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临床前开发和描述一种人体血衍生的高纯度X因子缩剂,用于治疗

Joanne Lloyd¹, Peter Feldman¹

¹Research & Development Department, Bio Products Laboratory, Dagger Lane, Elstree, UK.

Haemophilia : the official journal of the World Federation of Hemophilia

|September 28, 2023

概括

开发了一种新的高纯度X因子缩剂,用于遗传性X因子缺乏症. 这种血衍生的治疗药物证明了安全性和稳定性,为孤儿药物批准铺平了道路.

科学领域:

血液学血液学血液学
生物技术是生物技术.
药理学药理学是指药理学的学科.

背景情况:

遗传性X因子缺乏症是一种罕见的出血障碍,治疗选择有限.
临床前开发的重点是新的血衍生X因子缩物.

研究的目的:

描述高纯度X因子缩物的临床前开发和表征.
为治疗遗传性X因子缺乏症获得孤儿药物营销许可.

主要方法:

从人体血中开发了X因子的染色学净化过程.
进行了全面的体外,体内和体外安全性和疗效测试.

主要成果:

达到6000倍的净化和100倍的度,导致94-96%的纯X因子.
证明有效的病毒减少,没有血栓性,毒性和免疫性潜力.
在受控的室温和48小时的复制后活动下,已确认产品的稳定性至少为三年.

结论:

成功完成第一个高度净化X因子缩物的有针对性的临床前开发.
解决了与血衍生产品相关的关键安全问题.
开发方法可以为创建其他超孤儿药物提供信息.

关键词:

血液中的蛋白质血液中的蛋白质这是X因子缺乏症.孤儿药物的生产生产.药品制剂是药品制剂中的一种.治疗药物治疗药物

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