JPWO2020061177A5 - - Google Patents
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- JPWO2020061177A5 JPWO2020061177A5 JP2021515578A JP2021515578A JPWO2020061177A5 JP WO2020061177 A5 JPWO2020061177 A5 JP WO2020061177A5 JP 2021515578 A JP2021515578 A JP 2021515578A JP 2021515578 A JP2021515578 A JP 2021515578A JP WO2020061177 A5 JPWO2020061177 A5 JP WO2020061177A5
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- 230000009368 gene silencing by RNA Effects 0.000 claims description 58
- 239000003795 chemical substances by application Substances 0.000 claims description 55
- 102100037429 17-beta-hydroxysteroid dehydrogenase 13 Human genes 0.000 claims description 33
- 101000806241 Homo sapiens 17-beta-hydroxysteroid dehydrogenase 13 Proteins 0.000 claims description 32
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 27
- 238000000034 method Methods 0.000 claims description 25
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 claims description 22
- 201000010099 disease Diseases 0.000 claims description 21
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 claims description 18
- 239000000203 mixture Substances 0.000 claims description 13
- 238000011282 treatment Methods 0.000 claims description 13
- 230000000692 anti-sense effect Effects 0.000 claims description 12
- 230000014509 gene expression Effects 0.000 claims description 11
- 239000003814 drug Substances 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 229940124597 therapeutic agent Drugs 0.000 claims description 9
- 108091081021 Sense strand Proteins 0.000 claims description 8
- 101150000579 Hsd17b13 gene Proteins 0.000 claims description 6
- 208000035475 disorder Diseases 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 5
- 230000000295 complement effect Effects 0.000 claims description 4
- 208000019423 liver disease Diseases 0.000 claims description 4
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 108091030071 RNAI Proteins 0.000 claims 25
- 125000003729 nucleotide group Chemical group 0.000 claims 18
- 239000002773 nucleotide Substances 0.000 claims 10
- SXUXMRMBWZCMEN-UHFFFAOYSA-N 2'-O-methyl uridine Natural products COC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 SXUXMRMBWZCMEN-UHFFFAOYSA-N 0.000 claims 8
- FPUGCISOLXNPPC-IOSLPCCCSA-N cordysinin B Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-IOSLPCCCSA-N 0.000 claims 8
- 239000000126 substance Substances 0.000 claims 8
- QPHRQMAYYMYWFW-FJGDRVTGSA-N 1-[(2r,3s,4r,5r)-3-fluoro-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]pyrimidine-2,4-dione Chemical compound O[C@]1(F)[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 QPHRQMAYYMYWFW-FJGDRVTGSA-N 0.000 claims 4
- FPUGCISOLXNPPC-UHFFFAOYSA-N 2'-O-Methyladenosine Natural products COC1C(O)C(CO)OC1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-UHFFFAOYSA-N 0.000 claims 4
- RFCQJGFZUQFYRF-UHFFFAOYSA-N 2'-O-Methylcytidine Natural products COC1C(O)C(CO)OC1N1C(=O)N=C(N)C=C1 RFCQJGFZUQFYRF-UHFFFAOYSA-N 0.000 claims 4
- OVYNGSFVYRPRCG-UHFFFAOYSA-N 2'-O-Methylguanosine Natural products COC1C(O)C(CO)OC1N1C(NC(N)=NC2=O)=C2N=C1 OVYNGSFVYRPRCG-UHFFFAOYSA-N 0.000 claims 4
- RFCQJGFZUQFYRF-ZOQUXTDFSA-N 2'-O-methylcytidine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)N=C(N)C=C1 RFCQJGFZUQFYRF-ZOQUXTDFSA-N 0.000 claims 4
