CN116348139A - 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 - Google Patents

肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 Download PDF

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CN116348139A
CN116348139A CN202180065042.1A CN202180065042A CN116348139A CN 116348139 A CN116348139 A CN 116348139A CN 202180065042 A CN202180065042 A CN 202180065042A CN 116348139 A CN116348139 A CN 116348139A
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seq
amino acid
acid sequence
antibody
heavy chain
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罗梅什·R·苏布拉马尼亚
穆罕默德·T·卡塔纳尼
蒂莫西·威登
科迪·A·德雅尔丹
布伦丹·奎因
约翰·纳吉姆
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Dyne Therapeutics Inc
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Dyne Therapeutics Inc
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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CN202180065042.1A 2020-07-23 2021-07-09 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 Pending CN116348139A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063055768P 2020-07-23 2020-07-23
US63/055,768 2020-07-23
US202063061839P 2020-08-06 2020-08-06
US63/061,839 2020-08-06
US202163143828P 2021-01-30 2021-01-30
US63/143,828 2021-01-30
US202163181456P 2021-04-29 2021-04-29
US63/181,456 2021-04-29
PCT/US2021/040987 WO2022020106A1 (en) 2020-07-23 2021-07-09 Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

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CN116348139A true CN116348139A (zh) 2023-06-27

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CN202180065042.1A Pending CN116348139A (zh) 2020-07-23 2021-07-09 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途

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US (1) US20230272065A1 (https=)
EP (1) EP4185314A4 (https=)
JP (1) JP7851294B2 (https=)
KR (1) KR20230046297A (https=)
CN (1) CN116348139A (https=)
AU (1) AU2021313057A1 (https=)
BR (1) BR112023001003A2 (https=)
CA (1) CA3186746A1 (https=)
IL (1) IL299666A (https=)
MX (1) MX2023000961A (https=)
WO (1) WO2022020106A1 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
CA3108282A1 (en) 2018-08-02 2020-02-06 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
KR20230023612A (ko) 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제
CN116194470B (zh) * 2020-07-23 2025-10-03 达因疗法公司 肌肉靶向复合物及其用途
EP4185320A4 (en) * 2020-07-23 2025-10-08 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES IN THE TREATMENT OF MUSCLE ATROPHY
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2024525608A (ja) * 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic 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
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
EP4551230A2 (en) * 2022-07-06 2025-05-14 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2025522877A (ja) * 2022-07-06 2025-07-17 ダイン セラピューティクス，インコーポレーテッド Ｃｎｓ標的化複合体およびその使用
AU2024299328A1 (en) 2023-07-21 2026-01-22 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
WO2025255283A1 (en) * 2024-06-07 2025-12-11 Dyne Therapeutics, Inc. Dosing of muscle targeting complexes for treating facioscapulohumeral muscular dystrophy

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017009644A1 (en) * 2015-07-15 2017-01-19 King's College London Kinase inhibitors for use in the treatment of fascioscapulohumeral dystrophy
US20180273942A1 (en) * 2015-01-16 2018-09-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA039366B1 (ru) * 2015-06-24 2022-01-19 Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер
FR3049951A1 (fr) * 2016-04-12 2017-10-13 Univ Rabelais Francois Nouvelles constructions peptidiques et leur utilisation dans le traitement de la toxoplasmose
DK3473270T5 (da) 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
MX2019008199A (es) * 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
TWI869346B (zh) * 2018-05-30 2025-01-11 瑞士商諾華公司 Ｅｎｔｐｄ２抗體、組合療法、及使用該等抗體和組合療法之方法
SG11202100928QA (en) * 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2020028842A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy
AU2019314538B2 (en) * 2018-08-02 2025-03-13 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028836A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
IL312067B2 (en) * 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180273942A1 (en) * 2015-01-16 2018-09-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
WO2017009644A1 (en) * 2015-07-15 2017-01-19 King's College London Kinase inhibitors for use in the treatment of fascioscapulohumeral dystrophy

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US20230272065A1 (en) 2023-08-31
BR112023001003A2 (pt) 2023-03-28
JP2023535444A (ja) 2023-08-17
WO2022020106A1 (en) 2022-01-27
KR20230046297A (ko) 2023-04-05
JP7851294B2 (ja) 2026-04-24
CA3186746A1 (en) 2022-01-27
EP4185314A4 (en) 2024-08-07
IL299666A (en) 2023-03-01
EP4185314A1 (en) 2023-05-31
AU2021313057A1 (en) 2023-03-16

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