JP2020534268A5 - - Google Patents
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- JP2020534268A5 JP2020534268A5 JP2020514926A JP2020514926A JP2020534268A5 JP 2020534268 A5 JP2020534268 A5 JP 2020534268A5 JP 2020514926 A JP2020514926 A JP 2020514926A JP 2020514926 A JP2020514926 A JP 2020514926A JP 2020534268 A5 JP2020534268 A5 JP 2020534268A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- rnai agent
- agent according
- nucleotides
- sense strand
- 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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- 108091030071 RNAI Proteins 0.000 claims description 98
- 239000003795 chemical substances by application Substances 0.000 claims description 98
- 230000009368 gene silencing by RNA Effects 0.000 claims description 98
- 125000003729 nucleotide group Chemical group 0.000 claims description 95
- 239000002773 nucleotide Substances 0.000 claims description 81
- 108091081021 Sense strand Proteins 0.000 claims description 48
- 239000000203 mixture Substances 0.000 claims description 46
- 230000000692 anti-sense effect Effects 0.000 claims description 28
- 238000000034 method Methods 0.000 claims description 25
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 18
- 239000003446 ligand Substances 0.000 claims description 18
- 230000008685 targeting Effects 0.000 claims description 18
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound 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 16
- 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 16
- 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 16
- 101000693085 Homo sapiens Angiopoietin-related protein 3 Proteins 0.000 claims description 13
- 201000010099 disease Diseases 0.000 claims description 13
- 230000014509 gene expression Effects 0.000 claims description 11
- 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 claims description 8
- 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 8
- 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 claims description 8
- 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 8
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims description 8
- 108010007622 LDL Lipoproteins Proteins 0.000 claims description 8
- 102000007330 LDL Lipoproteins Human genes 0.000 claims description 8
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims description 8
- 230000001476 alcoholic effect Effects 0.000 claims description 8
- 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 8
- 230000037396 body weight Effects 0.000 claims description 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 description 8
- 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 claims description 8
- 229940029575 guanosine Drugs 0.000 claims description 8
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims description 8
- 229940045145 uridine Drugs 0.000 claims description 8
- 102100025668 Angiopoietin-related protein 3 Human genes 0.000 claims description 7
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 7
- 208000035150 Hypercholesterolemia Diseases 0.000 claims description 6
- 208000006454 hepatitis Diseases 0.000 claims description 6
- 231100000283 hepatitis Toxicity 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 208000035475 disorder Diseases 0.000 claims description 5
- 208000024891 symptom Diseases 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
- FPUGCISOLXNPPC-UHFFFAOYSA-N 2'-O-Methyladenosine Natural products COC1C(O)C(CO)OC1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-UHFFFAOYSA-N 0.000 claims description 4
- 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 description 4
- 201000001320 Atherosclerosis Diseases 0.000 claims description 4
- 208000024172 Cardiovascular disease Diseases 0.000 claims description 4
- 208000030673 Homozygous familial hypercholesterolemia Diseases 0.000 claims description 4
- 208000031226 Hyperlipidaemia Diseases 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
- 208000008589 Obesity Diseases 0.000 claims description 4
- 230000002159 abnormal effect Effects 0.000 claims description 4
- 208000029078 coronary artery disease Diseases 0.000 claims description 4
- 208000016097 disease of metabolism Diseases 0.000 claims description 4
- 230000002526 effect on cardiovascular system Effects 0.000 claims description 4
- 208000006575 hypertriglyceridemia Diseases 0.000 claims description 4
- 150000002632 lipids Chemical class 0.000 claims description 4
- 208000030159 metabolic disease Diseases 0.000 claims description 4
- 230000004060 metabolic process Effects 0.000 claims description 4
- 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 description 4
- 235000020824 obesity Nutrition 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- UFTFJSFQGQCHQW-UHFFFAOYSA-N triformin Chemical compound O=COCC(OC=O)COC=O UFTFJSFQGQCHQW-UHFFFAOYSA-N 0.000 claims description 4
- 208000001072 type 2 diabetes mellitus Diseases 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 3
- JLWUWXCKSOIFPS-KQYNXXCUSA-N (2r,3r,4r,5r)-5-(2,6-diaminopurin-9-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-ol Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC(N)=NC(N)=C2N=C1 JLWUWXCKSOIFPS-KQYNXXCUSA-N 0.000 claims description 2
- FJLGEFLZQAZZCD-MCBHFWOFSA-N (3R,5S)-fluvastatin Chemical compound C12=CC=CC=C2N(C(C)C)C(\C=C\[C@@H](O)C[C@@H](O)CC(O)=O)=C1C1=CC=C(F)C=C1 FJLGEFLZQAZZCD-MCBHFWOFSA-N 0.000 claims description 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 description 2
- 102000005427 Asialoglycoprotein Receptor Human genes 0.000 claims description 2
- XUKUURHRXDUEBC-KAYWLYCHSA-N Atorvastatin Chemical compound C=1C=CC=CC=1C1=C(C=2C=CC(F)=CC=2)N(CC[C@@H](O)C[C@@H](O)CC(O)=O)C(C(C)C)=C1C(=O)NC1=CC=CC=C1 XUKUURHRXDUEBC-KAYWLYCHSA-N 0.000 claims description 2
- XUKUURHRXDUEBC-UHFFFAOYSA-N Atorvastatin Natural products C=1C=CC=CC=1C1=C(C=2C=CC(F)=CC=2)N(CCC(O)CC(O)CC(O)=O)C(C(C)C)=C1C(=O)NC1=CC=CC=C1 XUKUURHRXDUEBC-UHFFFAOYSA-N 0.000 claims description 2
- 206010014476 Elevated cholesterol Diseases 0.000 claims description 2
- 206010014486 Elevated triglycerides Diseases 0.000 claims description 2
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 claims description 2
- TUZYXOIXSAXUGO-UHFFFAOYSA-N Pravastatin Natural products C1=CC(C)C(CCC(O)CC(O)CC(O)=O)C2C(OC(=O)C(C)CC)CC(O)C=C21 TUZYXOIXSAXUGO-UHFFFAOYSA-N 0.000 claims description 2
- JVWLUVNSQYXYBE-UHFFFAOYSA-N Ribitol Natural products OCC(C)C(O)C(O)CO JVWLUVNSQYXYBE-UHFFFAOYSA-N 0.000 claims description 2
- RYMZZMVNJRMUDD-UHFFFAOYSA-N SJ000286063 Natural products C12C(OC(=O)C(C)(C)CC)CC(C)C=C2C=CC(C)C1CCC1CC(O)CC(=O)O1 RYMZZMVNJRMUDD-UHFFFAOYSA-N 0.000 claims description 2
- 239000002253 acid Substances 0.000 claims description 2
- 239000002214 arabinonucleotide Substances 0.000 claims description 2
- 108010006523 asialoglycoprotein receptor Proteins 0.000 claims description 2
- 229960005370 atorvastatin Drugs 0.000 claims description 2
- 230000000295 complement effect Effects 0.000 claims description 2
- 239000005547 deoxyribonucleotide Substances 0.000 claims description 2
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 2
