SG10202001856WA - Compositions for modulating c9orf72 expression - Google Patents

Compositions for modulating c9orf72 expression

Info

Publication number
SG10202001856WA
SG10202001856WA SG10202001856WA SG10202001856WA SG10202001856WA SG 10202001856W A SG10202001856W A SG 10202001856WA SG 10202001856W A SG10202001856W A SG 10202001856WA SG 10202001856W A SG10202001856W A SG 10202001856WA SG 10202001856W A SG10202001856W A SG 10202001856WA
Authority
SG
Singapore
Prior art keywords
compositions
c9orf72 expression
modulating
modulating c9orf72
expression
Prior art date
Application number
SG10202001856WA
Inventor
Frank Rigo
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of SG10202001856WA publication Critical patent/SG10202001856WA/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/22—Anxiolytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
SG10202001856WA 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression SG10202001856WA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562148691P 2015-04-16 2015-04-16
US201562232941P 2015-09-25 2015-09-25
US201562239400P 2015-10-09 2015-10-09

Publications (1)

Publication Number Publication Date
SG10202001856WA true SG10202001856WA (en) 2020-04-29

Family

ID=57126261

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10202001856WA SG10202001856WA (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
SG11201708468YA SG11201708468YA (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201708468YA SG11201708468YA (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression

Country Status (30)

Country Link
US (4) US9605263B2 (en)
EP (2) EP3283080B1 (en)
JP (3) JP6606557B2 (en)
KR (2) KR102104163B1 (en)
CN (2) CN113265404A (en)
AU (2) AU2016249002B2 (en)
BR (1) BR112017016068A2 (en)
CA (1) CA2978103A1 (en)
CL (4) CL2017002567A1 (en)
CO (1) CO2017007671A2 (en)
CY (1) CY1122981T1 (en)
DK (1) DK3283080T3 (en)
ES (1) ES2791995T3 (en)
HK (1) HK1250255A1 (en)
HR (1) HRP20200843T1 (en)
HU (1) HUE049233T2 (en)
IL (2) IL253557B (en)
LT (1) LT3283080T (en)
MX (2) MX366128B (en)
MY (1) MY181815A (en)
NZ (1) NZ733882A (en)
PH (2) PH12017501875A1 (en)
PL (1) PL3283080T3 (en)
PT (1) PT3283080T (en)
RS (1) RS60230B1 (en)
RU (1) RU2736574C2 (en)
SA (3) SA520412450B1 (en)
SG (2) SG10202001856WA (en)
SI (1) SI3283080T1 (en)
WO (1) WO2016168592A2 (en)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2616051T3 (en) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Method for the synthesis of modified nucleic acids in the phosphorus atom
AU2010270714B2 (en) 2009-07-06 2015-08-13 Wave Life Sciences Ltd. Novel nucleic acid prodrugs and methods use thereof
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
CN107011400B (en) 2012-07-13 2021-05-25 波涛生命科学有限公司 Asymmetric auxiliary group
US10577604B2 (en) 2012-10-15 2020-03-03 Ionis Pharmaceuticals, Inc. Methods for monitoring C9ORF72 expression
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
PT2906696T (en) 2012-10-15 2019-12-16 Univ California Methods for modulating c9orf72 expression
CN112080502A (en) 2013-10-11 2020-12-15 Ionis制药公司 Compositions for modulating expression of C9ORF72
KR102423317B1 (en) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 Chiral design
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
AU2017248637A1 (en) * 2016-04-13 2018-09-27 Ionis Pharmaceuticals, Inc. Methods for reducing C9ORF72 expression
US11795453B2 (en) * 2016-10-31 2023-10-24 Emendobio, Inc. Compositions for genome editing
JP2020534253A (en) * 2017-08-08 2020-11-26 ウェーブ ライフ サイエンシーズ リミテッド Oligonucleotide composition and its method
WO2019094694A1 (en) 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
SG11202006526YA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
EP3831942A4 (en) 2018-08-03 2022-05-11 National University Corporation Tokyo Medical And Dental University Tdp-43 aggregation inhibitor
BR112021010660A2 (en) 2018-12-06 2021-08-24 Biogen Ma Inc. Neurofilament protein to guide therapeutic intervention in amyotrophic lateral sclerosis
EP3946374A4 (en) * 2019-03-29 2023-07-19 University of Massachusetts Oligonucleotide-based modulation of c9orf72
US11629347B2 (en) 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
AU2020363344A1 (en) * 2019-10-06 2022-05-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

Family Cites Families (99)

