JP2017533721A5 - - Google Patents
Download PDFInfo
- 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
Links
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)
| 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)
| 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 |
-
2015
- 2015-11-16 AU AU2015346042A patent/AU2015346042A1/en not_active Abandoned
- 2015-11-16 WO PCT/US2015/060938 patent/WO2016077837A1/en not_active Ceased
- 2015-11-16 US US15/525,232 patent/US10822369B2/en active Active
- 2015-11-16 EP EP15859580.1A patent/EP3218483A4/en active Pending
- 2015-11-16 CA CA2964979A patent/CA2964979A1/en not_active Abandoned
- 2015-11-16 JP JP2017525921A patent/JP2017533721A/ja active Pending
-
2020
- 2020-09-15 US US17/021,510 patent/US12030910B2/en active Active
-
2024
- 2024-05-20 US US18/668,732 patent/US20250109164A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2017533721A5 (OSRAM) | ||
| JP2013226147A5 (OSRAM) | ||
| IL300119A (en) | Oligonucleotides for inducing paternal ube3a expression | |
| Grimson et al. | Early origins and evolution of microRNAs and Piwi-interacting RNAs in animals | |
| JP2012029693A5 (OSRAM) | ||
| Chung et al. | MicroRNA and nephropathy: emerging concepts | |
| JP2013516489A5 (OSRAM) | ||
| JP2018512110A5 (OSRAM) | ||
| JP2018512041A5 (OSRAM) | ||
| JP2009219504A5 (OSRAM) | ||
| MX2024005653A (es) | Agentes de acido ribonucleico (arn) de cadena doble modificados. | |
| WO2016094845A3 (en) | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides | |
| AR116102A1 (es) | Compuestos y métodos para la reducción de la expresión de lrrk2 | |
| JP2012510297A5 (OSRAM) | ||
| JP2017505623A5 (OSRAM) | ||
| RU2019124314A (ru) | Сопряженные антисмысловые соединения и их применение | |
| JP2009532392A5 (OSRAM) | ||
| JP2016116520A5 (OSRAM) | ||
| JP2017524348A5 (OSRAM) | ||
| JP2015511821A5 (OSRAM) | ||
| JP2016521753A5 (OSRAM) | ||
| RU2014121304A (ru) | Ингибирование экспрессии вирусных генов | |
| 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 |