UY36357A - 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
UY36357A
UY36357A UY0001036357A UY36357A UY36357A UY 36357 A UY36357 A UY 36357A UY 0001036357 A UY0001036357 A UY 0001036357A UY 36357 A UY36357 A UY 36357A UY 36357 A UY36357 A UY 36357A
Authority
UY
Uruguay
Prior art keywords
oxidase
methods
hydroxyacyde
glycolate
inhibition
Prior art date
Application number
UY0001036357A
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English (en)
Inventor
Querbes William
Fitzgerald Kevin
Bettencourt Brian
Liebow Abigail
David V Erbe
Original Assignee
Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of UY36357A publication Critical patent/UY36357A/es

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    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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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    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12YENZYMES
    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03Oxidoreductases 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)

Abstract

La invención se refiere a agentes de ARNi, por ejemplo, agentes de ARNi bicatenarios, que se dirigen al gen HAO1 y métodos de uso de tales agentes de ARNi para inhibir la expresión de HAO1 y métodos de tratamiento de sujetos que tienen, por ejemplo, PH1.
UY0001036357A 2014-10-10 2015-10-13 Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa)) UY36357A (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)

Publication Number Publication Date
UY36357A true UY36357A (es) 2016-11-30

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UY0001036357A UY36357A (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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US (3) US10478500B2 (es)
EP (2) EP4039809A1 (es)
JP (3) JP6836988B2 (es)
KR (2) KR102558604B1 (es)
CN (2) CN108064154B (es)
AU (2) AU2015330726B9 (es)
BR (1) BR112017006469A2 (es)
CA (2) CA3187050A1 (es)
CO (1) CO2017004728A2 (es)
CR (1) CR20170190A (es)
CY (1) CY1125364T1 (es)
DK (1) DK3204015T3 (es)
DO (1) DOP2017000094A (es)
EA (1) EA037115B1 (es)
EC (1) ECSP17028310A (es)
ES (1) ES2905889T3 (es)
HR (1) HRP20211971T1 (es)
HU (2) HUE057327T2 (es)
IL (3) IL310466A (es)
JO (2) JOP20200115A1 (es)
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MX (2) MX2017004634A (es)
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TN (1) TN2017000125A1 (es)
TW (3) TWI755351B (es)
UA (1) UA126266C2 (es)
UY (1) UY36357A (es)
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