EA202191210A1 - Способы лечения - Google Patents

Способы лечения

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Publication number
EA202191210A1
EA202191210A1 EA202191210A EA202191210A EA202191210A1 EA 202191210 A1 EA202191210 A1 EA 202191210A1 EA 202191210 A EA202191210 A EA 202191210A EA 202191210 A EA202191210 A EA 202191210A EA 202191210 A1 EA202191210 A1 EA 202191210A1
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EA
Eurasian Patent Office
Prior art keywords
nucleic acid
treatment methods
cell
acid conjugate
delivering
Prior art date
Application number
EA202191210A
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English (en)
Inventor
Джеймс Хейес
Ричард Дж. Холланд
Адам Джадж
Киэу Монг Лам
Original Assignee
Дженевент Сайенсез Гмбх
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Application filed by Дженевент Сайенсез Гмбх filed Critical Дженевент Сайенсез Гмбх
Publication of EA202191210A1 publication Critical patent/EA202191210A1/ru

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    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/32Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • AHUMAN NECESSITIES
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    • A61K47/545Heterocyclic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
    • A61K47/548Phosphates or phosphonates, e.g. bone-seeking
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    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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Abstract

В изобретении представлены способы и композиции для доставки нуклеиновой кислоты в целевую клетку. Способ включает приведение клетки в контакт с 1) полимером, дестабилизирующим мембрану; и 2) конъюгатом нуклеиновой кислоты. Конъюгат нуклеиновой кислоты включает в себя нацеливающий лиганд, связанный с необязательным линкером и нуклеиновой кислотой.
EA202191210A 2018-11-02 2019-11-04 Способы лечения EA202191210A1 (ru)

Applications Claiming Priority (2)

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US201862755196P 2018-11-02 2018-11-02
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