AU2017326372B2 - Modified oligonucleotides and methods of use - Google Patents

Modified oligonucleotides and methods of use Download PDF

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Publication number
AU2017326372B2
AU2017326372B2 AU2017326372A AU2017326372A AU2017326372B2 AU 2017326372 B2 AU2017326372 B2 AU 2017326372B2 AU 2017326372 A AU2017326372 A AU 2017326372A AU 2017326372 A AU2017326372 A AU 2017326372A AU 2017326372 B2 AU2017326372 B2 AU 2017326372B2
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dap
oligonucleotide
domain
nucleosides
modified
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AU2017326372A1 (en
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Leonid Beigelman
Sergei Gryaznov
Jin Hong
Saúl Martínez-Monero
Vivek Rajwanshi
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Janssen Biopharma Inc
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Janssen Biopharma Inc
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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 against viruses
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
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    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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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    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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AU2017326372A 2016-09-14 2017-09-14 Modified oligonucleotides and methods of use Active AU2017326372B2 (en)

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US201662394739P 2016-09-14 2016-09-14
US201662394738P 2016-09-14 2016-09-14
US62/394,738 2016-09-14
US62/394,739 2016-09-14
PCT/US2017/051644 WO2018053185A1 (en) 2016-09-14 2017-09-14 Modified oligonucleotides and methods of use

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EP (1) EP3512949A1 (OSRAM)
JP (3) JP6970187B2 (OSRAM)
KR (3) KR102533038B1 (OSRAM)
CN (1) CN109952378B (OSRAM)
AU (2) AU2017326372B2 (OSRAM)
BR (1) BR112019004911A2 (OSRAM)
CA (1) CA3037042A1 (OSRAM)
IL (2) IL315071A (OSRAM)
MA (1) MA46422A (OSRAM)
MX (2) MX2019002960A (OSRAM)
TW (1) TWI769177B (OSRAM)
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WO2022153358A1 (ja) 2021-01-12 2022-07-21 日本電気株式会社 秘密計算システム、秘密計算サーバ装置、秘密計算方法および秘密計算プログラム
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IL315071A (en) 2024-10-01
MA46422A (fr) 2019-07-24
IL265359B1 (en) 2024-10-01
KR102533038B1 (ko) 2023-05-17
US20180135054A1 (en) 2018-05-17
EP3512949A1 (en) 2019-07-24
CN109952378A (zh) 2019-06-28
US20250092400A1 (en) 2025-03-20
IL265359B2 (en) 2025-02-01
RU2019111020A (ru) 2020-10-15
JP2019529406A (ja) 2019-10-17
AU2017326372A1 (en) 2019-04-04
KR20240010761A (ko) 2024-01-24
US12077757B2 (en) 2024-09-03
MX2023014145A (es) 2023-12-13
RU2019111020A3 (OSRAM) 2021-06-22
WO2018053185A1 (en) 2018-03-22
TW201825103A (zh) 2018-07-16
BR112019004911A2 (pt) 2019-06-25
MX2019002960A (es) 2019-09-18
KR20230033736A (ko) 2023-03-08
JP2022023936A (ja) 2022-02-08
JP6970187B2 (ja) 2021-11-24
CN109952378B (zh) 2024-09-17
CA3037042A1 (en) 2018-03-22
KR20190047025A (ko) 2019-05-07
JP7170820B2 (ja) 2022-11-14
JP2023011793A (ja) 2023-01-24
IL265359A (en) 2019-05-30
KR102627418B1 (ko) 2024-01-19
US10793859B2 (en) 2020-10-06
JP7478210B2 (ja) 2024-05-02
TWI769177B (zh) 2022-07-01
AU2024201485A1 (en) 2024-03-28
US20210040483A1 (en) 2021-02-11

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