ATE348104T1 - Azyklische linker enthaltende oligonukleotide und deren verwendungen - Google Patents
Azyklische linker enthaltende oligonukleotide und deren verwendungenInfo
- Publication number
- ATE348104T1 ATE348104T1 AT02769804T AT02769804T ATE348104T1 AT E348104 T1 ATE348104 T1 AT E348104T1 AT 02769804 T AT02769804 T AT 02769804T AT 02769804 T AT02769804 T AT 02769804T AT E348104 T1 ATE348104 T1 AT E348104T1
- Authority
- AT
- Austria
- Prior art keywords
- nucleic acid
- target nucleic
- prevention
- function
- oligonucleotides containing
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 3
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 4
- 150000007523 nucleic acids Chemical class 0.000 abstract 4
- 102000039446 nucleic acids Human genes 0.000 abstract 4
- 230000002265 prevention Effects 0.000 abstract 3
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 238000004458 analytical method Methods 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 230000015556 catabolic process Effects 0.000 abstract 1
- 238000003776 cleavage reaction Methods 0.000 abstract 1
- 238000006731 degradation reaction Methods 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 230000010076 replication Effects 0.000 abstract 1
- 238000010839 reverse transcription Methods 0.000 abstract 1
- 230000007017 scission Effects 0.000 abstract 1
- 238000002560 therapeutic procedure Methods 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (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)
- Saccharide Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US33071901P | 2001-10-29 | 2001-10-29 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE348104T1 true ATE348104T1 (de) | 2007-01-15 |
Family
ID=23291016
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT02769804T ATE348104T1 (de) | 2001-10-29 | 2002-10-29 | Azyklische linker enthaltende oligonukleotide und deren verwendungen |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20050233455A1 (de) |
| EP (1) | EP1440079B1 (de) |
| JP (1) | JP2005508634A (de) |
| AT (1) | ATE348104T1 (de) |
| CA (1) | CA2465129A1 (de) |
| DE (1) | DE60216798T2 (de) |
| MX (1) | MXPA04004056A (de) |
| NZ (1) | NZ532610A (de) |
| WO (1) | WO2003037909A1 (de) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7563618B2 (en) | 2001-03-23 | 2009-07-21 | Geron Corporation | Oligonucleotide conjugates |
| WO2006045202A1 (en) | 2004-10-29 | 2006-05-04 | Topigen Pharmaceuticals Inc. | Antisense oligonucleotides for treating allergy and neoplastic cell proliferation |
| EP1987166A4 (de) * | 2006-02-03 | 2010-06-02 | Univ Columbia | Binäre deoxyribozym-sonden zur nukleinsäure-analyse |
| AU2007252192B2 (en) | 2006-05-19 | 2013-10-17 | Topigen Pharmaceuticals Inc. | Oligonucleotides affecting expression of phosphodiesterases |
| EP2560001B1 (de) * | 2006-09-21 | 2016-04-13 | University of Rochester | Zusammensetzungen und Verfahren für eine Proteinverlagerungstherapie für myotone Dystrophie |
| KR101629017B1 (ko) | 2007-05-22 | 2016-06-10 | 아크투루스 쎄라퓨틱스, 인크. | 히드록시메틸 치환된 ｒｎａ 올리고뉴클레오티드 및 ｒｎａ 복합체 |
| EP2285961A4 (de) | 2008-05-15 | 2012-10-17 | Topigen Pharmaceuticals Inc | Oligonukleotide zur behandlung von entzündungen und neoplastischer zellvermehrung |
| SG171879A1 (en) | 2008-12-03 | 2011-07-28 | Marina Biotech Inc | Usirna complexes |
| US20110262989A1 (en) * | 2010-04-21 | 2011-10-27 | Nanomr, Inc. | Isolating a target analyte from a body fluid |
| US10913767B2 (en) | 2010-04-22 | 2021-02-09 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
| WO2011139387A1 (en) | 2010-05-03 | 2011-11-10 | Opko Curna, Llc | Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt) |
| US9243246B2 (en) * | 2010-08-24 | 2016-01-26 | Sirna Therapeutics, Inc. | Single-stranded RNAi agents containing an internal, non-nucleic acid spacer |
| EP2753694B1 (de) * | 2011-09-05 | 2017-08-23 | Stichting Katholieke Universiteit | Antisense-oligonukleotide zur behandlung von leberscher kongenitaler amaurose |
| AU2012308320C1 (en) | 2011-09-14 | 2018-08-23 | Translate Bio Ma, Inc. | Multimeric oligonucleotide compounds |
| CN104039961A (zh) * | 2012-01-07 | 2014-09-10 | 株式会社博纳克 | 具有氨基酸骨架的单链核酸分子 |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| EA201492122A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| AU2013262699A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating ATP2A2 expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| BR112014028631A2 (pt) | 2012-05-16 | 2017-10-17 | Rana Therapeutics Inc | composições e métodos para modulação da expressão da família de genes da hemoglobina |
