Ultragenyx公布DTX301 AAV8基因疗法治疗鸟氨酸氨甲酰基转移酶缺乏症三期研究36周积极数据

美股速递03-12

Ultragenyx Pharmaceutical Inc（简称Ultragenyx）近日宣布，其针对鸟氨酸氨甲酰基转移酶（OTC）缺乏症研发的DTX301 AAV8基因疗法，在三期临床研究中取得了36周的积极数据。这一进展标志着该基因疗法在长期疗效与安全性方面展现出令人鼓舞的结果。

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