局部基因疗法KB105治疗转谷氨酰胺酶-1缺乏常染色体隐性遗传性先天性鱼鳞病

2019-06-16 Allan MedSci原创

Krystal是一家开发皮肤病药物的基因治疗公司,近日宣布它已向美国FDA提交了一份研究性新药(IND)申请,以启动KB105的I/II期临床试验,KB105是一种基于HSV-1的基因疗法,旨在为患有转谷氨酰胺酶-1(TGM1)缺陷的常染色体隐性遗传性先天性鱼鳞病(ARCI)的患者提供TGM1基因。

Krystal是一家开发皮肤病药物的基因治疗公司,近日宣布它已向美国FDA提交了一份研究性新药(IND)申请,以启动KB105I/II临床试验,KB105是一种基于HSV-1的基因疗法,旨在为患有转谷氨酰胺酶-1TGM1)缺陷的常染色体隐性遗传性先天性鱼鳞病(ARCI)的患者提供TGM1基因。

Krystal利用其皮肤靶向递送(STAR-D)平台技术,将TGM1基因传递给分裂和非分裂的皮肤细胞,使其能够产生该患者缺乏的TGM1蛋白。


原始出处:

http://www.firstwordpharma.com/node/1647663#axzz5r0RYRQBl

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    2019-06-18 huirong
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    2019-06-18 chg122