Loading...

News List

FDA批准首个可重复给药基因疗法,每年63万美元,治疗“最痛苦疾病”

2023-05-22


FDA批准首个可重复给药基因疗法,每年63万美元,治疗“最痛苦疾病”

2023年5月19日,FDA批准了 Krystal Biotech 公司开发的基于单纯疱疹病毒1型(HSV-1)的局部基因疗法Beremagene Geperpavec(B-VEC),商品名Vyjuvke。用于治疗6月龄及以上的伴有COL7A1基因突变的营养不良大疱性表皮松解症(DEB)患者的伤口。营养不良型大疱性表皮松解症(Dystrophic Epidermolysis Bullosa,DEB)是一种罕见的遗传性皮肤疾病,由COL7A1基因突变引起,COL7A1基因编码VII型胶原蛋白(C7),患者皮肤易出现大疱、血疱,这些患者也被称为“蝴蝶宝宝”,形容他们的皮肤像蝴蝶一样脆弱,一碰就破。这是世界上最痛苦的疾病之一,而且患者易出现感染、皮肤癌,甚至死亡。

Pre: Wang Ben's team at Zhejiang University developed fluorine-containing polymer assembly drugs to achieve chemotherapeutic-immune cascade cancer therapy

Next: FDA approves First repeatable drug gene Therapy, $630,000 a Year, for 'most painful diseases'

We would like to use cookies to better understand your use of this website, which will help improve your experience on future visits to the website. You can change this setting in your browser settings. For more information about the use of cookies, please refer to our Privacy Policy.