CN113302290A - 治疗威尔逊氏病的组合物和方法 - Google Patents

治疗威尔逊氏病的组合物和方法 Download PDF

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CN113302290A
CN113302290A CN201980089235.3A CN201980089235A CN113302290A CN 113302290 A CN113302290 A CN 113302290A CN 201980089235 A CN201980089235 A CN 201980089235A CN 113302290 A CN113302290 A CN 113302290A
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nucleic acid
acid molecule
seq
atp7b
sequence
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卡提克·拉马穆尔蒂
斯蒂芬妮·塔利亚泰拉
安妮·坦侬豪斯
安德鲁·杨
陈思颖
张弛
斯蒂芬妮·马丁
大卫·奥伯科夫勒
维多利亚·黄
李建民
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Coding Therapy Co
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Coding Therapy Co
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CN201980089235.3A 2018-11-16 2019-11-15 治疗威尔逊氏病的组合物和方法 Pending CN113302290A (zh)

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US201862768744P 2018-11-16 2018-11-16
US62/768,744 2018-11-16
PCT/US2019/061808 WO2020102723A1 (en) 2018-11-16 2019-11-15 Compositions and methods for treating wilson's disease

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US (1) US20210369870A1 (https=)
EP (2) EP4257155A3 (https=)
JP (1) JP7532360B2 (https=)
CN (1) CN113302290A (https=)
CA (1) CA3118936A1 (https=)
TW (1) TW202039854A (https=)
WO (1) WO2020102723A1 (https=)

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CN110290814A (zh) * 2016-12-30 2019-09-27 宾夕法尼亚州立大学托管会 用于治疗威尔逊病的基因疗法
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子
WO2026007662A1 (zh) * 2024-07-02 2026-01-08 苏州星核迪赛生物技术有限公司 一种靶向肝实质细胞的核酸递送载体及其应用
CN121319202A (zh) * 2025-12-15 2026-01-13 凌意(杭州)生物科技有限公司 Atp7b抗体及其应用

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WO2022208342A1 (en) * 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN113699211A (zh) * 2021-07-20 2021-11-26 深圳市儿童医院 一种用于儿童威尔森氏症快速筛查的参数组合和试剂盒
EP4398896A4 (en) * 2021-09-07 2025-07-02 Alexion Pharma Inc METHODS FOR TREATING DISEASES OR DISORDERS ASSOCIATED WITH COPPER METABOLISM
US20250144239A1 (en) * 2021-10-18 2025-05-08 Logicbio Therapeutics, Inc. Gene therapy for the treatment of wilson's disease
KR20250102134A (ko) * 2022-10-11 2025-07-04 메이라지티엑스 유케이 Ii 리미티드 Atp7b 유전자 요법
WO2024183769A1 (en) * 2023-03-06 2024-09-12 Lingyi Biotech Co., Ltd. A truncated atp7b and the use thereof
JP2026508707A (ja) * 2023-03-22 2026-03-11 スカイライン、セラピューティクス、リミテッド ウィルソン病の遺伝子療法のための組換えａａｖ
WO2025002226A1 (zh) * 2023-06-28 2025-01-02 迈威(上海)生物科技股份有限公司 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用
WO2025026198A1 (en) * 2023-07-28 2025-02-06 Lingyi Biotech Co., Ltd. Polynucleotides for the treatment of wilson disease
CN117757797B (zh) * 2023-09-25 2024-08-16 北京圭特细胞科技有限公司 一种编码人III型胶原蛋白α链的核酸及其应用
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease

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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110290814A (zh) * 2016-12-30 2019-09-27 宾夕法尼亚州立大学托管会 用于治疗威尔逊病的基因疗法
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子
WO2026007662A1 (zh) * 2024-07-02 2026-01-08 苏州星核迪赛生物技术有限公司 一种靶向肝实质细胞的核酸递送载体及其应用
CN121319202A (zh) * 2025-12-15 2026-01-13 凌意(杭州)生物科技有限公司 Atp7b抗体及其应用
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