AU2014259953B2 - Compounds and methods for enhanced cellular uptake - Google Patents

Compounds and methods for enhanced cellular uptake Download PDF

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AU2014259953B2
AU2014259953B2 AU2014259953A AU2014259953A AU2014259953B2 AU 2014259953 B2 AU2014259953 B2 AU 2014259953B2 AU 2014259953 A AU2014259953 A AU 2014259953A AU 2014259953 A AU2014259953 A AU 2014259953A AU 2014259953 B2 AU2014259953 B2 AU 2014259953B2
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certain embodiments
modified oligonucleotide
modified
compound
nucleoside
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AU2014259953A1 (en
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Balkrishen Bhat
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Regulus Therapeutics Inc
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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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    • C12N2310/141—MicroRNAs, miRNAs
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/999—Small molecules not provided for elsewhere

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2014259953A 2013-05-01 2014-04-30 Compounds and methods for enhanced cellular uptake Active AU2014259953B2 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201361818447P 2013-05-01 2013-05-01
US201361818441P 2013-05-01 2013-05-01
US61/818,447 2013-05-01
US61/818,441 2013-05-01
US201361822115P 2013-05-10 2013-05-10
US61/822,115 2013-05-10
US201361895708P 2013-10-25 2013-10-25
US61/895,708 2013-10-25
PCT/US2014/036136 WO2014179445A1 (en) 2013-05-01 2014-04-30 Compounds and methods for enhanced cellular uptake

Publications (2)

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AU2014259953A1 AU2014259953A1 (en) 2015-11-05
AU2014259953B2 true AU2014259953B2 (en) 2020-07-02

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US (3) US9506030B2 (enExample)
EP (1) EP2992096B1 (enExample)
JP (1) JP6649248B2 (enExample)
CN (1) CN105164261B (enExample)
AU (1) AU2014259953B2 (enExample)
CA (1) CA2909868C (enExample)
IL (1) IL241973B (enExample)
NZ (1) NZ630921A (enExample)
TW (1) TWI680767B (enExample)
WO (1) WO2014179445A1 (enExample)

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