JP2011522036A5 - - Google Patents

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JP2011522036A5
JP2011522036A5 JP2011512270A JP2011512270A JP2011522036A5 JP 2011522036 A5 JP2011522036 A5 JP 2011522036A5 JP 2011512270 A JP2011512270 A JP 2011512270A JP 2011512270 A JP2011512270 A JP 2011512270A JP 2011522036 A5 JP2011522036 A5 JP 2011522036A5
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pharmaceutical composition
ear
otic
ears
composition
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JP2011512270A
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JP5524189B2 (ja
JP2011522036A (ja
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Priority claimed from PCT/IL2009/000570 external-priority patent/WO2009147684A2/en
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JP2011512270A 2008-06-06 2009-06-07 耳障害治療のための組成物および方法 Expired - Fee Related JP5524189B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US13116208P 2008-06-06 2008-06-06
US61/131,162 2008-06-06
PCT/IL2009/000570 WO2009147684A2 (en) 2008-06-06 2009-06-07 Compositions and methods for treatment of ear disorders

Publications (3)

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JP2011522036A JP2011522036A (ja) 2011-07-28
JP2011522036A5 true JP2011522036A5 (https=) 2012-07-19
JP5524189B2 JP5524189B2 (ja) 2014-06-18

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JP2011512270A Expired - Fee Related JP5524189B2 (ja) 2008-06-06 2009-06-07 耳障害治療のための組成物および方法

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US (2) US8431692B2 (https=)
EP (1) EP2293800B1 (https=)
JP (1) JP5524189B2 (https=)
WO (1) WO2009147684A2 (https=)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7825099B2 (en) * 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
WO2009001359A2 (en) 2007-06-27 2008-12-31 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of pro-apoptotic genes
JP5524189B2 (ja) 2008-06-06 2014-06-18 クォーク ファーマシューティカルズ インコーポレーティッド 耳障害治療のための組成物および方法
EP2510098B1 (en) 2009-12-09 2015-02-11 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the cns
CA2801535C (en) 2010-06-04 2017-05-30 Hough Ear Institute Composition and method for inner ear sensory hair cell regeneration or replacement
EP2632553B1 (en) 2010-10-28 2020-01-01 Pacira Pharmaceuticals, Inc. A sustained release formulation of a non-steroidal anti-inflammatory drug
KR20140047141A (ko) * 2011-08-03 2014-04-21 쿠아크 파마수티칼스 인코퍼레이티드 청력 및 균형 장애를 치료하기 위한 이중-가닥 올리고뉴클레오티드 화합물들
JP6118331B2 (ja) 2011-11-03 2017-04-19 クォーク ファーマシューティカルズ インコーポレーティッドQuark Pharmaceuticals,Inc. 神経保護のための方法および組成物
US20140323549A1 (en) 2011-11-08 2014-10-30 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
EP2802657B1 (en) * 2012-01-12 2018-05-02 Quark Pharmaceuticals, Inc. Combination therapy for treating hearing and balance disorders
DK2895608T3 (en) 2012-09-12 2019-01-21 Quark Pharmaceuticals Inc DOUBLE-STRENGTHED OIGONUCLEOTIDE MOLECULES FOR P53 AND PROCEDURES FOR USING IT
US9932578B2 (en) 2012-09-12 2018-04-03 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to P53 and methods of use thereof
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
KR20160019553A (ko) 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
CN106103717A (zh) 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
US20190002887A1 (en) 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
EP3541815A4 (en) 2016-11-18 2020-07-15 Pacira Pharmaceuticals, Inc. ZINC MELXICAM COMPLEX MICROPARTICLE MULTIVESICULAR LIPOSOME FORMULATIONS AND METHODS OF MAKING THE SAME
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
EP3565605A1 (en) * 2017-01-03 2019-11-13 ethris GmbH Ornithine transcarbamylase coding polyribonucleotides and formulations thereof
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
CA3128615A1 (en) * 2019-03-01 2020-09-10 Flagship Pioneering Innovations Vi, Llc Topical delivery of polyribonucleic acids
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
WO2021041922A1 (en) 2019-08-30 2021-03-04 The Broad Institute, Inc. Crispr-associated mu transposase systems
WO2021146641A1 (en) 2020-01-17 2021-07-22 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
WO2025059533A1 (en) 2023-09-13 2025-03-20 The Broad Institute, Inc. Crispr enzymes and systems
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof

