JPWO2020190740A5 - - Google Patents
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- Publication number
- JPWO2020190740A5 JPWO2020190740A5 JP2021555448A JP2021555448A JPWO2020190740A5 JP WO2020190740 A5 JPWO2020190740 A5 JP WO2020190740A5 JP 2021555448 A JP2021555448 A JP 2021555448A JP 2021555448 A JP2021555448 A JP 2021555448A JP WO2020190740 A5 JPWO2020190740 A5 JP WO2020190740A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- modified oligonucleotide
- oligomeric compound
- seq
- nucleobases
- 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 54
- 108091034117 Oligonucleotide Proteins 0.000 claims description 32
- 239000008194 pharmaceutical composition Substances 0.000 claims description 30
- 239000002777 nucleoside Substances 0.000 claims description 17
- 125000003835 nucleoside group Chemical group 0.000 claims description 13
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims description 10
- 239000003085 diluting agent Substances 0.000 claims description 10
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
- 150000004713 phosphodiesters Chemical class 0.000 claims description 4
- 150000003839 salts Chemical class 0.000 claims description 3
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- XAEFZNCEHLXOMS-UHFFFAOYSA-M potassium benzoate Chemical compound [K+].[O-]C(=O)C1=CC=CC=C1 XAEFZNCEHLXOMS-UHFFFAOYSA-M 0.000 claims description 2
- 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 12
- 239000002953 phosphate buffered saline Substances 0.000 claims 12
- 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 4
- 125000000824 D-ribofuranosyl group Chemical group [H]OC([H])([H])[C@@]1([H])OC([H])(*)[C@]([H])(O[H])[C@]1([H])O[H] 0.000 claims 1
- 159000000000 sodium salts Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 description 16
- 101000944018 Homo sapiens Potassium channel subfamily T member 1 Proteins 0.000 description 9
- 102100033508 Potassium channel subfamily T member 1 Human genes 0.000 description 9
- 230000000926 neurological effect Effects 0.000 description 9
- 210000004027 cell Anatomy 0.000 description 6
- 125000002619 bicyclic group Chemical group 0.000 description 4
- 206010015037 epilepsy Diseases 0.000 description 4
- 206010010904 Convulsion Diseases 0.000 description 2
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 description 2
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 description 2
- 230000000692 anti-sense effect Effects 0.000 description 2
- 208000008233 autosomal dominant nocturnal frontal lobe epilepsy Diseases 0.000 description 2
- 210000003169 central nervous system Anatomy 0.000 description 2
- 230000000295 complement effect Effects 0.000 description 2
- 150000003833 nucleoside derivatives Chemical class 0.000 description 2
- 235000021092 sugar substitutes Nutrition 0.000 description 2
- 239000003765 sweetening agent Substances 0.000 description 2
- 208000024891 symptom Diseases 0.000 description 2
- 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 description 1
- 208000019901 Anxiety disease Diseases 0.000 description 1
- 208000014644 Brain disease Diseases 0.000 description 1
- 208000016192 Demyelinating disease Diseases 0.000 description 1
- 206010012305 Demyelination Diseases 0.000 description 1
- 208000032274 Encephalopathy Diseases 0.000 description 1
- 206010021118 Hypotonia Diseases 0.000 description 1
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical group C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 description 1
- 208000007379 Muscle Hypotonia Diseases 0.000 description 1
- 206010061334 Partial seizures Diseases 0.000 description 1
- 230000001668 ameliorated effect Effects 0.000 description 1
- 230000036506 anxiety Effects 0.000 description 1
- 230000006931 brain damage Effects 0.000 description 1
- 231100000874 brain damage Toxicity 0.000 description 1
- 208000029028 brain injury Diseases 0.000 description 1
- 230000003920 cognitive function Effects 0.000 description 1
- 230000001934 delay Effects 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- -1 linker nucleoside Chemical class 0.000 description 1
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 description 1
- 208000004141 microcephaly Diseases 0.000 description 1
- 230000001617 migratory effect Effects 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 description 1
- 108020004707 nucleic acids Proteins 0.000 description 1
- 102000039446 nucleic acids Human genes 0.000 description 1
- 150000007523 nucleic acids Chemical class 0.000 description 1
- 229910052708 sodium Inorganic materials 0.000 description 1
- 239000011734 sodium Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024168231A JP2025000805A (ja) | 2019-03-15 | 2024-09-27 | ｋｃｎｔ１発現を低減するための化合物及び方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962819344P | 2019-03-15 | 2019-03-15 | |
