India, July 6 -- Opus Genetics Inc. (IRD), a clinical-stage biopharmaceutical company, on Monday announced achieving alignment with the U.S. Food and Drug Administration (FDA) on the Phase 3 registrational trial on OPGx-LCA5 in treating inherited retinal dystrophy in a Type B Rare Disease Evidence Principles meeting.
OPGx-LCA5 is a gene therapy that employs an adeno-associated virus 8 (AAV8) vector, developed to treat Leber congenital amaurosis (LCA) caused by biallelic mutations in the LCA5 gene encoding the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy (IRD) that leads to dissociation of retinal architecture and visual function.
The drug previously received Rare Pediat...