Posted by Michael Wonder on 22 Aug 2024
Opus Genetics receives rare paediatric disease designation from the US FDA for ocular gene therapy OPGx-LCA5 to treat rare inherited retinal disease LCA5
20 August 2024 - Opus Genetics today announced the US FDA has granted rare paediatric disease designation for its ocular gene therapy OPGx-LCA5 to treat patients with the inherited retinal disease LCA5.
OPGx-LCA5 is an adenovirus associated serotype 8 vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis resulting from biallelic mutations in the LCA5 gene.
