JP2016533717A5 - - Google Patents
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- JP2016533717A5 JP2016533717A5 JP2016521622A JP2016521622A JP2016533717A5 JP 2016533717 A5 JP2016533717 A5 JP 2016533717A5 JP 2016521622 A JP2016521622 A JP 2016521622A JP 2016521622 A JP2016521622 A JP 2016521622A JP 2016533717 A5 JP2016533717 A5 JP 2016533717A5
- Authority
- JP
- Japan
- Prior art keywords
- compound according
- modified
- modified oligonucleotide
- compound
- linked nucleosides
- 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 88
- 108091034117 Oligonucleotide Proteins 0.000 claims description 44
- 239000002777 nucleoside Substances 0.000 claims description 44
- 125000003835 nucleoside group Chemical group 0.000 claims description 30
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 14
- 230000000295 complement effect Effects 0.000 claims description 13
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 12
- 239000000203 mixture Substances 0.000 claims description 7
- 230000000692 anti-sense effect Effects 0.000 claims description 5
- 125000002619 bicyclic group Chemical group 0.000 claims description 5
- 239000000126 substance Substances 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 239000003085 diluting agent Substances 0.000 claims description 3
- 208000015122 neurodegenerative disease Diseases 0.000 claims description 3
- 150000003839 salts Chemical class 0.000 claims description 3
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 150000004713 phosphodiesters Chemical class 0.000 claims description 2
- 238000010382 chemical cross-linking Methods 0.000 claims 2
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical group P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 239000002953 phosphate buffered saline Substances 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 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
- 125000003545 alkoxy group Chemical group 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 239000008177 pharmaceutical agent Substances 0.000 claims 1
- 159000000000 sodium salts Chemical group 0.000 claims 1
- 238000000034 method Methods 0.000 description 10
- ABEXEQSGABRUHS-UHFFFAOYSA-N 16-methylheptadecyl 16-methylheptadecanoate Chemical compound CC(C)CCCCCCCCCCCCCCCOC(=O)CCCCCCCCCCCCCCC(C)C ABEXEQSGABRUHS-UHFFFAOYSA-N 0.000 description 7
- 241000764238 Isis Species 0.000 description 7
- 238000005417 image-selected in vivo spectroscopy Methods 0.000 description 7
- 238000012739 integrated shape imaging system Methods 0.000 description 7
- 241001465754 Metazoa Species 0.000 description 6
- 230000001154 acute effect Effects 0.000 description 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 6
- 241000700159 Rattus Species 0.000 description 5
- 241000699670 Mus sp. Species 0.000 description 3
- 201000010099 disease Diseases 0.000 description 3
- 208000035475 disorder Diseases 0.000 description 3
- 201000011240 Frontotemporal dementia Diseases 0.000 description 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 2
- 230000001934 delay Effects 0.000 description 2
- 108020004999 messenger RNA Proteins 0.000 description 2
- 125000006239 protecting group Chemical group 0.000 description 2
- 125000004400 (C1-C12) alkyl group Chemical group 0.000 description 1
- KZNQNBZMBZJQJO-UHFFFAOYSA-N 1-(2-azaniumylacetyl)pyrrolidine-2-carboxylate Chemical compound NCC(=O)N1CCCC1C(O)=O KZNQNBZMBZJQJO-UHFFFAOYSA-N 0.000 description 1
- IVNJKQPHHPMONX-WCCKRBBISA-N 2-aminoacetic acid;(2s)-2-amino-5-(diaminomethylideneamino)pentanoic acid Chemical compound NCC(O)=O.OC(=O)[C@@H](N)CCCNC(N)=N IVNJKQPHHPMONX-WCCKRBBISA-N 0.000 description 1
- 241000699666 Mus <mouse, genus> Species 0.000 description 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 1
- 240000007817 Olea europaea Species 0.000 description 1
- 206010034010 Parkinsonism Diseases 0.000 description 1
