JP2020537653A5 - - Google Patents
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- JP2020537653A5 JP2020537653A5 JP2020521451A JP2020521451A JP2020537653A5 JP 2020537653 A5 JP2020537653 A5 JP 2020537653A5 JP 2020521451 A JP2020521451 A JP 2020521451A JP 2020521451 A JP2020521451 A JP 2020521451A JP 2020537653 A5 JP2020537653 A5 JP 2020537653A5
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- 125000003729 nucleotide group Chemical group 0.000 claims description 76
- 239000002773 nucleotide Substances 0.000 claims description 61
- 108091030071 RNAI Proteins 0.000 claims description 50
- 239000003795 chemical substances by application Substances 0.000 claims description 50
- 230000009368 gene silencing by RNA Effects 0.000 claims description 50
- 108091081021 Sense strand Proteins 0.000 claims description 31
- 230000000692 anti-sense effect Effects 0.000 claims description 29
- 239000003446 ligand Substances 0.000 claims description 19
- 230000008685 targeting Effects 0.000 claims description 17
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims description 12
- 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 12
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 12
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Natural products C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims description 10
- 102100026292 Asialoglycoprotein receptor 1 Human genes 0.000 claims description 7
- 101000785944 Homo sapiens Asialoglycoprotein receptor 1 Proteins 0.000 claims description 7
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims description 7
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims description 6
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims description 6
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims description 6
- 201000010099 disease Diseases 0.000 claims description 6
- 208000035475 disorder Diseases 0.000 claims description 6
- 229940029575 guanosine Drugs 0.000 claims description 6
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims description 6
- 229940045145 uridine Drugs 0.000 claims description 6
- 101150075175 Asgr1 gene Proteins 0.000 claims description 5
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- FPUGCISOLXNPPC-IOSLPCCCSA-N cordysinin B Chemical group 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 description 5
- 230000002401 inhibitory effect Effects 0.000 claims description 5
- 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 description 4
- 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 description 4
- 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 description 4
- 208000010125 myocardial infarction Diseases 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 3
- 208000029078 coronary artery disease Diseases 0.000 claims description 3
- 201000001320 Atherosclerosis Diseases 0.000 claims description 2
- 208000024172 Cardiovascular disease Diseases 0.000 claims description 2
- 208000032928 Dyslipidaemia Diseases 0.000 claims description 2
- 208000035150 Hypercholesterolemia Diseases 0.000 claims description 2
- 208000031226 Hyperlipidaemia Diseases 0.000 claims description 2
- 229930010555 Inosine Natural products 0.000 claims description 2
- 208000017170 Lipid metabolism disease Diseases 0.000 claims description 2
- 208000001145 Metabolic Syndrome Diseases 0.000 claims description 2
- 208000008589 Obesity Diseases 0.000 claims description 2
- 208000018262 Peripheral vascular disease Diseases 0.000 claims description 2
- 201000000690 abdominal obesity-metabolic syndrome Diseases 0.000 claims description 2
- 239000002214 arabinonucleotide Substances 0.000 claims description 2
- 208000026106 cerebrovascular disease Diseases 0.000 claims description 2
- 239000005547 deoxyribonucleotide Substances 0.000 claims description 2
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 2
- 206010012601 diabetes mellitus Diseases 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 208000006575 hypertriglyceridemia Diseases 0.000 claims description 2
- 229960003786 inosine Drugs 0.000 claims description 2
- 125000001921 locked nucleotide group Chemical group 0.000 claims description 2
- 230000004060 metabolic process Effects 0.000 claims description 2
- 235000020824 obesity Nutrition 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 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 4
- 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 2
- FPUGCISOLXNPPC-UHFFFAOYSA-N 2'-O-Methyladenosine Natural products COC1C(O)C(CO)OC1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-UHFFFAOYSA-N 0.000 claims 2
- 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 2
- 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 2
- 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 2
- 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 2
- 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 2
- 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 2
- HPHXOIULGYVAKW-IOSLPCCCSA-N 2'-O-methylinosine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(N=CNC2=O)=C2N=C1 HPHXOIULGYVAKW-IOSLPCCCSA-N 0.000 claims 1
- HPHXOIULGYVAKW-UHFFFAOYSA-N 2'-O-methylinosine Natural products COC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 HPHXOIULGYVAKW-UHFFFAOYSA-N 0.000 claims 1
- 208000035657 Abasia Diseases 0.000 claims 1
