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FDA批准MRT5201进行单次剂量递增的I/II期临床试验，用于鸟氨酸氨甲酰基转移酶缺乏症

2019-06-27 Allan MedSci原创

鸟氨酸氨甲酰基转移酶缺乏症MRT5201mRNA
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Translate是一家临床阶段信使RNA（mRNA）治疗公司，近日宣布，美国FDA已批准MRT5201进行单次剂量递增的I/II期临床试验，用于治疗鸟氨酸氨甲酰基转移酶缺乏症。

鸟氨酸氨甲酰基转移酶缺乏症（OTC）属少见先天性遗传代谢的罕见病，患者尿素循环中断、血氨增高、精氨酸降低、谷氨酸酰胺及丙氨酸升高。

Translate是一家临床阶段信使RNA（mRNA）治疗公司，近日宣布，美国FDA已批准MRT5201进行单次剂量递增的I/II期临床试验，用于治疗鸟氨酸氨甲酰基转移酶缺乏症。MRT5201旨在通过提供全功能OTC酶的mRNA来直接解决OTC缺乏。Translate首席执行官Ronald Renaud说：“我们很高兴能够推进MRT5201在人体内进行OTC缺乏症治疗的临床试验，这对我们团队来说是一个重要的里程碑”。


原始出处：

http://www.firstwordpharma.com/node/1649715#axzz5s0nRyue8

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    2020-06-01 医生2402

    #I期临床#

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    2019-11-28 kksonne

    #II期临床试验#

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    2020-01-25 智者为医08

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    2019-08-20 839640778

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    2019-06-29 guihongzh

    #II期临床#

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