- 125000000217 alkyl group Chemical group 0.000 description 1
- 210000004227 basal ganglia Anatomy 0.000 description 1
- 208000025434 cerebellar degeneration Diseases 0.000 description 1
- 230000001054 cortical effect Effects 0.000 description 1
- 230000007850 degeneration Effects 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 150000007523 nucleic acids Chemical group 0.000 description 1
- 230000007918 pathogenicity Effects 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 208000011580 syndromic disease Diseases 0.000 description 1
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361890108P | 2013-10-11 | 2013-10-11 | |
| US61/890,108 | 2013-10-11 | ||
| US201361891313P | 2013-10-15 | 2013-10-15 | |
| US61/891,313 | 2013-10-15 | ||
| US201361919540P | 2013-12-20 | 2013-12-20 | |
| US61/919,540 | 2013-12-20 | ||
| US201461927903P | 2014-01-15 | 2014-01-15 | |
| US61/927,903 | 2014-01-15 | ||
| US201461980502P | 2014-04-16 | 2014-04-16 | |
| US61/980,502 | 2014-04-16 | ||
| PCT/US2014/060194 WO2015054676A2 (en) | 2013-10-11 | 2014-10-11 | Compositions for modulating c9orf72 expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020047126A Division JP2020115865A (ja) | 2013-10-11 | 2020-03-18 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016533717A JP2016533717A (ja) | 2016-11-04 |
| JP2016533717A5 true JP2016533717A5 (cg-RX-API-DMAC7.html) | 2017-11-24 |
| JP6679476B2 JP6679476B2 (ja) | 2020-04-15 |
Family
ID=52813758
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016521622A Expired - Fee Related JP6679476B2 (ja) | 2013-10-11 | 2014-10-11 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
| JP2020047126A Ceased JP2020115865A (ja) | 2013-10-11 | 2020-03-18 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020047126A Ceased JP2020115865A (ja) | 2013-10-11 | 2020-03-18 | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
Country Status (16)
Families Citing this family (39)
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| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ | 不斉補助基 |
| WO2014062691A2 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| DK2906696T4 (da) | 2012-10-15 | 2023-02-27 | Ionis Pharmaceuticals Inc | Fremgangsmåder til modulering af c9orf72-ekspression |
| CN112080502A (zh) * | 2013-10-11 | 2020-12-15 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
| PT3094728T (pt) | 2014-01-16 | 2022-05-19 | Wave Life Sciences Ltd | Desenho quiral |
| EP3750907A3 (en) * | 2014-03-18 | 2021-04-28 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| PL3283080T3 (pl) * | 2015-04-16 | 2020-07-27 | Ionis Pharmaceuticals, Inc. | Kompozycja do modulowania ekspresji c9orf72 |
| CN109477106B (zh) * | 2015-07-10 | 2022-10-04 | Ionis制药公司 | 二酰基甘油酰基转移酶2(dgat2)的调节剂 |
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| CN118006607A (zh) | 2017-09-22 | 2024-05-10 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
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| KR20210102294A (ko) | 2018-12-06 | 2021-08-19 | 바이오젠 엠에이 인코포레이티드 | 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질 |
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| AU2020252020A1 (en) * | 2019-03-29 | 2021-11-11 | University Of Massachusetts | Oligonucleotide-based modulation of C90RF72 |
| EP3966328A4 (en) * | 2019-05-06 | 2023-10-18 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
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| WO2021195510A1 (en) * | 2020-03-27 | 2021-09-30 | University Of Massachusetts | Dual-acting sirna based modulation of c9orf72 |
| EP4133073A2 (en) * | 2020-04-09 | 2023-02-15 | Association Institut de Myologie | Antisense sequences for treating amyotrophic lateral sclerosis |
| KR20230043914A (ko) | 2020-07-28 | 2023-03-31 | 아이오니스 파마수티컬즈, 인코포레이티드 | App 발현을 감소시키기 위한 화합물 및 방법 |
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