HORA-PDE6B has been developed for the treatment of retinitis pigmentosa (RP) caused by defects in the PDE6B gene
HORA-PDE6B 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 rods (where the PDE6β subunit is specifically expressed) and cones.
HORA-PDE6B is designed as a gene replacement product, i.e., to provide a non-mutated copy of the human PDE6B gene with which to replace the defective gene, thereby facilitating expression of functional PDE6ß protein in rods to halt or significantly delay retinal degeneration in PDE6ß-deficient patients.
A phase I/II has started December 2017. Description of the protocol can be found on clinicaltrials.gov