DK3137091T3 - Konjugater af modificerede antisense-oligonukleotider og anvendelse deraf til modulation af pkk-ekspression - Google Patents

Konjugater af modificerede antisense-oligonukleotider og anvendelse deraf til modulation af pkk-ekspression Download PDF

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Publication number
DK3137091T3
DK3137091T3 DK15786489.3T DK15786489T DK3137091T3 DK 3137091 T3 DK3137091 T3 DK 3137091T3 DK 15786489 T DK15786489 T DK 15786489T DK 3137091 T3 DK3137091 T3 DK 3137091T3
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Prior art keywords
conjugates
module
antisense oligonucleotides
modified antisense
pkk expression
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DK15786489.3T
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Danish (da)
English (en)
Inventor
Punit P Seth
Eric E Swayze
Susan M Freier
Huynh-Hoa Bui
Thazha P Prakash
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Ionis Pharmaceuticals Inc
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    • 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
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/555—Medicinal 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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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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    • 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
    • C12N15/1137—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 against enzymes
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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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/30—Chemical structure
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    • C12N2310/3525—MOE, methoxyethoxy

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DK15786489.3T 2014-05-01 2015-05-01 Konjugater af modificerede antisense-oligonukleotider og anvendelse deraf til modulation af pkk-ekspression DK3137091T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201461987478P 2014-05-01 2014-05-01
US201462058629P 2014-10-01 2014-10-01
US201462088459P 2014-12-05 2014-12-05
PCT/US2015/028765 WO2015168532A2 (en) 2014-05-01 2015-05-01 Compositions and methods for modulating pkk expression

Publications (1)

Publication Number Publication Date
DK3137091T3 true DK3137091T3 (da) 2021-01-25

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Country Link
US (4) US10294477B2 (https=)
EP (3) EP3862362A3 (https=)
JP (4) JP6665111B2 (https=)
KR (1) KR102366078B1 (https=)
CN (1) CN106232125B (https=)
AU (2) AU2015252917B2 (https=)
BR (1) BR112016022855B1 (https=)
CA (1) CA2943705A1 (https=)
DK (1) DK3137091T3 (https=)
ES (1) ES2849600T3 (https=)
HU (1) HUE052709T2 (https=)
IL (2) IL247862B (https=)
MX (2) MX377141B (https=)
RU (1) RU2703411C2 (https=)
WO (1) WO2015168532A2 (https=)

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