリーiRNA組成物およびその使用方法

Country Status (10)

Country Link
US (1) US20240360455A1 (OSRAM)
EP (1) EP4416284A1 (OSRAM)
JP (1) JP2024539629A (OSRAM)
KR (1) KR20240101590A9 (OSRAM)
CN (1) CN118369427A (OSRAM)
AU (1) AU2022364838A1 (OSRAM)
CA (1) CA3234887A1 (OSRAM)
IL (1) IL311864A (OSRAM)
MX (1) MX2024004437A (OSRAM)
WO (1) WO2023064530A1 (OSRAM)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
UY40699A (es) 2023-04-05 2024-08-30 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de pln
CN121793987A (zh) 2023-06-30 2026-04-03 艾维迪提生物科学公司 使用pln靶向性抗体-寡核苷酸缀合物的组合物和方法
IL326940A (en) 2023-09-21 2026-05-01 Alnylam Pharmaceuticals Inc Activin A Receptor Type 1C (ACVR1C) IRNA Compositions and Methods of Using Them
WO2025073871A1 (en) 2023-10-03 2025-04-10 Bicycletx Limited Bicyclic peptide ligand complexes specific for tfr1
WO2025259743A1 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dual conjugate compounds for extrahepatic delivery
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
WO2026007966A1 (zh) * 2024-07-01 2026-01-08 武汉人福创新药物研发中心有限公司 抑制INHBE基因表达的siRNA、偶联物及其应用
CN121450642A (zh) * 2024-08-01 2026-02-03 北京尧景基因技术有限公司 抑制PLN基因表达的siRNA及其缀合物和应用
WO2026046355A1 (zh) * 2024-08-31 2026-03-05 齐鲁制药有限公司 dsRNA分子、药物组合物及其在肝外递送中的应用

Family Cites Families (149)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US105A (en) 1836-12-15 knight
US5218A (en) 1847-08-07 Improvement in plows
US2816110A (en) 1956-11-23 1957-12-10 Merck & Co Inc Methods for the production of substituted pteridines
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
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
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5149782A (en) 1988-08-19 1992-09-22 Tanox Biosystems, Inc. Molecular conjugates containing cell membrane-blending agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DK0942000T3 (da) 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
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
ATE167523T1 (de) 1990-05-11 1998-07-15 Microprobe Corp Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
EP0556301B1 (en) 1990-11-08 2001-01-10 Hybridon, Inc. Incorporation of multiple reporter groups on synthetic oligonucleotides
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US5677195A (en) 1991-11-22 1997-10-14 Affymax Technologies N.V. Combinatorial strategies for polymer synthesis
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5159079A (en) 1991-12-20 1992-10-27 Eli Lilly And Company 2-piperidones as intermediates for 5-deaza-10-oxo- and 5-deaza-10-thio-5,6,7,8-tetrahydrofolic acids
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
WO1993023569A1 (en) 1992-05-11 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
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
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent animal diseases
RU95114435A (ru) 1992-12-14 1997-05-20 Ханивелл Инк. (Us) Система с бесщеточным двигателем постоянного тока
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
WO1994022864A1 (en) 1993-03-30 1994-10-13 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
CA2170869C (en) 1993-09-03 1999-09-14 Phillip Dan Cook Amine-derivatized nucleosides and oligonucleosides
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
ATE277640T1 (de) 1994-03-07 2004-10-15 Dow Chemical Co Bioaktive und/oder gezielte dendrimere-konjugate enthaltend genetisches material
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US6054299A (en) 1994-04-29 2000-04-25 Conrad; Charles A. Stem-loop cloning vector and method
WO2000022114A1 (en) 1998-10-09 2000-04-20 Ingene, Inc. PRODUCTION OF ssDNA $i(IN VIVO)
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
US5801155A (en) 1995-04-03 1998-09-01 Epoch Pharmaceuticals, Inc. Covalently linked oligonucleotide minor grove binder conjugates
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
US5672662A (en) 1995-07-07 1997-09-30 Shearwater Polymers, Inc. Poly(ethylene glycol) and related polymers monosubstituted with propionic or butanoic acids and functional derivatives thereof for biotechnical applications
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US7144869B2 (en) 1995-12-13 2006-12-05 Mirus Bio Corporation Nucleic acid injected into hapatic vein lumen and delivered to primate liver
US8217015B2 (en) 2003-04-04 2012-07-10 Arrowhead Madison Inc. Endosomolytic polymers
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6444806B1 (en) 1996-04-30 2002-09-03 Hisamitsu Pharmaceutical Co., Inc. Conjugates and methods of forming conjugates of oligonucleotides and carbohydrates
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
WO1998051278A2 (en) 1997-05-14 1998-11-19 Inex Pharmaceuticals Corporation High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
CA2303299C (en) 1997-09-12 2016-02-23 Exiqon A/S Oligonucleotide analogues
WO1999054459A2 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
US6335437B1 (en) 1998-09-07 2002-01-01 Isis Pharmaceuticals, Inc. Methods for the preparation of conjugated oligomers
BR9914772A (pt) 1998-10-09 2001-12-11 Ingene Inc Conjunto de elementos genéticos, vetor, célulahospedeira, conjunto para a produção de umasequência de ácido nucléico, método para aprodução in vivo ou in vitro de uma sequência deácido nucléico, transcrição de cdna, molécula deácido nucléico inibidor, transcrição de mrna,molécula heteroduplex e composiçãofarmacêutica
PT1152009E (pt) 1999-02-12 2005-03-31 Sankyo Co Novos analogos de nucleosidos e oligonucleotidos
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US8211468B2 (en) 1999-06-07 2012-07-03 Arrowhead Madison Inc. Endosomolytic polymers
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
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
US6395437B1 (en) 1999-10-29 2002-05-28 Advanced Micro Devices, Inc. Junction profiling using a scanning voltage micrograph
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
US8138383B2 (en) 2002-03-11 2012-03-20 Arrowhead Madison Inc. Membrane active heteropolymers
US8008355B2 (en) 2002-03-11 2011-08-30 Roche Madison Inc. Endosomolytic poly(vinyl ether) polymers
EP2258401B1 (en) 2002-05-06 2014-07-09 Endocyte, Inc. Folate-receptor targeted imaging agents
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US8101348B2 (en) 2002-07-10 2012-01-24 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. RNA-interference by single-stranded RNA molecules
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
JP4597976B2 (ja) 2003-04-17 2010-12-15 アルナイラム ファーマシューティカルズ インコーポレイテッド 修飾iRNA剤
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
JP2007523649A (ja) 2004-02-10 2007-08-23 サーナ・セラピューティクス・インコーポレイテッド 多機能短鎖干渉核酸(多機能siNA)を用い、RNA干渉を介した遺伝子発現の阻害
ATE541928T1 (de) 2005-03-31 2012-02-15 Calando Pharmaceuticals Inc Inhibitoren der untereinheit 2 der ribonukleotidreduktase und ihre verwendungen
WO2007091269A2 (en) 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
CN101460178B (zh) 2006-04-07 2016-12-07 艾德拉药物股份有限公司 用于tlr7和tlr8的稳定化的免疫调节性rna(simra)化合物
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
US8017109B2 (en) 2006-08-18 2011-09-13 Roche Madison Inc. Endosomolytic poly(acrylate) polymers
EP2357231A2 (en) 2007-07-09 2011-08-17 Idera Pharmaceuticals, Inc. Stabilized immune modulatory RNA (SIMRA) compounds
CN102006890A (zh) 2007-12-04 2011-04-06 阿尔尼拉姆医药品有限公司 靶向脂质
NZ588583A (en) 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
CA2732229C (en) 2008-07-25 2023-10-17 Alnylam Pharmaceuticals, Inc. Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand
US20100222417A1 (en) * 2008-11-26 2010-09-02 Alnylam Pharmaceuticals Compostions and methods for enhancing oligonucleotide delivery across and into epithelial tissues
CA2764158A1 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
TR201811076T4 (tr) 2009-06-10 2018-08-27 Arbutus Biopharma Corp Geliştirilmiş lipit formulasyonu.
SI2470656T1 (sl) 2009-08-27 2015-07-31 Idera Pharmaceuticals, Inc. Sestavek za inhibicijo ekspresije genov in njegova uporaba
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
EP2723865B1 (en) 2011-06-21 2019-03-27 Alnylam Pharmaceuticals, Inc. METHODS FOR DETERMINING ACTIVITY OF RNAi IN A SUBJECT
EP3640332A1 (en) * 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
IL303831A (en) 2011-11-18 2023-08-01 Alnylam Pharmaceuticals Inc Adapted RNAI factors
US9114354B2 (en) 2012-06-04 2015-08-25 Z124 Heat transfer device for water recovery system
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
PE20201287A1 (es) 2017-09-14 2020-11-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso
EP3918073A4 (en) * 2019-01-29 2023-11-22 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION
US20220211743A1 (en) * 2019-05-17 2022-07-07 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides

