JP2013516190A5 - - Google Patents

Download PDF

Info

Publication number
JP2013516190A5
JP2013516190A5 JP2012548108A JP2012548108A JP2013516190A5 JP 2013516190 A5 JP2013516190 A5 JP 2013516190A5 JP 2012548108 A JP2012548108 A JP 2012548108A JP 2012548108 A JP2012548108 A JP 2012548108A JP 2013516190 A5 JP2013516190 A5 JP 2013516190A5
Authority
JP
Japan
Prior art keywords
c3pi
c3ps
c3oh
molecule
group
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
JP2012548108A
Other languages
English (en)
Japanese (ja)
Other versions
JP5824463B2 (ja
JP2013516190A (ja
Filing date
Publication date
Priority claimed from PCT/US2010/049047 external-priority patent/WO2011084193A1/en
Application filed filed Critical
Publication of JP2013516190A publication Critical patent/JP2013516190A/ja
Publication of JP2013516190A5 publication Critical patent/JP2013516190A5/ja
Application granted granted Critical
Publication of JP5824463B2 publication Critical patent/JP5824463B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2012548108A 2010-01-07 2011-01-06 非ヌクレオチドオーバーハングを含むオリゴヌクレオチド化合物 Expired - Fee Related JP5824463B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US29287810P 2010-01-07 2010-01-07
US61/292,878 2010-01-07
USPCT/US2010/049047 2010-09-16
PCT/US2010/049047 WO2011084193A1 (en) 2010-01-07 2010-09-16 Oligonucleotide compounds comprising non-nucleotide overhangs
PCT/US2011/020298 WO2011085056A1 (en) 2010-01-07 2011-01-06 Oligonucleotide compounds comprising non-nucleotide overhangs

Publications (3)

Publication Number Publication Date
JP2013516190A JP2013516190A (ja) 2013-05-13
JP2013516190A5 true JP2013516190A5 (enExample) 2014-01-30
JP5824463B2 JP5824463B2 (ja) 2015-11-25

Family

ID=44305677

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012548108A Expired - Fee Related JP5824463B2 (ja) 2010-01-07 2011-01-06 非ヌクレオチドオーバーハングを含むオリゴヌクレオチド化合物

Country Status (6)

Country Link
US (2) US8859751B2 (enExample)
EP (1) EP2521783B1 (enExample)
JP (1) JP5824463B2 (enExample)
CN (1) CN102770541B (enExample)
ES (1) ES2640091T3 (enExample)
WO (2) WO2011084193A1 (enExample)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2781896C (en) 2009-12-09 2021-03-30 Nitto Denko Corporation Modulation of hsp47 expression
AU2011270896B9 (en) * 2010-06-24 2015-05-07 Quark Pharmaceuticals, Inc. Double stranded RNA compounds to RhoA and use thereof
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
AU2012223366B2 (en) 2011-03-03 2017-02-23 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
US9796979B2 (en) 2011-03-03 2017-10-24 Quark Pharmaceuticals Inc. Oligonucleotide modulators of the toll-like receptor pathway
EP2773758B1 (en) 2011-11-03 2017-06-07 Quark Pharmaceuticals, Inc. Compositions for use in neuroprotection
US20140323549A1 (en) 2011-11-08 2014-10-30 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
WO2013098813A1 (en) 2012-01-01 2013-07-04 Qbi Enterprises Ltd. Endo180-targeted particles for selective delivery of therapeutic and diagnostic agents
US9382542B2 (en) * 2012-01-04 2016-07-05 Quark Pharmaceuticals, Inc. Double-stranded RNA compounds to CASP2 and uses thereof
US9707235B1 (en) * 2012-01-13 2017-07-18 University Of Kentucky Research Foundation Protection of cells from degeneration and treatment of geographic atrophy
CN108148838B (zh) 2012-05-22 2026-07-31 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
WO2014043289A2 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to ddit4 and methods of use thereof
EP2895608B1 (en) 2012-09-12 2018-12-05 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to p53 and methods of use thereof
EP2927320B1 (en) * 2012-11-29 2019-09-04 Gifu University Rna interference agent, method for producing same, and use thereof
EP3027222A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
EP3027223A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
KR101696704B1 (ko) 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
DK3149172T3 (en) 2014-05-29 2018-08-20 Quark Pharmaceuticals Inc METHODS AND COMPOSITIONS FOR THE PREVENTION OF ISKEMIC REPERFUSION LESSON IN BODIES
US20160272970A1 (en) * 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
EP4101930A1 (en) 2015-09-17 2022-12-14 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
JP7027311B2 (ja) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ,インコーポレーテッド MyD88又はTLR3を標的とするRNA複合体を使用した加齢黄斑変性の治療
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
KR102825945B1 (ko) 2016-02-02 2025-06-30 올릭스 주식회사 Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
JP7049262B2 (ja) 2016-04-11 2022-04-06 オリックス ファーマシューティカルズ,インコーポレーテッド 結合組織成長因子を標的とするrna複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference
WO2020191177A1 (en) * 2019-03-21 2020-09-24 Sudhir Agrawal Antisense oligonucleotides for allele specificity
KR102421750B1 (ko) * 2020-05-26 2022-07-19 올릭스 주식회사 MyD88을 표적으로 하는 RNAi 제제 및 이의 용도
EP4176061A1 (en) * 2021-01-30 2023-05-10 E-Therapeutics plc Nucleic acids containing abasic nucleotides
CN116829717A (zh) * 2021-01-30 2023-09-29 e-生物有限公司 含有无碱基核苷酸的核酸
BR112023014443A2 (pt) * 2021-01-30 2024-01-23 E Therapeutics Plc Ácidos nucleicos contendo nucleotídeos abásicos
CA3232053A1 (en) * 2021-10-08 2023-04-13 E-Therapeutics Plc Nucleic acids containing abasic nucleosides
KR20250043472A (ko) * 2022-07-27 2025-03-28 이-테라퓨틱스 피엘씨 핵산 화합물
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
KR20250043473A (ko) * 2022-07-27 2025-03-28 이-테라퓨틱스 피엘씨 핵산 화합물
WO2024165571A2 (en) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibitors of expression and/or function
WO2025125576A2 (en) * 2023-12-15 2025-06-19 E-Therapeutics Plc Inhibitors of expression and/or function
EP4686757A1 (en) * 2024-07-31 2026-02-04 e-therapeutics PLC Inhibitors of expression and/or function

