HORA-PDE6B is in development for the treatment of retinitis pigmentosa (RP) caused by mutations in the PDE6B gene
HORA-001 provides a full-length non mutated copy of a functional human PDE6B gene delivered in the form of a sterile rAAV vector suspension injected directly into the subretinal space, where it rapidly induces robust transgene expression and synthesis of functional proteins in photoreceptive rods and cones. By effectively providing these cells with a functional protein HORA-PDE6B may significantly delay or halt retinal degeneration in PDE6ß-deficient patients.
A phase I/II was initiated in December 2017. Description of the protocol can be found on clinicaltrials.gov