| US62/819,344 | 2019-03-15 | ||
| US201962884501P | 2019-08-08 | 2019-08-08 | |
| US62/884,501 | 2019-08-08 | ||
| PCT/US2020/022680 WO2020190740A1 (en) | 2019-03-15 | 2020-03-13 | Compounds and methods for reducing kcnt1 expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024168231A Division JP2025000805A (ja) | 2019-03-15 | 2024-09-27 | ｋｃｎｔ１発現を低減するための化合物及び方法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022526267A JP2022526267A (ja) | 2022-05-24 |
| JP2022526267A5 JP2022526267A5 (https=) | 2023-08-24 |
| JPWO2020190740A5 true JPWO2020190740A5 (https=) | 2023-08-24 |
| JP7564817B2 JP7564817B2 (ja) | 2024-10-09 |
Family
ID=72520445
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021555448A Active JP7564817B2 (ja) | 2019-03-15 | 2020-03-13 | ｋｃｎｔ１発現を低減するための化合物及び方法 |
| JP2024168231A Pending JP2025000805A (ja) | 2019-03-15 | 2024-09-27 | ｋｃｎｔ１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 |
|---|---|---|---|
| JP2024168231A Pending JP2025000805A (ja) | 2019-03-15 | 2024-09-27 | ｋｃｎｔ１発現を低減するための化合物及び方法 |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US20220177893A1 (https=) |
| EP (1) | EP3938514A4 (https=) |
| JP (2) | JP7564817B2 (https=) |
| KR (1) | KR20210141983A (https=) |
| CN (3) | CN121674396A (https=) |
| AU (1) | AU2020241693B2 (https=) |
| BR (1) | BR112021015494A2 (https=) |
| CA (1) | CA3133247A1 (https=) |
| CL (1) | CL2021002398A1 (https=) |
| CO (1) | CO2021013371A2 (https=) |
| CR (1) | CR20210519A (https=) |
| IL (1) | IL285546A (https=) |
| JO (1) | JOP20210254A1 (https=) |
| MX (1) | MX2021011132A (https=) |
| PE (1) | PE20220168A1 (https=) |
| PH (1) | PH12021552220A1 (https=) |
| SG (1) | SG11202108625WA (https=) |
| TW (1) | TW202102675A (https=) |
| WO (1) | WO2020190740A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10952978B2 (en) | 2017-08-28 | 2021-03-23 | Enanta Pharmaceuticals, Inc. | Hepatitis B antiviral agents |
| US11198693B2 (en) | 2018-11-21 | 2021-12-14 | Enanta Pharmaceuticals, Inc. | Functionalized heterocycles as antiviral agents |
| WO2021188414A1 (en) | 2020-03-16 | 2021-09-23 | Enanta Pharmaceuticals, Inc. | Functionalized heterocyclic compounds as antiviral agents |
| MX2024006709A (es) * | 2021-12-01 | 2024-08-19 | Atalanta Therapeutics Inc | Composiciones y metodos para el tratamiento de epilepsias. |
| WO2025036916A1 (en) * | 2023-08-16 | 2025-02-20 | Les Laboratoires Servier | Oligonucleotides for modulating kcnt1 expression |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050288242A1 (en) * | 2001-05-18 | 2005-12-29 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of RAS gene expression using short interfering nucleic acid (siNA) |
| US7601501B2 (en) * | 2006-08-11 | 2009-10-13 | The Scripps Research Institute | Controlling osteogenesis by inhibition of osteogenic suppressors |
| EP3260540A1 (en) * | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| US20200129538A1 (en) | 2017-06-13 | 2020-04-30 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1 |
-
2020
- 2020-03-13 PE PE2021001518A patent/PE20220168A1/es unknown
- 2020-03-13 CR CR20210519A patent/CR20210519A/es unknown
- 2020-03-13 CN CN202511762645.3A patent/CN121674396A/zh active Pending
- 2020-03-13 CN CN202080020884.0A patent/CN113661241A/zh active Pending
- 2020-03-13 CA CA3133247A patent/CA3133247A1/en active Pending
- 2020-03-13 EP EP20773689.3A patent/EP3938514A4/en active Pending
- 2020-03-13 KR KR1020217032716A patent/KR20210141983A/ko not_active Withdrawn
- 2020-03-13 CN CN202311150264.0A patent/CN117106778A/zh active Pending
- 2020-03-13 MX MX2021011132A patent/MX2021011132A/es unknown
- 2020-03-13 WO PCT/US2020/022680 patent/WO2020190740A1/en not_active Ceased
- 2020-03-13 AU AU2020241693A patent/AU2020241693B2/en not_active Ceased
- 2020-03-13 BR BR112021015494-9A patent/BR112021015494A2/pt unknown
- 2020-03-13 JP JP2021555448A patent/JP7564817B2/ja active Active
- 2020-03-13 SG SG11202108625WA patent/SG11202108625WA/en unknown
- 2020-03-13 PH PH1/2021/552220A patent/PH12021552220A1/en unknown
- 2020-03-13 US US17/437,507 patent/US20220177893A1/en not_active Abandoned
- 2020-03-16 TW TW109108639A patent/TW202102675A/zh unknown
-
2021
- 2021-08-11 IL IL285546A patent/IL285546A/en unknown
- 2021-09-14 JO JOP/2021/0254A patent/JOP20210254A1/ar unknown
- 2021-09-14 CL CL2021002398A patent/CL2021002398A1/es unknown
- 2021-10-06 CO CONC2021/0013371A patent/CO2021013371A2/es unknown
-
2024
- 2024-09-27 JP JP2024168231A patent/JP2025000805A/ja active Pending
-
2025
- 2025-06-27 US US19/252,993 patent/US20250320505A1/en active Pending
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