KR101774526B1 - 헌팅틴 발현의 조절 - Google Patents

헌팅틴 발현의 조절 Download PDF

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Publication number
KR101774526B1
KR101774526B1 KR1020127009137A KR20127009137A KR101774526B1 KR 101774526 B1 KR101774526 B1 KR 101774526B1 KR 1020127009137 A KR1020127009137 A KR 1020127009137A KR 20127009137 A KR20127009137 A KR 20127009137A KR 101774526 B1 KR101774526 B1 KR 101774526B1
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South Korea
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delete delete
certain embodiments
oligonucleotide
huntingtin
isis
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KR20120120511A (ko
Inventor
젠 흉
씨. 프랭크 벤네트
수잔 엠 프레이어
홀리 코다세위츠
리사 스탄네크
돈 더블유. 클리브랜드
셍 에이치. 쳉
라먀 쉬하부딘
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아이오니스 파마수티컬즈, 인코포레이티드
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    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/20—Hypnotics; Sedatives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/22—Anxiolytics
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    • 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
    • A61P3/00—Drugs for disorders of the metabolism
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    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • C—CHEMISTRY; METALLURGY
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    • 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
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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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 ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • 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

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KR1020127009137A 2009-09-11 2010-09-10 헌팅틴 발현의 조절 Active KR101774526B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US24185309P 2009-09-11 2009-09-11
US61/241,853 2009-09-11
PCT/US2010/048532 WO2011032045A1 (en) 2009-09-11 2010-09-10 Modulation of huntingtin expression

Related Child Applications (1)

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KR1020177024113A Division KR20170102069A (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절

Publications (2)

Publication Number Publication Date
KR20120120511A KR20120120511A (ko) 2012-11-01
KR101774526B1 true KR101774526B1 (ko) 2017-09-04

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Application Number Title Priority Date Filing Date
KR1020127009137A Active KR101774526B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020177024113A Ceased KR20170102069A (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020197026749A Active KR102173836B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020187037590A Active KR102175230B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020207031044A Active KR102279458B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절

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KR1020177024113A Ceased KR20170102069A (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020197026749A Active KR102173836B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020187037590A Active KR102175230B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절
KR1020207031044A Active KR102279458B1 (ko) 2009-09-11 2010-09-10 헌팅틴 발현의 조절

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Country Link
US (8) US8906873B2 (enExample)
EP (3) EP3626823A1 (enExample)
JP (5) JP5809146B2 (enExample)
KR (5) KR101774526B1 (enExample)
CN (3) CN111705058A (enExample)
AU (4) AU2010292003B2 (enExample)
BR (1) BR112012005448B1 (enExample)
CA (2) CA2931725C (enExample)
DK (1) DK2475675T3 (enExample)
ES (2) ES2615534T3 (enExample)
HU (1) HUE033113T2 (enExample)
IL (4) IL218482A (enExample)
MX (3) MX380175B (enExample)
NZ (3) NZ599032A (enExample)
PL (1) PL2475675T3 (enExample)
PT (1) PT2475675T (enExample)
RU (2) RU2562861C2 (enExample)
SI (1) SI2475675T1 (enExample)
WO (1) WO2011032045A1 (enExample)

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