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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- Prior art keywords
- modified
- modified oligonucleotide
- compound
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- sugar
- Prior art date
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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 (enExample) | 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 (4) | US10443052B2 (enExample) |
| EP (1) | EP2906258A4 (enExample) |
| JP (3) | JP6570447B2 (enExample) |
| KR (1) | KR20150070278A (enExample) |
| CN (2) | CN110951731A (enExample) |
| AU (2) | AU2013331434B2 (enExample) |
| BR (1) | BR112015008399A8 (enExample) |
| CA (1) | CA2888486A1 (enExample) |
| HK (1) | HK1212597A1 (enExample) |
| IL (1) | IL238266A0 (enExample) |
| MX (2) | MX370924B (enExample) |
| RU (2) | RU2020127664A (enExample) |
| WO (1) | WO2014062691A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
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| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
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| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| US10443052B2 (en) | 2012-10-15 | 2019-10-15 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C9ORF72 expression |
| FI2906696T4 (fi) | 2012-10-15 | 2023-03-18 | Menetelmiä c9orf72:n ilmentymisen moduloimiseksi | |
| EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| RS59283B1 (sr) * | 2013-01-22 | 2019-10-31 | Deutsches Zentrum Fuer Neurodegenerative Erkrankungen E V | Proteini sa dipeptidnim ponovkom kao terapeutsko ciljno mesto kod neurodegenerativnih bolesti sa ekspanzijom heksanukleotidnog ponovka |
| MY192689A (en) | 2013-10-11 | 2022-09-01 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
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| SI3119888T1 (sl) | 2014-03-19 | 2021-11-30 | Ionis Pharmaceuticals, Inc. | Sestavki za moduliranje izražanja ataksina 2 |
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| US10538762B2 (en) | 2014-10-14 | 2020-01-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat |
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| US12344839B2 (en) | 2020-03-27 | 2025-07-01 | University Of Massachusetts | Dual-acting siRNA based modulation of C9orf72 |
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