BET x RPE65

本研究是一项单臂、开放标签、多中心的I/II期临床研究，旨在评价FT-001对RPE65双等位基因变异相关的视网膜变性受试者的安全性、耐受性和有效性。

The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.