HORA-RLBP1

HORA-RLBP1 is developed for the treatment of retinitis punctata albescens due to mutations in the RLBP1 gene.

HORA-RLBP1 is delivered in the form of a sterile suspension of viral particles injected directly into the subretinal space, where it allows transgene expression in the RPE.

HORA-RLBP1 medicinal product is intended as a gene replacement therapy, i.e., to provide the cell with a non-mutated copy of the human RLBP1 gene, that can express a functional CRALBP protein to halt or, at least, significantly slow the rate of retinal degeneration in RPA patients.