BR112022004461A2 - Conjugado de galnac-oligonucleotídeo para entrega ao fígado e método de fabricação do mesmo - Google Patents

Conjugado de galnac-oligonucleotídeo para entrega ao fígado e método de fabricação do mesmo

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Publication number
BR112022004461A2
BR112022004461A2 BR112022004461A BR112022004461A BR112022004461A2 BR 112022004461 A2 BR112022004461 A2 BR 112022004461A2 BR 112022004461 A BR112022004461 A BR 112022004461A BR 112022004461 A BR112022004461 A BR 112022004461A BR 112022004461 A2 BR112022004461 A2 BR 112022004461A2
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Brazil
Prior art keywords
liver
galnac
delivery
manufacturing
oligonucleotide conjugate
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Application number
BR112022004461A
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English (en)
Inventor
Koizumi Makoto
Iwamoto Mitsuhiro
Sekiguchi Yukiko
Ito Kentaro
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Daiichi Sankyo Co Ltd
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Publication date
Application filed by Daiichi Sankyo Co Ltd filed Critical Daiichi Sankyo Co Ltd
Publication of BR112022004461A2 publication Critical patent/BR112022004461A2/pt

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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2267/03—Animal model, e.g. for test or diseases
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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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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

CONJUGADO DE GalNAc-OLIGONUCLEOTÍDEO PARA ENTREGA AO FÍGADO E MÉTODO DE FABRICAÇÃO DO MESMO. A presente invenção provê um conjugado de um oligonucleotídeo tendo uma sequência de ácido nucleico que se espera tenha um efeito farmacológico em células parenquimais hepáticas com uma unidade de GalNAc biantenária, ou um sal farmaceuticamente aceitável do mesmo, e um medicamento ou similar contendo o mesmo como um componente ativo.
BR112022004461A 2019-09-10 2020-09-09 Conjugado de galnac-oligonucleotídeo para entrega ao fígado e método de fabricação do mesmo BR112022004461A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2019164564 2019-09-10
PCT/JP2020/034022 WO2021049504A1 (ja) 2019-09-10 2020-09-09 肝臓送達用ＧａｌＮＡｃ－オリゴヌクレオチドコンジュゲートおよび製造方法

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BR112022004461A2 true BR112022004461A2 (pt) 2022-07-19

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BR112022004461A BR112022004461A2 (pt) 2019-09-10 2020-09-09 Conjugado de galnac-oligonucleotídeo para entrega ao fígado e método de fabricação do mesmo

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US (1) US20220378920A1 (pt)
EP (1) EP4029520A1 (pt)
JP (1) JPWO2021049504A1 (pt)
KR (1) KR20220061972A (pt)
CN (1) CN114616332A (pt)
AU (1) AU2020345317A1 (pt)
BR (1) BR112022004461A2 (pt)
CA (1) CA3150715A1 (pt)
IL (1) IL291251A (pt)
TW (1) TW202123973A (pt)
WO (1) WO2021049504A1 (pt)

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WO2024075760A1 (ja) * 2022-10-05 2024-04-11 第一三共株式会社 ２分岐型ｎ－アセチル－ｄ－ガラクトサミン構造を有する化合物の製造方法

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JPWO2021049504A1 (pt) 2021-03-18
CN114616332A (zh) 2022-06-10
US20220378920A1 (en) 2022-12-01
TW202123973A (zh) 2021-07-01
CA3150715A1 (en) 2021-03-18
AU2020345317A1 (en) 2022-04-21
IL291251A (en) 2022-05-01
KR20220061972A (ko) 2022-05-13
WO2021049504A1 (ja) 2021-03-18
EP4029520A1 (en) 2022-07-20

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