KR20230128470A - 안면견갑상완 근이영양증(fshd)을 치료하기 위한 조성물및 방법 - Google Patents

안면견갑상완 근이영양증(fshd)을 치료하기 위한 조성물및 방법 Download PDF

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KR20230128470A
KR20230128470A KR1020237022057A KR20237022057A KR20230128470A KR 20230128470 A KR20230128470 A KR 20230128470A KR 1020237022057 A KR1020237022057 A KR 1020237022057A KR 20237022057 A KR20237022057 A KR 20237022057A KR 20230128470 A KR20230128470 A KR 20230128470A
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dux4
aav
vector
promoter
nucleic acid
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스캇 퀀턴 하퍼
아프루즈 라시노네자드
니콜라스 세바스티앙 웨인
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더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020237022057A 2020-11-30 2021-11-30 안면견갑상완 근이영양증(fshd)을 치료하기 위한 조성물및 방법 Pending KR20230128470A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063119268P 2020-11-30 2020-11-30
US63/119,268 2020-11-30
PCT/US2021/061109 WO2022115745A1 (en) 2020-11-30 2021-11-30 Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)

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KR20230128470A true KR20230128470A (ko) 2023-09-05

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KR1020237022057A Pending KR20230128470A (ko) 2020-11-30 2021-11-30 안면견갑상완 근이영양증(fshd)을 치료하기 위한 조성물및 방법

Country Status (8)

Country Link
US (1) US20240026356A1 (https=)
EP (1) EP4251752A1 (https=)
JP (1) JP2023551279A (https=)
KR (1) KR20230128470A (https=)
AU (1) AU2021385595A1 (https=)
CA (1) CA3203585A1 (https=)
IL (1) IL303230A (https=)
WO (1) WO2022115745A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
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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
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2019419494B2 (en) * 2018-12-31 2026-03-26 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2025507958A (ja) 2022-03-04 2025-03-21 ロックアネイビオ， インコーポレイテッド 操作された核内低分子ＲＮＡ（ｓｎＲＮＡ）を含む組成物および方法
WO2025225719A1 (en) * 2024-04-26 2025-10-30 Chugai Seiyaku Kabushiki Kaisha Treatment and prevention of muscular dystrophy and its related diseases or disorders with satralizumab

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CA3203585A1 (en) 2022-06-02
AU2021385595A9 (en) 2023-08-17
IL303230A (en) 2023-07-01
JP2023551279A (ja) 2023-12-07
AU2021385595A1 (en) 2023-06-29
US20240026356A1 (en) 2024-01-25
WO2022115745A1 (en) 2022-06-02
EP4251752A1 (en) 2023-10-04

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