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
- 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 (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 (3) | US10443052B2 (enExample) |
| EP (1) | EP2906258A4 (enExample) |
| JP (3) | JP6570447B2 (enExample) |
| KR (1) | KR20150070278A (enExample) |
| CN (2) | CN104968783B (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) | RU2730677C2 (enExample) |
| WO (1) | WO2014062691A2 (enExample) |
Families Citing this family (38)
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| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
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| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| US10443052B2 (en) | 2012-10-15 | 2019-10-15 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C9ORF72 expression |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| ES2762326T5 (es) | 2012-10-15 | 2023-04-27 | Ionis Pharmaceuticals Inc | Métodos para modular la expresión de C9ORF72 |
| LT2948777T (lt) * | 2013-01-22 | 2019-09-25 | Deutsches Zentrum Für Neurodegenerative Erkrankungen E.V. | Baltymai su pasikartojančiais dipeptidais, kaip terapinis taikinys neurodegeneracinių ligų su padidėjusiu heksanukleotidų pasikartojimu atveju |
| MX2016004651A (es) * | 2013-10-11 | 2016-08-05 | Ionis Pharmaceuticals Inc | Composiciones para modular la expresion de c9orf72. |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| AU2015231294B2 (en) | 2014-03-18 | 2020-10-29 | University Of Massachusetts | rAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| EP3119888B1 (en) | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| WO2016024205A1 (en) * | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene |
| WO2016060919A1 (en) | 2014-10-14 | 2016-04-21 | 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 |
| US10793855B2 (en) | 2015-01-06 | 2020-10-06 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of C9ORF72 antisense transcript |
| WO2016167780A1 (en) * | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| EP3722424A1 (en) | 2015-04-16 | 2020-10-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| MY192997A (en) * | 2015-07-10 | 2022-09-20 | Ionis Pharmaceuticals Inc | Modulators of diacyglycerol acyltransferase 2 (dgat2) |
| JP7089763B2 (ja) * | 2015-10-05 | 2022-06-23 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | トリヌクレオチドリピート伸長が関与する遺伝的疾患の予防または治療における一本鎖アンチセンスオリゴヌクレオチドの使用 |
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| US20190142856A1 (en) * | 2016-04-13 | 2019-05-16 | Ionis Pharmaceuticals, Inc. | Methods for reducing c9orf72 expression |
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| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JP7246729B2 (ja) * | 2017-05-18 | 2023-03-28 | 国立大学法人京都大学 | 脊髄小脳変性症３６型の予防又は治療用組成物 |
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2013
- 2013-10-15 US US14/436,024 patent/US10443052B2/en active Active
- 2013-10-15 KR KR1020157012430A patent/KR20150070278A/ko not_active Abandoned
- 2013-10-15 BR BR112015008399A patent/BR112015008399A8/pt not_active Application Discontinuation
- 2013-10-15 RU RU2015117680A patent/RU2730677C2/ru active
- 2013-10-15 EP EP13847957.1A patent/EP2906258A4/en active Pending
- 2013-10-15 WO PCT/US2013/065073 patent/WO2014062691A2/en not_active Ceased
- 2013-10-15 MX MX2015004774A patent/MX370924B/es active IP Right Grant
- 2013-10-15 RU RU2020127664A patent/RU2020127664A/ru unknown
- 2013-10-15 CA CA2888486A patent/CA2888486A1/en not_active Abandoned
- 2013-10-15 AU AU2013331434A patent/AU2013331434B2/en not_active Ceased
- 2013-10-15 HK HK16100488.1A patent/HK1212597A1/zh unknown
- 2013-10-15 CN CN201380053819.8A patent/CN104968783B/zh not_active Expired - Fee Related
- 2013-10-15 JP JP2015537009A patent/JP6570447B2/ja not_active Expired - Fee Related
- 2013-10-15 CN CN201911095323.2A patent/CN110951731A/zh active Pending
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2015
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- 2019-04-17 JP JP2019078283A patent/JP6974386B2/ja not_active Expired - Fee Related
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- 2021-08-02 JP JP2021126493A patent/JP2021176329A/ja active Pending
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2023
- 2023-06-06 US US18/330,084 patent/US20240200067A1/en active Pending
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