JP2018184423A5 - - Google Patents
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- JP2018184423A5 JP2018184423A5 JP2018128926A JP2018128926A JP2018184423A5 JP 2018184423 A5 JP2018184423 A5 JP 2018184423A5 JP 2018128926 A JP2018128926 A JP 2018128926A JP 2018128926 A JP2018128926 A JP 2018128926A JP 2018184423 A5 JP2018184423 A5 JP 2018184423A5
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- 239000008194 pharmaceutical composition Substances 0.000 claims 29
- 230000000692 anti-sense effect Effects 0.000 claims 16
- 150000001875 compounds Chemical class 0.000 claims 16
- 108091034117 Oligonucleotide Proteins 0.000 claims 13
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 10
- 239000002777 nucleoside Substances 0.000 claims 9
- 239000000074 antisense oligonucleotide Substances 0.000 claims 4
- 238000012230 antisense oligonucleotides Methods 0.000 claims 4
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 4
- 125000003835 nucleoside group Chemical group 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 101150081851 SMN1 gene Proteins 0.000 claims 2
- 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 claims 2
- 210000002161 motor neuron Anatomy 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 230000004083 survival effect Effects 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- -1 Methoxyethyl nucleoside Chemical class 0.000 claims 1
- 208000008425 Protein deficiency Diseases 0.000 claims 1
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 230000002068 genetic effect Effects 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21803109P | 2009-06-17 | 2009-06-17 | |
| US61/218,031 | 2009-06-17 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016216200A Division JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019227997A Division JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018184423A JP2018184423A (ja) | 2018-11-22 |
| JP2018184423A5 true JP2018184423A5 (cg-RX-API-DMAC7.html) | 2019-05-16 |
| JP6637121B2 JP6637121B2 (ja) | 2020-01-29 |
Family
ID=43356760
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2018128926A Active JP6637121B2 (ja) | 2009-06-17 | 2018-07-06 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Country Status (25)
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