JP2019525918A5 - - Google Patents
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- Publication number
- JP2019525918A5 JP2019525918A5 JP2019500807A JP2019500807A JP2019525918A5 JP 2019525918 A5 JP2019525918 A5 JP 2019525918A5 JP 2019500807 A JP2019500807 A JP 2019500807A JP 2019500807 A JP2019500807 A JP 2019500807A JP 2019525918 A5 JP2019525918 A5 JP 2019525918A5
- Authority
- JP
- Japan
- Prior art keywords
- modified oligonucleotide
- oligomeric compound
- modified
- nucleosides
- compound according
- 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
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- 150000001875 compounds Chemical class 0.000 claims description 208
- 108091034117 Oligonucleotide Proteins 0.000 claims description 138
- 239000002777 nucleoside Substances 0.000 claims description 96
- 125000003835 nucleoside group Chemical group 0.000 claims description 68
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims description 33
- 102100021947 Survival motor neuron protein Human genes 0.000 claims description 33
- 108020004999 messenger RNA Proteins 0.000 claims description 28
- 230000000295 complement effect Effects 0.000 claims description 26
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 24
- 239000000203 mixture Substances 0.000 claims description 20
- 150000002632 lipids Chemical class 0.000 claims description 18
- 150000004713 phosphodiesters Chemical class 0.000 claims description 10
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 8
- 208000002320 spinal muscular atrophy Diseases 0.000 claims description 8
- 125000000217 alkyl group Chemical group 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- 235000012000 cholesterol Nutrition 0.000 claims description 4
- GVJHHUAWPYXKBD-UHFFFAOYSA-N d-alpha-tocopherol Natural products OC1=C(C)C(C)=C2OC(CCCC(C)CCCC(C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-UHFFFAOYSA-N 0.000 claims description 4
- 239000003085 diluting agent Substances 0.000 claims description 4
- 125000001183 hydrocarbyl group Chemical group 0.000 claims description 4
- 229960001295 tocopherol Drugs 0.000 claims description 4
- 229930003799 tocopherol Natural products 0.000 claims description 4
- 235000010384 tocopherol Nutrition 0.000 claims description 4
- 239000011732 tocopherol Substances 0.000 claims description 4
- GVJHHUAWPYXKBD-IEOSBIPESA-N α-tocopherol Chemical compound OC1=C(C)C(C)=C2O[C@@](CCC[C@H](C)CCC[C@H](C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-IEOSBIPESA-N 0.000 claims description 4
- BHQCQFFYRZLCQQ-UHFFFAOYSA-N (3alpha,5alpha,7alpha,12alpha)-3,7,12-trihydroxy-cholan-24-oic acid Natural products OC1CC2CC(O)CCC2(C)C2C1C1CCC(C(CCC(O)=O)C)C1(C)C(O)C2 BHQCQFFYRZLCQQ-UHFFFAOYSA-N 0.000 claims description 3
- 239000004380 Cholic acid Substances 0.000 claims description 3
- BHQCQFFYRZLCQQ-OELDTZBJSA-N cholic acid Chemical compound C([C@H]1C[C@H]2O)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 BHQCQFFYRZLCQQ-OELDTZBJSA-N 0.000 claims description 3
- 229960002471 cholic acid Drugs 0.000 claims description 3
- 235000019416 cholic acid Nutrition 0.000 claims description 3
- KXGVEGMKQFWNSR-UHFFFAOYSA-N deoxycholic acid Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(CCC(O)=O)C)C1(C)C(O)C2 KXGVEGMKQFWNSR-UHFFFAOYSA-N 0.000 claims description 3
- 150000004671 saturated fatty acids Chemical class 0.000 claims description 3
- UFTFJSFQGQCHQW-UHFFFAOYSA-N triformin Chemical compound O=COCC(OC=O)COC=O UFTFJSFQGQCHQW-UHFFFAOYSA-N 0.000 claims description 3
- 150000004670 unsaturated fatty acids Chemical class 0.000 claims description 3
- 230000001268 conjugating effect Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 229920006395 saturated elastomer Polymers 0.000 claims description 2
- 229930195734 saturated hydrocarbon Natural products 0.000 claims description 2
