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FDA授予OTL-103再生医学高级治疗（RMAT）认定用于治疗Wiskott-Aldrich综合征

2019-08-04 Allan MedSci原创

Wiskott-Aldrich综合征OTL-103再生医学高级治疗（RMAT）
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Orchard是一家领先的生物制药公司，致力于通过创新的基因疗法改善罕见疾病患者的生活。Orchard近日宣布，其OTL-103已经获得FDA的再生医学高级治疗（RMAT）认定。OTL-103是基于离体自体造血干细胞（HSC）的基因疗法，用于治疗Wiskott-Aldrich综合征（WAS）。

Orchard是一家领先的生物制药公司，致力于通过创新的基因疗法改善罕见疾病患者的生活。Orchard近日宣布，其OTL-103已经获得FDA的再生医学高级治疗（RMAT）认定。OTL-103是基于离体自体造血干细胞（HSC）的基因疗法，用于治疗Wiskott-Aldrich综合征（WAS）。

Wiskott-Aldrich综合征（WAS）是由X染色体上WAS基因突变所导致的，兼有原发性免疫缺陷病和遗传性血液病表现的特殊类型疾病。虽然典型WAS的临床表现特征比较明显，经相关基因（WAS）及其产物蛋白（WASP）检测可以确诊，但由于发病率较低，病例严重程度差异显著。


原始出处：

http://www.firstwordpharma.com/node/1656499#axzz5vaProhET

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    2019-09-09 guojianrong

    #Wiskott-Aldrich综合征#

    0

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    2019-08-06 diushouji

    #ICH#

    0

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    2019-08-06 zhouqu_8

    #综合征#

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    2019-08-06 zhangjingnwpu

    #ALD#

    0

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