业界动态MHRA
英国药品和保健产品监管局（MHRA）批准用于五岁及以上患有严重遗传性血液病儿童的基因编辑疗法
UK MHRA · 2026-10-30 09:18
摘要
在MHRA扩大其英国授权范围后，五岁及以上患有重度镰状细胞病和输血依赖型β-地中海贫血的儿童现在可以使用Casgevy（exagamglogene autotemcel）治疗。
English
MHRA approves gene-editing treatment for children aged five and over with serious inherited blood disorders
Children aged five and over with severe sickle-cell disease and transfusion-dependent β-thalassemia, can now be treated with Casgevy (exagamglogene autotemcel), following an extension of its UK authorisation by the MHRA.