- 229930195735 unsaturated hydrocarbon Natural products 0.000 claims description 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 3
- 239000002953 phosphate buffered saline Substances 0.000 claims 3
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical compound 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 claims 1
- OHLUUHNLEMFGTQ-UHFFFAOYSA-N N-methylacetamide Chemical compound CNC(C)=O OHLUUHNLEMFGTQ-UHFFFAOYSA-N 0.000 claims 1
- XAEFZNCEHLXOMS-UHFFFAOYSA-M potassium benzoate Chemical compound [K+].[O-]C(=O)C1=CC=CC=C1 XAEFZNCEHLXOMS-UHFFFAOYSA-M 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 229910052708 sodium Inorganic materials 0.000 claims 1
- 239000011734 sodium Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 40
- 210000003169 central nervous system Anatomy 0.000 description 10
- YIMATHOGWXZHFX-WCTZXXKLSA-N (2r,3r,4r,5r)-5-(hydroxymethyl)-3-(2-methoxyethoxy)oxolane-2,4-diol Chemical group COCCO[C@H]1[C@H](O)O[C@H](CO)[C@H]1O YIMATHOGWXZHFX-WCTZXXKLSA-N 0.000 description 8
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 description 7
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 7
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 6
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical group O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 6
- 229910052739 hydrogen Inorganic materials 0.000 description 6
- 239000001257 hydrogen Substances 0.000 description 6
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical group CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 6
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 description 5
- 210000000056 organ Anatomy 0.000 description 5
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 4
- 125000004435 hydrogen atom Chemical group [H]* 0.000 description 4
- 229930024421 Adenine Natural products 0.000 description 3
- 208000003954 Spinal Muscular Atrophies of Childhood Diseases 0.000 description 3
- 229960000643 adenine Drugs 0.000 description 3
- GFFGJBXGBJISGV-UHFFFAOYSA-N adenyl group Chemical group N1=CN=C2N=CNC2=C1N GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 3
- -1 bicyclic nucleoside Chemical class 0.000 description 3
- 229940104302 cytosine Drugs 0.000 description 3
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 description 3
- 229940113082 thymine Drugs 0.000 description 3
- IHLOTZVBEUFDMD-UUOKFMHZSA-N 7-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-2,2-dioxo-1h-imidazo[4,5-c][1,2,6]thiadiazin-4-one Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(NS(=O)(=O)NC2=O)=C2N=C1 IHLOTZVBEUFDMD-UUOKFMHZSA-N 0.000 description 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 description 2
- 229910019142 PO4 Inorganic materials 0.000 description 2
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 description 2
- 239000010452 phosphate Substances 0.000 description 2
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 description 2
- 235000021122 unsaturated fatty acids Nutrition 0.000 description 2
- 229940035893 uracil Drugs 0.000 description 2
- PMDCZENCAXMSOU-UHFFFAOYSA-N N-Ethylacetamide Natural products CCNC(C)=O PMDCZENCAXMSOU-UHFFFAOYSA-N 0.000 description 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 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 201000006913 intermediate spinal muscular atrophy Diseases 0.000 description 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 230000003584 silencer Effects 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022199992A JP2023027285A (ja) | 2016-07-15 | 2022-12-15 | Ｓｍｎ２の調節のための化合物及び方法 |
| JP2025003382A JP2025063137A (ja) | 2016-07-15 | 2025-01-09 | Ｓｍｎ２の調節のための化合物及び方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662363195P | 2016-07-15 | 2016-07-15 | |
| US62/363,195 | 2016-07-15 | ||