* 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 (en) 1984-07-19 1989-02-17 Centre Nat Rech Scient NOVEL OLIGONUCLEOTIDES, THEIR PREPARATION PROCESS AND THEIR APPLICATIONS AS MEDIATORS IN DEVELOPING THE EFFECTS OF INTERFERONS
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DK0497875T3 (en) 1989-10-24 2000-07-03 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
DE69032425T2 (en) 1990-05-11 1998-11-26 Microprobe Corp Immersion test strips for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
ES2103918T3 (en) 1991-10-17 1997-10-01 Ciba Geigy Ag BICYCLE NUCLEOSIDES, OLIGONUCLEOTIDES, PROCEDURE FOR THEIR OBTAINING AND INTERMEDIATE PRODUCTS.
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
CA2126691C (en) 1991-12-24 2003-05-06 Phillip Dan Cook Gapped 2' modified oligonucleotides
FR2687679B1 (en) 1992-02-05 1994-10-28 Centre Nat Rech Scient OLIGOTHIONUCLEOTIDES.
EP0577558A2 (en) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclic nucleosides having bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
ES2107205T3 (en) 1993-03-30 1997-11-16 Sanofi Sa ANALOGS OF ACICLIC NUCLEOSIDES AND OLIGONUCLEOTIDE SEQUENCES THAT CONTAIN 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
JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
EP2341057A3 (en) 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide 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
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
DE60027203T2 (en) 1999-12-30 2007-01-04 K.U. Leuven Research & Development CYCLOHEXENE NUCLEIC
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
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004044136A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2’-modified nucleosides 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
EP2305812A3 (en) 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
ES2382807T3 (en) 2003-08-28 2012-06-13 Takeshi Imanishi New artificial nucleic acids of the N-O link type with cross-linking
JP5379347B2 (en) 2003-09-18 2013-12-25 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 4'-thionucleosides and oligomeric compounds
US20050261233A1 (en) 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
AU2005248147A1 (en) 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
JP2008501693A (en) 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク． Double-stranded composition with individually regulated strands for use in gene regulation
JP2008501335A (en) 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク． Chimeric gapped oligomer composition
US20080261905A1 (en) 2004-11-08 2008-10-23 K.U. Leuven Research And Development Modified Nucleosides for Rna Interference
JP5425474B2 (en) 2006-01-26 2014-02-26 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 Composition and use thereof for huntingtin
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
AU2007258117B2 (en) 2006-05-05 2013-05-30 Isis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
ES2389737T3 (en) 2006-05-11 2012-10-31 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
CN101563458A (en) 2006-12-14 2009-10-21 诺瓦提斯公司 Compositions and methods to treat muscular and cardiovascular disorders
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
EP2014769B1 (en) 2007-06-18 2010-03-31 Commissariat à l'Energie Atomique Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated 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 (en) * 2007-11-05 2010-07-29 Baltic Technology Dev Ltd Use of oligonucleotides with modified bases in hybridization of nucleic acids.
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
US8604192B2 (en) 2008-09-24 2013-12-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acids analogs
EP2356129B1 (en) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
KR20120050429A (en) 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Lipid formulated dsrna targeting the pcsk9 gene
CA2767231A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Cell-based bioprocessing
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011135396A1 (en) 2010-04-30 2011-11-03 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
KR20180105730A (en) * 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 Modulation of dystrophia myotonica-protein kinase (dmpk) expression
CA2817960C (en) * 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CA2822462A1 (en) 2010-12-20 2012-06-28 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
CA2831915C (en) 2011-04-01 2021-01-12 Isis Pharmaceuticals, Inc. Modulation of signal transducer and activator of transcription 3 (stat3) expression
UA118951C2 (en) * 2011-04-21 2019-04-10 Айоніс Фармасьютікалз, Інк. Modulation of hepatitis b virus (hbv) expression
DK2751284T3 (en) * 2011-08-31 2017-03-27 Univ Manchester PROCEDURE FOR DIAGNOSTICING A NEURODEGENERATIVE DISEASE
WO2013036833A1 (en) 2011-09-09 2013-03-14 Mayo Foundation For Medical Education And Research Detecting frontotemporal dementia and amyotrophic lateral sclerosis
US8901098B2 (en) * 2011-10-25 2014-12-02 Isis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
EP2780474A4 (en) * 2011-11-17 2015-06-17 Rheonix Inc System and methods for selective molecular analysis
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
EP2788087A4 (en) 2011-12-06 2015-08-26 Ohio State Innovation Foundation Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
EP3228712A1 (en) * 2012-04-23 2017-10-11 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
NZ631071A (en) * 2012-05-24 2017-02-24 Ionis Pharmaceuticals Inc Methods and compositions for modulating apolipoprotein(a) expression
US10577604B2 (en) * 2012-10-15 2020-03-03 Ionis Pharmaceuticals, Inc. Methods for monitoring C9ORF72 expression
US10443052B2 (en) * 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
PT2906696T (en) * 2012-10-15 2019-12-16 Univ California Methods for modulating c9orf72 expression
PL2948777T3 (en) * 2013-01-22 2019-12-31 Deutsches Zentrum Für Neurodegenerative Erkrankungen E.V. Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
CN112080502A (en) * 2013-10-11 2020-12-15 Ionis制药公司 Compositions for modulating expression of C9ORF72
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
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
AU2017248637A1 (en) 2016-04-13 2018-09-27 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

