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

compostos e métodos para modular a expressão de tmprss6

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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
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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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    • C12N15/09—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • 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
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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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

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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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