KR102273926B1 - 타우 발현 조절 조성물 - Google Patents

타우 발현 조절 조성물 Download PDF

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KR102273926B1
KR102273926B1 KR1020167003896A KR20167003896A KR102273926B1 KR 102273926 B1 KR102273926 B1 KR 102273926B1 KR 1020167003896 A KR1020167003896 A KR 1020167003896A KR 20167003896 A KR20167003896 A KR 20167003896A KR 102273926 B1 KR102273926 B1 KR 102273926B1
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tau
certain embodiments
antisense
pharmaceutical composition
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KR20160034340A (ko
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홀리 코르다시에비치
에릭 이. 스웨이즈
수잔 엠. 프레이어
후인-호아 부이
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바이오젠 엠에이 인코포레이티드
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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
    • 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
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • 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
    • C07H21/02—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 ribosyl 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
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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
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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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    • C12N2310/00—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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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
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  • Biotechnology (AREA)
  • Public Health (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Thiazole And Isothizaole Compounds (AREA)
  • Pyridine Compounds (AREA)
KR1020167003896A 2013-07-19 2014-07-21 타우 발현 조절 조성물 Active KR102273926B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020217020564A KR102424991B1 (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201361856551P 2013-07-19 2013-07-19
US61/856,551 2013-07-19
US201361879621P 2013-09-18 2013-09-18
US61/879,621 2013-09-18
US201361885371P 2013-10-01 2013-10-01
US61/885,371 2013-10-01
US201462014486P 2014-06-19 2014-06-19
US62/014,486 2014-06-19
PCT/US2014/047486 WO2015010135A2 (en) 2013-07-19 2014-07-21 Compositions for modulating tau expression

Related Child Applications (1)

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KR1020217020564A Division KR102424991B1 (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물

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KR20160034340A KR20160034340A (ko) 2016-03-29
KR102273926B1 true KR102273926B1 (ko) 2021-07-09

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KR1020217020564A Active KR102424991B1 (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020237013823A Ceased KR20230062658A (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020227025324A Active KR102526525B1 (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020247016358A Ceased KR20240093742A (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020257018963A Pending KR20250091306A (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물

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KR1020237013823A Ceased KR20230062658A (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020227025324A Active KR102526525B1 (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020247016358A Ceased KR20240093742A (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물
KR1020257018963A Pending KR20250091306A (ko) 2013-07-19 2014-07-21 타우 발현 조절 조성물

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US (4) US9683235B2 (enExample)
EP (2) EP3910060A1 (enExample)
JP (4) JP6684707B2 (enExample)
KR (6) KR102273926B1 (enExample)
CN (3) CN113667667A (enExample)
AU (2) AU2014290395B2 (enExample)
BR (1) BR112016000654B1 (enExample)
CA (2) CA2918600C (enExample)
CY (1) CY1124292T1 (enExample)
DK (1) DK3022217T3 (enExample)
ES (1) ES2871533T3 (enExample)
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HU (1) HUE055144T2 (enExample)
IL (4) IL279572B2 (enExample)
LT (1) LT3022217T (enExample)
MX (2) MX379535B (enExample)
NZ (2) NZ755605A (enExample)
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RU (1) RU2735551C2 (enExample)
SI (1) SI3022217T1 (enExample)
SM (1) SMT202100371T1 (enExample)
TW (4) TWI772856B (enExample)
WO (1) WO2015010135A2 (enExample)

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