JP2016526529A5 - - Google Patents
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- JP2016526529A5 JP2016526529A5 JP2015560881A JP2015560881A JP2016526529A5 JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5 JP 2015560881 A JP2015560881 A JP 2015560881A JP 2015560881 A JP2015560881 A JP 2015560881A JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- strand
- independently selected
- nucleotide analogs
- natural
- 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.)
- Granted
Links
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361835634P | 2013-06-16 | 2013-06-16 | |
| US61/835,634 | 2013-06-16 | ||
| US201361836672P | 2013-06-19 | 2013-06-19 | |
| US61/836,672 | 2013-06-19 | ||
| PCT/JP2014/003208 WO2014203518A1 (en) | 2013-06-16 | 2014-06-16 | Double-stranded antisense nucleic acid with exon-skipping effect |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016526529A JP2016526529A (ja) | 2016-09-05 |
| JP2016526529A5 true JP2016526529A5 (enExample) | 2017-08-17 |
| JP6604544B2 JP6604544B2 (ja) | 2019-11-13 |
Family
ID=52104269
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015560881A Active JP6604544B2 (ja) | 2013-06-16 | 2014-06-16 | エクソンスキッピング効果を有する二本鎖アンチセンス核酸 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US10190117B2 (enExample) |
| EP (1) | EP3010514B1 (enExample) |
| JP (1) | JP6604544B2 (enExample) |
| CN (1) | CN105324119A (enExample) |
| AU (1) | AU2014282666A1 (enExample) |
| CA (1) | CA2915443A1 (enExample) |
| WO (1) | WO2014203518A1 (enExample) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| CN104583402A (zh) | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节mecp2表达的组合物和方法 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Smn遺伝子ファミリー発現を調節するための組成物及び方法 |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| EP3052632A4 (en) | 2013-10-04 | 2017-03-29 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| AU2015367378A1 (en) | 2014-12-17 | 2017-06-15 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| EP3353306A4 (en) | 2015-09-25 | 2019-06-05 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| CN109475568B (zh) * | 2016-05-06 | 2026-07-17 | Ionis制药公司 | Glp-1受体配体部分缀合的寡核苷酸及其用途 |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| AU2017291960B2 (en) * | 2016-07-05 | 2024-03-14 | Biomarin Technologies B.V. | Pre-mRNA splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| WO2018041973A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| US11433089B2 (en) | 2016-09-23 | 2022-09-06 | National University Corporation Tokyo Medical And Dental University | Blood-brain barrier permeable heteroduplex nucleic acid |
| US11260134B2 (en) | 2016-09-29 | 2022-03-01 | National University Corporation Tokyo Medical And Dental University | Double-stranded nucleic acid complex having overhang |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| WO2018237245A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS |
| JP6974872B2 (ja) | 2017-06-30 | 2021-12-01 | 国立大学法人 東京医科歯科大学 | ヘテロ二本鎖型antimiR |
| AU2018364621A1 (en) * | 2017-11-08 | 2020-06-18 | Astrazeneca Ab | GLP-1 receptor ligand moiety conjugated oligonucleotides and uses thereof |
| US11674141B2 (en) | 2018-02-28 | 2023-06-13 | National University Corporation Tokyo Medical And Dental University | Ischemic-lesion-site-specific gene therapy |
| EP3766972B1 (en) * | 2018-03-14 | 2025-04-23 | Institute of Science Tokyo | Nucleic acid complex |
| US11851654B2 (en) | 2018-03-19 | 2023-12-26 | National University Corporation Tokyo Medical And Dental University | Nucleic acid with reduced toxicity |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| BR112021002440A2 (pt) | 2018-08-10 | 2021-05-04 | University Of Massachusetts | oligonucleotídeos modificados visando snps |
| WO2020041769A1 (en) | 2018-08-23 | 2020-02-27 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113614232A (zh) * | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| CN113677374A (zh) * | 2019-04-08 | 2021-11-19 | 国立大学法人东京医科齿科大学 | 肌疾病治疗用药物组合物 |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| EP4043565A4 (en) * | 2019-10-11 | 2024-01-24 | National University Corporation Tokyo Medical and Dental University | MODIFIED HETERONUCLEIC ACID |
| EP4090744A4 (en) * | 2020-01-17 | 2024-04-10 | Anastasia Khvorova | UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS |
| EP4123023A4 (en) * | 2020-03-16 | 2024-07-17 | National University Corporation Tokyo Medical and Dental University | Heteronucleic acid containing morpholino nucleic acid |
| CA3174079A1 (en) | 2020-05-26 | 2021-12-02 | Anastasia Khvorova | Synthetic oligonucleotides having regions of block and cluster modifications |
| AU2021382146A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| EP4389891A4 (en) * | 2021-08-19 | 2025-10-29 | Univ Nat Corp Tokyo Medical & Dental | MODIFIED HETERONUCLEIC ACID CONTAINING MORPHOLINO NUCLEIC ACID |
| WO2023105898A1 (ja) * | 2021-12-07 | 2023-06-15 | リードファーマ株式会社 | アンチセンスオリゴヌクレオチド複合体 |
| CN115806984B (zh) * | 2022-10-18 | 2023-10-10 | 昆明理工大学 | 环状rna及载体和载体的应用 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| EP4782540A1 (en) | 2023-09-19 | 2026-07-29 | National University Corporation Tokai National Higher Education And Research System | Single-stranded polynucleotide |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH09110894A (ja) * | 1995-10-17 | 1997-04-28 | Soyaku Gijutsu Kenkyusho:Kk | ハイブリッドdna/rnaオリゴヌクレオチド及び抗ウイルス剤 |
| JP3781879B2 (ja) | 1996-11-18 | 2006-05-31 | 武 今西 | 新規ヌクレオチド類縁体 |
| ES2192672T3 (es) | 1996-11-18 | 2003-10-16 | Takeshi Imanishi | Nuevos analogos de nucleotidos. |
| 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 |
| EP1015469B2 (en) | 1997-09-12 | 2015-11-18 | Exiqon A/S | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US20010024808A1 (en) * | 1998-09-10 | 2001-09-27 | Millennium Pharmaceuticals, Inc., A Delaware Corporation | Leptin induced genes |
| US6933105B2 (en) * | 2000-01-31 | 2005-08-23 | Millennium Pharmaceuticals, Inc. | Resistance sequences and uses thereof |
| EP1294899B3 (en) * | 2000-03-31 | 2011-07-06 | Millennium Pharmaceuticals, Inc. | 16836, a human phospholipase c family member and uses thereof |
| CA2504694C (en) * | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| AU2003294281B2 (en) | 2002-11-13 | 2010-05-20 | Kastle Therapeutics, Llc | Antisense modulation of apolipoprotein B expression |
| DK1661905T3 (da) | 2003-08-28 | 2012-07-23 | Takeshi Imanishi | Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype |
| WO2005113571A2 (en) * | 2004-05-13 | 2005-12-01 | The University Of North Carolina At Chapel Hill | Methods for the delivery of oligomeric compounds |
| WO2006059507A1 (ja) * | 2004-11-30 | 2006-06-08 | Sankyo Company, Limited | 11β-HSD1アンチセンス化合物 |
| ES2556128T3 (es) * | 2006-03-23 | 2016-01-13 | Roche Innovation Center Copenhagen A/S | ARN interfiriente pequeño internamente segmentado |
| EP2505646A1 (en) | 2006-05-05 | 2012-10-03 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of CRP |
| WO2008029619A1 (fr) | 2006-09-07 | 2008-03-13 | Daiichi Sankyo Company, Limited | Oligonucléotide antisens ena ayant une action spécifique de la séquence |
| EP2076597A2 (en) | 2006-10-09 | 2009-07-08 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of pcsk9 |
| US8470791B2 (en) * | 2007-03-22 | 2013-06-25 | Santaris Pharma A/S | RNA antagonist compounds for the inhibition of Apo-B100 expression |
| US8399248B2 (en) * | 2007-05-03 | 2013-03-19 | Merck Sharp & Dohme Corp. | Methods of using MIR34 as a biomarker for TP53 functional status |
| DE102007021870A1 (de) | 2007-05-10 | 2008-11-20 | Clariant International Limited | Wässrige Pigmentpräparationen |
| US20110054011A1 (en) | 2007-08-30 | 2011-03-03 | Mccullagh Keith | RNA Antagonist Compounds for the Modulation of FABP4/AP2 |
| WO2010093788A2 (en) * | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| US20130190383A1 (en) * | 2010-04-26 | 2013-07-25 | Marina Biotech, Inc. | Nucleic acid compounds with conformationally restricted monomers and uses thereof |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| AU2012246822B2 (en) * | 2011-04-22 | 2017-05-25 | Biomarin Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1) |
| CN104126010B (zh) * | 2011-12-16 | 2021-04-06 | 国立大学法人东京医科齿科大学 | 嵌合的双链核酸 |
-
2014
- 2014-06-16 CN CN201480034378.1A patent/CN105324119A/zh active Pending
- 2014-06-16 WO PCT/JP2014/003208 patent/WO2014203518A1/en not_active Ceased
- 2014-06-16 JP JP2015560881A patent/JP6604544B2/ja active Active
- 2014-06-16 EP EP14814254.0A patent/EP3010514B1/en active Active
- 2014-06-16 US US14/898,630 patent/US10190117B2/en active Active
- 2014-06-16 CA CA2915443A patent/CA2915443A1/en not_active Abandoned
- 2014-06-16 AU AU2014282666A patent/AU2014282666A1/en not_active Abandoned
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