HORA-RPE65 has been developed for the treatment of inherited retinal dystrophies caused by mutations in the RPE65 gene

HORA-RPE65 is delivered in the form of a sterile vector suspension injected directly into the subretinal space, where it induces transgene expression in retinal pigment epithelium cells.

HORA-RPE65 is a gene replacement product. It provides the cell with a non-mutated copy of the human RPE65 gene, which can express functional RPE65 protein to halt or at least significantly delay retinal degeneration in patients with inherited retinal dystrophies caused by RPE65 gene mutations.