| PCT/US2017/042463 WO2018014041A2 (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of smn2 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022199992A Division JP2023027285A (ja) | 2016-07-15 | 2022-12-15 | Ｓｍｎ２の調節のための化合物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019525918A JP2019525918A (ja) | 2019-09-12 |
| JP2019525918A5 true JP2019525918A5 (https=) | 2020-08-27 |
| JP7197463B2 JP7197463B2 (ja) | 2022-12-27 |
Family
ID=60952211
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019500807A Active JP7197463B2 (ja) | 2016-07-15 | 2017-07-17 | Ｓｍｎ２の調節のための化合物及び方法 |
| JP2022199992A Pending JP2023027285A (ja) | 2016-07-15 | 2022-12-15 | Ｓｍｎ２の調節のための化合物及び方法 |
| JP2025003382A Pending JP2025063137A (ja) | 2016-07-15 | 2025-01-09 | Ｓｍｎ２の調節のための化合物及び方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022199992A Pending JP2023027285A (ja) | 2016-07-15 | 2022-12-15 | Ｓｍｎ２の調節のための化合物及び方法 |
| JP2025003382A Pending JP2025063137A (ja) | 2016-07-15 | 2025-01-09 | Ｓｍｎ２の調節のための化合物及び方法 |
Country Status (27)
| Country | Link |
|---|---|
| US (3) | US20190323006A1 (https=) |
| EP (2) | EP3484524B1 (https=) |
| JP (3) | JP7197463B2 (https=) |
| KR (1) | KR102556825B1 (https=) |
| CN (1) | CN109844115B (https=) |
| AU (2) | AU2017297622A1 (https=) |
| BR (1) | BR112019000356A2 (https=) |
| CA (1) | CA3030864A1 (https=) |
| CL (2) | CL2019000115A1 (https=) |
| CO (1) | CO2019001236A2 (https=) |
| DK (1) | DK3484524T3 (https=) |
| ES (1) | ES2935658T3 (https=) |
| FI (1) | FI3484524T3 (https=) |
| HR (1) | HRP20221378T1 (https=) |
| HU (1) | HUE060831T2 (https=) |
| IL (2) | IL308256A (https=) |
| LT (1) | LT3484524T (https=) |
| MX (1) | MX2019000619A (https=) |
| NZ (1) | NZ750713A (https=) |
| PE (1) | PE20190513A1 (https=) |
| PL (1) | PL3484524T3 (https=) |
| PT (1) | PT3484524T (https=) |
| RS (1) | RS63928B1 (https=) |
| SG (1) | SG11201900238UA (https=) |
| SI (1) | SI3484524T1 (https=) |
| SM (1) | SMT202200489T1 (https=) |
| WO (1) | WO2018014041A2 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2983676B1 (en) * | 2013-04-12 | 2019-03-20 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| US10053465B2 (en) | 2015-08-26 | 2018-08-21 | Incyte Corporation | Pyrrolopyrimidine derivatives as TAM inhibitors |
| MD3436461T2 (ro) | 2016-03-28 | 2024-05-31 | Incyte Corp | Compuși pirolotriazină ca inhibitori TAM |
| SI3687996T1 (sl) | 2017-09-27 | 2022-04-29 | Incyte Corporation | Soli pirolotriazinskih derivatov, ki so uporabne kot zaviralci TAM |
| MD3813800T2 (ro) | 2018-06-29 | 2025-10-31 | Incyte Corp | Formulări de inhibitor al AXL/MER |
| AU2020316997A1 (en) * | 2019-07-19 | 2022-03-10 | Biogen Ma Inc. | Methods of treating or preventing spinal muscular atrophy |
| US20220280548A1 (en) * | 2019-08-15 | 2022-09-08 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| US20220370491A1 (en) * | 2019-09-18 | 2022-11-24 | National University Corporation Tokyo Medical And Dental University | Nucleic acid complex |
| WO2021174036A1 (en) * | 2020-02-28 | 2021-09-02 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating scn1a expression |
| CR20220485A (es) * | 2020-02-28 | 2022-11-10 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| EP4114401A1 (en) | 2020-03-06 | 2023-01-11 | Incyte Corporation | Combination therapy comprising axl/mer and pd-1/pd-l1 inhibitors |
| KR20240032184A (ko) * | 2021-04-22 | 2024-03-11 | 몰레큘라 악시옴, 엘엘씨 | Sos 유전자 발현을 조절하기 위한 조성물 및 방법 |
| FR3128873B1 (fr) | 2021-11-10 | 2026-01-16 | Biophytis | Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale |
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| US4751219A (en) | 1985-02-05 | 1988-06-14 | Nederlandse Centrale Organisatie Voor Toegepast-Natuur-Wetenschappelijk Onderzoek | Synthetic glycolipides, a process for the preparation thereof and several uses for these synthetic glycolipides |
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