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AR102257A1 - Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa)) - Google Patents

Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa))

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Publication number
AR102257A1
AR102257A1 ARP150103309A ARP150103309A AR102257A1 AR 102257 A1 AR102257 A1 AR 102257A1 AR P150103309 A ARP150103309 A AR P150103309A AR P150103309 A ARP150103309 A AR P150103309A AR 102257 A1 AR102257 A1 AR 102257A1
Authority
AR
Argentina
Prior art keywords
seq
nucleotides
double stranded
hao1
cell
Prior art date
Application number
ARP150103309A
Other languages
English (en)
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR102257A1 publication Critical patent/AR102257A1/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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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    • C12N15/09—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • 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

Reivindicación 1: Un agente de ARNi bicatenario capaz de inhibir la expresión de HAO1 en una célula, en donde dicho agente de ARNi bicatenario comprende una cadena sentido y una cadena antisentido que forma una región bicatenaria, en donde dicha cadena sentido comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de cualquiera de las secuencias de nucleótidos de SEQ ID Nº 1, SEQ ID Nº 2, SEQ ID Nº 5 o SEQ ID Nº 6 y dicha cadena antisentido comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de cualquiera de las secuencias de nucleótidos de SEQ ID Nº 3, SEQ ID Nº 4, SEQ ID Nº 7 o SEQ ID Nº 8; en donde sustancialmente todos los nucleótidos de dicha cadena sentido y sustancialmente todos los nucleótidos de dicha cadena antisentido son nucleótidos modificados, y en donde dicha cadena sentido se conjuga con un ligando unido al término 3. Reivindicación 68: Una composición farmacéutica que comprende el agente de ARNi bicatenario de acuerdo con cualquiera de las reivindicaciones 1, 7, 56 y 58 - 64 o el agente polinucleótido antisentido modificado de acuerdo con la reivindicación 65 o el vector de acuerdo con la reivindicación 66. Reivindicación 74: Un método de inhibición de la expresión de HAO1 en una célula, que comprende: (a) poner en contacto la célula con el agente de ARNi bicatenario de acuerdo con cualquiera de las reivindicaciones 1, 7, 56 y 58 - 64 o el agente polinucleótido antisentido modificado de acuerdo con la reivindicación 65 o el vector de acuerdo con la reivindicación 66 o a composición farmacéutica de acuerdo con cualquiera de las reivindicaciones 68 - 73; y (b) mantener la célula producida en la etapa (a) durante un tiempo suficiente para obtener degradación del trascripto de mARN de un gen HAO1, inhibiendo así la expresión del gen HAO1 en la célula. Reivindicación 78: Un método de tratamiento de un sujeto que tiene un trastorno asociado con HAO1, que comprende la administración al sujeto de una cantidad terapéuticamente efectiva del agente de ARNi bicatenario de acuerdo con cualquiera de las reivindicaciones 1, 7, 56 y 58 - 64 o el agente polinucleótido antisentido modificado de acuerdo con la reivindicación 65 o el vector de acuerdo con la reivindicación 66 o una composición farmacéutica de acuerdo con cualquiera de las reivindicaciones 68 - 73, tratando así a dicho sujeto.
ARP150103309A 2014-10-10 2015-10-13 Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa)) AR102257A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462062751P 2014-10-10 2014-10-10

Publications (1)

Publication Number Publication Date
AR102257A1 true AR102257A1 (es) 2017-02-15

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
ARP150103309A AR102257A1 (es) 2014-10-10 2015-10-13 Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa))

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AR (1) AR102257A1 (es)
MA (1) MA41049B1 (es)

Also Published As

Publication number Publication date
MA41049B1 (fr) 2022-02-28
MA41049A (fr) 2017-08-15

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