JP2016526529A5 - - Google Patents
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- Publication number
- 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
- 125000003729 nucleotide group Chemical group 0.000 description 9
- 239000002773 nucleotide Substances 0.000 description 7
- 108091034117 Oligonucleotide Proteins 0.000 description 1
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 (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| JP6128529B2 (ja) | 2011-07-19 | 2017-05-17 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | 官能化核酸の合成のための方法 |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| US10059941B2 (en) | 2012-05-16 | 2018-08-28 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| US9598458B2 (en) | 2012-07-13 | 2017-03-21 | Wave Life Sciences Japan, Inc. | Asymmetric auxiliary group |
| JP2016534035A (ja) | 2013-10-04 | 2016-11-04 | ラナ セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症を治療するための組成物及び方法 |
| JP6718872B2 (ja) | 2014-12-17 | 2020-07-08 | プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップProQR Therapeutics II B.V. | 標的化rna編集 |
| 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 |
| US11116843B2 (en) | 2015-09-25 | 2021-09-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| HK1258758A1 (zh) | 2016-01-31 | 2019-11-22 | University Of Massachusetts | 分支的寡核苷酸 |
| IL262728B2 (en) * | 2016-05-06 | 2023-10-01 | Astrazeneca Ab | Oligonucleotides conjugated to the GLP-1 receptor ligand complex and their uses |
| JP7074345B2 (ja) | 2016-06-22 | 2022-05-24 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 一本鎖rna編集オリゴヌクレオチド |
| JP2019522972A (ja) * | 2016-07-05 | 2019-08-22 | バイオマリン テクノロジーズ ベー.フェー. | 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレmRNAスプライススイッチング又は調節オリゴヌクレオチド |
| EP3496758A4 (en) | 2016-08-12 | 2020-11-11 | University of Massachusetts | CONJUGATE OLIGONUCLEOTIDES |
| IL264923B2 (en) | 2016-09-01 | 2025-07-01 | Proqr Therapeutics Ii Bv | Single-stranded RNA-editing oligonucleotides with chemical modifications |
| JP7043078B2 (ja) | 2016-09-23 | 2022-03-29 | 国立大学法人 東京医科歯科大学 | 血液脳関門通過型ヘテロ2本鎖核酸 |
| WO2018062510A1 (ja) | 2016-09-29 | 2018-04-05 | 国立大学法人東京医科歯科大学 | オーバーハングを有する二本鎖核酸複合体 |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | 2テイル自己デリバリー型siRNAおよび関連方法 |
| 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 |
| EP3707156A4 (en) * | 2017-11-08 | 2021-08-04 | Ionis Pharmaceuticals, Inc. | GLP-1 RECEPTOR LIGAND PARTLY 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 |
| WO2019177061A1 (ja) * | 2018-03-14 | 2019-09-19 | 国立大学法人東京医科歯科大学 | 核酸複合体 |
| EP3769769A4 (en) | 2018-03-19 | 2022-05-04 | 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 |
| EP3833763A4 (en) | 2018-08-10 | 2023-07-19 | University of Massachusetts | MODIFIED OLIGONUCLEOTIDES FOR TARGETING 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 |
| JP7687682B2 (ja) * | 2019-04-08 | 2025-06-03 | 国立大学法人東京科学大学 | 筋疾患治療用医薬組成物 |
| US12024706B2 (en) | 2019-08-09 | 2024-07-02 | University Of Massachusetts | Modified oligonucleotides targeting SNPs |
| 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 |
| WO2021187392A1 (ja) * | 2020-03-16 | 2021-09-23 | 国立大学法人東京医科歯科大学 | モルホリノ核酸を含むヘテロ核酸 |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| WO2022106695A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
| US11702659B2 (en) | 2021-06-23 | 2023-07-18 | University Of Massachusetts | Optimized anti-FLT1 oligonucleotide compounds for 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 |
| JP7412668B2 (ja) * | 2021-12-07 | 2024-01-15 | 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 |
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 |
| AU4966997A (en) | 1996-11-18 | 1998-06-10 | Takeshi Imanishi | Novel nucleotide analogues |
| JP3781879B2 (ja) | 1996-11-18 | 2006-05-31 | 武 今西 | 新規ヌクレオチド類縁体 |
| 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 |
| EP2253639A1 (en) | 1997-09-12 | 2010-11-24 | 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 |
| AU2001233176A1 (en) * | 2000-01-31 | 2001-08-07 | Shengfang Jin | Resistance sequences and uses thereof |
| ATE433489T1 (de) * | 2000-03-31 | 2009-06-15 | Millennium Pharm Inc | 16836, ein mitglied der humanen phospholipase c familie und seine verwendungen |
| WO2004041889A2 (en) * | 2002-11-05 | 2004-05-21 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| EP2336318B1 (en) | 2002-11-13 | 2013-04-24 | Genzyme Corporation | Antisense modulation of apolipoprotein b expression |
| ES2382807T3 (es) | 2003-08-28 | 2012-06-13 | Takeshi Imanishi | Nuevos ácidos nucleicos artificiales del tipo de enlace N-O con reticulación |
| US20080199960A1 (en) | 2004-05-13 | 2008-08-21 | Juliano Rudolph L | 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 |
| CA2644347C (en) * | 2006-03-23 | 2017-05-30 | Santaris Pharma A/S | Small internally segmented interfering rna |
| ATE514777T1 (de) | 2006-05-05 | 2011-07-15 | Isis Pharmaceuticals Inc | Verbindungen und verfahren zur modulation von genexpression |
| WO2008029619A1 (fr) | 2006-09-07 | 2008-03-13 | Daiichi Sankyo Company, Limited | Oligonucléotide antisens ena ayant une action spécifique de la séquence |
| ES2603379T3 (es) | 2006-10-09 | 2017-02-27 | Roche Innovation Center Copenhagen A/S | Compuestos antagonistas de ARN para la modulación de PCSK9 |
| US8470791B2 (en) * | 2007-03-22 | 2013-06-25 | Santaris Pharma A/S | RNA antagonist compounds for the inhibition of Apo-B100 expression |
| WO2008137862A2 (en) * | 2007-05-03 | 2008-11-13 | Rosetta Inpharmatics Llc | Methods of using mir34 as a biomarker for tp53 functional status |
| DE102007021870A1 (de) | 2007-05-10 | 2008-11-20 | Clariant International Limited | Wässrige Pigmentpräparationen |
| WO2009027527A2 (en) | 2007-08-30 | 2009-03-05 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of fabp4/ap2 |
| US20100249214A1 (en) * | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
| EP2563922A1 (en) | 2010-04-26 | 2013-03-06 | 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 |
| EP2699269A1 (en) * | 2011-04-22 | 2014-02-26 | Prosensa Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1) |
| CN112386605A (zh) * | 2011-12-16 | 2021-02-23 | 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 US US14/898,630 patent/US10190117B2/en active Active
- 2014-06-16 AU AU2014282666A patent/AU2014282666A1/en not_active Abandoned
- 2014-06-16 EP EP14814254.0A patent/EP3010514B1/en active Active
- 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 CA CA2915443A patent/CA2915443A1/en not_active Abandoned
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