JP2020022483A5 - - Google Patents

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JP2020022483A5
JP2020022483A5 JP2019190648A JP2019190648A JP2020022483A5 JP 2020022483 A5 JP2020022483 A5 JP 2020022483A5 JP 2019190648 A JP2019190648 A JP 2019190648A JP 2019190648 A JP2019190648 A JP 2019190648A JP 2020022483 A5 JP2020022483 A5 JP 2020022483A5
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modified oligonucleotide
pharmaceutical composition
pharmaceutically acceptable
pbs
buffered saline
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JP2019190648A
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JP6924242B2 (ja
JP2020022483A (ja
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JP2019190648A 2015-04-16 2019-10-18 C9orf72発現を調節するための組成物 Expired - Fee Related JP6924242B2 (ja)

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JP2021124953A JP2021184720A (ja) 2015-04-16 2021-07-30 C9orf72発現を調節するための組成物

Applications Claiming Priority (7)

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US201562148691P 2015-04-16 2015-04-16
US62/148,691 2015-04-16
US201562232941P 2015-09-25 2015-09-25
US62/232,941 2015-09-25
US201562239400P 2015-10-09 2015-10-09
US62/239,400 2015-10-09
JP2017546618A JP6606557B2 (ja) 2015-04-16 2016-04-15 C9orf72発現を調節するための組成物

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JP2020022483A JP2020022483A (ja) 2020-02-13
JP2020022483A5 true JP2020022483A5 (enExample) 2020-05-14
JP6924242B2 JP6924242B2 (ja) 2021-08-25

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JP2017546618A Expired - Fee Related JP6606557B2 (ja) 2015-04-16 2016-04-15 C9orf72発現を調節するための組成物
JP2019190648A Expired - Fee Related JP6924242B2 (ja) 2015-04-16 2019-10-18 C9orf72発現を調節するための組成物
JP2021124953A Ceased JP2021184720A (ja) 2015-04-16 2021-07-30 C9orf72発現を調節するための組成物

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US (4) US9605263B2 (enExample)
EP (2) EP3283080B1 (enExample)
JP (3) JP6606557B2 (enExample)
KR (2) KR102258516B1 (enExample)
CN (2) CN113265404A (enExample)
AU (2) AU2016249002B2 (enExample)
BR (1) BR112017016068A2 (enExample)
CA (1) CA2978103A1 (enExample)
CL (4) CL2017002567A1 (enExample)
CO (1) CO2017007671A2 (enExample)
CY (1) CY1122981T1 (enExample)
DK (1) DK3283080T3 (enExample)
ES (1) ES2791995T3 (enExample)
HR (1) HRP20200843T1 (enExample)
HU (1) HUE049233T2 (enExample)
IL (2) IL253557B (enExample)
LT (1) LT3283080T (enExample)
MX (2) MX366128B (enExample)
MY (1) MY181815A (enExample)
NZ (1) NZ733882A (enExample)
PH (2) PH12017501875B1 (enExample)
PL (1) PL3283080T3 (enExample)
PT (1) PT3283080T (enExample)
RS (1) RS60230B1 (enExample)
RU (1) RU2736574C2 (enExample)
SA (3) SA520412451B1 (enExample)
SG (2) SG10202001856WA (enExample)
SI (1) SI3283080T1 (enExample)
SM (1) SMT202000165T1 (enExample)
WO (1) WO2016168592A2 (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
FI2906696T4 (fi) 2012-10-15 2023-03-18 Menetelmiä c9orf72:n ilmentymisen moduloimiseksi
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
MY192689A (en) * 2013-10-11 2022-09-01 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
WO2017180835A1 (en) * 2016-04-13 2017-10-19 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
WO2018081728A1 (en) * 2016-10-31 2018-05-03 Emendobio Inc. Compositions for genome editing
AU2018316159A1 (en) 2017-08-08 2020-02-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2019094694A1 (en) 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
MY203356A (en) 2018-01-12 2024-06-25 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
WO2019140236A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
AU2019310040A1 (en) * 2018-07-23 2021-02-11 Enclear Therapies, Inc. Methods of treating neurological disorders
JP7383298B2 (ja) 2018-08-03 2023-11-20 国立大学法人 東京医科歯科大学 Tdp-43凝集阻害剤
US12281305B2 (en) * 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
MA54383A (fr) 2018-12-06 2021-10-13 Biogen Ma Inc Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique
AU2020252020A1 (en) * 2019-03-29 2021-11-11 University Of Massachusetts Oligonucleotide-based modulation of C90RF72
EP3966328A4 (en) * 2019-05-06 2023-10-18 University Of Massachusetts Anti-c9orf72 oligonucleotides and related methods
CA3156176A1 (en) * 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP4048794A4 (en) * 2019-10-22 2024-04-17 Applied Genetic Technologies Corporation Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases
IL293824A (en) 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Compositions of RNA material (c9orf72) human chromosome 9 open reading frame 72 and methods of using them
WO2024036186A2 (en) * 2022-08-09 2024-02-15 The University Of Massachusetts Identification of allele-specific target sequences for c9orf72

Family Cites Families (100)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
EP0455905B1 (en) 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5681940A (en) 1994-11-02 1997-10-28 Icn Pharmaceuticals Sugar modified nucleosides and oligonucleotides
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
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
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
US5998148A (en) 1999-04-08 1999-12-07 Isis Pharmaceuticals Inc. Antisense modulation of microtubule-associated protein 4 expression
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP2003519231A (ja) 1999-12-30 2003-06-17 カー・ユ・ルーベン・リサーチ・アンド・ディベロップメント シクロヘキセン核酸
USH2191H1 (en) 2000-10-24 2007-06-05 Snp Consortium Identification and mapping of single nucleotide polymorphisms in the human genome
US20050019915A1 (en) 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
US20050261233A1 (en) 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
EP1766052A4 (en) 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH
WO2006047842A2 (en) 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
EP2422819B1 (en) 2006-01-26 2017-03-01 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
EP2505646A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2076597A2 (en) 2006-10-09 2009-07-08 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
BRPI0719995A2 (pt) 2006-12-14 2014-03-18 Novartis Ag Composições e métodos para tratar distúrbios musculares & cardiovasculares
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
DE602007005629D1 (de) 2007-06-18 2010-05-12 Commissariat Energie Atomique Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US20090123928A1 (en) 2007-10-11 2009-05-14 The Johns Hopkins University Genomic Landscapes of Human Breast and Colorectal Cancers
MX2010004984A (es) * 2007-11-05 2010-07-29 Baltic Technology Dev Ltd Uso de oligonucleotidos con bases modificadas en hibridacion de acidos nucleicos.
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2010036696A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
RU2011146158A (ru) * 2009-04-15 2013-05-27 АйЭсАйЭс ФАРМАСЬЮТИКАЛЗ, ИНК. Модуляция воспалительных реакций фактором хi
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
JP2014501489A (ja) 2009-07-06 2014-01-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド 細胞ベースのバイオプロセシング
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2563921B1 (en) 2010-04-30 2016-11-23 Cellectis Method for modulating double-strand break-induced homologous recombination
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
ES2756326T3 (es) * 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
EP3521451A1 (en) * 2010-11-17 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
AU2011349464B2 (en) 2010-12-20 2016-07-07 The General Hospital Corporation Polycomb-associated non-coding RNAS
US20140050778A1 (en) 2010-12-28 2014-02-20 University Of Rochester Nucleic acid binding compounds, methods of making, and use thereof
KR20190092590A (ko) 2011-04-01 2019-08-07 아이오니스 파마수티컬즈, 인코포레이티드 전사 신호 전달 인자 및 활성 인자 3(stat3) 발현 조절
SI3505528T1 (sl) * 2011-04-21 2021-04-30 Glaxo Group Limited Modulacija izražanja virusa hepatitisa B (HBV)
WO2013030588A1 (en) * 2011-08-31 2013-03-07 The University Of Manchester Method for diagnosing a neurodegenerative disease
US20140255936A1 (en) 2011-09-09 2014-09-11 Mayo Foundation For Medical Education And Research Detecting frontotemporal dementia and amyotrophic lateral sclerosis
AU2012328680A1 (en) * 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
EP2780474A4 (en) * 2011-11-17 2015-06-17 Rheonix Inc SYSTEM AND METHOD FOR SELECTIVE MOLECULAR ANALYSIS
WO2013082548A1 (en) * 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
CA2858576A1 (en) 2011-12-06 2013-06-13 Research Institute At Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
PT2841578T (pt) * 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
RU2624028C2 (ru) * 2012-05-24 2017-06-30 Ионис Фармасьютикалз, Инк. Способы и композиции для модулирования экспрессии аполипопротеина (а)
FI2906696T4 (fi) * 2012-10-15 2023-03-18 Menetelmiä c9orf72:n ilmentymisen moduloimiseksi
EP2906697A4 (en) * 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
RS59283B1 (sr) * 2013-01-22 2019-10-31 Deutsches Zentrum Fuer Neurodegenerative Erkrankungen E V Proteini sa dipeptidnim ponovkom kao terapeutsko ciljno mesto kod neurodegenerativnih bolesti sa ekspanzijom heksanukleotidnog ponovka
MY192689A (en) 2013-10-11 2022-09-01 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
WO2016024205A1 (en) * 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
US10538762B2 (en) 2014-10-14 2020-01-21 The Board Of Regents Of The University Of Texas System Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
WO2017180835A1 (en) 2016-04-13 2017-10-19 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
US10399845B2 (en) 2017-09-22 2019-09-03 Emco Wheaton Retail Corporation Testable overfill prevention valve for a liquid storage container

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