JP2017533721A5 - - Google Patents

Download PDF

Info

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
Authority
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
Other languages
English (en)
Japanese (ja)
Other versions
JP2017533721A (ja
Filing date
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

Links

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 (cg-RX-API-DMAC7.html) 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 (cg-RX-API-DMAC7.html)
EP (1) EP3218483A4 (cg-RX-API-DMAC7.html)
JP (1) JP2017533721A (cg-RX-API-DMAC7.html)
AU (1) AU2015346042A1 (cg-RX-API-DMAC7.html)
CA (1) CA2964979A1 (cg-RX-API-DMAC7.html)
WO (1) WO2016077837A1 (cg-RX-API-DMAC7.html)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9580708B2 (en) 2011-09-14 2017-02-28 Rana Therapeutics, Inc. Multimeric oligonucleotides 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
EP2895200B1 (en) 2012-09-14 2019-11-06 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
KR102620328B1 (ko) 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
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
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
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
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
KR102487356B1 (ko) * 2016-07-29 2023-01-12 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 종양 관련 대식세포를 표적화하는 항체 및 이의 용도
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
SMT202400071T1 (it) 2017-08-25 2024-03-13 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di condizioni e malattie
EP3740500A4 (en) * 2018-01-17 2022-01-19 The Florey Institute of Neuroscience and Mental Health COMPOSITIONS AND METHODS TO INCREASE EXPRESSION OF SCN2A
SG11202007644PA (en) 2018-02-14 2020-09-29 Deep Genomics Incorporated Oligonucleotide therapy for wilson disease
US12060558B2 (en) 2018-05-04 2024-08-13 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
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
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
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
WO2023086295A2 (en) * 2021-11-10 2023-05-19 University Of Rochester Antisense oligonucleotides for modifying protein expression
AU2023275606A1 (en) * 2022-05-25 2024-11-28 Yale University Methods of treating, ameliorating and/or preventing polycystic kidney disease and polycystic liver 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
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
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
JP2828642B2 (ja) 1987-06-24 1998-11-25 ハワード フローレイ インスティテュト オブ イクスペリメンタル フィジオロジー アンド メディシン ヌクレオシド誘導体
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
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
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
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
AU1341092A (en) 1991-01-25 1992-08-27 United States Biochemical Corporation 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
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
AU3222793A (en) 1991-11-26 1993-06-28 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
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
AU1039397A (en) 1995-11-22 1997-06-27 Johns Hopkins University, The Ligands to enhance cellular uptake of biomolecules
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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
CN100406065C (zh) 2000-12-01 2008-07-30 细胞工厂治疗公司 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法
US20040110144A1 (en) * 2002-12-09 2004-06-10 Isis Pharmaceuticals Inc. Modulation of EMAP-II expression
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides 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
US7427672B2 (en) 2003-08-28 2008-09-23 Takeshi Imanishi Artificial nucleic acids of n-o bond crosslinkage type
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
CN102908630B (zh) 2006-01-27 2014-11-19 Isis制药公司 6-修饰的双环核酸类似物
EP2066684B1 (en) 2006-05-11 2012-07-18 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
ES2388590T3 (es) 2007-05-30 2012-10-16 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos con puente aminometileno N-sustituido.
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
WO2009126933A2 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
NZ708313A (en) 2012-12-21 2019-08-30 Nz Inst Plant & Food Res Ltd Regulation of gene expression via uorf of gdp-l-galactose phosphorylase (ggp) gene
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
EP3033423A4 (en) 2013-08-16 2017-04-26 Rana Therapeutics Inc. Epigenetic regulators of frataxin
WO2015023937A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Heterochromatin forming non-coding rnas
AU2014306416B2 (en) 2013-08-16 2021-02-25 Translate Bio Ma, Inc. Compositions and methods for modulating RNA
WO2015023941A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
WO2015023939A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin

Similar Documents

Publication Publication Date Title
JP2017533721A5 (cg-RX-API-DMAC7.html)
JP2013226147A5 (cg-RX-API-DMAC7.html)
IL300119A (en) Oligonucleotides for inducing paternal ube3a expression
Grimson et al. Early origins and evolution of microRNAs and Piwi-interacting RNAs in animals
JP2012029693A5 (cg-RX-API-DMAC7.html)
Chung et al. MicroRNA and nephropathy: emerging concepts
JP2013516489A5 (cg-RX-API-DMAC7.html)
Wei Coupling activators for the oligonucleotide synthesis via phosphoramidite approach
JP2018512110A5 (cg-RX-API-DMAC7.html)
JP2018512041A5 (cg-RX-API-DMAC7.html)
JP2009219504A5 (cg-RX-API-DMAC7.html)
JP2017093448A5 (cg-RX-API-DMAC7.html)
MX2024005653A (es) Agentes de acido ribonucleico (arn) de cadena doble modificados.
JP2012510297A5 (cg-RX-API-DMAC7.html)
JP2017505623A5 (cg-RX-API-DMAC7.html)
RU2019124314A (ru) Сопряженные антисмысловые соединения и их применение
JP2009532392A5 (cg-RX-API-DMAC7.html)
JP2016116520A5 (cg-RX-API-DMAC7.html)
JP2017524348A5 (cg-RX-API-DMAC7.html)
JP2015511821A5 (cg-RX-API-DMAC7.html)
HRP20191471T1 (hr) Molekule nukleinske kiseline modificirane saharidom
JP2016521753A5 (cg-RX-API-DMAC7.html)
IL262728B1 (en) Oligonucleotides conjugated to the ligand complex of the glp-1 receptor and their uses
RU2016131028A (ru) Композиции, содержащие асимметричную интерферирующую рнк, которые подвергают сайленсингу k-ras, и способы их применения
El-Shorafa et al. Dysregulation of micro-RNA contributes to the risk of unexplained recurrent pregnancy loss