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
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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
JP2017525921A
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English (en)
Japanese (ja)
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JP2017533721A (ja
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Publication date
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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

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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)

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

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2756080B1 (en) 2011-09-14 2019-02-20 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
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
CA2884608C (en) 2012-09-14 2025-05-20 Translate Bio Ma, Inc. MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
JP6867945B2 (ja) 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
US20180055869A1 (en) * 2015-02-13 2018-03-01 Translate Bio Ma, Inc. Compositions and methods for modulating rna
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
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
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
EP3377629B1 (en) 2015-11-18 2023-07-05 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
US11083745B2 (en) 2015-12-14 2021-08-10 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome
US11186634B2 (en) 2016-07-29 2021-11-30 INSERM (Institut National de la Santé et de la Recherche Médicale) Antibodies targeting tumor associated macrophages and uses thereof
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
WO2019143831A1 (en) * 2018-01-17 2019-07-25 Rogcon U.R., Inc. Compositions and methods for increasing expression of scn2a
MX2020008533A (es) 2018-02-14 2020-11-06 Deep Genomics Incorporated Terapia con oligonucleotidos para la enfermedad de wilson.
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US20220154182A1 (en) * 2019-03-20 2022-05-19 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
WO2021229036A1 (en) * 2020-05-13 2021-11-18 F. Hoffmann-La Roche Ag Oligonucleotide agonists targeting progranulin
EP4240857A4 (en) * 2020-11-05 2024-12-25 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
CN119546758A (zh) * 2022-05-25 2025-02-28 耶鲁大学 治疗、改善和/或预防多囊肾病和多囊肝病的方法
WO2024050261A1 (en) * 2022-08-29 2024-03-07 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
EP4665854A1 (en) 2024-01-29 2025-12-24 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

Family Cites Families (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
CA1340032C (en) 1987-06-24 1998-09-08 Jim Haralambidis Lucleoside derivatives
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
CA2088258C (en) 1990-07-27 2004-09-14 Phillip Dan Cook Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
PT100052A (pt) 1991-01-25 1993-03-31 Us Biochemical Corp Regulacao da translacao do acido nucleico
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
WO1993010820A1 (en) 1991-11-26 1993-06-10 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
EP0673559A1 (en) 1992-12-14 1995-09-27 Honeywell Inc. Motor system with individually controlled redundant windings
US6824976B1 (en) 1993-04-02 2004-11-30 Rigel Pharmaceuticals, Inc. Method for selective inactivation of viral replication
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5994517A (en) 1995-11-22 1999-11-30 Paul O. P. Ts'o Ligands to enhance cellular uptake of biomolecules
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
DE60033927T2 (de) 1999-05-04 2007-11-29 Santaris Pharma A/S L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
JP2004536027A (ja) 2000-12-01 2004-12-02 ジョーンズ・ホプキンス・ユニーバーシティー グリコシル化/ガラクトシル化ペプチドの接合体、二官能性リンカー、およびヌクレオチドのモノマー/ポリマー、および関連する組成物および使用方法
US20040110144A1 (en) * 2002-12-09 2004-06-10 Isis Pharmaceuticals Inc. Modulation of EMAP-II expression
AU2003290596B2 (en) 2002-11-05 2011-05-12 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ, インコーポレーテッド 4’−チオヌクレオシドおよびオリゴマー化合物
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
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
PL2314594T3 (pl) 2006-01-27 2014-12-31 Isis Pharmaceuticals Inc Zmodyfikowane w pozycji 6 analogi bicykliczne kwasów nukleinowych
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US8278426B2 (en) 2007-06-08 2012-10-02 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP3156077B1 (en) 2007-12-04 2022-03-09 Arbutus Biopharma Corporation Targeting lipids
JP5788312B2 (ja) 2008-04-11 2015-09-30 アルニラム ファーマスーティカルズ インコーポレイテッドAlnylam Pharmaceuticals, Inc. 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
WO2014097226A2 (en) 2012-12-21 2014-06-26 The New Zealand Institute For Plant And Food Research Limited Regulation of gene expression
US10590412B2 (en) * 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
CN105682687A (zh) 2013-08-16 2016-06-15 Rana医疗有限公司 异染色质形成性非编码rna
EP3033422A4 (en) 2013-08-16 2017-08-02 Rana Therapeutics Inc. Oligonucleotides targeting euchromatin regions of genes
SG11201600987TA (en) 2013-08-16 2016-03-30 Rana Therapeutics Inc Compositions and methods for modulating rna
WO2015023938A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Epigenetic regulators of frataxin
US20160201064A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin

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