Similar Documents

Publication Publication Date Title
US20250250565A1 (en) Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides
CN114681621B (zh) 肽寡核苷酸缀合物
US8518908B2 (en) Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting of nucleic acids
DE69829760T3 (de) Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
DE69432934T2 (de) Oligonukleotidmodulation der expression von multiwirkstoff-resistenz -assoziiertem protein
US20030134808A1 (en) Oligonucleotide analogues
HUE028389T2 (en) For the synthesis of thiocarbons protecting groups RNSS
TW202307207A (zh) 用於抑制黃嘌呤脫氫酶(XDH)之表現之RNAi藥劑、其醫藥組合物及使用方法
CN112105625A (zh) 核苷酸前体、核苷酸类似物以及含其的寡聚化合物
CN119662650A (zh) 用于抑制α-ENaC表达的RNAi剂及使用方法
CN108472504A (zh) 肽寡核苷酸缀合物
CN121194983A (zh) 5'-膦酸酯修饰的核苷类似物及其制备的寡核苷酸
JP4791665B2 (ja) 接合体ならびにそれらの製造方法および生体膜を横切り分子を輸送するためのそれらの使用
JP2024509783A5 (OSRAM)
CN118496291A (zh) 经修饰的双链寡核苷酸及其用途
JP2025542331A (ja) 新規rna治療薬及びその使用
EP1007735A1 (en) Anti-sense oligonucleotide pharmacological agents
CN117858949A (zh) 用于抑制粘蛋白5AC(MUC5AC)的表达的RNAi试剂、其组合物及其使用方法
TW202528539A (zh) 抑制受磷蛋白之表現的siRNA
TW202019441A (zh) 用於經口遞送寡核苷酸之方法
WO1991018898A1 (en) Nucleic acid homolog
WO2025007274A1 (zh) 一种缀合物及其中间体化合物和用途
EP4105328A1 (en) Antisense-oligonucleotides for prevention of kidney dysfunction promoted by endothelial dysfunction by ephrin-b2 suppression
WO2026077086A1 (zh) 修饰的双链寡核苷酸及其缀合物和组合物
WO2025264948A2 (en) Inhbe rna interference agents