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
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JP
Japan
Prior art keywords
nucleotides
strand
independently selected
nucleotide analogs
natural
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JP2015560881A
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English (en)
Japanese (ja)
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JP2016526529A (ja
JP6604544B2 (ja
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Priority claimed from PCT/JP2014/003208 external-priority patent/WO2014203518A1/en
Publication of JP2016526529A publication Critical patent/JP2016526529A/ja
Publication of JP2016526529A5 publication Critical patent/JP2016526529A5/ja
Application granted granted Critical
Publication of JP6604544B2 publication Critical patent/JP6604544B2/ja
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JP2015560881A 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 Active JP6604544B2 (ja)

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

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ID=52104269

Family Applications (1)

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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 (43)

* Cited by examiner, † Cited by third party
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
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
WO2013012758A1 (en) 2011-07-19 2013-01-24 Ontorii, Inc. Methods for the synthesis of functionalized nucleic acids
EA201492123A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии семейства генов smn
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US20150152410A1 (en) 2012-05-16 2015-06-04 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
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
EP3712269A1 (en) 2014-12-17 2020-09-23 ProQR Therapeutics II B.V. Targeted rna editing
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
CA2995110A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017053999A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
US20190134214A1 (en) 2016-05-06 2019-05-09 Astrazeneca Ab GLP-1 Receptor Ligand Moiety Conjugated Oligonucleotides and Uses Thereof
US10988763B2 (en) 2016-06-22 2021-04-27 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
MX2018016253A (es) * 2016-07-05 2019-09-09 Biomarin Tech Bv Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos.
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
PL3507366T3 (pl) 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
WO2018056442A1 (ja) 2016-09-23 2018-03-29 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
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
WO2018134301A1 (en) * 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
EP3647423A4 (en) 2017-06-30 2021-03-24 National University Corporation Tokyo Medical and Dental University STRAIGHT DOUBLE-STRANDED ANTI-MIRROR
EP3707156A4 (en) * 2017-11-08 2021-08-04 Ionis Pharmaceuticals, Inc. OLIGONUCLEOTIDES CONJUGATED TO A LIGAND FRACTION OF THE GLP-1 RECEPTOR AND THEIR USES
WO2019167995A1 (ja) 2018-02-28 2019-09-06 国立大学法人東京医科歯科大学 虚血病変部位特異的な遺伝子治療法
JP7394392B2 (ja) * 2018-03-14 2023-12-08 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
JP7618229B2 (ja) 2018-08-10 2025-01-21 ユニバーシティー オブ マサチューセッツ Ｓｎｐを標的化する修飾オリゴヌクレオチド
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
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
JPWO2021070959A1 (enExample) * 2019-10-11 2021-04-15
US20230061751A1 (en) * 2020-01-17 2023-03-02 University Of Massachusetts Universal dynamic pharmacokinetic-modifying anchors
US20230174981A1 (en) * 2020-03-16 2023-06-08 National University Corporation Tokyo Medical And Dental University Heteronucleic acid containing morpholino nucleic acid
CA3202708A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
TW202315943A (zh) 2021-06-23 2023-04-16 麻薩諸塞大學 用於治療子癇前症及其它血管新生病症的最佳化抗flt1寡核苷酸化合物
JPWO2023022229A1 (enExample) * 2021-08-19 2023-02-23
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)

* Cited by examiner, † Cited by third party
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
DE69719220T2 (de) 1996-11-18 2004-01-22 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
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
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
EP1294899B3 (en) * 2000-03-31 2011-07-06 Millennium Pharmaceuticals, Inc. 16836, a human phospholipase c family member and uses thereof
WO2004041889A2 (en) * 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004044181A2 (en) 2002-11-13 2004-05-27 Isis Pharmaceuticals, Inc. 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
US8329888B2 (en) * 2006-03-23 2012-12-11 Santaris Pharma A/S Small internally segmented interfering RNA
JP5825754B2 (ja) 2006-05-05 2015-12-02 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 Ａｐｏｂの発現を調節するための化合物および方法
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
US20100216864A1 (en) 2006-10-09 2010-08-26 Ellen Marie Straarup 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
WO2009027527A2 (en) 2007-08-30 2009-03-05 Santaris Pharma A/S 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
WO2011139710A1 (en) 2010-04-26 2011-11-10 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
CA2833223A1 (en) 2011-04-22 2012-10-26 Prosensa Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1)
EP2791335B1 (en) * 2011-12-16 2018-11-14 National University Corporation Tokyo Medical and Dental University Chimeric double-stranded nucleic acid

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