EA036417B1 - Композиции и способы для снижения экспрессии tau - Google Patents

Композиции и способы для снижения экспрессии tau Download PDF

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EA036417B1
EA036417B1 EA201891468A EA201891468A EA036417B1 EA 036417 B1 EA036417 B1 EA 036417B1 EA 201891468 A EA201891468 A EA 201891468A EA 201891468 A EA201891468 A EA 201891468A EA 036417 B1 EA036417 B1 EA 036417B1
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Prior art keywords
seq
tau
oligonucleotide
disease
mrna
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EA201891468A
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Russian (ru)
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EA201891468A1 (ru
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Мануэла Полидоро Офенгейм
Ян Вайлер
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Новартис Аг
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EA201891468A 2015-12-21 2016-12-19 Композиции и способы для снижения экспрессии tau EA036417B1 (ru)

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US201562270165P 2015-12-21 2015-12-21
PCT/IB2016/057794 WO2017109679A1 (en) 2015-12-21 2016-12-19 Compositions and methods for decreasing tau expression

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EA201891468A1 EA201891468A1 (ru) 2018-12-28
EA036417B1 true EA036417B1 (ru) 2020-11-09

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Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2831231A1 (en) 2012-03-30 2015-02-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
TWI657819B (zh) 2013-07-19 2019-05-01 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
CN111868068A (zh) * 2018-03-13 2020-10-30 詹森药业有限公司 用于在tau蛋白病变治疗中使用的经修饰的寡核苷酸
CA3103054A1 (en) 2018-07-03 2020-01-09 F. Hoffmann-La Roche Ag Oligonucleotides for modulating tau expression
BR112022019606A2 (pt) * 2020-03-30 2022-11-16 Alnylam Pharmaceuticals Inc Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas
CN113817728A (zh) * 2020-06-19 2021-12-21 四川大学华西医院 一种有效干扰Tau蛋白表达的重组慢病毒及其应用
EP4355878A4 (en) * 2021-06-16 2025-04-09 Empirico Inc. TREATMENT OF MTRES1-RELATED DISEASES AND DISORDERS
WO2023288111A2 (en) * 2021-07-16 2023-01-19 Aptah Bio, Inc. Polynucleotide compositions and methods for gene expression regulation
EP4608981A1 (en) * 2022-10-25 2025-09-03 Empirico Inc. Gene therapy treatment of mtres1 related diseases and disorders
CN120958129A (zh) * 2023-01-18 2025-11-14 阿普塔生物公司 用于治疗癌症的多核苷酸组合物和方法
WO2024155739A1 (en) * 2023-01-18 2024-07-25 Aptah Bio, Inc. Polynucleotide compositions and methods for treatment of neurodegenerative diseases
AU2024258561A1 (en) * 2023-04-20 2025-10-30 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
US20250283084A1 (en) * 2024-03-08 2025-09-11 Eli Lilly And Company Mapt antisense oligonucleotide
US20250302980A1 (en) * 2024-03-31 2025-10-02 Arrowhead Pharmaceuticals, Inc. RNAi Agents for Inhibiting Expression of Microtubule Associated Protein Tau (MAPT), Compositions Thereof, and Methods of Use

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013148283A1 (en) * 2012-03-30 2013-10-03 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
WO2015010135A2 (en) * 2013-07-19 2015-01-22 Isis Pharmaceuticals, Inc. Compositions for modulating tau expression