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4475196A (en) 1981-03-06 1984-10-02 Zor Clair G Instrument for locating faults in aircraft passenger reading light and attendant call control system
US4447233A (en) 1981-04-10 1984-05-08 Parker-Hannifin Corporation Medication infusion pump
US4439196A (en) 1982-03-18 1984-03-27 Merck & Co., Inc. Osmotic drug delivery system
US4447224A (en) 1982-09-20 1984-05-08 Infusaid Corporation Variable flow implantable infusion apparatus
US4487603A (en) 1982-11-26 1984-12-11 Cordis Corporation Implantable microinfusion pump system
US4486194A (en) 1983-06-08 1984-12-04 James Ferrara Therapeutic device for administering medicaments through the skin
US4666828A (en) 1984-08-15 1987-05-19 The General Hospital Corporation Test for Huntington's disease
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4801531A (en) 1985-04-17 1989-01-31 Biotechnology Research Partners, Ltd. Apo AI/CIII genomic polymorphisms predictive of atherosclerosis
US4959217A (en) 1986-05-22 1990-09-25 Syntex (U.S.A.) Inc. Delayed/sustained release of macromolecules
US4925678A (en) 1987-04-01 1990-05-15 Ranney David F Endothelial envelopment drug carriers
US5080646A (en) 1988-10-03 1992-01-14 Alza Corporation Membrane for electrotransport transdermal drug delivery
US5272057A (en) 1988-10-14 1993-12-21 Georgetown University Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase
US5270030A (en) 1988-12-29 1993-12-14 Bio-Technology General Corp. Fibrin binding domain polypeptide and method of producing
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5192659A (en) 1989-08-25 1993-03-09 Genetype Ag Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes
US5167616A (en) 1989-12-14 1992-12-01 Alza Corporation Iontophoretic delivery method
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5225182A (en) 1991-10-31 1993-07-06 Sharma Yash P Vectored drug delivery system using a cephaloplastin linking agent and a methed of using the system
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
CA2135646A1 (en) 1992-05-11 1993-11-25 Kenneth G. Draper Method and reagent for inhibiting viral replication
US6235886B1 (en) 1993-09-03 2001-05-22 Isis Pharmaceuticals, Inc. Methods of synthesis and use
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
EP1253199A1 (en) 1993-09-02 2002-10-30 Ribozyme Pharmaceuticals, Inc. Abasic moieties containing enzymatic nucleic acids
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US20050042647A1 (en) 1996-06-06 2005-02-24 Baker Brenda F. Phosphorous-linked oligomeric compounds and their use in gene modulation
US20050032067A1 (en) 2002-11-05 2005-02-10 Prakash Thazha P. Non-phosphorous-linked oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
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
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
AU776150B2 (en) 1999-01-28 2004-08-26 Medical College Of Georgia Research Institute, Inc. Composition and method for (in vivo) and (in vitro) attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
BRPI0008131B8 (pt) 1999-02-12 2021-05-25 Daiichi Sankyo Co Ltd composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno
WO2000049035A1 (en) 1999-02-19 2000-08-24 The General Hospital Corporation Gene silencing
JP2000290185A (ja) * 1999-04-08 2000-10-17 Sankyo Co Ltd Tggg配列を持つ修飾オリゴデオキシリボヌクレオチドを含有する抗エイズ剤
IL145778A0 (en) 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
EP1235842A4 (en) 1999-10-15 2003-04-23 Univ Massachusetts Rna interference pathway genes as tools for targeted genetic interference
GB9925459D0 (en) 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
ATE421584T1 (de) 1999-11-02 2009-02-15 Novartis Vaccines & Diagnostic Cpg rezeptor (cpg-r) und darauf bezogene methoden
