JP2024537098A5 - - Google Patents

Info

Publication number
JP2024537098A5
JP2024537098A5 JP2024520007A JP2024520007A JP2024537098A5 JP 2024537098 A5 JP2024537098 A5 JP 2024537098A5 JP 2024520007 A JP2024520007 A JP 2024520007A JP 2024520007 A JP2024520007 A JP 2024520007A JP 2024537098 A5 JP2024537098 A5 JP 2024537098A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
ligand
oligonucleotide compound
composition according
modified nucleotide
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
JP2024520007A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024537098A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/045664 external-priority patent/WO2023059629A1/en
Publication of JP2024537098A publication Critical patent/JP2024537098A/ja
Publication of JP2024537098A5 publication Critical patent/JP2024537098A5/ja
Pending legal-status Critical Current

Links

JP2024520007A 2021-10-05 2022-10-04 オリゴヌクレオチド化合物の遺伝子サイレンシング活性を増強するための組成物及び方法 Pending JP2024537098A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163252596P 2021-10-05 2021-10-05
US63/252,596 2021-10-05
PCT/US2022/045664 WO2023059629A1 (en) 2021-10-05 2022-10-04 Compositions and methods for enhancing gene silencing activity of oligonucleotide compounds

Publications (2)

Publication Number Publication Date
JP2024537098A JP2024537098A (ja) 2024-10-10
JP2024537098A5 true JP2024537098A5 (https=) 2025-10-10

Family

ID=84332288

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024520007A Pending JP2024537098A (ja) 2021-10-05 2022-10-04 オリゴヌクレオチド化合物の遺伝子サイレンシング活性を増強するための組成物及び方法

Country Status (8)

Country Link
US (1) US20250011779A1 (https=)
EP (1) EP4413135A1 (https=)
JP (1) JP2024537098A (https=)
CN (1) CN118556125A (https=)
AU (1) AU2022359289A1 (https=)
CA (1) CA3234369A1 (https=)
MX (1) MX2024004306A (https=)
WO (1) WO2023059629A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202513799A (zh) * 2023-06-02 2025-04-01 大陸商維亞臻生物技術(蘇州)有限公司 用於免疫類疾病的雙鏈核苷酸化合物及其用途
WO2025231397A1 (en) * 2024-05-03 2025-11-06 Tenaya Therapeutics, Inc. Modified hek cell lines for improved aav production

Family Cites Families (27)

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Publication number Priority date Publication date Assignee Title
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5976567A (en) 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
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
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
CA2487274A1 (en) 2002-05-06 2003-11-13 Nucleonics Inc. Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
CA2685127C (en) 2007-04-23 2019-01-08 Alnylam Pharmaceuticals, Inc. Glycoconjugates of rna interference agents
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
EP3564374A1 (en) 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
EP3356415B1 (en) 2015-09-29 2024-05-01 Amgen Inc. Asgr inhibitors for reduzing cholesterol levels

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