HORA-PDE6B

HORA-PDE6B is developed for the treatment of retinitis pigmentosa (RP) due to a defect in the PDE6B gene.

HORA-PDE6B is delivered in the form of a sterile suspension of viral particles injected directly into the subretinal space, where it allows rapid and strong transgene expression in both rods (where the PDE6β subunit is specifically expressed) and cones.

HORA-PDE6B product is intended as a gene replacement therapy, i.e., to provide a non-mutated copy of the human PDE6B gene to replace the defective gene, with the goal of expressing a functional PDE6ß protein in rods and in turn rescue retinal degeneration in PDE6ß-deficient patients.