EA202190418A1 - Мышечно-специфические комлексы и их применение в лечении прогрессирующей оссифицирующей фибродисплазии - Google Patents
Мышечно-специфические комлексы и их применение в лечении прогрессирующей оссифицирующей фибродисплазииInfo
- Publication number
- 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
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- muscle
- ossified
- fibrodysplasia
- progressive
- treatment
- Prior art date
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- 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
- A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6835—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6849—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
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- A—HUMAN NECESSITIES
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- A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
- A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
- A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
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- A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
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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.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
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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 |
Publications (1)
Publication Number | Publication Date |
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EA202190418A1 true EA202190418A1 (ru) | 2021-06-23 |
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EA202190418A EA202190418A1 (ru) | 2018-08-02 | 2019-08-02 | Мышечно-специфические комлексы и их применение в лечении прогрессирующей оссифицирующей фибродисплазии |
Country Status (9)
Country | Link |
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US (1) | US20210322563A1 (ru) |
EP (1) | EP3829645A4 (ru) |
JP (1) | JP2021533196A (ru) |
KR (1) | KR20210086600A (ru) |
CN (1) | CN113164620A (ru) |
CA (1) | CA3108328A1 (ru) |
EA (1) | EA202190418A1 (ru) |
IL (1) | IL280534A (ru) |
WO (1) | WO2020028831A1 (ru) |
Families Citing this family (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
CA3108289A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
WO2021142260A1 (en) * | 2020-01-10 | 2021-07-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of acvr1 |
US20230330562A1 (en) * | 2020-09-03 | 2023-10-19 | Dyne Therapeutics, Inc. | Methods of preparing protein-oligonucleotide complexes |
US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11672872B2 (en) | 2021-07-09 | 2023-06-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor antibody and uses thereof |
US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
US11827702B2 (en) | 2021-09-01 | 2023-11-28 | Biogen Ma Inc. | Anti-transferrin receptor antibodies and uses thereof |
US11931421B2 (en) | 2022-04-15 | 2024-03-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
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US8859752B2 (en) * | 2006-04-18 | 2014-10-14 | The Trustees Of The University Of Pennsylvania | SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP) |
EP4324480A3 (en) * | 2013-05-20 | 2024-05-08 | F. Hoffmann-La Roche AG | Anti-transferrin receptor antibodies and methods of use |
US20160075772A1 (en) * | 2014-09-12 | 2016-03-17 | Regeneron Pharmaceuticals, Inc. | Treatment of Fibrodysplasia Ossificans Progressiva |
WO2017221883A1 (ja) * | 2016-06-20 | 2017-12-28 | 武田薬品工業株式会社 | 抗体-薬物コンジュゲート |
JP2020505330A (ja) * | 2017-01-06 | 2020-02-20 | アビディティー バイオサイエンシーズ エルエルシー | エクソンスキッピングを誘導する核酸ポリペプチド組成物および方法 |
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- 2019-08-02 WO PCT/US2019/044947 patent/WO2020028831A1/en active Application Filing
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- 2019-08-02 CN CN201980064589.2A patent/CN113164620A/zh not_active Withdrawn
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CN113164620A (zh) | 2021-07-23 |
IL280534A (en) | 2021-03-25 |
EP3829645A1 (en) | 2021-06-09 |
US20210322563A1 (en) | 2021-10-21 |
JP2021533196A (ja) | 2021-12-02 |
CA3108328A1 (en) | 2020-02-06 |
KR20210086600A (ko) | 2021-07-08 |
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