WO2024138833A1 - 一种改造的重组凝血因子viii及其应用 - Google Patents

一种改造的重组凝血因子viii及其应用

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Publication number
WO2024138833A1
WO2024138833A1 PCT/CN2023/075157 CN2023075157W WO2024138833A1 WO 2024138833 A1 WO2024138833 A1 WO 2024138833A1 CN 2023075157 W CN2023075157 W CN 2023075157W WO 2024138833 A1 WO2024138833 A1 WO 2024138833A1
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WO
WIPO (PCT)
Prior art keywords
factor viii
blood coagulation
coagulation factor
recombinant blood
modified recombinant
Prior art date
Application number
PCT/CN2023/075157
Other languages
English (en)
French (fr)
Inventor
刘光猛
潘杏
余可
贾宁
方文晶
张钰
杜亮
王梦蝶
黄黎
何晓斌
Original Assignee
福因医药科技(武汉)有限公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 福因医药科技(武汉)有限公司 filed Critical 福因医药科技(武汉)有限公司
Publication of WO2024138833A1 publication Critical patent/WO2024138833A1/zh

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Abstract

一种改造的重组凝血因子VIII及其应用，所述重组凝血因子VIII的B结构域包括SQ序列，且所述SQ序列中插入一个或多个特定的vWF结合位点序列。改造的FVIII蛋白在细胞和体内的分泌后水平及凝血活性显著提高，并且仍能够被rAAV载体包装。相比于原始的FVIII-SQ蛋白，改造的重组凝血因子VIII在被rAAV载体递送后，动物体内的FVIII浓度有着显著的提高。这将有效地提高药物的治疗效果，解决高剂量带来的毒性等问题，对于血友病基因治疗领域具有重要意义。
PCT/CN2023/075157 2022-12-30 2023-02-09 一种改造的重组凝血因子viii及其应用 WO2024138833A1 (zh)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN202211725868.9 2022-12-30

Publications (1)

Publication Number Publication Date
WO2024138833A1 true WO2024138833A1 (zh) 2024-07-04

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