Krystal Biotech Inc. (KRYS) 宣布，其基因疗法Vyjuvek®已获得英国药品和保健品监管局（MHRA）的批准，适用于治疗营养不良型大疱性表皮松解症（DEB）患者。这一监管决定标志着该疗法在美国之外的关键市场取得了重要进展，为受这一罕见、严重遗传性皮肤疾病困扰的患者带来了新的治疗选择。Vyjuvek®是一种基于疱疹病毒载体的局部基因疗法，旨在通过功能性COL7A1基因的递送，帮助患者皮肤产生所需的VII型胶原蛋白，从而促进伤口愈合并减少水疱形成。英国监管机构的批准，是基于其先前在美国获得FDA批准以及一系列临床试验所展示出的积极疗效与安全性数据。此次获批有望进一步拓宽Krystal Biotech的市场覆盖，并强化其在基因治疗领域的地位。
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