JP2024105284A5 - - Google Patents
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- Publication number
- JP2024105284A5 JP2024105284A5 JP2024065950A JP2024065950A JP2024105284A5 JP 2024105284 A5 JP2024105284 A5 JP 2024105284A5 JP 2024065950 A JP2024065950 A JP 2024065950A JP 2024065950 A JP2024065950 A JP 2024065950A JP 2024105284 A5 JP2024105284 A5 JP 2024105284A5
- Authority
- JP
- Japan
- Prior art keywords
- rnai construct
- seq
- nucleotides
- pnpla3
- duplex
- 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
Links
- 108091030071 RNAI Proteins 0.000 claims 40
- 230000009368 gene silencing by RNA Effects 0.000 claims 40
- 125000003729 nucleotide group Chemical group 0.000 claims 26
- 230000000692 anti-sense effect Effects 0.000 claims 16
- 239000002773 nucleotide Substances 0.000 claims 16
- 108091081021 Sense strand Proteins 0.000 claims 11
- 102100031251 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Human genes 0.000 claims 10
- 101001129184 Homo sapiens 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Proteins 0.000 claims 10
- 108700028369 Alleles Proteins 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 4
- 210000005229 liver cell Anatomy 0.000 claims 3
- 108091093094 Glycol nucleic acid Proteins 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 230000005764 inhibitory process Effects 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- KBCBKZHEGSUDBQ-OFBQIVOGSA-N 9-[(2R,3S,4R,5R)-3,4-dihydroxy-5-(hydroxymethyl)-3-methoxyoxolan-2-yl]-1H-purin-6-one Chemical compound CO[C@@]1([C@@H](O[C@@H]([C@H]1O)CO)N1C=NC=2C(O)=NC=NC1=2)O KBCBKZHEGSUDBQ-OFBQIVOGSA-N 0.000 claims 1
- 208000035657 Abasia Diseases 0.000 claims 1
- -1 bicyclic nucleic acid Chemical class 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 238000011534 incubation Methods 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862777714P | 2018-12-10 | 2018-12-10 | |
| US62/777,714 | 2018-12-10 | ||
| PCT/US2019/065481 WO2020123508A2 (en) | 2018-12-10 | 2019-12-10 | Rnai constructs for inhibiting pnpla3 expression and methods of use thereof |
| JP2021532216A JP2022511550A (ja) | 2018-12-10 | 2019-12-10 | ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021532216A Division JP2022511550A (ja) | 2018-12-10 | 2019-12-10 | ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024105284A JP2024105284A (ja) | 2024-08-06 |
| JP2024105284A5 true JP2024105284A5 (https=) | 2025-03-07 |
Family
ID=69024749
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021532216A Pending JP2022511550A (ja) | 2018-12-10 | 2019-12-10 | ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト |
| JP2024065950A Pending JP2024105284A (ja) | 2018-12-10 | 2024-04-16 | ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021532216A Pending JP2022511550A (ja) | 2018-12-10 | 2019-12-10 | ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト |
Country Status (22)
| Country | Link |
|---|---|
| US (2) | US20220017906A1 (https=) |
| EP (1) | EP3894555A2 (https=) |
| JP (2) | JP2022511550A (https=) |
| KR (1) | KR20210102932A (https=) |
| CN (2) | CN113166761B (https=) |
| AR (1) | AR117297A1 (https=) |
| AU (1) | AU2019396419B2 (https=) |
| BR (1) | BR112021011061A2 (https=) |
| CA (1) | CA3121510A1 (https=) |
| CL (1) | CL2021001489A1 (https=) |
| CO (1) | CO2021008997A2 (https=) |
| CR (1) | CR20210371A (https=) |
| EA (1) | EA202191629A1 (https=) |
| IL (1) | IL283473A (https=) |
| JO (1) | JOP20210142A1 (https=) |
| MX (2) | MX2021006784A (https=) |
| PE (1) | PE20211225A1 (https=) |
| PH (1) | PH12021551326A1 (https=) |
| SG (1) | SG11202105711SA (https=) |
| TW (1) | TW202039843A (https=) |
| UY (1) | UY38503A (https=) |