- 235000014113 dietary fatty acids Nutrition 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 229930195729 fatty acid Natural products 0.000 claims description 2
- 239000000194 fatty acid Substances 0.000 claims description 2
- 150000004665 fatty acids Chemical class 0.000 claims description 2
- 229960003765 fluvastatin Drugs 0.000 claims description 2
- 125000001921 locked nucleotide group Chemical group 0.000 claims description 2
- HEBKCHPVOIAQTA-UHFFFAOYSA-N meso ribitol Natural products OCC(O)C(O)C(O)CO HEBKCHPVOIAQTA-UHFFFAOYSA-N 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 229960002797 pitavastatin Drugs 0.000 claims description 2
- VGYFMXBACGZSIL-MCBHFWOFSA-N pitavastatin Chemical compound OC(=O)C[C@H](O)C[C@H](O)\C=C\C1=C(C2CC2)N=C2C=CC=CC2=C1C1=CC=C(F)C=C1 VGYFMXBACGZSIL-MCBHFWOFSA-N 0.000 claims description 2
- 229960002965 pravastatin Drugs 0.000 claims description 2
- TUZYXOIXSAXUGO-PZAWKZKUSA-N pravastatin Chemical compound C1=C[C@H](C)[C@H](CC[C@@H](O)C[C@@H](O)CC(O)=O)[C@H]2[C@@H](OC(=O)[C@@H](C)CC)C[C@H](O)C=C21 TUZYXOIXSAXUGO-PZAWKZKUSA-N 0.000 claims description 2
- HEBKCHPVOIAQTA-ZXFHETKHSA-N ribitol Chemical compound OC[C@H](O)[C@H](O)[C@H](O)CO HEBKCHPVOIAQTA-ZXFHETKHSA-N 0.000 claims description 2
- 229960000672 rosuvastatin Drugs 0.000 claims description 2
- BPRHUIZQVSMCRT-VEUZHWNKSA-N rosuvastatin Chemical compound CC(C)C1=NC(N(C)S(C)(=O)=O)=NC(C=2C=CC(F)=CC=2)=C1\C=C\[C@@H](O)C[C@@H](O)CC(O)=O BPRHUIZQVSMCRT-VEUZHWNKSA-N 0.000 claims description 2
- 229960002855 simvastatin Drugs 0.000 claims description 2
- RYMZZMVNJRMUDD-HGQWONQESA-N simvastatin Chemical compound C([C@H]1[C@@H](C)C=CC2=C[C@H](C)C[C@@H]([C@H]12)OC(=O)C(C)(C)CC)C[C@@H]1C[C@@H](O)CC(=O)O1 RYMZZMVNJRMUDD-HGQWONQESA-N 0.000 claims description 2
- 229940124597 therapeutic agent Drugs 0.000 claims description 2
- 208000019423 liver disease Diseases 0.000 claims 1
- 125000003835 nucleoside group Chemical group 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000463 material Substances 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 239000002777 nucleoside Substances 0.000 description 1
- 150000003833 nucleoside derivatives Chemical class 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023028733A JP7660154B2 (ja) | 2017-09-14 | 2023-02-27 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
| JP2025058240A JP2025092670A (ja) | 2017-09-14 | 2025-03-31 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762558819P | 2017-09-14 | 2017-09-14 | |
| US62/558,819 | 2017-09-14 | ||
| US201762583919P | 2017-11-09 | 2017-11-09 | |
| US62/583,919 | 2017-11-09 | ||
| US201862651284P | 2018-04-02 | 2018-04-02 | |
| US62/651,284 | 2018-04-02 | ||
| US201862694976P | 2018-07-07 | 2018-07-07 | |
| US62/694,976 | 2018-07-07 | ||
| PCT/US2018/050848 WO2019055633A1 (en) | 2017-09-14 | 2018-09-13 | RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023028733A Division JP7660154B2 (ja) | 2017-09-14 | 2023-02-27 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020534268A JP2020534268A (ja) | 2020-11-26 |
| JP2020534268A5 true JP2020534268A5 (enExample) | 2021-10-14 |
| JP7281452B2 JP7281452B2 (ja) | 2023-05-25 |
Family
ID=65630620
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020514926A Active JP7281452B2 (ja) | 2017-09-14 | 2018-09-13 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
| JP2023028733A Active JP7660154B2 (ja) | 2017-09-14 | 2023-02-27 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
