JP2015536132A5 - - Google Patents
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- JP2015536132A5 JP2015536132A5 JP2015537009A JP2015537009A JP2015536132A5 JP 2015536132 A5 JP2015536132 A5 JP 2015536132A5 JP 2015537009 A JP2015537009 A JP 2015537009A JP 2015537009 A JP2015537009 A JP 2015537009A JP 2015536132 A5 JP2015536132 A5 JP 2015536132A5
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- JP
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- Prior art keywords
- modified
- modified oligonucleotide
- compound
- compound according
- sugar
- 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.)
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- 150000001875 compounds Chemical class 0.000 claims description 81
- 108091034117 Oligonucleotide Proteins 0.000 claims description 61
- 239000002777 nucleoside Substances 0.000 claims description 35
- 125000003835 nucleoside group Chemical group 0.000 claims description 22
- 235000000346 sugar Nutrition 0.000 claims description 22
- 108020004707 nucleic acids Proteins 0.000 claims description 13
- 150000007523 nucleic acids Chemical class 0.000 claims description 13
- 102000039446 nucleic acids Human genes 0.000 claims description 13
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 13
- 230000000295 complement effect Effects 0.000 claims description 7
- 235000021092 sugar substitutes Nutrition 0.000 claims description 6
- 239000003765 sweetening agent Substances 0.000 claims description 6
- 125000002619 bicyclic group Chemical group 0.000 claims description 5
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 3
- 238000012986 modification Methods 0.000 claims description 3
- 230000004770 neurodegeneration Effects 0.000 claims description 3
- 208000015122 neurodegenerative disease Diseases 0.000 claims description 3
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 3
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 2
- 238000010382 chemical cross-linking Methods 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- 208000027089 Parkinsonian disease Diseases 0.000 claims 1
- 206010034010 Parkinsonism Diseases 0.000 claims 1
- 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 Chemical class 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 claims 1
- 210000004227 basal ganglia Anatomy 0.000 claims 1
- 208000025434 cerebellar degeneration Diseases 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 25
- 238000012230 antisense oligonucleotides Methods 0.000 description 25
- 230000000694 effects Effects 0.000 description 5
- 101000989501 Homo sapiens Guanine nucleotide exchange factor C9orf72 Proteins 0.000 description 4
- 238000011260 co-administration Methods 0.000 description 4
- 102000045815 human C9orf72 Human genes 0.000 description 4
- 230000000692 anti-sense effect Effects 0.000 description 2
- -1 intron Proteins 0.000 description 2
- 108020005345 3' Untranslated Regions Proteins 0.000 description 1
- 108020003589 5' Untranslated Regions Proteins 0.000 description 1
- 230000002730 additional effect Effects 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 238000000034 method Methods 0.000 description 1
- 230000003584 silencer Effects 0.000 description 1
- 230000002195 synergetic effect Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019078283A JP6974386B2 (ja) | 2012-10-15 | 2019-04-17 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261714132P | 2012-10-15 | 2012-10-15 | |
| US61/714,132 | 2012-10-15 | ||
| PCT/US2013/065073 WO2014062691A2 (en) | 2012-10-15 | 2013-10-15 | Compositions for modulating c9orf72 expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019078283A Division JP6974386B2 (ja) | 2012-10-15 | 2019-04-17 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015536132A JP2015536132A (ja) | 2015-12-21 |
| JP2015536132A5 true JP2015536132A5 (https=) | 2016-12-01 |
| JP6570447B2 JP6570447B2 (ja) | 2019-09-04 |
Family
ID=50488880
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015537009A Expired - Fee Related JP6570447B2 (ja) | 2012-10-15 | 2013-10-15 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
| JP2019078283A Expired - Fee Related JP6974386B2 (ja) | 2012-10-15 | 2019-04-17 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
| JP2021126493A Pending JP2021176329A (ja) | 2012-10-15 | 2021-08-02 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019078283A Expired - Fee Related JP6974386B2 (ja) | 2012-10-15 | 2019-04-17 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
| JP2021126493A Pending JP2021176329A (ja) | 2012-10-15 | 2021-08-02 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US10443052B2 (https=) |
| EP (1) | EP2906258A4 (https=) |
| JP (3) | JP6570447B2 (https=) |
| KR (1) | KR20150070278A (https=) |
| CN (2) | CN110951731A (https=) |
| AU (2) | AU2013331434B2 (https=) |
| BR (1) | BR112015008399A8 (https=) |
| CA (1) | CA2888486A1 (https=) |
| HK (1) | HK1212597A1 (https=) |
| IL (1) | IL238266A0 (https=) |
| MX (2) | MX370924B (https=) |
| RU (2) | RU2730677C2 (https=) |
| WO (1) | WO2014062691A2 (https=) |
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| RU2572826C2 (ru) | 2008-12-02 | 2016-01-20 | Чиралджен, Лтд. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
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| HK1212597A1 (zh) | 2012-10-15 | 2016-06-17 | Isis Pharmaceuticals, Inc. | 用於調節c90rf72表達的組合物 |
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| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
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2013
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- 2013-10-15 WO PCT/US2013/065073 patent/WO2014062691A2/en not_active Ceased
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