细胞疗法MGTA-456治疗遗传性代谢疾病的全新II期数据

2020-02-20 Allan MedSci原创

Magenta是一家临床阶段的生物技术公司,近日公布了其细胞疗法MGTA-456治疗遗传性代谢疾病的最新II期数据。MGTA-456是一种细胞疗法,旨在提供与患者良好匹配的高剂量造血干细胞(HSC),以确保患者的免疫和血液系统重建。

Magenta是一家临床阶段的生物技术公司,近日公布了其细胞疗法MGTA-456治疗遗传性代谢疾病的最新II期数据。MGTA-456是一种细胞疗法,旨在提供与患者良好匹配的高剂量造血干细胞HSC),以确保患者的免疫血液系统重建。

II临床试验正在探索MGTA-456治疗遗传性代谢异常(IMD)的有效性和安全性,IMD包括脑性肾上腺髓质营养不良(cALD)、IH型黏多糖贮积症(MPS IHurler综合征)、异色性白细胞营养不良(MLD)或球状细胞白细胞营养不良(GLDKrabbe病)。这些是罕见的且快速进展的疾病,如果不治疗则会致命。

Magenta首席医学官John Davis博士表示:现有治疗方法和正在研究的基因疗法均未能有效治疗遗传性代谢异常。但细胞疗法MGTA-456的临床试验证明,MGTA-456能够有效改善患者症状


原始出处:

https://www.firstwordpharma.com/node/1702382?tsid=4

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    2020-02-21 飛歌

    学习了很有用不錯

    0