第61届ASH:Mustang Bio宣布MB-107和MB-106慢病毒基因疗法的全新数据

2019-11-07 Allan MedSci原创

Mustang Bio是一家临床阶段的生物制药公司,致力于使用先进的细胞和基因疗法治疗血液学癌症。在第61届美国血液病学会(ASH)年会上,Mustang Bio的MB-107和MB-106慢病毒基因疗法治疗X连锁严重综合免疫缺陷症(XSCID)的I/II期临床数据将被披露。ASH将于2019年12月7日至10日在佛罗里达州奥兰多举行。

Mustang Bio是一家临床阶段的生物制药公司,致力于使用先进的细胞和基因疗法治疗血液学癌症。在第61届美国血液病学会(ASH)年会上,Mustang BioMB-107MB-106慢病毒基因疗法治疗X连锁严重综合免疫缺陷症(XSCID)的I/II临床数据将被披露。ASH将于2019127日至10日在佛罗里达州奥兰多举行。

X连锁重症联合免疫缺陷症(X-SCID)是由IL2RG中半合子的致病性变异引起的细胞与体液联合免疫缺陷综合征。在典型的X-SCID中,缺乏IL2RG功能导致几乎完全缺乏T和自然杀伤(NK)淋巴细胞和非功能性B淋巴细胞。除非通过骨髓移植或基因治疗实现免疫系统的重建,否则X-SCID在生命的头两年几乎全都是致命的。


原始出处:

http://www.firstwordpharma.com/node/1679094#axzz64Xy4dOaT

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    2020-07-14 sunylz
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    2019-11-08 kksonne
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    2019-11-08 ymljack