林业发展局接受了Rocket的BLA基因疗法,以治疗罕见的免疫障碍;批准目标是2026年3月。 FDA accepted Rocket's gene therapy BLA for rare immune disorder; approval target March 2026.

林业发展局已接受火箭制药公司为Kresladi重新提交BLA,这是严重LAD-I的基因疗法,是一种罕见的遗传性免疫障碍。 The FDA has accepted Rocket Pharmaceuticals' resubmitted BLA for Kresladi, a gene therapy for severe LAD-I, a rare genetic immune disorder. 审查的目标行动日期是2026年3月28日。 The review has a target action date of March 28, 2026. 如果获得批准,Rocket可以收到一份儿科疾病优先审查券。 If approved, Rocket could receive a Rare Pediatric Disease Priority Review Voucher. 临床数据显示,12个月存活率达到100%,感染减少,伤口愈合改善,没有与治疗有关的严重副作用。 Clinical data showed 100% survival at 12 months, reduced infections, and improved wound healing with no serious treatment-related side effects. 治疗旨在提供治疗,无需捐赠者移植。 The therapy aims to provide a cure without needing a donor transplant.