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
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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 (OSRAM) 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 (OSRAM)
EP (1) EP3218483A4 (OSRAM)
JP (1) JP2017533721A (OSRAM)
AU (1) AU2015346042A1 (OSRAM)
CA (1) CA2964979A1 (OSRAM)
WO (1) WO2016077837A1 (OSRAM)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016219052B2 (en) * 2015-02-13 2022-06-02 Translate Bio Ma, Inc. Compositions and methods for modulating RNA
US10758558B2 (en) 2015-02-13 2020-09-01 Translate Bio Ma, 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
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
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
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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
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
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US11713463B2 (en) 2018-01-17 2023-08-01 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for increasing expression of SCN2A
AU2019222767A1 (en) 2018-02-14 2020-08-27 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
EP3788169A4 (en) 2018-05-04 2022-08-10 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2020191212A1 (en) * 2019-03-20 2020-09-24 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
MX2022014151A (es) 2020-05-11 2022-11-30 Stoke Therapeutics Inc Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.
JP2023526267A (ja) 2020-05-13 2023-06-21 エフ. ホフマン-ラ ロシュ アーゲー プログラニュリン標的オリゴヌクレオチドアゴニスト
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
WO2023086295A2 (en) * 2021-11-10 2023-05-19 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
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
EP4671372A2 (en) 2024-01-29 2025-12-31 Arnatar Therapeutics, Inc Translation-enhancing nucleic acid compounds: Upward regulation 1 coupled with ASO (ACT-UP1) and their uses

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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
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
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. 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
IE920229A1 (en) 1991-01-25 1992-07-29 Us Biochemical Corp Regulation of nucleic acid translation
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
JP3739785B2 (ja) 1991-11-26 2006-01-25 アイシス ファーマシューティカルズ,インコーポレイティド 修飾されたピリミジンを含有するオリゴマーを使用する増強された三重らせんおよび二重らせんの成形
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals 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
JP2000501414A (ja) 1995-11-22 2000-02-08 ザ・ジョンズ・ホプキンス・ユニバーシティー 生体分子の細胞取り込みを高めるリガンド
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
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
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
US6906182B2 (en) 2000-12-01 2005-06-14 Cell Works Therapeutics, Inc. Conjugates of glycosylated/galactosylated peptide, bifunctional linker, and nucleotidic monomers/polymers, and related compositions and method of use
US20040110144A1 (en) * 2002-12-09 2004-06-10 Isis Pharmaceuticals Inc. Modulation of EMAP-II expression
WO2004044136A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2’-modified nucleosides for use in gene modulation
AU2003291753B2 (en) 2002-11-05 2010-07-08 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
ATE555118T1 (de) 2003-08-28 2012-05-15 Takeshi Imanishi Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung
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
JP5342881B2 (ja) 2006-01-27 2013-11-13 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 6−修飾された二環式核酸類似体
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
WO2008101157A1 (en) 2007-02-15 2008-08-21 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
EP2173760B2 (en) 2007-06-08 2015-11-04 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
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
JP2013541334A (ja) 2010-09-15 2013-11-14 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 修飾されたiRNA剤
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
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
CN105658797A (zh) 2013-08-16 2016-06-08 Rana医疗有限公司 用于调节rna的组合物和方法
US20160201063A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Epigenetic regulators of frataxin
WO2015023939A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
CA2921457A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Heterochromatin forming non-coding rnas
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド

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