KR20220107222A - 마이크로디스트로핀 유전자 치료 작제물 및 이의 용도 - Google Patents

마이크로디스트로핀 유전자 치료 작제물 및 이의 용도 Download PDF

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KR20220107222A
KR20220107222A KR1020227021099A KR20227021099A KR20220107222A KR 20220107222 A KR20220107222 A KR 20220107222A KR 1020227021099 A KR1020227021099 A KR 1020227021099A KR 20227021099 A KR20227021099 A KR 20227021099A KR 20220107222 A KR20220107222 A KR 20220107222A
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South Korea
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nucleic acid
seq
acid sequence
dystrophin
aav
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Korean (ko)
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춘핑 치아오
데빈 맥두걸드
예 리우
올리비에 다노스
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리젠엑스바이오 인크.
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KR1020227021099A 2019-11-28 2020-11-27 마이크로디스트로핀 유전자 치료 작제물 및 이의 용도 Pending KR20220107222A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962941719P 2019-11-28 2019-11-28
US62/941,719 2019-11-28
US202063024933P 2020-05-14 2020-05-14
US63/024,933 2020-05-14
PCT/US2020/062484 WO2021108755A2 (en) 2019-11-28 2020-11-27 Microdystrophin gene therapy constructs and uses thereof

Publications (1)

Publication Number Publication Date
KR20220107222A true KR20220107222A (ko) 2022-08-02

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US (4) US12697399B2 (enExample)
EP (1) EP4065596A2 (enExample)
JP (2) JP7822933B2 (enExample)
KR (1) KR20220107222A (enExample)
AU (1) AU2020392243A1 (enExample)
BR (1) BR112022009895A2 (enExample)
CA (1) CA3159516A1 (enExample)
CL (1) CL2022001372A1 (enExample)
MX (1) MX2022006427A (enExample)
TW (1) TWI908746B (enExample)
WO (1) WO2021108755A2 (enExample)

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CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用
EP4694904A2 (en) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting aav capsids, methods of manufacturing and using same
AR133921A1 (es) 2023-09-25 2025-11-12 Sarepta Therapeutics Inc Distrofinas humanas adaptadas con mayor afinidad de unión a la actina para el tratamiento de las distrofias musculares
TW202613313A (zh) 2024-05-31 2026-04-01 美商薩羅塔治療公司 肌向性ｒａａｖ
WO2026075999A2 (en) * 2024-10-01 2026-04-09 Sarepta Therapeutics, Inc. Dystrophin and micro-dystrophin antibodies and fragments thereof
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