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 |
| AU2005299218B2 (en) | 2004-10-29 | 2012-04-12 | Pharmaxis Ltd | 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 |
| KR20090035662A (ko) | 2006-05-19 | 2009-04-10 | 토피겐 파마슈티컬스 인코포레이티드 | 포스포디에스테라아제의 발현에 영향을 미치는 올리고누클레오티드 |
| CA2664189C (en) * | 2006-09-21 | 2017-11-21 | University Of Rochester | Compositions and methods related to protein displacement therapy for myotonic dystrophy |
| NZ580712A (en) * | 2007-05-22 | 2011-12-22 | Marina Biotech Inc | Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers |
| CN102099474B (zh) | 2008-05-15 | 2014-05-28 | 托皮根药品公司 | 用于治疗炎症和赘生性细胞增殖的寡核苷酸 |
| WO2010065756A2 (en) | 2008-12-03 | 2010-06-10 | Mdrna, 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 |
| RU2018110642A (ru) * | 2010-05-03 | 2019-02-27 | Курна, Инк. | Лечение заболеваний, связанных с сиртуином (sirt), путем ингибирования природного антисмыслового транскрипта к сиртуину (sirt) |
| EP2609198B8 (de) * | 2010-08-24 | 2018-03-28 | Sirna Therapeutics, Inc. | Einsträngige rnai-mittel mit einem internen nukleinsäurefreien spacer |
| EP2753694B1 (de) | 2011-09-05 | 2017-08-23 | Stichting Katholieke Universiteit | Antisense-oligonukleotide zur behandlung von leberscher kongenitaler amaurose |
| CA2848753C (en) | 2011-09-14 | 2022-07-26 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| ES2645996T3 (es) * | 2012-01-07 | 2017-12-11 | Bonac Corporation | Molécula de ácido nucleico monocatenario que tiene una cadena principal de aminoácidos |
| KR20150030205A (ko) | 2012-05-16 | 2015-03-19 | 라나 테라퓨틱스, 인크. | Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법 |
| BR112014028644A2 (pt) | 2012-05-16 | 2017-08-15 | Rana Therapeutics Inc | Composições e métodos para modulação da expressão de atp2a2 |
| EA201492121A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии семейства генов гемоглобина |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| JP2015518713A (ja) | 2012-05-16 | 2015-07-06 | ラナ セラピューティクス インコーポレイテッド | Ｕｔｒｎ発現を調節するための組成物及び方法 |
| US20150152410A1 (en) | 2012-05-16 | 2015-06-04 | Rana Therapeutics, Inc. | Compositions and methods for modulating mecp2 expression |
| EP2857513A4 (de) | 2012-05-26 | 2016-05-25 | Bonac Corp | Einsträngiges nukleinsäuremolekül zur regelung der expression eines gens mit transportfunktion |
| AU2013315225B2 (en) * | 2012-09-14 | 2018-11-08 | Translate Bio Ma, Inc. | Multimeric oligonucleotide compounds |
| EP3052632A4 (de) | 2013-10-04 | 2017-03-29 | Rana Therapeutics, Inc. | Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als) |
| 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 |
| SG10201913570XA (en) | 2013-12-27 | 2020-03-30 | Bonac Corp | Artificial match-type mirna for controlling gene expression and use therefor |
| CA2946719C (en) | 2014-03-25 | 2023-09-26 | Arcturus Therapeutics, Inc. | Una oligomers having reduced off-target effects in gene silencing |
| US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
| EP3122365B1 (de) | 2014-03-25 | 2023-05-03 | Arcturus Therapeutics, Inc. | Transthyretin-allel-selektive una-oligomere zum gen-silencing |
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| WO2016104775A1 (ja) | 2014-12-27 | 2016-06-30 | 株式会社ボナック | 遺伝子発現制御のための天然型ｍｉＲＮＡおよびその用途 |
| GB201503408D0 (en) | 2015-02-27 | 2015-04-15 | Proqr Therapeutics N V | Oligonucleotides |
| ES2926369T3 (es) | 2015-03-27 | 2022-10-25 | Bonac Corp | Molécula de ácido nucleico monocatenario que tiene función de suministro y capacidad de control de la expresión génica |
| EP3277289A4 (de) | 2015-04-01 | 2018-12-05 | Arcturus Therapeutics, Inc. | Therapeutische una-oligomere und verwendungen davon |
| 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 |
| WO2017218884A1 (en) | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
| AU2018346507B2 (en) | 2017-10-04 | 2025-03-13 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| AU2019408408A1 (en) * | 2018-12-21 | 2021-06-03 | Valerio Therapeutics | New conjugated nucleic acid molecules and their uses |
| CA3142337A1 (en) * | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| EP3980436A4 (de) | 2019-06-06 | 2023-12-20 | Avidity Biosciences, Inc. | Nukleinsäurepolypeptidzusammensetzungen und verwendungen davon |
| CN115279379B (zh) | 2020-02-28 | 2025-05-09 | 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 |
| AU1430097A (en) * | 1996-01-16 | 1997-08-11 | 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 |
| CA2326823A1 (en) * | 1998-04-20 | 1999-10-28 | Ribozyme Pharmaceuticals, Inc. | Nucleic acid molecules with novel chemical compositions capable of modulating gene expression |
| EP0978561A1 (de) * | 1998-08-07 | 2000-02-09 | Hoechst Marion Roussel Deutschland GmbH | Antisense Oligonukleotide zur Hemmung von VEGF-Expression |
-
2002
- 2002-10-29 AT AT02769804T patent/ATE348104T1/de not_active IP Right Cessation
- 2002-10-29 EP EP02769804A patent/EP1440079B1/de not_active Expired - Lifetime
- 2002-10-29 US US10/494,141 patent/US20050233455A1/en not_active Abandoned
- 2002-10-29 WO PCT/CA2002/001628 patent/WO2003037909A1/en not_active Ceased
- 2002-10-29 NZ NZ532610A patent/NZ532610A/en unknown
- 2002-10-29 DE DE60216798T patent/DE60216798T2/de not_active Expired - Lifetime
- 2002-10-29 JP JP2003540190A patent/JP2005508634A/ja active Pending
- 2002-10-29 CA CA002465129A patent/CA2465129A1/en not_active Abandoned
- 2002-10-29 MX MXPA04004056A patent/MXPA04004056A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| NZ532610A (en) | 2008-03-28 |
| EP1440079B1 (de) | 2006-12-13 |
| WO2003037909A1 (en) | 2003-05-08 |
| MXPA04004056A (es) | 2004-09-10 |
| JP2005508634A (ja) | 2005-04-07 |
| DE60216798T2 (de) | 2007-10-18 |
| DE60216798D1 (de) | 2007-01-25 |
| CA2465129A1 (en) | 2003-05-08 |
| EP1440079A1 (de) | 2004-07-28 |
| US20050233455A1 (en) | 2005-10-20 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |