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JOP20210142A1 - تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها - Google Patents

تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها

Info

Publication number
JOP20210142A1
JOP20210142A1 JOP/2021/0142A JOP20210142A JOP20210142A1 JO P20210142 A1 JOP20210142 A1 JO P20210142A1 JO P20210142 A JOP20210142 A JO P20210142A JO P20210142 A1 JOP20210142 A1 JO P20210142A1
Authority
JO
Jordan
Prior art keywords
rnai constructs
pnpla3 expression
inhibiting pnpla3
inhibiting
liver disease
Prior art date
Application number
JOP/2021/0142A
Other languages
English (en)
Inventor
Michael Ollmann
Ingrid Rulifson
Justin K Murray
Oliver Homann
Original Assignee
Amgen Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Amgen Inc filed Critical Amgen Inc
Publication of JOP20210142A1 publication Critical patent/JOP20210142A1/ar

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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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Abstract

يتعلق الاختراع الحالي بتركيبات RNAi لتقليل التعبير الوراثي عن جين PNPLA3. تم أيضاً وصف طرق استخدام هذه التركيبات RNAi لعلاج أو منع أمراض الكبد، مرض الكبد الدهني غير الكحولي (NAFLD).
JOP/2021/0142A 2018-12-10 2019-12-10 تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها JOP20210142A1 (ar)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862777714P 2018-12-10 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

Publications (1)

Publication Number Publication Date
JOP20210142A1 true JOP20210142A1 (ar) 2023-01-30

Family

ID=69024749

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2021/0142A JOP20210142A1 (ar) 2018-12-10 2019-12-10 تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها

Country Status (23)

Country Link
US (1) US20220017906A1 (ar)
EP (1) EP3894555A2 (ar)
JP (2) JP2022511550A (ar)
KR (1) KR20210102932A (ar)
CN (1) CN113166761B (ar)
AR (1) AR117297A1 (ar)
AU (1) AU2019396419A1 (ar)
BR (1) BR112021011061A2 (ar)
CA (1) CA3121510A1 (ar)
CL (1) CL2021001489A1 (ar)
CO (1) CO2021008997A2 (ar)
CR (1) CR20210371A (ar)
EA (1) EA202191629A1 (ar)
IL (1) IL283473A (ar)
JO (1) JOP20210142A1 (ar)
MX (1) MX2021006784A (ar)
NZ (1) NZ776661A (ar)
PE (1) PE20211225A1 (ar)
PH (1) PH12021551326A1 (ar)
SG (1) SG11202105711SA (ar)
TW (1) TW202039843A (ar)
UY (1) UY38503A (ar)
WO (1) WO2020123508A2 (ar)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR20220133248A (ko) 2020-01-28 2022-10-04 리제너론 파마슈티칼스 인코포레이티드 인간화 pnpla3 좌위를 포함하는 비-인간 동물 및 사용 방법
WO2022221430A1 (en) 2021-04-14 2022-10-20 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
EP4402262A1 (en) * 2021-09-01 2024-07-24 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
EP4630556A1 (en) * 2022-12-07 2025-10-15 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of patatin-like phospholipase domain-containing 3 (pnpla3)
AU2023408479A1 (en) * 2022-12-19 2025-05-01 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar)
WO2024173593A1 (en) * 2023-02-14 2024-08-22 Basecure Therapeutics Modified double stranded rna agents
WO2024182446A2 (en) * 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
WO2024249328A2 (en) * 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
DE69233087T2 (de) 1991-11-22 2003-12-24 Affymetrix, Inc. (N.D.Ges.D.Staates Delaware) Verfahren zur Herstellung von Polymerarrays
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
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
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
AU747597B2 (en) 1998-07-20 2002-05-16 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme 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)
JP4868739B2 (ja) 2002-05-06 2012-02-01 アルナイラム ファーマシューティカルズ， インコーポレイテッド 核酸の送達法
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
AU2004233092C9 (en) 2003-04-17 2010-10-28 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
CA3043911A1 (en) 2007-12-04 2009-07-02 Arbutus Biopharma Corporation 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
WO2014205451A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
AU2016219263B2 (en) * 2015-02-13 2022-12-01 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)
JP2020522510A (ja) * 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
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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