BR112022024420A2 - Composições de irna de xantina desidrogenase (xdh) e métodos de uso das mesmas - Google Patents

Composições de irna de xantina desidrogenase (xdh) e métodos de uso das mesmas

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Publication number
BR112022024420A2
BR112022024420A2 BR112022024420A BR112022024420A BR112022024420A2 BR 112022024420 A2 BR112022024420 A2 BR 112022024420A2 BR 112022024420 A BR112022024420 A BR 112022024420A BR 112022024420 A BR112022024420 A BR 112022024420A BR 112022024420 A2 BR112022024420 A2 BR 112022024420A2
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BR
Brazil
Prior art keywords
xdh
methods
xanthine dehydrogenase
irna compositions
gene
Prior art date
Application number
BR112022024420A
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English (en)
Inventor
D Mcininch James
Liu Jingxuan
K Schlegel Mark
Castoreno Adam
Borodovsky Anna
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.)
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of BR112022024420A2 publication Critical patent/BR112022024420A2/pt

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12Y107/00—Oxidoreductases acting on other nitrogenous compounds as donors (1.7)
    • C12Y107/02—Oxidoreductases acting on other nitrogenous compounds as donors (1.7) with a cytochrome as acceptor (1.7.2)
    • C12Y107/02002—Nitrite reductase (cytochrome; ammonia-forming)(1.7.2.2)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y117/00—Oxidoreductases acting on CH or CH2 groups (1.17)
    • C12Y117/01—Oxidoreductases acting on CH or CH2 groups (1.17) with NAD+ or NADP+ as acceptor (1.17.1)
    • C12Y117/01004—Xanthine dehydrogenase (1.17.1.4)

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
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  • Plant Pathology (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

COMPOSIÇÕES DE IRNA DE XANTINA DESIDROGENASE (XDH) E MÉTODOS DE USO DAS MESMAS. A presente invenção refere-se a agentes de RNAi, por exemplo, agentes de dsRNA, visando o gene da xantina desidrogenase (XDH). A invenção também se refere a métodos de uso de tais agentes de RNAi para inibir a expressão de um gene XDH e a métodos de tratamento ou prevenção de uma doença associada a XDH em um indivíduo.
BR112022024420A 2020-06-18 2021-06-17 Composições de irna de xantina desidrogenase (xdh) e métodos de uso das mesmas BR112022024420A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063040587P 2020-06-18 2020-06-18
US202163153983P 2021-02-26 2021-02-26
PCT/US2021/037748 WO2021257782A1 (en) 2020-06-18 2021-06-17 XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
BR112022024420A2 true BR112022024420A2 (pt) 2023-01-17

Family

ID=76859777

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022024420A BR112022024420A2 (pt) 2020-06-18 2021-06-17 Composições de irna de xantina desidrogenase (xdh) e métodos de uso das mesmas

Country Status (12)

Country Link
US (2) US11326166B1 (pt)
EP (1) EP4168546A1 (pt)
JP (1) JP2023530461A (pt)
KR (1) KR20230026455A (pt)
CN (1) CN116209760A (pt)
AU (1) AU2021292296A1 (pt)
BR (1) BR112022024420A2 (pt)
CA (1) CA3184289A1 (pt)
IL (1) IL299063A (pt)
MX (1) MX2022015149A (pt)
TW (1) TW202214856A (pt)
WO (1) WO2021257782A1 (pt)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022263931A2 (en) 2021-06-17 2022-12-22 Oslo Universitetssykehus Hf Compositions and methods for treating cancer
WO2022269346A2 (en) * 2021-06-21 2022-12-29 Horizon Therapeutics Ireland Dac Compositions and methods of modulating xanthine dehydrogenase
US11549112B1 (en) 2021-06-21 2023-01-10 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
WO2023202686A1 (zh) * 2022-04-22 2023-10-26 上海舶望制药有限公司 用于抑制黄嘌呤脱氢酶(xdh)的组合物和方法
WO2024140596A1 (zh) * 2022-12-26 2024-07-04 大睿生物医药科技(上海)有限公司 调控XDH基因活性的siRNA分子

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