MX2023002853A - Agentes de arni para inhibir la expresion de dux4, composiciones de dichos agentes, y metodos de uso. - Google Patents

Agentes de arni para inhibir la expresion de dux4, composiciones de dichos agentes, y metodos de uso.

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Publication number
MX2023002853A
MX2023002853A MX2023002853A MX2023002853A MX2023002853A MX 2023002853 A MX2023002853 A MX 2023002853A MX 2023002853 A MX2023002853 A MX 2023002853A MX 2023002853 A MX2023002853 A MX 2023002853A MX 2023002853 A MX2023002853 A MX 2023002853A
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MX
Mexico
Prior art keywords
dux4
rnai agents
compositions
methods
gene
Prior art date
Application number
MX2023002853A
Other languages
English (en)
Inventor
Tao Pei
Anthony Nicholas
Xiaokai Li
Zhao Xu
Zhi- Ming Ding
Susan Phan
Teng Ai
Susan Ramos-Hunter
Dyke Jonathan Van
Casi M Schienebeck
Original Assignee
Arrowhead Pharmaceuticals 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.)
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of MX2023002853A publication Critical patent/MX2023002853A/es

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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
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  • Saccharide Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se describen agentes de ARNi, composiciones que incluyen agentes de ARNi, y métodos para inhibir un gen doble homebox 4 (DUX4). Los agentes de ARNi contra DUX4 y los conjugados de agentes de ARNi que se divulgan en la presente divulgación inhiben la expresión de un gen DUX4. También se describen composiciones farmacéuticas que incluyen uno o más agentes de ARNi contra DUX4, opcionalmente con uno o más agentes terapéuticos adicionales. El transporte de los agentes de ARNi contra DUX4 descritos hasta las células de músculo esquelético in vivo, causa la inhibición de la expresión del gen DUX4 y la disminución en niveles de DUX4, lo cual puede causar un efecto beneficioso a los sujetos, incluidos los sujetos humanos, que padecen ciertas enfermedades o trastornos relacionados con las células de músculo esquelético incluida la distrofia muscular facioescapulohumeral (FSHD).
MX2023002853A 2020-09-11 2021-09-10 Agentes de arni para inhibir la expresion de dux4, composiciones de dichos agentes, y metodos de uso. MX2023002853A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063077272P 2020-09-11 2020-09-11
US202163214742P 2021-06-24 2021-06-24
PCT/US2021/049871 WO2022056266A2 (en) 2020-09-11 2021-09-10 Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
MX2023002853A true MX2023002853A (es) 2023-03-31

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US (2) US20230416737A1 (es)
EP (1) EP4211243A2 (es)
JP (1) JP2023541404A (es)
KR (1) KR20230064620A (es)
AU (1) AU2021342155A1 (es)
CA (1) CA3189065A1 (es)
CL (1) CL2023000662A1 (es)
IL (1) IL301187A (es)
MX (1) MX2023002853A (es)
TW (1) TW202227627A (es)
UY (1) UY39417A (es)
WO (1) WO2022056266A2 (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020006169A2 (pt) * 2017-10-05 2020-10-20 Fulcrum Therapeutics, Inc. inibidores de quinase p38 que reduzem dux4 e expressão de gene a jusante para o tratamento de fshd
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
CN112912499A (zh) 2018-08-02 2021-06-04 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF DYSTROPHINOPATHIES
JP2023521042A (ja) 2020-04-02 2023-05-23 ミレキュール インコーポレイテッド 操作されたオリゴヌクレオチドを使用する標的化された阻害
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024011135A2 (en) * 2022-07-06 2024-01-11 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

Family Cites Families (49)

