BR112022019889A2 - Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente - Google Patents

Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente

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Publication number
BR112022019889A2
BR112022019889A2 BR112022019889A BR112022019889A BR112022019889A2 BR 112022019889 A2 BR112022019889 A2 BR 112022019889A2 BR 112022019889 A BR112022019889 A BR 112022019889A BR 112022019889 A BR112022019889 A BR 112022019889A BR 112022019889 A2 BR112022019889 A2 BR 112022019889A2
Authority
BR
Brazil
Prior art keywords
genetically modified
modified oligonucleotides
targeted inhibition
activity
disclosed
Prior art date
Application number
BR112022019889A
Other languages
English (en)
Portuguese (pt)
Inventor
Place Robert
Saleh Anthony
Williams Tishan
Original Assignee
Mirecule 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 Mirecule Inc filed Critical Mirecule Inc
Publication of BR112022019889A2 publication Critical patent/BR112022019889A2/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
    • 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
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P31/12—Antivirals
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • G16B15/30—Drug targeting using structural data; Docking or binding prediction
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    • C12N2310/35—Nature of the modification
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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BR112022019889A 2020-04-02 2021-04-02 Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente BR112022019889A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063004045P 2020-04-02 2020-04-02
PCT/US2021/025639 WO2021203043A2 (en) 2020-04-02 2021-04-02 Targeted inhibition using engineered oligonucleotides

Publications (1)

Publication Number Publication Date
BR112022019889A2 true BR112022019889A2 (pt) 2023-03-07

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BR112022019889A BR112022019889A2 (pt) 2020-04-02 2021-04-02 Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente

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Country Link
US (5) US20230132602A1 (https=)
EP (1) EP4127175A4 (https=)
JP (1) JP2023521042A (https=)
KR (1) KR20230023612A (https=)
CN (1) CN116096890A (https=)
AU (1) AU2021246530A1 (https=)
BR (1) BR112022019889A2 (https=)
CA (1) CA3174286A1 (https=)
CO (1) CO2022015141A2 (https=)
IL (1) IL296957A (https=)
MX (1) MX2022012265A (https=)
WO (1) WO2021203043A2 (https=)

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US20230407302A1 (en) * 2022-02-04 2023-12-21 New York University Novel efficacious microrna-30c analogs reduce apolipoprotein b secretion in human liver cells
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