Katherine A. High

Katherine High is a pioneer in hemophilia gene therapy who led development of Luxturna (voretigene neparvovec), the first FDA-approved AAV gene therapy for a genetic disease (RPE65 retinal dystrophy). Her work on liver-directed factor IX gene therapy has demonstrated durable correction of hemophilia, with gene therapy products now approved or in late-stage development.

Katherine High是血友病基因治疗的先驱，领导了Luxturna（voretigene neparvovec）的开发，这是第一个FDA批准的用于遗传疾病（RPE65视网膜营养不良）的AAV基因治疗。她在肝脏定向因子IX基因治疗方面的工作证明了血友病的持久矫正，基因治疗产品现已获批或处于后期开发阶段。