Family Cites Families (104)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3817837A (en) 1971-05-14 1974-06-18 Syva Corp Enzyme amplification assay
US3939350A (en) 1974-04-29 1976-02-17 Board Of Trustees Of The Leland Stanford Junior University Fluorescent immunoassay employing total reflection for activation
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4275149A (en) 1978-11-24 1981-06-23 Syva Company Macromolecular environment control in specific receptor assays
US4277437A (en) 1978-04-05 1981-07-07 Syva Company Kit for carrying out chemically induced fluorescence immunoassay
US4366241A (en) 1980-08-07 1982-12-28 Syva Company Concentrating zone method in heterogeneous immunoassays
AU651569B2 (en) 1990-01-11 1994-07-28 Isis Pharmaceuticals, Inc. Compositions and methods for detecting and modulating RNA activity and gene expression
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020052331A1 (en) 1990-08-14 2002-05-02 Brenda Baker Compositions and methods for antisense inhibition of protein translation
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
US7408027B1 (en) 1991-12-06 2008-08-05 Max-Planck-Gesellschaft Zur Forderung Der Wissenchaften Tools for the diagnosis and treatment of Alzheimer's disease
DE69233723T2 (de) 1991-12-06 2009-02-05 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Verwendung von Proteinkinasen zur Diagnose und Behandlung der Alzheimer Krankheit
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
FI951601A7 (fi) * 1992-10-05 1995-05-29 Isis Pharmaceuticals Inc Rasgeenin antisens-oligonukleotidi-inhibitio
CA2116460A1 (en) 1993-03-02 1994-09-03 Mitsubishi Chemical Corporation Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being
KR940021073A (ko) 1993-03-02 1994-10-17 미야베 요시가즈 알쯔하이머병의 예방 또는 치료제, 그 스크리닝 방법 및 인간 유래의 타우 단백질 키나아제 i
AU6786594A (en) 1993-05-11 1994-12-12 University Of North Carolina At Chapel Hill, The Antisense oligonucleotides which combat aberrant splicing and methods of using the same
CN1214688A (zh) 1996-02-14 1999-04-21 伊希斯药物有限公司 糖修饰的缺口寡核苷酸
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
EP2253639A1 (en) 1997-09-12 2010-11-24 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
AU3875599A (en) 1998-05-01 1999-11-23 Mayo Foundation For Medical Education And Research Pathogeniv tau mutations
US6664443B1 (en) 1998-05-01 2003-12-16 Mayo Foundation For Medical Education And Research Pathogenic Tau mutations in transgenic mice
KR20010083111A (ko) * 1998-07-14 2001-08-31 길리스 스티브 전립선암의 치료 및 진단을 위한 조성물 및 방법
EP1152009B2 (en) 1999-02-12 2017-09-06 Daiichi Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
WO2000064262A1 (en) 1999-04-26 2000-11-02 The University Of North Carolina At Chapel Hill Antisense human fucosyltransferase sequences and methods of use thereof
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
CN101284873A (zh) * 1999-06-30 2008-10-15 科里克萨有限公司 用于肺癌治疗和诊断的组合物和方法
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
AU2001229619A1 (en) 2000-01-21 2001-07-31 Pharmacia And Upjohn Company Transgenic mouse model of human neurodegenerative disease
MXPA02008239A (es) * 2000-02-28 2004-08-12 Decode Genetics Ehf Gen humano de la esquizofrenia.
WO2002055681A2 (en) 2001-01-11 2002-07-18 Bayer Ag Regulation of human tau-tubulin kinase
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003247521A1 (en) 2002-06-26 2004-01-19 Isis Pharmaceuticals, Inc. Antisense inhibition via rnase h-independent reduction in mrna
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
US20080176812A1 (en) 2002-08-05 2008-07-24 Davidson Beverly L Allele-specific silencing of disease genes
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
AU2003257850A1 (en) 2002-08-14 2004-03-03 Mitsubishi Kagaku Iatron, Inc. ANTIBODY SPECIFIC TO CENTRAL Tau-PROTEIN
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US7906326B2 (en) 2003-05-07 2011-03-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides associated with alzheimer's disease and uses thereof
US8163896B1 (en) 2002-11-14 2012-04-24 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US20070077553A1 (en) 2003-10-30 2007-04-05 Rosetta Genomics Bioinformatically detectable group of novel vaccinia regulatory genes and uses thereof
US20130130231A1 (en) 2002-11-26 2013-05-23 Isaac Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20060257851A1 (en) 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
US7696334B1 (en) 2002-12-05 2010-04-13 Rosetta Genomics, Ltd. Bioinformatically detectable human herpesvirus 5 regulatory gene
AU2003904328A0 (en) 2003-08-13 2003-08-28 Garvan Institute Of Medical Research Diagnosis and treatment of neurodegenerative disorders