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US20050020525A1 (en) 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
JP2003526367A (ja) 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド Rna干渉の方法とrna干渉組成物
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US20070032441A1 (en) 2001-05-18 2007-02-08 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
US20040019001A1 (en) 2002-02-20 2004-01-29 Mcswiggen James A. RNA interference mediated inhibition of protein typrosine phosphatase-1B (PTP-1B) gene expression using short interfering RNA
KR100990055B1 (ko) 2001-11-21 2010-10-26 사이고 가오루 유전자 발현 억제 방법
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
US20050096289A1 (en) * 2002-02-07 2005-05-05 Hans Prydz Methods and compositions for modulating tissue factor
CA2475447A1 (en) * 2002-02-07 2003-08-14 Hans Prydz Short interfering rna molecules directed towards a tissue factor coding nucleic acid
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20050233342A1 (en) 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
AU2004221760B2 (en) 2003-03-21 2010-03-18 Roche Innovation Center Copenhagen A/S Short interfering RNA (siRNA) analogues
US20070167384A1 (en) 2003-04-02 2007-07-19 Dharmacon, Inc. Modified polynucleotides for use in rna interference
CA2528012C (en) 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
CA2527958C (en) 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
WO2005044976A2 (en) 2003-06-20 2005-05-19 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
EP2990410A1 (en) 2004-08-10 2016-03-02 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
NZ553162A (en) 2004-08-16 2010-03-26 Quark Biotech Inc Double stranded siRNA compounds that inhibit RRTP801
US20110313024A1 (en) 2004-08-20 2011-12-22 Leonid Beigelman RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2006047842A2 (en) 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
US20090176725A1 (en) * 2005-08-17 2009-07-09 Sirna Therapeutics Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
KR20080047463A (ko) * 2005-09-16 2008-05-28 콜리 파마슈티칼 게엠베하 뉴클레오티드 변형에 의한 짧은 간섭 리보핵산(sirna)의 면역자극 특성의 조절
US20070259827A1 (en) 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
EP2046993A4 (en) 2006-07-07 2010-11-17 Univ Massachusetts RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のsiRNAおよびその使用方法
WO2008104978A2 (en) 2007-02-28 2008-09-04 Quark Pharmaceuticals, Inc. Novel sirna structures
US7812002B2 (en) 2007-03-21 2010-10-12 Quark Pharmaceuticals, Inc. Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer
CA2701845A1 (en) 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures
US8188060B2 (en) * 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
TW201124160A (en) 2009-11-26 2011-07-16 Quark Pharmaceuticals Inc SiRNA compounds comprising terminal substitutions
CA2781896C (en) 2009-12-09 2021-03-30 Nitto Denko Corporation Modulation of hsp47 expression
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications

Similar Documents

Publication Publication Date Title
JP2014509511A5 (enExample)
NZ599237A (en) Sirna compounds comprising terminal substitutions
JP7348922B2 (ja) B型肝炎感染の治療用のPAPD5及びPAPD7 mRNAを低減させるための核酸分子
JP2016513976A5 (enExample)
JP2021531022A5 (enExample)
JP2021533804A5 (enExample)
JP2014511686A5 (enExample)
JP7515400B2 (ja) B型肝炎ウイルス感染を治療するためのfubp1阻害剤の使用
JP2009524696A5 (enExample)
JP2012050438A5 (enExample)
JP2019534009A5 (enExample)
JP2019503394A5 (enExample)
JP2016116520A5 (enExample)
JP2014527401A5 (enExample)
JP2009514877A5 (enExample)
JP2016520312A5 (enExample)
JP2009543783A5 (enExample)
JP2007505627A5 (enExample)
JP2005517436A5 (enExample)
JP2013510561A5 (enExample)
JP2016502858A5 (enExample)
CN105008533A (zh) 嵌合单链反义多核苷酸和双链反义试剂
JP2010512747A5 (enExample)
JP2013537404A5 (enExample)
JP2012505657A5 (enExample)