Also Published As

Publication number Publication date
AU2019250156B2 (en) 2021-11-25
MY181815A (en) 2021-01-08
PH12019502724A1 (en) 2021-03-15
AU2016249002B2 (en) 2019-07-18
CL2018003582A1 (en) 2019-04-05
WO2016168592A3 (en) 2016-11-24
CO2017007671A2 (en) 2018-01-31
JP6924242B2 (en) 2021-08-25
CY1122981T1 (en) 2021-10-29
EP3283080B1 (en) 2020-03-18
US9605263B2 (en) 2017-03-28
ES2791995T3 (en) 2020-11-06
HRP20200843T1 (en) 2020-08-21
PH12017501875B1 (en) 2018-02-26
CL2020002019A1 (en) 2020-12-11
KR102258516B1 (en) 2021-05-31
PH12017501875A1 (en) 2018-02-26
CN113265404A (en) 2021-08-17
WO2016168592A2 (en) 2016-10-20
CN107427532B (en) 2021-06-04
JP2021184720A (en) 2021-12-09
PL3283080T3 (en) 2020-07-27
KR102104163B1 (en) 2020-04-23
IL253557B (en) 2020-11-30
AU2019250156A1 (en) 2019-10-31
US20160304871A1 (en) 2016-10-20
JP6606557B2 (en) 2019-11-13
EP3722424A1 (en) 2020-10-14
SI3283080T1 (en) 2020-06-30
MX2019007834A (en) 2019-09-06
IL277624A (en) 2020-11-30
CL2017002567A1 (en) 2018-06-01
RU2736574C2 (en) 2020-11-18
RU2017139121A (en) 2019-05-16
NZ733882A (en) 2020-04-24
SA517390168B1 (en) 2020-08-26
US10815483B2 (en) 2020-10-27
RS60230B1 (en) 2020-06-30
EP3283080A4 (en) 2018-12-19
SA520412450B1 (en) 2022-01-24
IL253557A0 (en) 2017-09-28
US20170349897A1 (en) 2017-12-07
CA2978103A1 (en) 2016-10-20
KR20170136597A (en) 2017-12-11
SA520412451B1 (en) 2021-02-22
MX2017013199A (en) 2018-02-09
DK3283080T3 (en) 2020-04-06
BR112017016068A2 (en) 2018-04-03
LT3283080T (en) 2020-05-25
PT3283080T (en) 2020-06-08
MX366128B (en) 2019-06-28
EP3283080A2 (en) 2018-02-21
CL2020002018A1 (en) 2020-12-11
JP2018512127A (en) 2018-05-17
SG11201708468YA (en) 2017-11-29
HUE049233T2 (en) 2020-09-28
JP2020022483A (en) 2020-02-13
US20210230589A1 (en) 2021-07-29
HK1250255A1 (en) 2018-12-07
KR20200043513A (en) 2020-04-27
RU2017139121A3 (en) 2019-06-10
CN107427532A (en) 2017-12-01
AU2016249002A1 (en) 2017-08-10
US10138482B2 (en) 2018-11-27
US20190264204A1 (en) 2019-08-29

Similar Documents

Publication Publication Date Title
IL277624A (en) Compositions for modulating c9orf72 expression
IL273404A (en) Compositions for modulating sod-1 expression
HK1232251A1 (en) Compositions for modulating ataxin expression
GB201501598D0 (en) Compositions
GB201514328D0 (en) Compositions
GB201601572D0 (en) NO containing compositions
GB201515387D0 (en) Compositions
SG10202003691PA (en) Composition for modulating irak1
GB201517841D0 (en) NO containing compositions
GB201511469D0 (en) Compositions
GB201507673D0 (en) Compositions
GB201504995D0 (en) Compositions
GB201504045D0 (en) Compositions