JP2020518633A5 - - Google Patents
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- JP2020518633A5 JP2020518633A5 JP2019560355A JP2019560355A JP2020518633A5 JP 2020518633 A5 JP2020518633 A5 JP 2020518633A5 JP 2019560355 A JP2019560355 A JP 2019560355A JP 2019560355 A JP2019560355 A JP 2019560355A JP 2020518633 A5 JP2020518633 A5 JP 2020518633A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- subject
- composition according
- modified oligonucleotide
- nucleoside
- 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
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- 239000008194 pharmaceutical composition Substances 0.000 claims 43
- 239000002777 nucleoside Substances 0.000 claims 34
- 108091034117 Oligonucleotide Proteins 0.000 claims 25
- 125000003835 nucleoside group Chemical group 0.000 claims 16
- DDRJAANPRJIHGJ-UHFFFAOYSA-N creatinine Chemical compound CN1CC(=O)NC1=N DDRJAANPRJIHGJ-UHFFFAOYSA-N 0.000 claims 14
- 230000024924 glomerular filtration Effects 0.000 claims 14
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 13
- 208000003215 hereditary nephritis Diseases 0.000 claims 11
- 238000000034 method Methods 0.000 claims 11
- 208000024985 Alport syndrome Diseases 0.000 claims 10
- 229940109239 creatinine Drugs 0.000 claims 7
- 230000007423 decrease Effects 0.000 claims 7
- -1 2'-MOE nucleoside Chemical class 0.000 claims 5
- 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
- 239000005549 deoxyribonucleoside Substances 0.000 claims 4
- 230000002068 genetic effect Effects 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 210000002700 urine Anatomy 0.000 claims 4
- 102000012192 Cystatin C Human genes 0.000 claims 3
- 108010061642 Cystatin C Proteins 0.000 claims 3
- 102000015736 beta 2-Microglobulin Human genes 0.000 claims 3
- 108010081355 beta 2-Microglobulin Proteins 0.000 claims 3
- 239000003085 diluting agent Substances 0.000 claims 3
- 201000001474 proteinuria Diseases 0.000 claims 3
- 239000000243 solution Substances 0.000 claims 3
- KZMAWJRXKGLWGS-UHFFFAOYSA-N 2-chloro-n-[4-(4-methoxyphenyl)-1,3-thiazol-2-yl]-n-(3-methoxypropyl)acetamide Chemical compound S1C(N(C(=O)CCl)CCCOC)=NC(C=2C=CC(OC)=CC=2)=C1 KZMAWJRXKGLWGS-UHFFFAOYSA-N 0.000 claims 2
- 239000005541 ACE inhibitor Substances 0.000 claims 2
- 102000009027 Albumins Human genes 0.000 claims 2
- 108010088751 Albumins Proteins 0.000 claims 2
- 102000005862 Angiotensin II Human genes 0.000 claims 2
- 102000008873 Angiotensin II receptor Human genes 0.000 claims 2
- 108050000824 Angiotensin II receptor Proteins 0.000 claims 2
- 101800000733 Angiotensin-2 Proteins 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- 102000004190 Enzymes Human genes 0.000 claims 2
- CZGUSIXMZVURDU-JZXHSEFVSA-N Ile(5)-angiotensin II Chemical compound C([C@@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CC=1NC=NC=1)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CC=1C=CC=CC=1)C([O-])=O)NC(=O)[C@@H](NC(=O)[C@H](CCCNC(N)=[NH2+])NC(=O)[C@@H]([NH3+])CC([O-])=O)C(C)C)C1=CC=C(O)C=C1 CZGUSIXMZVURDU-JZXHSEFVSA-N 0.000 claims 2
- 206010061481 Renal injury Diseases 0.000 claims 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 2