US7888497B2 (en) 2003-08-13 2011-02-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
WO2005021749A1 (en) 2003-08-28 2005-03-10 Novartis Ag Interfering rna duplex having blunt-ends and 3’-modifications
CN1856573A (zh) * 2003-09-25 2006-11-01 千叶县政府 用于神经母细胞瘤预后诊断的微阵列和神经母细胞瘤预后诊断方法
WO2005040382A2 (de) 2003-10-28 2005-05-06 Alexander Cherkasky Cherkasky - fusionsproteine enthaltend antikorperbinde-, antigenbinde - mikrotubulibinde und immunantwortauslosende
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
IL179285A (en) * 2004-05-14 2011-04-28 Rosetta Genomics Ltd Micrornas and uses thereof
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
EP2206781B1 (en) 2004-06-28 2015-12-02 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7312035B2 (en) 2004-09-03 2007-12-25 Affymetrix, Inc. Methods of genetic analysis of yeast
US7833513B2 (en) 2004-12-03 2010-11-16 Rhode Island Hospital Treatment of Alzheimer's Disease
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
CA2587601C (en) 2004-12-03 2017-11-07 Rhode Island Hospital Diagnosis and treatment of alzheimer's disease
US20070048756A1 (en) 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
EP1937312B1 (en) 2005-08-30 2016-06-29 Ionis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
EP2007889A2 (en) 2006-04-03 2008-12-31 Santaris Pharma A/S Pharmaceutical compositions comprising anti-mirna antisense oligonucleotides
US20090280110A1 (en) 2006-06-27 2009-11-12 University Of Virginia Patent Foundation Cell Model for Alzheimer's Disease Pathology
EP2097527B1 (en) 2006-11-23 2018-08-01 Querdenker Aps Oligonucleotides for modulating target rna activity
JP5721426B2 (ja) 2007-04-05 2015-05-20 ザ ジェイ.ディヴィッド グラッドストン インスティテューツ 神経の過度な興奮を緩和する薬剤を識別する方法
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
CA2717792A1 (en) 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2010122538A1 (en) 2009-04-24 2010-10-28 Santaris Pharma A/S Pharmaceutical compositions for treatment of hcv patients that are non-responders to interferon
LT2499249T (lt) 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
WO2011094645A1 (en) 2010-01-28 2011-08-04 University Of Massachusetts Lowell Methods of diagnosing tau-associated neurodegenerative diseases
EP2550360B1 (en) 2010-03-24 2017-08-30 Mirrx Therapeutics A/s Bivalent antisense oligonucleotides
CA2817256A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9145557B2 (en) 2010-11-23 2015-09-29 Mirrx Therapeutics A/S Oligonucleotides for modulation of target RNA activity
CA2822462A1 (en) 2010-12-20 2012-06-28 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2702170B1 (en) * 2011-04-28 2015-12-16 Life Technologies Corporation Methods and compositions for multiplex pcr
WO2013013165A2 (en) 2011-07-21 2013-01-24 The Trustees Of The University Of Pennsylvania Use of lna compounds for therapeutic inhibition of mir-21 in systemic lupus erythematosus
EP2834260A4 (en) 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS
SG11201407017WA (en) 2012-06-04 2014-12-30 Irm Llc Site-specific labeling methods and molecules produced thereby
WO2014064187A1 (en) 2012-10-24 2014-05-01 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. A microtubuli-modifying compound
US9200068B2 (en) 2012-12-18 2015-12-01 Regents Of The University Of Minnesota Compositions and methods related to tauopathy
AU2013364158A1 (en) 2012-12-20 2015-07-09 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
BR112015023001B8 (pt) 2013-03-14 2022-08-09 Sarepta Therapeutics Inc Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
HRP20190382T1 (hr) 2013-03-14 2019-04-19 Sarepta Therapeutics, Inc. Pripravci koji preskaču ekson za liječenje mišićne distrofije
EP2971142B1 (en) 2013-03-14 2020-06-24 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
EP3007705A4 (en) 2013-06-12 2017-02-15 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
WO2015068075A2 (en) 2013-10-24 2015-05-14 Institut National De La Sante Et De La Recherche Medicale Detection of tau
CN104711324A (zh) * 2013-12-11 2015-06-17 芮屈生物技术(上海)有限公司 老年性痴呆病变前期TAU基因的mRNA水平原位杂交筛查试剂盒
EP3169784B1 (en) * 2014-07-16 2020-06-10 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
US20170211064A1 (en) 2014-07-29 2017-07-27 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
CN107636159B (zh) 2015-02-04 2022-06-14 百时美施贵宝公司 选择治疗性分子的方法
CN107922946A (zh) * 2015-02-04 2018-04-17 霍夫曼-拉罗奇有限公司 Tau反义低聚物及其用途
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013148283A1 (en) * 2012-03-30 2013-10-03 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
WO2015010135A2 (en) * 2013-07-19 2015-01-22 Isis Pharmaceuticals, Inc. Compositions for modulating tau expression

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