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BR112017015864A2 - compostos e métodos para modular a expressão de tmprss6 - Google Patents

compostos e métodos para modular a expressão de tmprss6

Info

Publication number
BR112017015864A2
BR112017015864A2 BR112017015864-7A BR112017015864A BR112017015864A2 BR 112017015864 A2 BR112017015864 A2 BR 112017015864A2 BR 112017015864 A BR112017015864 A BR 112017015864A BR 112017015864 A2 BR112017015864 A2 BR 112017015864A2
Authority
BR
Brazil
Prior art keywords
compounds
methods
tmprss6 expression
modulate
individual
Prior art date
Application number
BR112017015864-7A
Other languages
English (en)
Inventor
Guo Shuling
Aghajan Mariam
E. Swayze Eric
Original Assignee
Ionis Pharmaceuticals, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals, Inc. filed Critical Ionis Pharmaceuticals, Inc.
Publication of BR112017015864A2 publication Critical patent/BR112017015864A2/pt

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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
    • C12N15/1137Non-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
    • 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
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • AHUMAN NECESSITIES
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    • A61P7/06Antianaemics
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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
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    • 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
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    • C12N15/1138Non-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 receptors or cell surface proteins
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    • C12N2310/32Chemical structure of the sugar
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Abstract

divulgados neste documento são composições e compostos compreendendo oligonucleotídeos modificados para modular tmprss6 e modular uma doença, distúrbio e/ou condição de acúmulo de ferro em um indivíduo em necessidade do mesmo. doenças de acúmulo de ferro em um indivíduo, tais como policitemia, hemocromatose ou ß-talassemia podem ser tratadas, amenizadas, atrasadas ou evitadas com a administração de compostos antissenso direcionados para tmprss6.
BR112017015864-7A 2015-04-03 2016-04-04 compostos e métodos para modular a expressão de tmprss6 BR112017015864A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562142986P 2015-04-03 2015-04-03
US62/142,986 2015-04-03
PCT/US2016/025883 WO2016161429A1 (en) 2015-04-03 2016-04-04 Compounds and methods for modulating tmprss6 expression

Publications (1)

Publication Number Publication Date
BR112017015864A2 true BR112017015864A2 (pt) 2018-07-31

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US (4) US10415038B2 (pt)
EP (1) EP3277817A4 (pt)
JP (3) JP6833705B2 (pt)
KR (1) KR102539587B1 (pt)
CN (1) CN107429250B (pt)
AU (1) AU2016244106B2 (pt)
BR (1) BR112017015864A2 (pt)
CA (1) CA2978100C (pt)
HK (1) HK1249140A1 (pt)
IL (2) IL253509B2 (pt)
MX (1) MX2017012426A (pt)
RU (1) RU2017137410A (pt)
WO (1) WO2016161429A1 (pt)

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