OPGx-001

OPGx-001 is an adeno-associated virus 8 (AAV8) vectored gene therapy designed to deliver a functional LCA5 gene to retinal photoreceptors for the treatment of vision loss caused by biallelic mutations in the LCA5 gene, which results in Leber congenital amaurosis 5 (LCA5), a severe, early-onset inherited retinal degeneration. OPGx-001 provides gene augmentation by enabling expression of the lebercilin protein, aiming to preserve or restore visual function. The therapy is administered via subretinal injection. Developed initially at the University of Pennsylvania and now by Opus Genetics, OPGx-001 is currently in Phase 1/2 clinical evaluation and has demonstrated functional improvements in both adult and pediatric LCA5 patients in early data. There are currently no approved therapies for LCA5-associated vision loss[1][2][3][4][5][6].

Brand names

OPGx-LCA5OPGx-LCA-5OPGx-LCA 5

Other names

OPGx-001OPGx001OPGx 001OPGX-LCA5OPGX-LCA-5OPGX-LCA 5AAV8.hLCA5AAV-8.hLCA5AAV 8.hLCA5LCA5-LCALCA-5-LCALCA 5-LCA