JPWO2020132558A5 - - Google Patents
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- Publication number
- JPWO2020132558A5 JPWO2020132558A5 JP2021535612A JP2021535612A JPWO2020132558A5 JP WO2020132558 A5 JPWO2020132558 A5 JP WO2020132558A5 JP 2021535612 A JP2021535612 A JP 2021535612A JP 2021535612 A JP2021535612 A JP 2021535612A JP WO2020132558 A5 JPWO2020132558 A5 JP WO2020132558A5
- Authority
- JP
- Japan
- Prior art keywords
- modified
- modified oligonucleotide
- oligomeric compound
- optionally
- 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.)
- Granted
Links
- 150000001875 compounds Chemical class 0.000 claims description 55
- 108091034117 Oligonucleotide Proteins 0.000 claims description 50
- 239000002777 nucleoside Substances 0.000 claims description 46
- 125000003835 nucleoside group Chemical group 0.000 claims description 32
- 235000000346 sugar Nutrition 0.000 claims description 27
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 11
- 101001000631 Homo sapiens Peripheral myelin protein 22 Proteins 0.000 claims description 9
- 102100035917 Peripheral myelin protein 22 Human genes 0.000 claims description 9
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 230000001268 conjugating effect Effects 0.000 claims description 8
- 201000010099 disease Diseases 0.000 claims description 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 8
- 101001082860 Homo sapiens Peroxisomal membrane protein 2 Proteins 0.000 claims description 7
- 125000002619 bicyclic group Chemical group 0.000 claims description 7
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 claims description 5
- 235000021092 sugar substitutes Nutrition 0.000 claims description 5
- 239000003765 sweetening agent Substances 0.000 claims description 5
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 5
- 230000000295 complement effect Effects 0.000 claims description 4
- 239000003085 diluting agent Substances 0.000 claims description 4
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims description 4
- 239000002953 phosphate buffered saline Substances 0.000 claims description 4
- 150000004713 phosphodiesters Chemical class 0.000 claims description 4
- YIMATHOGWXZHFX-WCTZXXKLSA-N (2r,3r,4r,5r)-5-(hydroxymethyl)-3-(2-methoxyethoxy)oxolane-2,4-diol Chemical compound COCCO[C@H]1[C@H](O)O[C@H](CO)[C@H]1O YIMATHOGWXZHFX-WCTZXXKLSA-N 0.000 claims description 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 201000008991 Charcot-Marie-Tooth disease type 1E Diseases 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 2
- 208000011580 syndromic disease Diseases 0.000 claims description 2
- 210000001175 cerebrospinal fluid Anatomy 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
- 238000000034 method Methods 0.000 description 9
- 101000635955 Homo sapiens Myelin P2 protein Proteins 0.000 description 2
- 102100030738 Myelin P2 protein Human genes 0.000 description 2
- 208000024891 symptom Diseases 0.000 description 2
- 206010003694 Atrophy Diseases 0.000 description 1
- 208000016192 Demyelinating disease Diseases 0.000 description 1
- 206010012305 Demyelination Diseases 0.000 description 1
- 206010061159 Foot deformity Diseases 0.000 description 1
- 230000001668 ameliorated effect Effects 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 230000037444 atrophy Effects 0.000 description 1
- 230000007844 axonal damage Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 150000008163 sugars Chemical class 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024163353A JP2025000717A (ja) | 2018-12-21 | 2024-09-20 | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862783997P | 2018-12-21 | 2018-12-21 | |
| US62/783,997 | 2018-12-21 | ||
| PCT/US2019/068040 WO2020132558A1 (en) | 2018-12-21 | 2019-12-20 | Compounds and methods for reducing pmp22 expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024163353A Division JP2025000717A (ja) | 2018-12-21 | 2024-09-20 | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022515140A JP2022515140A (ja) | 2022-02-17 |
| JP2022515140A5 JP2022515140A5 (https=) | 2022-12-14 |
| JPWO2020132558A5 true JPWO2020132558A5 (https=) | 2022-12-14 |
| JP7561129B2 JP7561129B2 (ja) | 2024-10-03 |
Family
ID=71101859
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021535612A Active JP7561129B2 (ja) | 2018-12-21 | 2019-12-20 | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
| JP2024163353A Pending JP2025000717A (ja) | 2018-12-21 | 2024-09-20 | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024163353A Pending JP2025000717A (ja) | 2018-12-21 | 2024-09-20 | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20220112503A1 (https=) |
| EP (1) | EP3897837A4 (https=) |
| JP (2) | JP7561129B2 (https=) |
| WO (1) | WO2020132558A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA202091693A1 (ru) | 2018-01-12 | 2021-04-14 | Бристол-Маерс Сквибб Компани | Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения |
| BR112020013994A2 (pt) | 2018-01-12 | 2020-12-08 | Bristol-Myers Squibb Company | Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos |
| EP4168549A4 (en) * | 2020-06-19 | 2024-10-09 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR PMP22 MODULATION |
| AU2021390471A1 (en) * | 2020-12-01 | 2023-06-29 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of peripheral myelin protein-22 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030198983A1 (en) * | 2002-02-01 | 2003-10-23 | Affymetrix, Inc. | Methods of genetic analysis of human genes |
| EP1646874B1 (en) * | 2003-07-11 | 2010-03-03 | ZEILLINGER, Robert | A method for predicting the metastatic potential of breast cancer |
| WO2012177639A2 (en) * | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
| PT3277815T (pt) * | 2015-04-03 | 2021-11-11 | Beth Israel Deaconess Medical Ct Inc | Compostos de oligonucleótidos para o tratamento de pré-eclâmpsia e outros distúrbios angiogénicos |
| JP7054675B2 (ja) * | 2015-12-14 | 2022-04-14 | コールド スプリング ハーバー ラボラトリー | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
| AU2017229778A1 (en) * | 2016-03-09 | 2018-08-16 | Ionis Pharmaceuticals, Inc. | Methods and compositions for inhibiting PMP22 expression |
| KR101889072B1 (ko) * | 2017-09-15 | 2018-08-16 | 한국생명공학연구원 | 디지털 PCR을 이용한 유전성 강직성 대마비(Hereditary spastic paraplegia, HSP) 관련 유전자 SPG4의 거대결손 검증법 |
-
2019
- 2019-12-20 WO PCT/US2019/068040 patent/WO2020132558A1/en not_active Ceased
- 2019-12-20 EP EP19899128.3A patent/EP3897837A4/en active Pending
- 2019-12-20 US US17/416,392 patent/US20220112503A1/en active Pending
- 2019-12-20 JP JP2021535612A patent/JP7561129B2/ja active Active
-
2024
- 2024-09-20 JP JP2024163353A patent/JP2025000717A/ja active Pending
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