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 (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| 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 |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| WO2015051283A1 (en) | 2013-10-04 | 2015-04-09 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| CA2968336C (en) | 2014-12-17 | 2021-11-23 | Proqr Therapeutics Ii B.V. | Construct for site directed editing of an adenosine nucleotide in target rna |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| EP3334499A4 (en) | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| US11116843B2 (en) | 2015-09-25 | 2021-09-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| IL299516A (en) | 2016-05-06 | 2023-02-01 | Astrazeneca Ab | Oligonucleotides conjugated to the GLP-1 receptor ligand complex and their uses |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| CN109757108A (zh) * | 2016-07-05 | 2019-05-14 | 比奥马林技术公司 | 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸 |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| EP3507366B1 (en) | 2016-09-01 | 2020-10-07 | ProQR Therapeutics II B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| 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 |
| JP7129702B2 (ja) | 2016-09-29 | 2022-09-02 | 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 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| WO2019004420A1 (ja) | 2017-06-30 | 2019-01-03 | 国立大学法人東京医科歯科大学 | ヘテロ二本鎖型antimiR |
| JP7503492B2 (ja) * | 2017-11-08 | 2024-06-20 | アイオニス・ファーマシューティカルズ・インコーポレイテッド | Glp-1受容体リガンド部分コンジュゲート化オリゴヌクレオチド及びその使用 |
| 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 |
| JP7333079B2 (ja) | 2018-03-19 | 2023-08-24 | 国立大学法人 東京医科歯科大学 | 毒性が軽減した核酸 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| AU2019316640B2 (en) | 2018-08-10 | 2026-03-12 | University Of Massachusetts | Modified oligonucleotides targeting SNPs |
| EP3840759A4 (en) | 2018-08-23 | 2022-06-01 | University Of Massachusetts | O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES |
| AU2020207935A1 (en) | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| WO2020209285A1 (ja) | 2019-04-08 | 2020-10-15 | 国立大学法人東京医科歯科大学 | 筋疾患治療用医薬組成物 |
| 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 |
| JP7793144B2 (ja) * | 2019-10-11 | 2026-01-05 | 国立大学法人東京科学大学 | 修飾ヘテロ核酸 |
| 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 |
| US12534724B2 (en) | 2020-05-26 | 2026-01-27 | 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 |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| WO2023022229A1 (ja) * | 2021-08-19 | 2023-02-23 | 国立大学法人東京医科歯科大学 | モルホリノ核酸を含む修飾ヘテロ核酸 |
| 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 |
| ATE232880T1 (de) | 1996-11-18 | 2003-03-15 | Takeshi Imanishi | Neue nucleotidanaloga |
| JP3781879B2 (ja) | 1996-11-18 | 2006-05-31 | 武 今西 | 新規ヌクレオチド類縁体 |
| 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 |
| DE69829760T3 (de) | 1997-09-12 | 2016-04-14 | Exiqon A/S | Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga |
| 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 |
| ES2328448T7 (es) * | 2000-03-31 | 2012-03-16 | Millennium Pharmaceuticals Inc. | 16836, un miembro de la familia de la fosfolipasa c humana y sus usos. |
| EP1562971B1 (en) * | 2002-11-05 | 2014-02-12 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| JP4986109B2 (ja) * | 2002-11-13 | 2012-07-25 | ジェンザイム・コーポレーション | アポリポタンパク質b発現のアンチセンス調節 |
| DK1661905T3 (da) | 2003-08-28 | 2012-07-23 | Takeshi Imanishi | Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype |
| 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 |
| EP2002004B1 (en) * | 2006-03-23 | 2015-10-14 | Roche Innovation Center Copenhagen A/S | Small internally segmented interfering rna |
| CA3044969A1 (en) | 2006-05-05 | 2007-12-21 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating gene expression |
| WO2008029619A1 (fr) | 2006-09-07 | 2008-03-13 | Daiichi Sankyo Company, Limited | Oligonucléotide antisens ena ayant une action spécifique de la séquence |
| CA2666191C (en) | 2006-10-09 | 2017-07-11 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of pcsk9 |
| EP2126079A1 (en) | 2007-03-22 | 2009-12-02 | Santaris Pharma A/S | Rna antagonist compounds for the inhibition of apo-b100 expression |
| WO2008137867A2 (en) * | 2007-05-03 | 2008-11-13 | Rosetta Inpharmatics Llc | Compositions comprising mir34 therapeutic agents for treating cancer |
| DE102007021870A1 (de) | 2007-05-10 | 2008-11-20 | Clariant International Limited | Wässrige Pigmentpräparationen |
| AU2008292091A1 (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 |
| WO2011139710A1 (en) | 2010-04-26 | 2011-11-10 | Marina Biotech, Inc. | Nucleic acid compounds with conformationally restricted monomers and uses thereof |
| WO2012138487A2 (en) * | 2011-04-07 | 2012-10-11 | 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) |
| WO2013089283A1 (en) * | 2011-12-16 | 2013-06-20 | National University Corporation Tokyo Medical And Dental University | Chimeric double-stranded nucleic acid |
-
2014
- 2014-06-16 CA CA2915443A patent/CA2915443A1/en not_active Abandoned
- 2014-06-16 US US14/898,630 patent/US10190117B2/en active Active
- 2014-06-16 JP JP2015560881A patent/JP6604544B2/ja 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 WO PCT/JP2014/003208 patent/WO2014203518A1/en not_active Ceased
- 2014-06-16 CN CN201480034378.1A patent/CN105324119A/zh active Pending
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