- 239000002333 angiotensin II receptor antagonist Substances 0.000 claims 2
- 229950006323 angiotensin ii Drugs 0.000 claims 2
- 229940125364 angiotensin receptor blocker Drugs 0.000 claims 2
- 229940044094 angiotensin-converting-enzyme inhibitor Drugs 0.000 claims 2
- 210000004369 blood Anatomy 0.000 claims 2
- 239000008280 blood Substances 0.000 claims 2
- 208000020832 chronic kidney disease Diseases 0.000 claims 2
- CVSVTCORWBXHQV-UHFFFAOYSA-N creatine Chemical compound NC(=[NH2+])N(C)CC([O-])=O CVSVTCORWBXHQV-UHFFFAOYSA-N 0.000 claims 2
- 239000003112 inhibitor Substances 0.000 claims 2
- 239000002504 physiological saline solution Substances 0.000 claims 2
- 239000003087 receptor blocking agent Substances 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 238000010254 subcutaneous injection Methods 0.000 claims 2
- 239000007929 subcutaneous injection Substances 0.000 claims 2
- BIDNLKIUORFRQP-XYGFDPSESA-N (2s,4s)-4-cyclohexyl-1-[2-[[(1s)-2-methyl-1-propanoyloxypropoxy]-(4-phenylbutyl)phosphoryl]acetyl]pyrrolidine-2-carboxylic acid Chemical compound C([P@@](=O)(O[C@H](OC(=O)CC)C(C)C)CC(=O)N1[C@@H](C[C@H](C1)C1CCCCC1)C(O)=O)CCCC1=CC=CC=C1 BIDNLKIUORFRQP-XYGFDPSESA-N 0.000 claims 1
- XPCFTKFZXHTYIP-PMACEKPBSA-N Benazepril Chemical compound C([C@@H](C(=O)OCC)N[C@@H]1C(N(CC(O)=O)C2=CC=CC=C2CC1)=O)CC1=CC=CC=C1 XPCFTKFZXHTYIP-PMACEKPBSA-N 0.000 claims 1
- 239000002083 C09CA01 - Losartan Substances 0.000 claims 1
- 239000002080 C09CA02 - Eprosartan Substances 0.000 claims 1
- 239000002947 C09CA04 - Irbesartan Substances 0.000 claims 1
- 239000002053 C09CA06 - Candesartan Substances 0.000 claims 1
- 102000015225 Connective Tissue Growth Factor Human genes 0.000 claims 1
- 108010039419 Connective Tissue Growth Factor Proteins 0.000 claims 1
- 108010061435 Enalapril Proteins 0.000 claims 1
- 102100034459 Hepatitis A virus cellular receptor 1 Human genes 0.000 claims 1
- 101710185991 Hepatitis A virus cellular receptor 1 homolog Proteins 0.000 claims 1
- 102000019298 Lipocalin Human genes 0.000 claims 1
- 108050006654 Lipocalin Proteins 0.000 claims 1
- 108010007859 Lisinopril Proteins 0.000 claims 1
- 108091008065 MIR21 Proteins 0.000 claims 1
- YDGMGEXADBMOMJ-LURJTMIESA-N N(g)-dimethylarginine Chemical compound CN(C)C(\N)=N\CCC[C@H](N)C(O)=O YDGMGEXADBMOMJ-LURJTMIESA-N 0.000 claims 1
- 239000005480 Olmesartan Substances 0.000 claims 1
- 201000002429 X-linked Alport syndrome Diseases 0.000 claims 1
- PNNCWTXUWKENPE-UHFFFAOYSA-N [N].NC(N)=O Chemical compound [N].NC(N)=O PNNCWTXUWKENPE-UHFFFAOYSA-N 0.000 claims 1
- 210000003815 abdominal wall Anatomy 0.000 claims 1
- 229960004530 benazepril Drugs 0.000 claims 1
- 239000000090 biomarker Substances 0.000 claims 1
- 238000001574 biopsy Methods 0.000 claims 1
- 229960000932 candesartan Drugs 0.000 claims 1
- SGZAIDDFHDDFJU-UHFFFAOYSA-N candesartan Chemical compound CCOC1=NC2=CC=CC(C(O)=O)=C2N1CC(C=C1)=CC=C1C1=CC=CC=C1C1=NN=N[N]1 SGZAIDDFHDDFJU-UHFFFAOYSA-N 0.000 claims 1
- 229960000830 captopril Drugs 0.000 claims 1
- FAKRSMQSSFJEIM-RQJHMYQMSA-N captopril Chemical group SC[C@@H](C)C(=O)N1CCC[C@H]1C(O)=O FAKRSMQSSFJEIM-RQJHMYQMSA-N 0.000 claims 1