| WO (1) | WO2020123508A2 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA201792263A1 (ru) | 2015-04-13 | 2018-08-31 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA ПРОТИВ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| AR113490A1 (es) * | 2017-12-12 | 2020-05-06 | Amgen Inc | CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS |
| EP4096396A1 (en) | 2020-01-28 | 2022-12-07 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized pnpla3 locus and methods of use |
| US12084662B2 (en) * | 2021-04-14 | 2024-09-10 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating PNPLA3 expression |
| EP4347823A1 (en) * | 2021-06-02 | 2024-04-10 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| AU2022339846A1 (en) * | 2021-09-01 | 2024-03-14 | Aligos Therapeutics, Inc. | Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof |
| JP2025524135A (ja) * | 2022-07-27 | 2025-07-25 | イー－セラピューティクス・ピーエルシー | 核酸化合物 |
| JP2025542119A (ja) * | 2022-12-07 | 2025-12-25 | シャンハイ アルゴ バイオファーマシューティカル カンパニー，リミテッド | パタチン様ホスホリパーゼドメイン含有３（ｐｎｐｌａ３）の発現を阻害するための組成物及び方法 |
| AU2023408479A1 (en) * | 2022-12-19 | 2025-05-01 | Arnatar Therapeutics, Inc | Advanced rna targeting (arnatar) |
| CN121152879A (zh) * | 2023-02-14 | 2025-12-16 | 基愈治疗公司 | 经修饰的双链rna药剂 |
| WO2024182446A2 (en) * | 2023-02-28 | 2024-09-06 | Aligos Therapeutics, Inc. | Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof |
| IL324720A (en) * | 2023-05-26 | 2026-01-01 | Adarx Pharmaceuticals Inc | SOD1 modulators and methods of using them |
Family Cites Families (40)
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|---|---|---|---|---|
| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
| US5744101A (en) | 1989-06-07 | 1998-04-28 | Affymax Technologies N.V. | Photolabile nucleoside protecting groups |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| DE69233501T2 (de) | 1991-11-22 | 2006-02-23 | Affymetrix, Inc. (n.d.Ges.d.Staates Delaware), Santa Clara | Kombinatorische Strategien für die Polymersynthese |
| US5981505A (en) | 1993-01-26 | 1999-11-09 | The Trustees Of The University Of Pennsylvania | Compositions and methods for delivery of genetic material |
| US5837533A (en) | 1994-09-28 | 1998-11-17 | American Home Products Corporation | Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent |
| US5556752A (en) | 1994-10-24 | 1996-09-17 | Affymetrix, Inc. | Surface-bound, unimolecular, double-stranded DNA |
| US5840710A (en) | 1994-12-09 | 1998-11-24 | Genzyme Corporation | Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules |
| US5981501A (en) | 1995-06-07 | 1999-11-09 | Inex Pharmaceuticals Corp. | Methods for encapsulating plasmids in lipid bilayers |
| US5545531A (en) | 1995-06-07 | 1996-08-13 | Affymax Technologies N.V. | Methods for making a device for concurrently processing multiple biological chip assays |
| ATE285477T1 (de) | 1995-06-07 | 2005-01-15 | Inex Pharmaceutical Corp | Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer |
| US7422902B1 (en) | 1995-06-07 | 2008-09-09 | The University Of British Columbia | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
| US5854033A (en) | 1995-11-21 | 1998-12-29 | Yale University | Rolling circle replication reporter systems |
| US6217900B1 (en) | 1997-04-30 | 2001-04-17 | American Home Products Corporation | Vesicular complexes and methods of making and using the same |
| DE69834038D1 (de) | 1997-07-01 | 2006-05-18 | Isis Pharmaceutical Inc | Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre |
| WO2000003683A2 (en) | 1998-07-20 | 2000-01-27 | Inex Pharmaceuticals Corporation | Liposomal encapsulated nucleic acid-complexes |
| US7833992B2 (en) | 2001-05-18 | 2010-11-16 | Merck Sharpe & Dohme | Conjugates and compositions for cellular delivery |
| US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| US6693187B1 (en) | 2000-10-17 | 2004-02-17 | Lievre Cornu Llc | Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge |
| US20030130186A1 (en) | 2001-07-20 | 2003-07-10 | Chandra Vargeese | Conjugates and compositions for cellular delivery |
| US7109165B2 (en) | 2001-05-18 | 2006-09-19 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| US9181551B2 (en) | 2002-02-20 | 2015-11-10 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| WO2003093449A2 (en) | 2002-05-06 | 2003-11-13 | Nucleonics, Inc. | Methods for delivery of nucleic acids |
| DK1620544T3 (en) | 2003-04-17 | 2019-01-14 | Alnylam Pharmaceuticals Inc | MODIFIED iRNA AGENTS |
| US8017762B2 (en) | 2003-04-17 | 2011-09-13 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
| US7851615B2 (en) | 2003-04-17 | 2010-12-14 | Alnylam Pharmaceuticals, Inc. | Lipophilic conjugated iRNA agents |
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| CA2685127C (en) | 2007-04-23 | 2019-01-08 | Alnylam Pharmaceuticals, Inc. | Glycoconjugates of rna interference agents |
| WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| AR090905A1 (es) | 2012-05-02 | 2014-12-17 | Merck Sharp & Dohme | Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica |
| EP3564374A1 (en) | 2013-06-21 | 2019-11-06 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulation of target nucleic acids |
| WO2016130806A2 (en) * | 2015-02-13 | 2016-08-18 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| EP3350328A1 (en) * | 2015-09-14 | 2018-07-25 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof |
| US10036024B2 (en) * | 2016-06-03 | 2018-07-31 | Purdue Research Foundation | siRNA compositions that specifically downregulate expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD) |
| US11597927B2 (en) * | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11603532B2 (en) * | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| AR113490A1 (es) * | 2017-12-12 | 2020-05-06 | Amgen Inc | CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS |
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2019
- 2019-12-10 CN CN201980081364.8A patent/CN113166761B/zh active Active
- 2019-12-10 KR KR1020217021155A patent/KR20210102932A/ko not_active Ceased
- 2019-12-10 UY UY0001038503A patent/UY38503A/es not_active Application Discontinuation
- 2019-12-10 CN CN202511224281.3A patent/CN121320339A/zh active Pending
- 2019-12-10 SG SG11202105711SA patent/SG11202105711SA/en unknown
- 2019-12-10 WO PCT/US2019/065481 patent/WO2020123508A2/en not_active Ceased
- 2019-12-10 PH PH1/2021/551326A patent/PH12021551326A1/en unknown
- 2019-12-10 US US17/312,721 patent/US20220017906A1/en active Pending
- 2019-12-10 TW TW108145175A patent/TW202039843A/zh unknown
- 2019-12-10 JP JP2021532216A patent/JP2022511550A/ja active Pending
- 2019-12-10 EA EA202191629A patent/EA202191629A1/ru unknown
- 2019-12-10 PE PE2021000850A patent/PE20211225A1/es unknown
- 2019-12-10 AU AU2019396419A patent/AU2019396419B2/en active Active
- 2019-12-10 CR CR20210371A patent/CR20210371A/es unknown
- 2019-12-10 AR ARP190103607A patent/AR117297A1/es unknown
- 2019-12-10 MX MX2021006784A patent/MX2021006784A/es unknown
- 2019-12-10 EP EP19828524.9A patent/EP3894555A2/en active Pending
- 2019-12-10 BR BR112021011061-5A patent/BR112021011061A2/pt unknown
- 2019-12-10 CA CA3121510A patent/CA3121510A1/en active Pending
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2021
- 2021-05-26 IL IL283473A patent/IL283473A/en unknown
- 2021-06-08 MX MX2026000462A patent/MX2026000462A/es unknown
- 2021-06-08 CL CL2021001489A patent/CL2021001489A1/es unknown
- 2021-06-09 JO JOP/2021/0142A patent/JOP20210142A1/ar unknown
- 2021-07-08 CO CONC2021/0008997A patent/CO2021008997A2/es unknown
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2024
- 2024-04-16 JP JP2024065950A patent/JP2024105284A/ja active Pending
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2025
- 2025-01-17 US US19/029,290 patent/US20250320502A1/en active Pending