JP2019088329A5 - - Google Patents
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- JP2019088329A5 JP2019088329A5 JP2019030128A JP2019030128A JP2019088329A5 JP 2019088329 A5 JP2019088329 A5 JP 2019088329A5 JP 2019030128 A JP2019030128 A JP 2019030128A JP 2019030128 A JP2019030128 A JP 2019030128A JP 2019088329 A5 JP2019088329 A5 JP 2019088329A5
- Authority
- JP
- Japan
- Prior art keywords
- modified
- modified oligonucleotide
- seq
- compound
- nucleobase
- 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.)
- Pending
Links
- 150000001875 compounds Chemical class 0.000 claims 16
- 108091034117 Oligonucleotide Proteins 0.000 claims 12
- 239000002777 nucleoside Substances 0.000 claims 9
- 125000003835 nucleoside group Chemical group 0.000 claims 7
- 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 6
- 102100020948 Growth hormone receptor Human genes 0.000 claims 6
- 108010068542 Somatotropin Receptors Proteins 0.000 claims 6
- 230000000295 complement effect Effects 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 102000039446 nucleic acids Human genes 0.000 claims 5
- 206010000599 Acromegaly Diseases 0.000 claims 3
- 102000018997 Growth Hormone Human genes 0.000 claims 3
- 108010051696 Growth Hormone Proteins 0.000 claims 3
- 125000002619 bicyclic group Chemical group 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000000122 growth hormone Substances 0.000 claims 3
- 241000282412 Homo Species 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- 102000004218 Insulin-Like Growth Factor I Human genes 0.000 claims 1
- 108090000723 Insulin-Like Growth Factor I Proteins 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
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361842302P | 2013-07-02 | 2013-07-02 | |
| US61/842,302 | 2013-07-02 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016524320A Division JP6487913B2 (ja) | 2013-07-02 | 2014-07-01 | 成長ホルモン受容体のモジュレータ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019088329A JP2019088329A (ja) | 2019-06-13 |
| JP2019088329A5 true JP2019088329A5 (2) | 2019-08-29 |
Family
ID=52144275
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016524320A Expired - Fee Related JP6487913B2 (ja) | 2013-07-02 | 2014-07-01 | 成長ホルモン受容体のモジュレータ |
| JP2019030128A Pending JP2019088329A (ja) | 2013-07-02 | 2019-02-22 | 成長ホルモン受容体のモジュレータ |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016524320A Expired - Fee Related JP6487913B2 (ja) | 2013-07-02 | 2014-07-01 | 成長ホルモン受容体のモジュレータ |
Country Status (13)
| Country | Link |
|---|---|
| US (4) | US20170073689A1 (2) |
| EP (2) | EP3017044B1 (2) |
| JP (2) | JP6487913B2 (2) |
| KR (1) | KR102236784B1 (2) |
| CN (1) | CN105452461B (2) |
| AU (2) | AU2014284398B2 (2) |
| BR (1) | BR112015033069A2 (2) |
| CA (1) | CA2917229A1 (2) |
| ES (1) | ES2787600T3 (2) |
| IL (2) | IL243093B (2) |
| MX (1) | MX375446B (2) |
| RU (1) | RU2700244C2 (2) |
| WO (1) | WO2015002971A2 (2) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| CN106103717A (zh) | 2014-03-19 | 2016-11-09 | Ionis制药公司 | 用于调节共济失调蛋白2表达的组合物 |
| WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| CN110903337A (zh) * | 2014-05-01 | 2020-03-24 | Ionis制药公司 | 用于调节生长激素受体表达的组合物和方法 |
| US20190002887A1 (en) | 2015-12-31 | 2019-01-03 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| WO2019233922A1 (en) | 2018-06-05 | 2019-12-12 | F. Hoffmann-La Roche Ag | Oligonucleotides for modulating atxn2 expression |
| SG11202012759XA (en) | 2018-07-03 | 2021-01-28 | Hoffmann La Roche | Oligonucleotides for modulating tau expression |
| BR112021000308A2 (pt) | 2018-07-25 | 2021-04-13 | Ionis Pharmaceuticals, Inc. | Compostos e métodos para redução da expressão de atxn2 |
| TW202028222A (zh) * | 2018-11-14 | 2020-08-01 | 美商Ionis製藥公司 | Foxp3表現之調節劑 |
| US11286485B2 (en) | 2019-04-04 | 2022-03-29 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating ATXN2 expression |
| EP4048801A4 (en) * | 2019-10-24 | 2023-11-22 | Genevant Sciences Gmbh | Conjugates and methods for treating acromegaly |
| KR20260005258A (ko) * | 2023-04-21 | 2026-01-09 | 세카나 파머씨티컬스 지엠비에이치 엔 씨오. 케이지 | Nrp1 특이적 안티센스 올리고뉴클레오티드 및 질병 예방 및/또는 치료에 있어서의 그 용도 |
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-
2014
- 2014-07-01 EP EP14819490.5A patent/EP3017044B1/en active Active
- 2014-07-01 RU RU2016102833A patent/RU2700244C2/ru active
- 2014-07-01 AU AU2014284398A patent/AU2014284398B2/en not_active Ceased
- 2014-07-01 CN CN201480038401.4A patent/CN105452461B/zh active Active
- 2014-07-01 KR KR1020167001790A patent/KR102236784B1/ko not_active Expired - Fee Related
- 2014-07-01 MX MX2015017863A patent/MX375446B/es active IP Right Grant
- 2014-07-01 ES ES14819490T patent/ES2787600T3/es active Active
- 2014-07-01 CA CA2917229A patent/CA2917229A1/en active Pending
- 2014-07-01 WO PCT/US2014/045088 patent/WO2015002971A2/en not_active Ceased
- 2014-07-01 EP EP20159386.0A patent/EP3730614A3/en not_active Withdrawn
- 2014-07-01 JP JP2016524320A patent/JP6487913B2/ja not_active Expired - Fee Related
- 2014-07-01 BR BR112015033069A patent/BR112015033069A2/pt not_active Application Discontinuation
- 2014-07-01 US US14/902,446 patent/US20170073689A1/en not_active Abandoned
-
2015
- 2015-12-14 IL IL243093A patent/IL243093B/en active IP Right Grant
-
2018
- 2018-12-27 US US16/234,409 patent/US20190225970A1/en not_active Abandoned
-
2019
- 2019-02-22 JP JP2019030128A patent/JP2019088329A/ja active Pending
-
2020
- 2020-01-28 AU AU2020200570A patent/AU2020200570A1/en not_active Abandoned
- 2020-02-20 IL IL272828A patent/IL272828A/en unknown
- 2020-05-27 US US16/884,782 patent/US20210108211A1/en not_active Abandoned
-
2022
- 2022-05-19 US US17/748,961 patent/US20230147811A1/en active Pending
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