JP2016526529A5 - - Google Patents

Download PDF

Info

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
Application number
JP2015560881A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016526529A (ja
JP6604544B2 (ja
Filing date
Publication date
Application filed filed Critical
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
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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

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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics 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
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
WO2015051283A1 (en) 2013-10-04 2015-04-09 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
EP3234134B1 (en) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP2018528783A (ja) 2015-09-25 2018-10-04 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. コンジュゲートアンチセンス化合物及びその使用
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
WO2017192820A1 (en) 2016-05-06 2017-11-09 Ionis Pharmaceuticals, Inc. Glp-1 receptor ligand moiety conjugated oligonucleotides and uses thereof
WO2017220751A1 (en) 2016-06-22 2017-12-28 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.
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
EP3517610A4 (en) 2016-09-23 2020-05-27 National University Corporation Tokyo Medical and Dental University THROUGH HETERODUPLEX NUCLEIC ACID THROUGH BLOOD BRAIN CABINET
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
EP3571300A1 (en) 2017-01-19 2019-11-27 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
EP3647423A4 (en) 2017-06-30 2021-03-24 National University Corporation Tokyo Medical and Dental University DOUBLE STRANDED HETERO-ANTI-MIRNA
WO2019092618A2 (en) * 2017-11-08 2019-05-16 Ionis Pharmaceuticals, Inc Glp-1 receptor ligand moiety conjugated oligonucleotides and uses thereof
EP3760209B1 (en) 2018-02-28 2023-11-22 National University Corporation Tokyo Medical and Dental University Ischemic-lesion-site-specific gene therapy
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
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11279930B2 (en) 2018-08-23 2022-03-22 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
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4247949A1 (en) 2020-11-23 2023-09-27 Alpha Anomeric SAS Nucleic acid duplexes
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
JPWO2023022229A1 (enExample) * 2021-08-19 2023-02-23
US20250297249A1 (en) * 2021-12-07 2025-09-25 Liid Pharmaceuticals, Inc. Antisense oligonucleotide complex
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
ES2192672T3 (es) 1996-11-18 2003-10-16 Takeshi Imanishi Nuevos analogos de nucleotidos.
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
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
US20010024808A1 (en) * 1998-09-10 2001-09-27 Millennium Pharmaceuticals, Inc., A Delaware Corporation Leptin induced genes
WO2001055455A2 (en) * 2000-01-31 2001-08-02 Millennium Pharmaceuticals, Inc. Resistance sequences and uses thereof
DE60138952D1 (de) * 2000-03-31 2011-04-28 Millenium Pharmaceuticals 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
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
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
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna
JP2009536222A (ja) 2006-05-05 2009-10-08 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 Pcsk9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
CA2666191C (en) 2006-10-09 2017-07-11 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
JP2010525826A (ja) * 2007-05-03 2010-07-29 ロゼッタ インファーマティックス エルエルシー 癌を治療するためのmir34治療剤を含む組成物
DE102007021870A1 (de) 2007-05-10 2008-11-20 Clariant International Limited Wässrige Pigmentpräparationen
JP2010537958A (ja) 2007-08-30 2010-12-09 サンタリス ファーマ アー/エス Fabp4/ap2を調節するためのrnaアンタゴニスト化合物
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
WO2012138487A2 (en) * 2011-04-07 2012-10-11 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)
JP6112569B2 (ja) * 2011-12-16 2017-04-12 国立大学法人 東京医科歯科大学 キメラ2重鎖核酸

Similar Documents

Publication Publication Date Title
JP2016526529A5 (enExample)
JP2016528887A5 (enExample)
JP2017502668A5 (enExample)
JP2015133993A5 (enExample)
JP2016195614A5 (enExample)
HUE057725T2 (hu) Módosított nukleozidok, nukleotidok és nukleinsavak és ezek felhasználása
JP2016525888A5 (enExample)
JP2019515674A5 (enExample)
WO2018041973A9 (en) Chemically modified single-stranded rna-editing oligonucleotides
CN106573011A8 (zh) 取代的核苷、核苷酸和其类似物
EP4328322A3 (en) Probe library construction
ZA201901061B (en) 2'-substituted-n6-substituted purine nucleotides for rna virus treatment
EP3192869A4 (en) Isolated oligonucleotide and use thereof in nucleic acid sequencing
EP2791160A4 (en) NUCLEOSIDE, NUCLEOTIDE, AND MODIFIED NUCLEIC ACID COMPOSITIONS
ES2679373T3 (es) Nuevos ligandos de RIG-I y métodos para producirlos
MX381121B (es) Supresión del gen de la huntingtina inducida por la arni
EA201792663A1 (ru) Способы скрининга бактерий, архей, водорослей и дрожжей с использованием нуклеиновых кислот crispr
HUE063813T2 (hu) CRISPR hibrid DNS/RNS polinukleotidok és alkalmazási eljárások
MX379233B (es) Métodos y composiciones para el perfilado de adn.
ME03502B (me) Supstituisani nukleozidi, nukleotidi i njihovi analozi
EP3146075A4 (en) Ion sensor dna and rna sequencing by synthesis using nucleotide reversible terminators
MY196912A (en) Short pendant arm linkers for nucleotides in sequencing applications
MX374553B (es) Derivado de ácido nucleico que tiene actividad inmunoestimuladora.
JP2012223202A5 (enExample)
EP3381947A4 (en) DNA Polymerase VARIANT