JP2023521042A5 - - Google Patents

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Publication number
JP2023521042A5
JP2023521042A5 JP2022560272A JP2022560272A JP2023521042A5 JP 2023521042 A5 JP2023521042 A5 JP 2023521042A5 JP 2022560272 A JP2022560272 A JP 2022560272A JP 2022560272 A JP2022560272 A JP 2022560272A JP 2023521042 A5 JP2023521042 A5 JP 2023521042A5
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JP
Japan
Prior art keywords
sequence
seq
oligonucleotide
salt
manipulated
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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
Application number
JP2022560272A
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English (en)
Japanese (ja)
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JP2023521042A (ja
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Priority claimed from PCT/US2021/025639 external-priority patent/WO2021203043A2/en
Publication of JP2023521042A publication Critical patent/JP2023521042A/ja
Publication of JP2023521042A5 publication Critical patent/JP2023521042A5/ja
Pending legal-status Critical Current

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JP2022560272A 2020-04-02 2021-04-02 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 Pending JP2023521042A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063004045P 2020-04-02 2020-04-02
US63/004,045 2020-04-02
PCT/US2021/025639 WO2021203043A2 (en) 2020-04-02 2021-04-02 Targeted inhibition using engineered oligonucleotides

Publications (2)

Publication Number Publication Date
JP2023521042A JP2023521042A (ja) 2023-05-23
JP2023521042A5 true JP2023521042A5 (enExample) 2024-04-15

Family

ID=77927597

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022560272A Pending JP2023521042A (ja) 2020-04-02 2021-04-02 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

Country Status (12)

Country Link
US (5) US20230132602A1 (enExample)
EP (1) EP4127175A4 (enExample)
JP (1) JP2023521042A (enExample)
KR (1) KR20230023612A (enExample)
CN (1) CN116096890A (enExample)
AU (1) AU2021246530A1 (enExample)
BR (1) BR112022019889A2 (enExample)
CA (1) CA3174286A1 (enExample)
CO (1) CO2022015141A2 (enExample)
IL (1) IL296957A (enExample)
MX (1) MX2022012265A (enExample)
WO (1) WO2021203043A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117677698A (zh) 2021-07-14 2024-03-08 米雷楚来股份有限公司 用于神经肌肉障碍的寡核苷酸及其组合物
KR20240125036A (ko) 2021-12-23 2024-08-19 마이레큘, 인크. 폴리뉴클레오타이드의 전달을 위한 조성물
KR20230102595A (ko) * 2021-12-30 2023-07-07 주식회사 이노제닉스 포스파티딜이노시톨 3-키나아제 촉매 소단위 알파 유전자의 3'비번역 부위에 특이적으로 결합하는 핵산 절편 및 그 응용
US20230407302A1 (en) * 2022-02-04 2023-12-21 New York University Novel efficacious microrna-30c analogs reduce apolipoprotein b secretion in human liver cells
CN117402251B (zh) * 2023-12-15 2024-02-23 中国医学科学院基础医学研究所 一种抗小g蛋白rbj的抗体及其应用

