The FDA granted rare pediatric disease designation for HORA-PDE6b, a gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6B gene, according to a press release from eyeDNA Therapeutics.
The AAV5-based gene therapy is designed to delay or halt retinal degeneration in PDE6B-deficient patients through delivery of a non-mutated copy of the PDE6B gene into the subretinal space. The designation will allow eyeDNA Therapeutics to receive a priority review voucher if HORA-PDE6b is approved that can be used to advance an additional program or be sold (Read more...)