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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
compositions
c9orf72
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
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of SG10201808903UA publication Critical patent/SG10201808903UA/en

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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • A61P25/00—Drugs for disorders of the nervous system
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    • 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
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--

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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)

Applications Claiming Priority (5)

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

Publications (1)

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

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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

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Country Status (16)

Country Link
US (3) US10221414B2 (en)
EP (2) EP3055414A4 (en)
JP (2) JP6679476B2 (en)
KR (1) KR20160062069A (en)
CN (2) CN105637090B (en)
AU (2) AU2014331652B2 (en)
BR (1) BR112016007751A2 (en)
CA (1) CA2926408A1 (en)
CL (3) CL2016000839A1 (en)
IL (1) IL244759A0 (en)
MX (2) MX2016004651A (en)
MY (1) MY192689A (en)
PH (1) PH12016500603A1 (en)
RU (1) RU2748426C2 (en)
SG (2) SG10201808903UA (en)
WO (1) WO2015054676A2 (en)

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