Subretinal Gene Therapy Drug AGTC-501 for XLRP Phase 1/2 Multicenter Study (HORIZON): 24-Month Safety and Efficacy Results.
Yang Paul, Birch David, Lauer Andreas, Sisk Robert, Anand Rajiv, Pennesi Mark E et al.
Subretinal gene therapy (AGTC-501) for XLRP showed preliminary efficacy at a tolerated dose, but higher doses had concerning retinal side effects, guiding future treatment development.