- 229960003624 creatine Drugs 0.000 claims 1
- 239000006046 creatine Substances 0.000 claims 1
- 229960000873 enalapril Drugs 0.000 claims 1
- GBXSMTUPTTWBMN-XIRDDKMYSA-N enalapril Chemical compound C([C@@H](C(=O)OCC)N[C@@H](C)C(=O)N1[C@@H](CCC1)C(O)=O)CC1=CC=CC=C1 GBXSMTUPTTWBMN-XIRDDKMYSA-N 0.000 claims 1
- 229960004563 eprosartan Drugs 0.000 claims 1
- OROAFUQRIXKEMV-LDADJPATSA-N eprosartan Chemical compound C=1C=C(C(O)=O)C=CC=1CN1C(CCCC)=NC=C1\C=C(C(O)=O)/CC1=CC=CS1 OROAFUQRIXKEMV-LDADJPATSA-N 0.000 claims 1
- 229960002490 fosinopril Drugs 0.000 claims 1
- 230000012010 growth Effects 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 229960002198 irbesartan Drugs 0.000 claims 1
- YCPOHTHPUREGFM-UHFFFAOYSA-N irbesartan Chemical compound O=C1N(CC=2C=CC(=CC=2)C=2C(=CC=CC=2)C=2[N]N=NN=2)C(CCCC)=NC21CCCC2 YCPOHTHPUREGFM-UHFFFAOYSA-N 0.000 claims 1
- 230000003907 kidney function Effects 0.000 claims 1
- 229960002394 lisinopril Drugs 0.000 claims 1
- RLAWWYSOJDYHDC-BZSNNMDCSA-N lisinopril Chemical compound C([C@H](N[C@@H](CCCCN)C(=O)N1[C@@H](CCC1)C(O)=O)C(O)=O)CC1=CC=CC=C1 RLAWWYSOJDYHDC-BZSNNMDCSA-N 0.000 claims 1
- 229960004773 losartan Drugs 0.000 claims 1
- KJJZZJSZUJXYEA-UHFFFAOYSA-N losartan Chemical compound CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C=2[N]N=NN=2)C=C1 KJJZZJSZUJXYEA-UHFFFAOYSA-N 0.000 claims 1
- 210000000440 neutrophil Anatomy 0.000 claims 1
- VTRAEEWXHOVJFV-UHFFFAOYSA-N olmesartan Chemical compound CCCC1=NC(C(C)(C)O)=C(C(O)=O)N1CC1=CC=C(C=2C(=CC=CC=2)C=2NN=NN=2)C=C1 VTRAEEWXHOVJFV-UHFFFAOYSA-N 0.000 claims 1
- 229960005117 olmesartan Drugs 0.000 claims 1
- 210000000557 podocyte Anatomy 0.000 claims 1
- 229960001455 quinapril Drugs 0.000 claims 1
- JSDRRTOADPPCHY-HSQYWUDLSA-N quinapril Chemical compound C([C@@H](C(=O)OCC)N[C@@H](C)C(=O)N1[C@@H](CC2=CC=CC=C2C1)C(O)=O)CC1=CC=CC=C1 JSDRRTOADPPCHY-HSQYWUDLSA-N 0.000 claims 1
- 229960003401 ramipril Drugs 0.000 claims 1
- HDACQVRGBOVJII-JBDAPHQKSA-N ramipril Chemical compound C([C@@H](C(=O)OCC)N[C@@H](C)C(=O)N1[C@@H](C[C@@H]2CCC[C@@H]21)C(O)=O)CC1=CC=CC=C1 HDACQVRGBOVJII-JBDAPHQKSA-N 0.000 claims 1
- 239000011780 sodium chloride Substances 0.000 claims 1
- 159000000000 sodium salts Chemical class 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023023048A JP2023075111A (ja) | 2017-05-04 | 2023-02-17 | アルポート症候群を治療するための方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762501699P | 2017-05-04 | 2017-05-04 | |
| US62/501,699 | 2017-05-04 | ||
| PCT/US2018/031094 WO2018204788A1 (en) | 2017-05-04 | 2018-05-04 | Methods for treatment of alport syndrome |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023023048A Division JP2023075111A (ja) | 2017-05-04 | 2023-02-17 | アルポート症候群を治療するための方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020518633A JP2020518633A (ja) | 2020-06-25 |
| JP2020518633A5 true JP2020518633A5 (enExample) | 2021-06-10 |
Family
ID=62223298
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019560355A Pending JP2020518633A (ja) | 2017-05-04 | 2018-05-04 | アルポート症候群を治療するための方法 |
