AVROBIO的慢病毒基因疗法AVR-RD-04治疗胱氨酸症,获得FDA的孤儿药称号

2020-03-10 MedSci原创 MedSci原创

AVROBIO宣布,美国食品药品监督管理局(FDA)已为其基因疗法AVR-RD-04治疗胱氨酸症,授予了孤儿药称号。

AVROBIO宣布,美国食品药品监督管理局(FDA)已为其基因疗法AVR-RD-04治疗胱氨酸症,授予了孤儿药称号。

胱氨酸症是一种罕见的进行性疾病,是由于胱氨酸在溶酶体中积累引起,表现为眼部并发症(例如严重的畏光症)、肌肉萎缩和肾功能衰竭。超过90%未接受治疗的患者在20岁之前需要进行肾脏移植。目前针对胱氨酸症的标准治疗需要患者每天服用数十种药,会导致严重的口臭,却不能阻止疾病的进展。

AVR-RD-04是一种慢病毒载体递送的基因疗法,旨在通过单剂量注射患者自身造血干细胞来阻止或逆转胱氨酸症。在移植到患者体内之前,先收集干细胞并进行基因修饰,以表达功能性胱氨酸酶,这是一种运输蛋白,可减少胱氨酸在细胞内溶酶体中的积累,从而减轻胱氨酸病的症状。

目前该基因疗法正在进行1/2期临床试验,用于评估在胱氨酸症患者中的安全性和有效性。

原始出处:

https://www.firstwordpharma.com/node/1706685?tsid=4

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    2020-07-19 sunylz
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    2020-03-12 ymljack

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