- OVYNGSFVYRPRCG-KQYNXXCUSA-N 2'-O-methylguanosine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(N=C(N)NC2=O)=C2N=C1 OVYNGSFVYRPRCG-KQYNXXCUSA-N 0.000 claims 4
- SXUXMRMBWZCMEN-ZOQUXTDFSA-N 2'-O-methyluridine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 SXUXMRMBWZCMEN-ZOQUXTDFSA-N 0.000 claims 4
- PJWBTAIPBFWVHX-FJGDRVTGSA-N 4-amino-1-[(2r,3s,4r,5r)-3-fluoro-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]pyrimidin-2-one Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@](F)(O)[C@H](O)[C@@H](CO)O1 PJWBTAIPBFWVHX-FJGDRVTGSA-N 0.000 claims 4
- 239000003446 ligand Substances 0.000 claims 4
- 230000008685 targeting Effects 0.000 claims 4
- UUDVSZSQPFXQQM-GIWSHQQXSA-N (2r,3s,4r,5r)-2-(6-aminopurin-9-yl)-3-fluoro-5-(hydroxymethyl)oxolane-3,4-diol Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@]1(O)F UUDVSZSQPFXQQM-GIWSHQQXSA-N 0.000 claims 3
- BGTXMQUSDNMLDW-AEHJODJJSA-N 2-amino-9-[(2r,3s,4r,5r)-3-fluoro-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-3h-purin-6-one Chemical compound C1=2NC(N)=NC(=O)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@]1(O)F BGTXMQUSDNMLDW-AEHJODJJSA-N 0.000 claims 3
- BXJSAMKIFDDLGI-UHFFFAOYSA-N 2-(dimethylamino)-N-[2-[3-[[5-[3-(dimethylcarbamoyl)phenyl]-2-methoxyphenyl]sulfonylamino]anilino]ethyl]benzamide Chemical compound COc1ccc(cc1S(=O)(=O)Nc1cccc(NCCNC(=O)c2ccccc2N(C)C)c1)-c1cccc(c1)C(=O)N(C)C BXJSAMKIFDDLGI-UHFFFAOYSA-N 0.000 claims 2
- 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 2
- KKZFLSZAWCYPOC-VPENINKCSA-N Deoxyribose 5-phosphate Chemical group O[C@H]1C[C@H](O)[C@@H](COP(O)(O)=O)O1 KKZFLSZAWCYPOC-VPENINKCSA-N 0.000 claims 2
- 125000003158 alcohol group Chemical group 0.000 claims 2
- 230000001404 mediated effect Effects 0.000 claims 2
- RHLMXWCISNJNDH-UHFFFAOYSA-N n-[2-[3-[[5-[3-(dimethylcarbamoyl)phenyl]-2-methoxyphenyl]sulfonylamino]anilino]ethyl]-3-methylbenzamide Chemical compound COC1=CC=C(C=2C=C(C=CC=2)C(=O)N(C)C)C=C1S(=O)(=O)NC(C=1)=CC=CC=1NCCNC(=O)C1=CC=CC(C)=C1 RHLMXWCISNJNDH-UHFFFAOYSA-N 0.000 claims 2
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims 1
- 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 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 description 33
- 208000019425 cirrhosis of liver Diseases 0.000 description 28
- 230000007882 cirrhosis Effects 0.000 description 15
- 208000022309 Alcoholic Liver disease Diseases 0.000 description 14
- 230000001476 alcoholic effect Effects 0.000 description 14
- 230000000069 prophylactic effect Effects 0.000 description 10
- 206010016654 Fibrosis Diseases 0.000 description 8
- 238000010254 subcutaneous injection Methods 0.000 description 6
- 239000007929 subcutaneous injection Substances 0.000 description 6
- 210000004027 cell Anatomy 0.000 description 4
- 108020004999 messenger RNA Proteins 0.000 description 4
- 108090000623 proteins and genes Proteins 0.000 description 3
- 230000001225 therapeutic effect Effects 0.000 description 3
- 208000026594 alcoholic fatty liver disease Diseases 0.000 description 2
- 239000002552 dosage form Substances 0.000 description 2
- 230000005764 inhibitory process Effects 0.000 description 2
- 208000024891 symptom Diseases 0.000 description 2
- 101710194118 17-beta-hydroxysteroid dehydrogenase 13 Proteins 0.000 description 1
- 208000007082 Alcoholic Fatty Liver Diseases 0.000 description 1
- 206010009208 Cirrhosis alcoholic Diseases 0.000 description 1
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 1
- 206010016262 Fatty liver alcoholic Diseases 0.000 description 1
- 206010019728 Hepatitis alcoholic Diseases 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 241001465754 Metazoa Species 0.000 description 1
- 208000002353 alcoholic hepatitis Diseases 0.000 description 1
- 208000010002 alcoholic liver cirrhosis Diseases 0.000 description 1
- 208000010706 fatty liver disease Diseases 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 238000007726 management method Methods 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 230000001717 pathogenic effect Effects 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 102000004169 proteins and genes Human genes 0.000 description 1
- 230000002459 sustained effect Effects 0.000 description 1
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- 238000011269 treatment regimen Methods 0.000 description 1
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Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024076157A JP7741927B2 (ja) | 2018-09-19 | 2024-05-08 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
| JP2025147403A JP2025186315A (ja) | 2018-09-19 | 2025-09-05 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862733320P | 2018-09-19 | 2018-09-19 | |
| US62/733,320 | 2018-09-19 | ||
| US201862773707P | 2018-11-30 | 2018-11-30 | |
| US62/773,707 | 2018-11-30 | ||
| US201962890220P | 2019-08-22 | 2019-08-22 | |
| US62/890,220 | 2019-08-22 | ||
| PCT/US2019/051707 WO2020061177A1 (en) | 2018-09-19 | 2019-09-18 | Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024076157A Division JP7741927B2 (ja) | 2018-09-19 | 2024-05-08 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022501040A JP2022501040A (ja) | 2022-01-06 |
| JPWO2020061177A5 true JPWO2020061177A5 (https=) | 2023-11-24 |
| JP2022501040A5 JP2022501040A5 (https=) | 2023-11-24 |
| JP7488254B2 JP7488254B2 (ja) | 2024-05-21 |
Family
ID=69887755
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021515578A Active JP7488254B2 (ja) | 2018-09-19 | 2019-09-18 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
| JP2024076157A Active JP7741927B2 (ja) | 2018-09-19 | 2024-05-08 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
| JP2025147403A Pending JP2025186315A (ja) | 2018-09-19 | 2025-09-05 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024076157A Active JP7741927B2 (ja) | 2018-09-19 | 2024-05-08 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
| JP2025147403A Pending JP2025186315A (ja) | 2018-09-19 | 2025-09-05 | １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 |
Country Status (21)
| Country | Link |
|---|---|
| US (1) | US12534728B2 (https=) |
| EP (1) | EP3852769A4 (https=) |
| JP (3) | JP7488254B2 (https=) |
| KR (1) | KR102769565B1 (https=) |
| CN (1) | CN113164509B (https=) |
| AU (1) | AU2019342117A1 (https=) |
| BR (1) | BR112021005137A2 (https=) |
| CA (1) | CA3109553A1 (https=) |
| CL (1) | CL2021000669A1 (https=) |
| CO (1) | CO2021003446A2 (https=) |
| CR (1) | CR20210186A (https=) |
| EC (1) | ECSP21027262A (https=) |
| IL (1) | IL281597A (https=) |
| MX (1) | MX2021003077A (https=) |
| MY (1) | MY209138A (https=) |
| PE (1) | PE20211212A1 (https=) |
| PH (1) | PH12021550423A1 (https=) |
| SG (1) | SG11202101698WA (https=) |
| TW (2) | TW202540423A (https=) |
| UY (1) | UY38382A (https=) |
| WO (1) | WO2020061177A1 (https=) |
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| MX2018009853A (es) * | 2016-09-02 | 2018-11-09 | Arrowhead Pharmaceuticals Inc | Ligandos de direccion. |
| JP7507093B2 (ja) * | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法 |
| MX2021003077A (es) * | 2018-09-19 | 2021-05-27 | Arrowhead Pharmaceuticals Inc | Agentes de arni para inhibir la expresion de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y metodos de uso. |
| EP3899024A4 (en) * | 2018-12-21 | 2023-05-31 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
| MX2022015169A (es) * | 2020-06-01 | 2023-07-03 | Amgen Inc | Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos. |
| WO2022098748A1 (en) * | 2020-11-06 | 2022-05-12 | Inipharm, Inc. | Uses for hsd17b13 inhibitors |
| CA3201624A1 (en) | 2020-12-23 | 2022-06-30 | Regeneron Pharmaceuticals, Inc. | Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors |
| US20240229037A1 (en) * | 2021-04-22 | 2024-07-11 | Tuojie Biotech(Shanghai) Co., Ltd. | SIRNA TARGETING 17Beta-HYDROXYSTEROID DEHYDROGENASE TYPE 13 AND SIRNA CONJUGATE |
| CN118103038A (zh) | 2021-08-20 | 2024-05-28 | 英安塔制药有限公司 | 17β-羟基类固醇脱氢酶13型抑制剂及其使用方法 |
| CA3230382A1 (en) * | 2021-09-08 | 2023-03-16 | Aligos Therapeutics, Inc. | Modified short interfering nucleic acid (sina) molecules and uses thereof |
| IL312743A (en) * | 2021-11-18 | 2024-07-01 | Novartis Ag | Compounds targeting PMP22 for the treatment of Charcot-Marie-Shen disease |
| JP2024545602A (ja) * | 2021-11-19 | 2024-12-10 | ウェイブ ライフ サイエンシズ リミテッド | Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 |
| WO2023109935A1 (zh) * | 2021-12-16 | 2023-06-22 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其制备方法及应用 |
| MX2024007463A (es) | 2021-12-20 | 2024-09-02 | Regeneron Pharma | Metodos para identificar y evaluar la inflamacion hepatica y la fibrosis hepatica en un sujeto mediante la determinacion de una puntuacion estratificada basada en la expresion genetica. |
| WO2023138650A1 (zh) * | 2022-01-20 | 2023-07-27 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其应用及制备方法 |
| CN119789859A (zh) | 2022-04-08 | 2025-04-08 | 箭头药业股份有限公司 | 非酒精性脂肪肝病的治疗 |
| IL315794A (en) * | 2022-04-28 | 2024-11-01 | Enanta Pharm Inc | Pyrimidine containing 17-beta-hydrosteroid dehydrogenase type 13 inhibitors |
| CN116515835A (zh) * | 2022-04-29 | 2023-08-01 | 北京福元医药股份有限公司 | 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途 |
| TW202400791A (zh) * | 2022-05-06 | 2024-01-01 | 大陸商蘇州瑞博生物技術股份有限公司 | 一種核酸、含有該核酸的組合物與綴合物及製備方法和用途 |
| WO2024131916A1 (en) * | 2022-12-22 | 2024-06-27 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of 17beta-hydroxysteroid dehydrogenase type 13 (hsd17b13) |
| CN118773194A (zh) * | 2023-06-16 | 2024-10-15 | 施能康医药科技(苏州)有限公司 | 靶向17-β-羟基类固醇脱氢酶13的核酸及其用途 |
| IL326882A (en) * | 2023-08-30 | 2026-04-01 | Arrowhead Pharmaceuticals Inc | RNAi agents for inhibiting expression of inhibitory subunit beta E (INHBE), their pharmaceutical compositions and methods of use |
| US20250120952A1 (en) * | 2023-10-04 | 2025-04-17 | Enanta Pharmaceuticals, Inc. | 17-Beta-Hydroxysteroid Dehydrogenase Type 13 Inhibitors And Methods Of Use Thereof |
| WO2025074157A1 (en) | 2023-10-06 | 2025-04-10 | Glaxosmithkline Intellectual Property (No.3) Limited | Treatment of a non-alcoholic fatty liver disease |
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