- OOYGSFOGFJDDHP-KMCOLRRFSA-N kanamycin A sulfate Chemical group OS(O)(=O)=O.O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CN)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](N)[C@H](O)[C@@H](CO)O2)O)[C@H](N)C[C@@H]1N OOYGSFOGFJDDHP-KMCOLRRFSA-N 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 239000002777 nucleoside Substances 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 description 14
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 description 4
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 description 4
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 description 4
- 235000012000 cholesterol Nutrition 0.000 description 3
- 238000004519 manufacturing process Methods 0.000 description 3
- MXYRZDAGKTVQIL-IOSLPCCCSA-N (2r,3r,4s,5r)-2-(6-aminopurin-9-yl)-5-(hydroxymethyl)-2-methyloxolane-3,4-diol Chemical compound C1=NC2=C(N)N=CN=C2N1[C@]1(C)O[C@H](CO)[C@@H](O)[C@H]1O MXYRZDAGKTVQIL-IOSLPCCCSA-N 0.000 description 1
- 238000008214 LDL Cholesterol Methods 0.000 description 1
- 230000037396 body weight Effects 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024064086A JP2024086876A (ja) | 2017-10-17 | 2024-04-11 | アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762573206P | 2017-10-17 | 2017-10-17 | |
| US62/573,206 | 2017-10-17 | ||
| US201762608606P | 2017-12-21 | 2017-12-21 | |
| US62/608,606 | 2017-12-21 | ||
| US201862635277P | 2018-02-26 | 2018-02-26 | |
| US62/635,277 | 2018-02-26 | ||
| PCT/US2018/056077 WO2019079294A1 (en) | 2017-10-17 | 2018-10-16 | RNAI-BASED AGENTS AND COMPOSITIONS FOR INHIBITING ASIALOGLYCOPROTEIN RECEPTOR 1 RECEPTOR 1 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024064086A Division JP2024086876A (ja) | 2017-10-17 | 2024-04-11 | アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020537653A JP2020537653A (ja) | 2020-12-24 |
| JP2020537653A5 true JP2020537653A5 (enExample) | 2021-11-25 |
| JP7473472B2 JP7473472B2 (ja) | 2024-04-23 |
Family
ID=66173364
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020521451A Active JP7473472B2 (ja) | 2017-10-17 | 2018-10-16 | アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物 |
| JP2024064086A Pending JP2024086876A (ja) | 2017-10-17 | 2024-04-11 | アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024064086A Pending JP2024086876A (ja) | 2017-10-17 | 2024-04-11 | アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US11492624B2 (enExample) |
| EP (1) | EP3697909A4 (enExample) |
| JP (2) | JP7473472B2 (enExample) |
| CN (1) | CN111212909A (enExample) |
| AU (1) | AU2018352379A1 (enExample) |
| CA (1) | CA3079413A1 (enExample) |
| TW (1) | TW201918555A (enExample) |
| UY (1) | UY37936A (enExample) |
| WO (1) | WO2019079294A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109462981B (zh) * | 2016-09-02 | 2023-07-07 | 箭头药业股份有限公司 | 靶向配体 |
| CN116617390A (zh) * | 2022-02-11 | 2023-08-22 | 武汉大学 | Asgr1抑制剂在促进胆固醇外排和治疗非酒精性脂肪性肝病中的应用 |
| CN117645995A (zh) * | 2022-09-02 | 2024-03-05 | 北京福元医药股份有限公司 | 抑制去唾液酸糖蛋白受体基因表达的siRNA、其缀合物和药物组合物及用途 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
| EP1159441B8 (en) | 1999-03-10 | 2008-10-29 | Marie Curie Cancer Care | Delivery of nucleic acids and proteins to cells |
| CN1300312C (zh) * | 2004-08-23 | 2007-02-14 | 中国人民解放军军事医学科学院放射与辐射医学研究所 | 靶向去唾液酸糖蛋白受体的抗乙型肝炎病毒反义寡核苷酸的结构和用途 |
| BRPI0715375A2 (pt) | 2006-08-18 | 2013-06-18 | Hoffmann La Roche | policonjugados para distribuiÇço in vivo de polinucleotÍdeos |
| WO2008043561A2 (en) * | 2006-10-11 | 2008-04-17 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Influenza targets |
| EP2295543A1 (en) * | 2009-09-11 | 2011-03-16 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Method for the preparation of an influenza virus |
| ES2562817T3 (es) | 2010-02-24 | 2016-03-08 | Arrowhead Research Corporation | Composiciones para el suministro dirigido de ARNip |
| US8501930B2 (en) | 2010-12-17 | 2013-08-06 | Arrowhead Madison Inc. | Peptide-based in vivo siRNA delivery system |
| MX2014001971A (es) | 2011-08-26 | 2014-03-31 | Arrowhead Res Corp | Polimeros de poli(vinilester) para suministro de acido nucleico in vivo. |
| MX2014001965A (es) | 2012-04-18 | 2014-03-31 | Arrowhead Res Corp | Polimeros de poli(acrilato) para suministro de acido nucleico in vivo. |
| US10358497B2 (en) * | 2015-09-29 | 2019-07-23 | Amgen Inc. | Methods of treating cardiovascular disease with an ASGR inhibitor |
| JOP20210043A1 (ar) * | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| CN109069529B (zh) | 2016-03-07 | 2021-08-20 | 箭头药业股份有限公司 | 用于治疗性化合物的靶向配体 |
| UY37376A (es) * | 2016-08-26 | 2018-03-23 | Amgen Inc | Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso |
| CN109462981B (zh) * | 2016-09-02 | 2023-07-07 | 箭头药业股份有限公司 | 靶向配体 |
-
2018
- 2018-10-16 CN CN201880066319.0A patent/CN111212909A/zh active Pending
- 2018-10-16 JP JP2020521451A patent/JP7473472B2/ja active Active
- 2018-10-16 CA CA3079413A patent/CA3079413A1/en active Pending
- 2018-10-16 US US16/756,440 patent/US11492624B2/en active Active
- 2018-10-16 EP EP18868368.4A patent/EP3697909A4/en active Pending
- 2018-10-16 WO PCT/US2018/056077 patent/WO2019079294A1/en not_active Ceased
- 2018-10-16 AU AU2018352379A patent/AU2018352379A1/en not_active Abandoned
- 2018-10-17 TW TW107136576A patent/TW201918555A/zh unknown
- 2018-10-17 UY UY0001037936A patent/UY37936A/es not_active Application Discontinuation
-
2024
- 2024-04-11 JP JP2024064086A patent/JP2024086876A/ja active Pending
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