HORA-RLBP1 has been developed for the treatment of retinal dystrophy caused by mutations in the RLBP1 gene

HORA-RLBP1 is delivered in the form of a sterile vector suspension injected directly into the subretinal space, where it allows rapid and robust transgene expression in the targeted retinal cells.

HORA-RLBP1 is designed as a gene replacement product, i.e., to provide the cells with a non-mutated copy of the human RLBP1 gene, thereby enabling expression of functional CRALBP and improving visual function, or at the very least significantly slowing retinal degeneration, in patients with RLBP1 mutations.