Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.

ABSTRACT: Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 10¹¹ vector genomes (vg); n = 69) or low-dose (1.0 × 10¹⁰ vg; n = 34) subretinal injection of the AAV2-vector-based gene therapy timrepigene emparvovec versus non-treated control (n = 66). Most treatment-emergent adverse events were mild or moderate. The trial did not meet its primary endpoint of best-corrected visual acuity (BCVA) improvement. In the primary endpoint analysis, three of 65 participants (5%) in the high-dose group, one of 34 (3%) participants in the low-dose group and zero of 62 (0%

SUBMITTER: MacLaren RE 

PROVIDER: S-EPMC10579095 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature