SG10201808903UA - Compositions for modulating c9orf72 expression - Google Patents

Compositions for modulating c9orf72 expression

Info

Publication number
SG10201808903UA
SG10201808903UA SG10201808903UA SG10201808903UA SG10201808903UA SG 10201808903U A SG10201808903U A SG 10201808903UA SG 10201808903U A SG10201808903U A SG 10201808903UA SG 10201808903U A SG10201808903U A SG 10201808903UA SG 10201808903U A SG10201808903U A SG 10201808903UA
Authority
SG
Singapore
Prior art keywords
c9orf72
compositions
methods
mrna
expression
Prior art date
Application number
SG10201808903UA
Inventor
Susan Freier
Frank Rigo
Priyam Singh
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
Priority to US201361890108P priority Critical
Priority to US201361891313P priority
Priority to US201361919540P priority
Priority to US201461927903P priority
Priority to US201461980502P priority
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of SG10201808903UA publication Critical patent/SG10201808903UA/en

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Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine

Abstract

COMPOSITIONS FOR MODULATING CORF72 EXPRESSION Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds. (no figure) 213
SG10201808903UA 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression SG10201808903UA (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
US201361890108P true 2013-10-11 2013-10-11
US201361891313P true 2013-10-15 2013-10-15
US201361919540P true 2013-12-20 2013-12-20
US201461927903P true 2014-01-15 2014-01-15
US201461980502P true 2014-04-16 2014-04-16

Publications (1)

Publication Number Publication Date
SG10201808903UA true SG10201808903UA (en) 2018-11-29

Family

ID=52813758

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201808903UA SG10201808903UA (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression
SG11201602597YA SG11201602597YA (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201602597YA SG11201602597YA (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression

Country Status (15)

Country Link
US (1) US10221414B2 (en)
EP (1) EP3055414A4 (en)
JP (1) JP2016533717A (en)
KR (1) KR20160062069A (en)
CN (1) CN105637090A (en)
AU (1) AU2014331652A1 (en)
BR (1) BR112016007751A2 (en)
CA (1) CA2926408A1 (en)
CL (2) CL2016000839A1 (en)
IL (1) IL244759D0 (en)
MX (1) MX2016004651A (en)
PH (1) PH12016500603A1 (en)
RU (1) RU2016116849A (en)
SG (2) SG10201808903UA (en)
WO (1) WO2015054676A2 (en)

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US9598458B2 (en) 2012-07-13 2017-03-21 Wave Life Sciences Japan, Inc. Asymmetric auxiliary group
US9963699B2 (en) 2012-10-15 2018-05-08 Ionis Pharmaceuticals, Inc. Methods for modulating C9ORF72 expression
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SG11201605782UA (en) 2014-01-16 2016-08-30 Wave Life Sciences Ltd Chiral design
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AU2017248637A1 (en) * 2016-04-13 2018-09-27 Ionis Pharmaceuticals, Inc. Methods for reducing C9ORF72 expression

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Also Published As

Publication number Publication date
JP2016533717A (en) 2016-11-04
MX2016004651A (en) 2016-08-05
BR112016007751A2 (en) 2017-09-12
CL2016000839A1 (en) 2016-12-30
EP3055414A4 (en) 2017-07-19
CN105637090A (en) 2016-06-01
IL244759D0 (en) 2016-04-21
SG11201602597YA (en) 2016-05-30
US20160251655A1 (en) 2016-09-01
WO2015054676A2 (en) 2015-04-16
PH12016500603A1 (en) 2016-06-13
AU2014331652A1 (en) 2016-04-21
KR20160062069A (en) 2016-06-01
US10221414B2 (en) 2019-03-05
RU2016116849A (en) 2017-11-16
EP3055414A2 (en) 2016-08-17
WO2015054676A3 (en) 2015-06-04
CA2926408A1 (en) 2015-04-16
CL2018001753A1 (en) 2018-10-19

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