AU2003216323B2 - Inhibition of vascular endothelial growth factor (vegf) and vegf receptor gene expression using short interfereing nucleic acid (sina) - Google Patents

Inhibition of vascular endothelial growth factor (vegf) and vegf receptor gene expression using short interfereing nucleic acid (sina) Download PDF

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Publication number
AU2003216323B2
AU2003216323B2 AU2003216323A AU2003216323A AU2003216323B2 AU 2003216323 B2 AU2003216323 B2 AU 2003216323B2 AU 2003216323 A AU2003216323 A AU 2003216323A AU 2003216323 A AU2003216323 A AU 2003216323A AU 2003216323 B2 AU2003216323 B2 AU 2003216323B2
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sina molecule
sina
nucleotides
molecule
rna
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AU2003216323A1 (en
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Leonid Beigelman
James Mcswiggen
Pamela Pavco
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Sirna Therapeutics Inc
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Sirna Therapeutics Inc
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Priority claimed from PCT/US2002/017674 external-priority patent/WO2002096927A2/en
Priority claimed from US10/287,949 external-priority patent/US20040102389A1/en
Application filed by Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Priority claimed from PCT/US2003/005022 external-priority patent/WO2003070910A2/en
Publication of AU2003216323A1 publication Critical patent/AU2003216323A1/en
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Priority to AU2006203062A priority Critical patent/AU2006203062B2/en
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US39379602P 2002-07-03 2002-07-03
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US10/287,949 US20040102389A1 (en) 1995-10-26 2002-11-04 Nucleic acid-mediated treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
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US10/306,747 US20030216335A1 (en) 2001-11-30 2002-11-27 Method and reagent for the modulation of female reproductive diseases and conditions
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Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050054596A1 (en) * 2001-11-30 2005-03-10 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
AU2004266311B2 (en) * 2001-05-18 2009-07-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20050222066A1 (en) * 2001-05-18 2005-10-06 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20040138163A1 (en) * 2002-05-29 2004-07-15 Mcswiggen James RNA interference mediated inhibition of vascular edothelial growth factor and vascular edothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20070203333A1 (en) * 2001-11-30 2007-08-30 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050075304A1 (en) * 2001-11-30 2005-04-07 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
EP1495120B1 (en) * 2002-04-18 2012-10-10 Acuity Pharmaceuticals, Inc Means and methods for the specific modulation of target genes in the eye
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
AU2004308484A1 (en) * 2003-12-23 2005-07-14 The Trustees Of The University Of Pennsylvania Compositions and methods for combined therapy of disease
US20070254291A1 (en) * 2004-06-14 2007-11-01 Xiaoxia Cui Gene Targeting in Eukaryotic Cells by Group II Intron Ribonucleoprotein Particles
WO2005123937A2 (en) * 2004-06-14 2005-12-29 The University Of Texas At Austin Methods for expressing rnp particles in eukaryotic cells
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
DK2029746T3 (da) * 2006-06-12 2012-10-08 Exegenics Inc D B A Opko Health Inc Sammensætninger og fremgangsmåder til siRNA-hæmning af angiogenese
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
US20080287408A1 (en) * 2007-05-14 2008-11-20 Drugtech Corporation Endometriosis treatment
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2010005527A1 (en) 2008-06-30 2010-01-14 Angioblast Systems, Inc. Treatment of eye diseases and excessive neovascularization using a combined therapy
ES2600781T3 (es) * 2008-12-04 2017-02-10 Curna, Inc. Tratamiento para enfermedades relacionadas con el factor de crecimiento del endotelio vascular (vegf) mediante la inhibición de transcritos antisentido naturales de vegf
AU2009322279A1 (en) * 2008-12-04 2011-07-14 Opko Pharmaceuticals, Llc Compositions and methods for selective inhibition of pro-angiogenic VEGF isoforms
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US9222086B2 (en) 2009-09-23 2015-12-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
JP6002382B2 (ja) * 2011-12-07 2016-10-05 株式会社バイオシンクタンク 遺伝子発現阻害剤及び阻害方法
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
AU2018215440B2 (en) 2017-02-06 2024-10-24 Nissan Chemical Corporation Single-stranded oligonucleotide
EP4121018A4 (en) 2020-03-17 2024-07-03 Genevant Sciences Gmbh CATIONIC LIPIDS FOR LIPID DNANOPARTICLE DELIVERY OF THERAPEUTICS TO HEPATIC STELLA CELLS
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
KR20240146674A (ko) 2022-01-31 2024-10-08 제네반트 사이언시즈 게엠베하 지질 나노입자를 위한 이온화 가능한 양이온성 지질
WO2025052278A1 (en) 2023-09-05 2025-03-13 Genevant Sciences Gmbh Pyrrolidine based cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
WO2026022656A2 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Lipid particle formulations with permanently charged cationic lipids
WO2026022677A1 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Permanently charged cationic lipids

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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)
WO2002096927A2 (en) * 2001-05-29 2002-12-05 Ribozyme Pharmaceuticals, Incorporated Ribozyme based treatment of female reproductive diseases
JP2000501941A (ja) * 1995-12-08 2000-02-22 ハイブリドン・インコーポレイテッド 修飾ｖｅｇｆアンチセンスオリゴヌクレオチド
DE19956568A1 (de) * 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis

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