EA202190418A1 - Мышечно-специфические комлексы и их применение в лечении прогрессирующей оссифицирующей фибродисплазии - Google Patents

Мышечно-специфические комлексы и их применение в лечении прогрессирующей оссифицирующей фибродисплазии

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EA202190418A1
EA202190418A1 EA202190418A EA202190418A EA202190418A1 EA 202190418 A1 EA202190418 A1 EA 202190418A1 EA 202190418 A EA202190418 A EA 202190418A EA 202190418 A EA202190418 A EA 202190418A EA 202190418 A1 EA202190418 A1 EA 202190418A1
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Prior art keywords
muscle
ossified
fibrodysplasia
progressive
treatment
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EA202190418A
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English (en)
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Ромеш Р. Субраманиян
Мохаммед Т. Катанани
Тимоти Уиден
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Дайн Терапьютикс, Инк.
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Publication of EA202190418A1 publication Critical patent/EA202190418A1/ru

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Abstract

Аспекты настоящего изобретения относятся к комплексам, содержащим мышечно-специфическое средство, ковалентно связанное с молекулярной нагрузкой. В некоторых вариантах осуществления мышечно-специфическое средство специфически связывается с интернализующимся рецептором клеточной поверхности на мышечных клетках. В некоторых вариантах осуществления молекулярная нагрузка ингибирует активность ACVR1. В некоторых вариантах осуществления молекулярная нагрузка является олигонуклеотидом, таким как антисмысловой олигонуклеотид или олигонуклеотид РНКи.
EA202190418A 2018-08-02 2019-08-02 Мышечно-специфические комлексы и их применение в лечении прогрессирующей оссифицирующей фибродисплазии EA202190418A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862713973P 2018-08-02 2018-08-02
PCT/US2019/044947 WO2020028831A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating fibrodysplasia ossificans progressiva

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EA202190418A1 true EA202190418A1 (ru) 2021-06-23

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CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
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