JP2017533721A5 - - Google Patents

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Publication number
JP2017533721A5
JP2017533721A5 JP2017525921A JP2017525921A JP2017533721A5 JP 2017533721 A5 JP2017533721 A5 JP 2017533721A5 JP 2017525921 A JP2017525921 A JP 2017525921A JP 2017525921 A JP2017525921 A JP 2017525921A JP 2017533721 A5 JP2017533721 A5 JP 2017533721A5
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JP
Japan
Prior art keywords
pharmaceutical composition
antisense compound
composition according
modified
element region
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
Application number
JP2017525921A
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English (en)
Japanese (ja)
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JP2017533721A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2015/060938 external-priority patent/WO2016077837A1/en
Publication of JP2017533721A publication Critical patent/JP2017533721A/ja
Publication of JP2017533721A5 publication Critical patent/JP2017533721A5/ja
Pending legal-status Critical Current

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JP2017525921A 2014-11-14 2015-11-16 タンパク質の調節のための化合物及び方法 Pending JP2017533721A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201462080223P 2014-11-14 2014-11-14
US62/080,223 2014-11-14
US201562139626P 2015-03-27 2015-03-27
US62/139,626 2015-03-27
US201562156139P 2015-05-01 2015-05-01
US62/156,139 2015-05-01
US201562156812P 2015-05-04 2015-05-04
US62/156,812 2015-05-04
US201562233183P 2015-09-25 2015-09-25
US62/233,183 2015-09-25
PCT/US2015/060938 WO2016077837A1 (en) 2014-11-14 2015-11-16 Compounds and methods for the modulation of proteins

Publications (2)

Publication Number Publication Date
JP2017533721A JP2017533721A (ja) 2017-11-16
JP2017533721A5 true JP2017533721A5 (enExample) 2018-12-27

Family

ID=55955201

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017525921A Pending JP2017533721A (ja) 2014-11-14 2015-11-16 タンパク質の調節のための化合物及び方法

Country Status (6)

Country Link
US (3) US10822369B2 (enExample)
EP (1) EP3218483A4 (enExample)
JP (1) JP2017533721A (enExample)
AU (1) AU2015346042A1 (enExample)
CA (1) CA2964979A1 (enExample)
WO (1) WO2016077837A1 (enExample)

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EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CA2873779A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating mecp2 expression
JP2015529469A (ja) 2012-09-14 2015-10-08 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
CA2976576A1 (en) * 2015-02-13 2016-08-18 Translate Bio Ma, Inc. Compositions and methods for modulating rna
US10525076B2 (en) 2015-02-20 2020-01-07 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017087282A1 (en) 2015-11-18 2017-05-26 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
ES2882500T3 (es) 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Oligómeros antisentido para el tratamiento del síndrome de Dravet
JP7219207B2 (ja) * 2016-07-29 2023-02-07 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 腫瘍関連マクロファージを標的化する抗体及びその使用
JP2019535839A (ja) 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
RS65031B1 (sr) 2017-08-25 2024-02-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje stanja i bolesti
EP3740500A4 (en) * 2018-01-17 2022-01-19 The Florey Institute of Neuroscience and Mental Health COMPOSITIONS AND METHODS TO INCREASE EXPRESSION OF SCN2A
KR20200120675A (ko) 2018-02-14 2020-10-21 딥 지노믹스 인코포레이티드 윌슨병을 위한 올리고뉴클레오티드 요법
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス,インク. コレステリルエステル蓄積症の処置のための方法及び組成物
EP3942049A4 (en) 2019-03-20 2023-10-18 President And Fellows Of Harvard College ANTISENSE OLIGONUCLEOTIDE BASED PROGRANULIN AUGMENTATION THERAPY FOR NEURODEGENERATIVE DISEASES
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
TW202208628A (zh) 2020-05-13 2022-03-01 瑞士商赫孚孟拉羅股份公司 靶向顆粒蛋白前體之寡核苷酸促效劑
WO2022098840A1 (en) * 2020-11-05 2022-05-12 The Jackson Laboratory Modified antisense oligonucleotides targeting splicing factors
GB202107553D0 (en) * 2021-05-27 2021-07-14 Univ Oxford Innovation Ltd Method
CN113584038B (zh) * 2021-09-09 2023-11-14 深圳雅济科技有限公司 一种治疗视网膜疾病的反义寡核苷酸组合及其应用
EP4430187A2 (en) * 2021-11-10 2024-09-18 University of Rochester Antisense oligonucleotides for modifying protein expression
CA3255304A1 (en) * 2022-05-25 2023-11-30 Univ Yale METHODS FOR TREATMENT, ALLEVIATION AND/OR PREVENTION OF POLYCYSTIC RENAL DISEASE AND POLYCYSTIC HEPATIC DISEASE
EP4581143A1 (en) * 2022-08-29 2025-07-09 University of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
WO2025043278A1 (en) * 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions
WO2025165891A1 (en) 2024-01-29 2025-08-07 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

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AU1341092A (en) 1991-01-25 1992-08-27 United States Biochemical Corporation Regulation of nucleic acid translation
US6824976B1 (en) 1993-04-02 2004-11-30 Rigel Pharmaceuticals, Inc. Method for selective inactivation of viral replication
US20040110144A1 (en) * 2002-12-09 2004-06-10 Isis Pharmaceuticals Inc. Modulation of EMAP-II expression
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
US7718625B2 (en) 2005-01-27 2010-05-18 University Of South Florida Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof
CN104884622B (zh) 2012-12-21 2018-11-13 新西兰植物和食品研究院有限公司 基因表达的调控
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
EP3033425A4 (en) 2013-08-16 2017-07-26 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
US20160201063A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Epigenetic regulators of frataxin
CA2921459A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
AU2014306416B2 (en) 2013-08-16 2021-02-25 Translate Bio Ma, Inc. Compositions and methods for modulating RNA
CA2921457A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Heterochromatin forming non-coding rnas

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