Family Cites Families (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US6153737A (en) * 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT 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
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
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
US20050042647A1 (en) * 1996-06-06 2005-02-24 Baker Brenda F. Phosphorous-linked oligomeric compounds and their use in gene modulation
US5929042A (en) * 1997-03-03 1999-07-27 The Trustees Of Columbia University In The City Of New York Antisense compounds which prevent cell death and uses thereof
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
US6395713B1 (en) * 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
DE19807426A1 (de) 1998-02-23 1999-10-14 Otogene Biotechnologische Fors Verfahren zur Behandlung von Erkrankungen oder Störungen des Innenohrs
EP2314700A1 (en) 1999-01-28 2011-04-27 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
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
AU3369900A (en) 1999-02-19 2000-09-04 General Hospital Corporation, The Gene silencing
WO2000063364A2 (en) 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
CA2386270A1 (en) 1999-10-15 2001-04-26 University Of Massachusetts Rna interference pathway genes as tools for targeted genetic interference
GB9925459D0 (en) * 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
JP4566494B2 (ja) 1999-11-02 2010-10-20 ノバルティス バクシンズ アンド ダイアグノスティックス,インコーポレーテッド CpGレセプター(CpG−R)およびCpG−Rに関する方法
GB9927444D0 (en) * 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE10100586C1 (de) * 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
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
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)
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
PT1309726E (pt) * 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
AU2001289068A1 (en) 2000-09-20 2002-04-02 Isis Pharmaceuticals, Inc. Antisense modulation of caspase 2 expression
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
US20040259247A1 (en) * 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US20050260620A1 (en) * 2001-05-18 2005-11-24 Sirna Therapeutics, Inc. RNA interference mediated inhibition of retinolblastoma (RBI) gene expression using 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
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)
CN1608133A (zh) * 2001-10-26 2005-04-20 里伯药品公司 双链核糖核酸用于治疗正(+)链rna病毒感染的用途
DE10230997A1 (de) * 2001-10-26 2003-07-17 Ribopharma Ag Medikament zur Erhöhung der Wirksamkeit eines Rezeptor-vermittelt Apoptose in Tumorzellen auslösenden Arzneimittels
CN1612930A (zh) 2001-11-21 2005-05-04 三菱化学株式会社 抑制基因表达的方法
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US7795422B2 (en) * 2002-02-20 2010-09-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
BR0307913A (pt) * 2002-02-25 2005-01-11 Lyfjathroun Hf Uso de uma composição, composição farmacêutica para administração nasal, e, métodos para eliciar um efeito terapêutico, profilático e/ou diagnóstico em um mamìfero, para obter um inìcio rápido de um efeito terapêutico, profilático e/ou diagnóstico de uma substância ativa em um mamìfero e para melhorar a biodisponibilidade de um efeito terapêutico, profilático e/ou diagnóstico de uma substância ativa em um mamìfero
AU2003260370B2 (en) * 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
WO2004014933A1 (en) * 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20050233342A1 (en) * 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
CA2519860C (en) 2003-03-21 2018-01-16 Santaris Pharma A/S Short interfering rna (sirna) analogues
CA2526493A1 (en) 2003-05-22 2004-12-02 Theraptosis S.A. Means for preventing and treating cellular death and their biological applications
ES2357116T5 (es) * 2003-06-02 2021-09-03 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y especificad de FNAi
DK1633767T3 (en) * 2003-06-02 2019-03-25 Univ Massachusetts METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING
US8673634B2 (en) * 2003-11-13 2014-03-18 Massachusetts Eye & Ear Infirmary Method for the treatment of hearing loss
WO2005055921A2 (en) 2003-12-12 2005-06-23 Eran Eilat Compositions for treatment of ear disorders and methods of use thereof
US7293861B2 (en) 2004-01-21 2007-11-13 Silverbrook Research Pty Ltd Inkjet printer cartridge refill dispenser system with variably positioned outlets
US7842800B2 (en) * 2004-04-02 2010-11-30 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory bacterial and bacterial associated oligonucleotides and uses thereof
US8173600B2 (en) * 2004-04-30 2012-05-08 Chiesi Farmaceutici S.P.A. Caspase-2 inhibitors and their biological applications
EP1602926A1 (en) 2004-06-04 2005-12-07 University of Geneva Novel means and methods for the treatment of hearing loss and phantom hearing
JP4468989B2 (ja) * 2004-08-16 2010-05-26 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 Rtp801阻害剤の治療への使用
JP3867091B2 (ja) 2004-09-16 2007-01-10 株式会社アドメテック 交流磁場中で発熱する粉末材料およびその製造方法
AU2005288522B2 (en) * 2004-09-28 2012-06-28 Quark Pharmaceuticals, Inc. Oligoribonucleotides and methods of use thereof for treatment of alopecia, acute renal failure and other diseases
EP2230305A1 (en) 2005-07-21 2010-09-22 Alnylam Pharmaceuticals Inc. Rnai modulation of the rho-a gene and uses thereof
US7825099B2 (en) * 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
NL2000439C2 (nl) * 2006-01-20 2009-03-16 Quark Biotech Therapeutische toepassingen van inhibitoren van RTP801.
WO2007087451A2 (en) 2006-01-25 2007-08-02 University Of Massachusetts Compositions and methods for enhancing discriminatory rna interference
EP1989307B1 (en) 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
US7910566B2 (en) * 2006-03-09 2011-03-22 Quark Pharmaceuticals Inc. Prevention and treatment of acute renal failure and other kidney diseases by inhibition of p53 by siRNA
US7741469B2 (en) * 2006-04-05 2010-06-22 University Of Iowa Research Foundation Compositions for treating hearing loss and methods of use thereof
WO2007141796A2 (en) 2006-06-09 2007-12-13 Quark Pharmaceuticals, Inc. Therapeutic uses of inhibitors of rtp801l
TWI491953B (zh) 2006-07-18 2015-07-11 希畢克斯幻像有限公司 電泳顯示器
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
US20100292301A1 (en) * 2007-02-28 2010-11-18 Elena Feinstein Novel sirna structures
AU2008306455C1 (en) 2007-10-03 2014-04-17 Quark Pharmaceuticals, Inc. Novel siRNA structures
JP5524189B2 (ja) 2008-06-06 2014-06-18 クォーク ファーマシューティカルズ インコーポレーティッド 耳障害治療のための組成物および方法
ES2563984T3 (es) * 2008-10-22 2016-03-17 Quark Pharmaceuticals, Inc. Métodos para el tratamiento de trastornos oculares

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