TWI657819B - 用於調節τ蛋白表現之組合物 - Google Patents

用於調節τ蛋白表現之組合物 Download PDF

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TWI657819B
TWI657819B TW103124846A TW103124846A TWI657819B TW I657819 B TWI657819 B TW I657819B TW 103124846 A TW103124846 A TW 103124846A TW 103124846 A TW103124846 A TW 103124846A TW I657819 B TWI657819 B TW I657819B
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compound
dna
modified
antisense
certain embodiments
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TW201503892A (zh
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賀利 利達席微茲
艾瑞克Ｅ 史瓦瑟
蘇珊Ｍ 弗瑞爾
賀恩 荷亞 比伊
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美商Ionis製藥公司
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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
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    • A—HUMAN NECESSITIES
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    • 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
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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
    • C07H21/02—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
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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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
    • 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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  • 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)
TW103124846A 2013-07-19 2014-07-18 用於調節τ蛋白表現之組合物 TWI657819B (zh)

Applications Claiming Priority (8)

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

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TW201503892A TW201503892A (zh) 2015-02-01
TWI657819B true TWI657819B (zh) 2019-05-01

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TW111127593A TW202246503A (zh) 2013-07-19 2014-07-18 用於調節τ蛋白表現之組合物
TW108102121A TWI702046B (zh) 2013-07-19 2014-07-18 用於調節τ蛋白表現之組合物
TW109127080A TWI772856B (zh) 2013-07-19 2014-07-18 用於調節τ蛋白表現之組合物
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TW109127080A TWI772856B (zh) 2013-07-19 2014-07-18 用於調節τ蛋白表現之組合物

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US (4) US9683235B2 (enExample)
EP (2) EP3022217B1 (enExample)
JP (4) JP6684707B2 (enExample)
KR (6) KR20230062658A (enExample)
CN (3) CN108064227B (enExample)
AU (2) AU2014290395B2 (enExample)
BR (1) BR112016000654B1 (enExample)
CA (2) CA3149282C (enExample)
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DK (1) DK3022217T3 (enExample)
ES (1) ES2871533T3 (enExample)
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HU (1) HUE055144T2 (enExample)
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LT (1) LT3022217T (enExample)
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PL (1) PL3022217T3 (enExample)
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RS (1) RS62054B1 (enExample)
RU (1) RU2735551C2 (enExample)
SI (1) SI3022217T1 (enExample)
SM (1) SMT202100371T1 (enExample)
TW (4) TW202246503A (enExample)
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TWI702046B (zh) * 2013-07-19 2020-08-21 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
US12091662B2 (en) 2013-07-19 2024-09-17 Biogen Ma Inc. Compositions for modulating tau expression

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