MX2021005224A - Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1). - Google Patents

Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1).

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Publication number
MX2021005224A
MX2021005224A MX2021005224A MX2021005224A MX2021005224A MX 2021005224 A MX2021005224 A MX 2021005224A MX 2021005224 A MX2021005224 A MX 2021005224A MX 2021005224 A MX2021005224 A MX 2021005224A MX 2021005224 A MX2021005224 A MX 2021005224A
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Mexico
Prior art keywords
hao1
oxidase
methods
inhibition
compositions
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MX2021005224A
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English (en)
Inventor
Kevin Fitzgerald
Brian Bettencourt
William Querbes
David V Erbe
Abigail Liebow
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Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of MX2021005224A publication Critical patent/MX2021005224A/es

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    • 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • 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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    • C12Y101/03—Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
    • C12Y101/03015—(S)-2-Hydroxy-acid oxidase (1.1.3.15)
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Abstract

La invención se refiere a agentes de iARN, por ejemplo, agentes de iARN bicatenarios, que tienen como diana el gen HAO1, y a métodos de uso de tales agentes de iARN para inhibir la expresión de HAO1 y a métodos para tratar sujetos que padecen, por ejemplo, PH1. En la presente se describen agentes de iARN bicatenarios que inhiben la expresión de un gen HAO1 en una célula, tal como una célula en un sujeto, por ejemplo, un mamífero, tal como un ser humano que padece un trastorno asociado con HAO1 y usos de tales agentes de iARN bicatenarios. En ciertos aspectos de la invención, sustancialmente todos los nucleótidos de un iARN de la invención están modificados.
MX2021005224A 2014-10-10 2017-04-07 Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1). MX2021005224A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462062751P 2014-10-10 2014-10-10
US201562147976P 2015-04-15 2015-04-15
US201562214602P 2015-09-04 2015-09-04

Publications (1)

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MX2021005224A true MX2021005224A (es) 2021-06-18

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MX2017004634A MX2017004634A (es) 2014-10-10 2015-10-09 Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1).
MX2021005224A MX2021005224A (es) 2014-10-10 2017-04-07 Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1).

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EP (2) EP3204015B1 (es)
JP (3) JP6836988B2 (es)
KR (2) KR20230113654A (es)
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BR (1) BR112017006469A2 (es)
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JP2023503644A (ja) * 2019-11-27 2023-01-31 ディー・ティー・エックス・ファーマ・インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための化合物および方法
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