欧盟最高药物机构建议批准Wakyra(Wakyra),这是非盈利性开发的基因疗法,用于治疗男孩的罕见遗传病,但有待欧盟最终批准。 The EU’s top drug agency recommended approval of Waskyra, a non-profit-developed gene therapy for a rare genetic disorder in boys, pending final EU approval.

欧洲医药署的CHMP建议批准Wakyra, 这是威斯科特-阿尔德里奇综合症的一种基因疗法, The European Medicines Agency’s CHMP has recommended approval of Waskyra, a gene therapy for Wiskott-Aldrich syndrome, a rare genetic disorder affecting mostly boys. 由意大利Telethon基金会开发, 它使用病人自己的干细胞, 改用扁豆矢量来传递功能性原是基因。 Developed by Italy’s Fondazione Telethon, it uses a patient’s own stem cells modified with a lentiviral vector to deliver a functional WAS gene. 这种疗法在改造后的细胞重新恢复之前需要进行化疗,在临床试验中,严重感染和出血明显减少。 The therapy, which requires chemotherapy before reinfusion of corrected cells, has shown significant reductions in severe infections and bleeding in clinical trials. 这是第一个获得监管批准的非营利性基因疗法,美国林业发展局仍在审查其应用。 It is the first non-profit-led gene therapy to reach regulatory approval, with the U.S. FDA still reviewing its application. 欧盟的批准正在等待欧洲联盟委员会作出最后决定。 Approval in the EU is pending final decision by the European Commission.