HORA-RPE65

HORA-RPE65 is developed for the treatment of inherited retinal dystrophies due to mutations in the RPE65 gene, e.g. Leber Congenital Amaurosis Type 2.

HORA-RPE65 is delivered in the form of a sterile suspension of viral particles injected directly into the subretinal space where it allows transgene expression RPE cells.

HORA-RPE65 medicinal product is intended as a gene replacement therapy, i.e., to provide the cell with a non-mutated copy of the human RPE65 gene, that can express a functional RPE65 protein to halt or, at least, significantly slow the rate of retinal degeneration in patients with inherited retinal dystrophies due to mutations in the RPE65 gene, e.g. Leber Congenital Amaurosis Type 2.