* Cited by examiner, † Cited by third party
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
US5744300A (en) 1993-03-24 1998-04-28 Geron Corporation Methods and reagents for the identification and regulation of senescence-related genes
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US5994076A (en) 1997-05-21 1999-11-30 Clontech Laboratories, Inc. Methods of assaying differential expression
US6352829B1 (en) 1997-05-21 2002-03-05 Clontech Laboratories, Inc. Methods of assaying differential expression
US6489455B2 (en) 1997-05-21 2002-12-03 Clontech Laboratories, Inc. Methods of assaying differential expression
JP2002537828A (ja) 1999-03-10 2002-11-12 フォゲン リミティド 細胞への物質のデリバリー
WO2003035884A2 (de) 2001-10-18 2003-05-01 Heart Biosystems Gmbh Transiente immortalisierung von zellen durch onkogenproteine oder telomerproteine
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7250289B2 (en) 2002-11-20 2007-07-31 Affymetrix, Inc. Methods of genetic analysis of mouse
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7374927B2 (en) 2004-05-03 2008-05-20 Affymetrix, Inc. Methods of analysis of degraded nucleic acid samples
US7361468B2 (en) 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
WO2008022309A2 (en) 2006-08-18 2008-02-21 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
DK2539451T3 (da) 2010-02-24 2016-04-04 Arrowhead Res Corp Sammensætninger til målrettet tilførsel af siRNA
WO2012024535A2 (en) 2010-08-18 2012-02-23 Fred Hutchinson Cancer Research Center Methods for determining the presence or risk of developing facioscapulohumeral dystrophy (fshd)
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
EP2655621B1 (en) 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
MX2014001971A (es) 2011-08-26 2014-03-31 Arrowhead Res Corp Polimeros de poli(vinilester) para suministro de acido nucleico in vivo.
AU2012377385A1 (en) 2012-04-18 2014-01-23 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
WO2014071340A1 (en) 2012-11-05 2014-05-08 University Of Washington Through Its Center For Commercialization Methods and assays for facioscapulohumeral muscular dystrophy
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
WO2016170348A2 (en) 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
DK3302522T3 (da) 2015-06-02 2022-09-26 Academisch Ziekenhuis Leiden Midler og fremgangsmåde til behandling af facioscapulohumeral muskeldystrofi (fshd)
EP3353305A4 (en) 2015-09-25 2019-09-18 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US11345913B2 (en) 2016-04-02 2022-05-31 Research Institute At Nationwide Children's Hospital Modified U6 promoter system for tissue specific expression
KR102426487B1 (ko) 2016-06-06 2022-07-27 애로우헤드 파마슈티컬스 인코포레이티드 5'-시클로-포스포네이트 변형된 뉴클레오티드
CA3074723A1 (en) 2016-09-23 2018-03-29 University Of Massachusetts Silencing of dux4 by recombinant gene editing complexes
JOP20190084B1 (ar) * 2016-11-01 2023-09-17 Arrowhead Pharmaceuticals Inc ربائط إنتجرين ألفا- v بيتا-6 واستخداماتها
CA3099522A1 (en) 2017-09-19 2019-03-28 Children's National Medical Center Gapmers and methods of using the same for treatment of muscular dystrophy
US20200248179A1 (en) * 2017-10-02 2020-08-06 Research Institute At Nationwide Children's Hospital MiRNA Detargeting System for Tissue Specific Interference
BR112020006169A2 (pt) 2017-10-05 2020-10-20 Fulcrum Therapeutics, Inc. inibidores de quinase p38 que reduzem dux4 e expressão de gene a jusante para o tratamento de fshd
US10342786B2 (en) 2017-10-05 2019-07-09 Fulcrum Therapeutics, Inc. P38 kinase inhibitors reduce DUX4 and downstream gene expression for the treatment of FSHD
CN111526880A (zh) * 2017-11-01 2020-08-11 箭头药业股份有限公司 整联蛋白配体及其用途
CA3083526A1 (en) * 2017-12-06 2019-06-13 Andrew John Geall Compositions and methods of treating muscle atrophy and myotonic dystrophy
US10973820B2 (en) 2017-12-13 2021-04-13 Facio Intellectual Property B.V. Compounds for treatment of diseases related to DUX4 expression
WO2019210308A1 (en) * 2018-04-27 2019-10-31 Arrowhead Pharmaceuticals, Inc. Integrin targeting ligands and uses thereof
WO2020028134A1 (en) * 2018-07-30 2020-02-06 Fred Hutchinson Cancer Research Center Methods and compositions for treating cancer
CN112912499A (zh) 2018-08-02 2021-06-04 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
JP2021533199A (ja) * 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 筋強直症ジストロフィーを処置するための筋標的化複合体およびそれらの使用
CA3124963A1 (en) 2018-12-31 2020-07-09 Research Institute At Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
MX2022011499A (es) 2020-03-19 2022-10-07 Avidity Biosciences Inc Composiciones y metodos para tratar la distrofia muscular facioescapulohumeral.
US20230323456A1 (en) 2020-08-31 2023-10-12 Modalis Therapeutics Corporation Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene
TW202227626A (zh) 2020-09-01 2022-07-16 美商奧崔基尼克斯製藥公司 Ｄｕｘ４抑制劑及其使用方法

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US20230416737A1 (en) 2023-12-28
US11845937B2 (en) 2023-12-19
IL301187A (en) 2023-05-01
EP4211243A2 (en) 2023-07-19
KR20230064620A (ko) 2023-05-10
AU2021342155A1 (en) 2023-04-13
WO2022056266A3 (en) 2022-04-28
CL2023000662A1 (es) 2024-01-05
TW202227627A (zh) 2022-07-16
UY39417A (es) 2022-03-31
WO2022056266A8 (en) 2023-04-13
US20230265430A1 (en) 2023-08-24
CA3189065A1 (en) 2022-03-17
JP2023541404A (ja) 2023-10-02
WO2022056266A2 (en) 2022-03-17

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