Family Cites Families (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
EP1504126B1 (en) 2002-05-03 2014-02-26 Duke University A method of regulating gene expression
US8106180B2 (en) * 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
WO2005017145A1 (ja) 2003-08-13 2005-02-24 Japan Biological Informatics Consortium 機能性rnaが制御する被制御遺伝子の同定・予測方法及びその利用方法
US7795419B2 (en) * 2004-05-26 2010-09-14 Rosetta Genomics Ltd. Viral and viral associated miRNAs and uses thereof
US20070099196A1 (en) 2004-12-29 2007-05-03 Sakari Kauppinen Novel oligonucleotide compositions and probe sequences useful for detection and analysis of micrornas and their target mRNAs
JP2006248978A (ja) 2005-03-10 2006-09-21 Mebiopharm Co Ltd 新規なリポソーム製剤
WO2006133022A2 (en) 2005-06-03 2006-12-14 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
ITMI20051248A1 (it) 2005-07-01 2007-01-02 Vimar Spa Modem per bus per impianti elettrici civili ed industriali
CA2621441C (en) 2005-09-12 2014-05-20 The Ohio State University Research Foundation Compositions and methods for the diagnosis and therapy of bcl2-associated cancers
US8603991B2 (en) 2005-11-18 2013-12-10 Gradalis, Inc. Individualized cancer therapy
CN101448942B (zh) 2005-12-12 2014-04-23 北卡罗来纳大学查珀尔希尔分校 调节肌细胞增殖和分化的microrna
EP2484782B1 (en) 2006-01-05 2015-11-18 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers
WO2007143752A2 (en) 2006-06-09 2007-12-13 The Regents Of The University Of California Targets in breast cancer for prognosis or therapy
WO2007149521A2 (en) 2006-06-20 2007-12-27 The Johns Hopkins University Nucleotide motifs providing localization elements and methods of use
WO2008069940A2 (en) 2006-12-06 2008-06-12 Albert Einstein College Of Medicine Of Yeshiva University Multi-microrna methods and compositions
WO2008088858A2 (en) 2007-01-17 2008-07-24 The Johns Hopkins University Compositions and methods featuring micronas for treating neoplasia
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
NZ583025A (en) 2007-07-31 2012-06-29 Univ Texas Micro-rnas that control myosin expression and myofiber identity
WO2009075817A1 (en) 2007-12-06 2009-06-18 Minerva Biotechnologies Corporation Method for treating cancer using interference rna
JP2010094122A (ja) 2008-06-12 2010-04-30 Keio Gijuku バイオマーカーとしてのマイクロrnaを用いた頭頸部腫瘍の診断・治療選択
WO2010065630A2 (en) 2008-12-03 2010-06-10 The Johns Hopkins University Compositions and methods for treating hepatic neoplasia
AU2010258875A1 (en) 2009-06-08 2012-01-19 Miragen Therapeutics Chemical modification motifs for miRNA inhibitors and mimetics
US8796238B2 (en) 2009-09-17 2014-08-05 Sigma-Aldrich Co. Llc Short RNA mimetics
US20120302626A1 (en) * 2009-12-04 2012-11-29 Sandeep Dave Microrna and use thereof in identification of b cell malignancies
US8768630B2 (en) * 2010-02-19 2014-07-01 The Regents Of The University Of Michigan miRNA target prediction
MX373121B (es) 2010-05-14 2020-04-30 Dana Farber Cancer Inst Inc Composiciones y metodos para el tratamiento de leucemia.
EP2426203B1 (en) 2010-09-02 2016-07-13 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
JP2014506789A (ja) 2011-02-03 2014-03-20 マーナ セラピューティクス インコーポレイテッド miR−124の合成模倣体
WO2012106591A1 (en) 2011-02-03 2012-08-09 Mirna Therapeutics, Inc. Synthetic mimics of mir-34
AU2012286994B2 (en) * 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
US20140242093A1 (en) 2011-07-29 2014-08-28 Fred Hutchinson Cancer Research Center Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject
CA2850223A1 (en) 2011-10-06 2013-04-11 Eva Van Rooij Control of whole body energy homeostasis by microrna regulation
US8846633B2 (en) 2011-11-07 2014-09-30 Taipei Veterans General Hospital Method for inhibiting cancer stem cell like properties and chemoradioresistant properties of cancer or tumor cells with microRNA145
WO2013120038A2 (en) 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
WO2013120497A1 (en) * 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
CN112007045B (zh) * 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
WO2014179765A2 (en) * 2013-05-02 2014-11-06 Thomas Jefferson University Novel human mirnas for use in diagnosis, prognosis and therapy of human conditions and diseases
US9518300B2 (en) 2014-04-14 2016-12-13 Dana-Farber Cancer Institute, Inc. Composition and method for treating a hematological malignancy
ES2744186T3 (es) 2014-04-25 2020-02-24 Childrens Medical Center Composiciones y métodos para tratar las hemoglobinopatías
WO2016115490A1 (en) * 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
IL318703A (en) * 2015-06-29 2025-03-01 Caris Science Inc Therapeutic oligonucleotides
US20170009230A1 (en) 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof
DK3426781T3 (da) * 2016-03-07 2024-09-30 Us Health Mikro-rna'er og fremgangsmåder til anvendelse deraf
WO2017173411A1 (en) * 2016-04-02 2017-10-05 Research Institute At Nation Wide Children's Hospital Modified u6 promoter system for tissue specific expression
CA3099522C (en) * 2017-09-19 2024-10-08 The Governors Of The University Of Alberta GAPMERES AND METHODS OF USING THESE FOR THE TREATMENT OF MUSCULAR DYSTROPHY
EP3668997A4 (en) 2017-10-02 2021-05-19 Research Institute at Nationwide Children's Hospital SYSTEM FOR MIRNA DETARGETING FOR TISSUE-SPECIFIC INTERFERENCE
US10676744B2 (en) * 2017-10-13 2020-06-09 University Of South Carolina Modulation of adipose tissue macrophage phenotype by use of MicroRNA-30
US11268305B2 (en) 2018-05-17 2022-03-08 Kiekert Ag Latching system for a motor vehicle
WO2020028134A1 (en) * 2018-07-30 2020-02-06 Fred Hutchinson Cancer Research Center Methods and compositions for treating cancer
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
IL326747A (en) * 2018-11-02 2026-04-01 Biomarin Tech Bv Bispecific antisense oligonucleotides for dystrophin exon skipping
BR112021018793A2 (pt) 2019-03-29 2021-11-23 Mitsubishi Tanabe Pharma Corp Oligonucleotídeo modificado, composição farmacêutica compreendendo o mesmo composto para modular a expressão de dux4 e uso
US20220364086A1 (en) 2019-07-12 2022-11-17 Daiichi Sankyo Company, Limited ANTISENSE OLIGONUCLEOTIDE CAPABLE OF ALTERING SPLICING OF DUX4 pre-mRNA
US20230088865A1 (en) 2020-01-10 2023-03-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2021211325A1 (en) 2020-04-17 2021-10-21 Board Of Regents Of The Nevada System Of Higher Education, On Behalf Of The University Of Nevada, Reno Crispr-inhibition for facioscapulohumeral muscular dystrophy
GB202009618D0 (en) 2020-06-24 2020-08-05 Ucl Business Ltd Therapeutics for the treatment of FSHD
CN116348139A (zh) 2020-07-23 2023-06-27 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
AU2021313058A1 (en) 2020-07-23 2023-03-30 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
JP7736329B2 (ja) 2020-08-31 2025-09-09 株式会社モダリス Dux4遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法
WO2022043566A1 (en) 2020-08-31 2022-03-03 Merck Patent Gmbh Method and device for manufacturing of solid oral dosage forms
CA3189861A1 (en) 2020-09-01 2022-03-10 Sean Christopher DAUGHERTY Dux4 inhibitors and methods of use thereof
JP2023541427A (ja) 2020-09-11 2023-10-02 アローヘッド ファーマシューティカルズ インコーポレイテッド 骨格筋送達プラットフォーム及び使用方法
AU2021342155A1 (en) 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
JP2022055361A (ja) 2020-09-28 2022-04-07 田辺三菱製薬株式会社 Dux4の発現を調節するための医薬組成物

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