| JP2025058240A Pending JP2025092670A (ja) | 2017-09-14 | 2025-03-31 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023028733A Active JP7660154B2 (ja) | 2017-09-14 | 2023-02-27 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
| JP2025058240A Pending JP2025092670A (ja) | 2017-09-14 | 2025-03-31 | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 |
Country Status (24)
| Country | Link |
|---|---|
| US (2) | US10995335B2 (enExample) |
| EP (1) | EP3681513A4 (enExample) |
| JP (3) | JP7281452B2 (enExample) |
| KR (2) | KR102737464B1 (enExample) |
| CN (2) | CN111343994B (enExample) |
| AU (2) | AU2018331383B2 (enExample) |
| BR (1) | BR112020003126A2 (enExample) |
| CA (1) | CA3074320A1 (enExample) |
| CL (1) | CL2020000666A1 (enExample) |
| CO (1) | CO2020001740A2 (enExample) |
| EC (1) | ECSP20018305A (enExample) |
| IL (2) | IL273173B1 (enExample) |
| JO (1) | JOP20200050A1 (enExample) |
| MX (1) | MX2020001912A (enExample) |
| MY (1) | MY200087A (enExample) |
| NZ (1) | NZ763275A (enExample) |
| PE (1) | PE20201287A1 (enExample) |
| PH (1) | PH12020500309A1 (enExample) |
| SG (1) | SG11201912188RA (enExample) |
| TN (1) | TN2020000038A1 (enExample) |
| TW (2) | TWI887846B (enExample) |
| UY (1) | UY37881A (enExample) |
| WO (1) | WO2019055633A1 (enExample) |
| ZA (1) | ZA202103669B (enExample) |
Families Citing this family (78)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2012272970A1 (en) | 2011-06-21 | 2014-02-06 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof |
| EP3283631A1 (en) | 2015-04-13 | 2018-02-21 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| CN109462981B (zh) * | 2016-09-02 | 2023-07-07 | 箭头药业股份有限公司 | 靶向配体 |
| US12156523B2 (en) | 2017-03-17 | 2024-12-03 | Mmag Co., Ltd. | Plant disease control agent |
| LT3607069T (lt) | 2017-04-05 | 2023-01-10 | Silence Therapeutics Gmbh | Produktai ir kompozicijos |
| WO2021030522A1 (en) | 2019-08-13 | 2021-02-18 | Alnylam Pharmaceuticals, Inc. | SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| WO2021087036A1 (en) | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| WO2021096763A1 (en) | 2019-11-13 | 2021-05-20 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating an angiotensinogen- (agt-) associated disorder |
| BR112022011417A2 (pt) | 2019-12-13 | 2022-08-30 | Alnylam Pharmaceuticals Inc | Composições do agente de irna da fase de leitura aberta 72 do cromossomo humano 9 (c9orf72) e métodos de uso das mesmas |
| TW202138559A (zh) | 2019-12-16 | 2021-10-16 | 美商阿尼拉製藥公司 | 含類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）ｉＲＮＡ組成物及其使用方法 |
| AU2021224778A1 (en) | 2020-02-18 | 2022-09-29 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof |
| EP4114947A1 (en) | 2020-03-05 | 2023-01-11 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases |
| AU2021232014A1 (en) | 2020-03-06 | 2022-10-06 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (KHK) IRNA compositions and methods of use thereof |
| WO2021188611A1 (en) | 2020-03-18 | 2021-09-23 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| MX2022011550A (es) * | 2020-03-18 | 2023-01-04 | Dicerna Pharmaceuticals Inc | Composiciones y métodos para inhibir la expresión de angptl3. |
| WO2021195307A1 (en) | 2020-03-26 | 2021-09-30 | Alnylam Pharmaceuticals, Inc. | Coronavirus irna compositions and methods of use thereof |
| US12054718B2 (en) * | 2020-03-26 | 2024-08-06 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use |
| WO2021206917A1 (en) | 2020-04-07 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| WO2021206922A1 (en) | 2020-04-07 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof |
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