| JP2023023048A Pending JP2023075111A (ja) | 2017-05-04 | 2023-02-17 | アルポート症候群を治療するための方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023023048A Pending JP2023075111A (ja) | 2017-05-04 | 2023-02-17 | アルポート症候群を治療するための方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US20210100828A1 (enExample) |
| EP (1) | EP3618838A1 (enExample) |
| JP (2) | JP2020518633A (enExample) |
| KR (1) | KR20190141770A (enExample) |
| CN (2) | CN116585344A (enExample) |
| AU (1) | AU2018261792A1 (enExample) |
| BR (1) | BR112019022824A2 (enExample) |
| CA (1) | CA3062316A1 (enExample) |
| CL (1) | CL2019003153A1 (enExample) |
| MA (1) | MA50749A (enExample) |
| MX (1) | MX2019013158A (enExample) |
| SG (1) | SG11201910099WA (enExample) |
| WO (1) | WO2018204788A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022076452A1 (en) * | 2020-10-05 | 2022-04-14 | Father Flanagan's Boys' Home Doing Business As Boys Town National Research Hospital | Combination therapy for alport renal disease |
| CN120731923B (zh) * | 2025-09-05 | 2025-11-04 | 内蒙古医科大学附属医院(内蒙古自治区心血管研究所) | 一种Alport综合征小鼠NMD逃逸模型的构建方法及其应用 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20080152700A1 (en) * | 2004-06-01 | 2008-06-26 | Reza Sheikhnejad | Methods and compositions for the inhibition of gene expression |
| KR101877698B1 (ko) * | 2008-08-25 | 2018-07-12 | 엑스칼리아드 파마슈티컬즈, 인코포레이티드 | 결합 조직 성장 인자에 대한 안티센스 올리고뉴클레오타이드 및 그의 용도 |
| EP2825648B1 (en) * | 2012-03-15 | 2018-09-05 | CuRNA, Inc. | Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf |
| BR112014026639A8 (pt) * | 2012-04-25 | 2018-01-16 | Regulus Therapeutics Inc | compostos , métodos de inibição da atividade de mir-21 , método para diminuir a expressão de colágeno , método para tratar , prevenir ou atrasar o início de uma doença , método de tratamento de um distúrbio fibroproliferativo e usos de um composto. |
| UA116639C2 (uk) * | 2012-10-09 | 2018-04-25 | Рег'Юлес Терап'Ютікс Інк. | Способи лікування синдрому альпорта |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
-
2018
- 2018-05-04 SG SG11201910099W patent/SG11201910099WA/en unknown
- 2018-05-04 MA MA050749A patent/MA50749A/fr unknown
- 2018-05-04 AU AU2018261792A patent/AU2018261792A1/en not_active Abandoned
- 2018-05-04 WO PCT/US2018/031094 patent/WO2018204788A1/en not_active Ceased
- 2018-05-04 MX MX2019013158A patent/MX2019013158A/es unknown
- 2018-05-04 EP EP18726668.9A patent/EP3618838A1/en not_active Withdrawn
- 2018-05-04 KR KR1020197035568A patent/KR20190141770A/ko not_active Ceased
- 2018-05-04 CA CA3062316A patent/CA3062316A1/en active Pending
- 2018-05-04 US US16/607,971 patent/US20210100828A1/en not_active Abandoned
- 2018-05-04 CN CN202310591916.8A patent/CN116585344A/zh active Pending
- 2018-05-04 CN CN201880037914.1A patent/CN110740737A/zh active Pending
- 2018-05-04 BR BR112019022824A patent/BR112019022824A2/pt not_active IP Right Cessation
- 2018-05-04 JP JP2019560355A patent/JP2020518633A/ja active Pending
-
2019
- 2019-11-04 CL CL2019003153A patent/CL2019003153A1/es unknown
-
2021
- 2021-09-14 US US17/474,264 patent/US20220133769A1/en not_active Abandoned
-
2023
- 2023-02-17 JP JP2023023048A patent/JP2023075111A/ja active Pending
- 2023-08-09 US US18/446,743 patent/US20240115598A1/en not_active Abandoned
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