| WO2013180038A1 (ja) | 2012-05-26 | 2013-12-05 | 株式会社ボナック | デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子 |
| US20150247141A1 (en) | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| WO2015051283A1 (en) | 2013-10-04 | 2015-04-09 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015099122A1 (ja) | 2013-12-26 | 2015-07-02 | 学校法人東京医科大学 | 遺伝子発現制御のための人工ミミックｍｉＲＮＡおよびその用途 |
| CN106068324B (zh) | 2013-12-27 | 2020-12-29 | 株式会社博纳克 | 控制基因表达的人工匹配型miRNA及其用途 |
| US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
| AU2015236215B2 (en) | 2014-03-25 | 2020-03-19 | Arcturus Therapeutics, Inc. | UNA oligomers having reduced off-target effects in gene silencing |
| JP6771387B2 (ja) | 2014-03-25 | 2020-10-21 | アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的ｕｎａオリゴマー |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| KR20170098914A (ko) | 2014-12-27 | 2017-08-30 | 가부시키가이샤 보낙 | 유전자 발현 제어를 위한 천연형 miRNA 및 그의 용도 |
| GB201503408D0 (en) | 2015-02-27 | 2015-04-15 | Proqr Therapeutics N V | Oligonucleotides |
| WO2016158809A1 (ja) | 2015-03-27 | 2016-10-06 | 株式会社ボナック | デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子 |
| US10519447B2 (en) | 2015-04-01 | 2019-12-31 | Arcturus Therapeutics, Inc. | Therapeutic UNA oligomers and uses thereof |
| WO2016164896A2 (en) | 2015-04-10 | 2016-10-13 | Ionis Pharmaceuticals, Inc. | Modulation of smn expression |
| WO2017015671A1 (en) | 2015-07-23 | 2017-01-26 | Arcturus Therapeutics, Inc. | Compositions for treating amyloidosis |
| US11198867B2 (en) | 2016-06-16 | 2021-12-14 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of SMN expression |
| EP3691657A4 (de) | 2017-10-04 | 2021-07-21 | Avidity Biosciences, Inc. | Nukleinsäurepolypeptidzusammensetzungen und verwendungen davon |
| BR112021012066A2 (pt) * | 2018-12-21 | 2021-11-03 | Onxeo | Novas moléculas de ácido nucleico conjugadas e seus usos |
| CN114375296A (zh) | 2019-06-06 | 2022-04-19 | 艾维迪提生物科学公司 | 核酸多肽组合物及其用途 |
| US12006499B2 (en) * | 2019-06-06 | 2024-06-11 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| CN120505310A (zh) | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5399676A (en) * | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
| US5674683A (en) * | 1995-03-21 | 1997-10-07 | Research Corporation Technologies, Inc. | Stem-loop and circular oligonucleotides and method of using |
| WO1997026270A2 (en) * | 1996-01-16 | 1997-07-24 | Ribozyme Pharmaceuticals, Inc. | Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules |
| US6468983B2 (en) * | 1997-04-21 | 2002-10-22 | The Cleveland Clinic Foundation | RNase L activators and antisense oligonucleotides effective to treat telomerase-expressing malignancies |
| EP1071753A2 (de) * | 1998-04-20 | 2001-01-31 | Ribozyme Pharmaceuticals, Inc. | Nukeinsäure moleküle mit einer neuen chemischen zusammensetzung die fähig sind die expression von gene zu modulieren |
| EP0978561A1 (de) * | 1998-08-07 | 2000-02-09 | Hoechst Marion Roussel Deutschland GmbH | Antisense Oligonukleotide zur Hemmung von VEGF-Expression |
-
2002
- 2002-10-29 CA CA002465129A patent/CA2465129A1/en not_active Abandoned
- 2002-10-29 US US10/494,141 patent/US20050233455A1/en not_active Abandoned
- 2002-10-29 MX MXPA04004056A patent/MXPA04004056A/es unknown
- 2002-10-29 JP JP2003540190A patent/JP2005508634A/ja active Pending
- 2002-10-29 NZ NZ532610A patent/NZ532610A/en unknown
- 2002-10-29 DE DE60216798T patent/DE60216798T2/de not_active Expired - Lifetime
- 2002-10-29 EP EP02769804A patent/EP1440079B1/de not_active Expired - Lifetime
- 2002-10-29 WO PCT/CA2002/001628 patent/WO2003037909A1/en not_active Ceased
- 2002-10-29 AT AT02769804T patent/ATE348104T1/de not_active IP Right Cessation
Also Published As
| Publication number | Publication date |
|---|---|
| CA2465129A1 (en) | 2003-05-08 |
| US20050233455A1 (en) | 2005-10-20 |
| WO2003037909A1 (en) | 2003-05-08 |
| NZ532610A (en) | 2008-03-28 |
| MXPA04004056A (es) | 2004-09-10 |
| EP1440079B1 (de) | 2006-12-13 |
| JP2005508634A (ja) | 2005-04-07 |
| EP1440079A1 (de) | 2004-07-28 |
| DE60216798T2 (de) | 2007-10-18 |
| DE60216798D1 (de) | 2007-01-25 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |