KR20160062069A - Ｃ９ｏｒｆ７２ 발현을 조절하기 위한 조성물 - Google Patents

Ｃ９ｏｒｆ７２ 발현을 조절하기 위한 조성물 Download PDF

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Publication number
KR20160062069A
KR20160062069A KR1020167010455A KR20167010455A KR20160062069A KR 20160062069 A KR20160062069 A KR 20160062069A KR 1020167010455 A KR1020167010455 A KR 1020167010455A KR 20167010455 A KR20167010455 A KR 20167010455A KR 20160062069 A KR20160062069 A KR 20160062069A
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South Korea
Prior art keywords
certain embodiments
c9orf72
antisense
nucleoside
compound
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Abandoned
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KR1020167010455A
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English (en)
Korean (ko)
Inventor
수잔 엠. 프레이어
프랭크 리고
프리얌 싱
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아이오니스 파마수티컬즈, 인코포레이티드
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Publication of KR20160062069A publication Critical patent/KR20160062069A/ko
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
    • A61P25/14—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
    • A61P25/28—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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical 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
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
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    • 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
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    • 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
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    • 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
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    • 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 ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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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  • Pharmacology & Pharmacy (AREA)
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  • Wood Science & Technology (AREA)
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  • Urology & Nephrology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Food Science & Technology (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
KR1020167010455A 2013-10-11 2014-10-11 Ｃ９ｏｒｆ７２ 발현을 조절하기 위한 조성물 Abandoned KR20160062069A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201361890108P 2013-10-11 2013-10-11
US61/890,108 2013-10-11
US201361891313P 2013-10-15 2013-10-15
US61/891,313 2013-10-15
US201361919540P 2013-12-20 2013-12-20
US61/919,540 2013-12-20
US201461927903P 2014-01-15 2014-01-15
US61/927,903 2014-01-15
US201461980502P 2014-04-16 2014-04-16
US61/980,502 2014-04-16
PCT/US2014/060194 WO2015054676A2 (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression

Publications (1)

Publication Number Publication Date
KR20160062069A true KR20160062069A (ko) 2016-06-01

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KR1020167010455A Abandoned KR20160062069A (ko) 2013-10-11 2014-10-11 Ｃ９ｏｒｆ７２ 발현을 조절하기 위한 조성물

Country Status (16)

Country Link
US (3) US10221414B2 (enExample)
EP (2) EP4166667A3 (enExample)
JP (2) JP6679476B2 (enExample)
KR (1) KR20160062069A (enExample)
CN (2) CN112080502A (enExample)
AU (2) AU2014331652B2 (enExample)
BR (1) BR112016007751A2 (enExample)
CA (1) CA2926408A1 (enExample)
CL (3) CL2016000839A1 (enExample)
IL (1) IL244759A0 (enExample)
MX (2) MX2016004651A (enExample)
MY (1) MY192689A (enExample)
PH (1) PH12016500603A1 (enExample)
RU (1) RU2748426C2 (enExample)
SG (2) SG10201808903UA (enExample)
WO (1) WO2015054676A2 (enExample)

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