AU2019218987B2 - Modified compounds and uses thereof - Google Patents

Modified compounds and uses thereof Download PDF

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Publication number
AU2019218987B2
AU2019218987B2 AU2019218987A AU2019218987A AU2019218987B2 AU 2019218987 B2 AU2019218987 B2 AU 2019218987B2 AU 2019218987 A AU2019218987 A AU 2019218987A AU 2019218987 A AU2019218987 A AU 2019218987A AU 2019218987 B2 AU2019218987 B2 AU 2019218987B2
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Australia
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oligomeric compound
sugar moiety
modified
central region
nucleoside
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AU2019218987A1 (en
Inventor
Stanley T. Crooke
Xue-Hai Liang
Michael T. Migawa
Michael OESTERGAARD
Punit Seth
Wen SHEN
Eric E. Swayze
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals 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
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    • C12N15/09—Recombinant DNA-technology
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    • 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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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • 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
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2019218987A 2018-02-12 2019-02-12 Modified compounds and uses thereof Active AU2019218987B2 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201862629632P 2018-02-12 2018-02-12
US62/629,632 2018-02-12
US201862686632P 2018-06-18 2018-06-18
US62/686,632 2018-06-18
US201862713698P 2018-08-02 2018-08-02
US62/713,698 2018-08-02
US201862739088P 2018-09-28 2018-09-28
US62/739,088 2018-09-28
US201862742265P 2018-10-05 2018-10-05
US62/742,265 2018-10-05
US201862746511P 2018-10-16 2018-10-16
US62/746,511 2018-10-16
PCT/US2019/017725 WO2019157531A1 (en) 2018-02-12 2019-02-12 Modified compounds and uses thereof

Publications (2)

Publication Number Publication Date
AU2019218987A1 AU2019218987A1 (en) 2020-07-23
AU2019218987B2 true AU2019218987B2 (en) 2025-04-24

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AU2019218987A Active AU2019218987B2 (en) 2018-02-12 2019-02-12 Modified compounds and uses thereof

Country Status (6)

Country Link
US (4) US11332733B2 (https=)
EP (1) EP3752612A4 (https=)
JP (1) JP7317029B2 (https=)
AU (1) AU2019218987B2 (https=)
CA (1) CA3090901A1 (https=)
WO (1) WO2019157531A1 (https=)

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