Opus Genetics received rare pediatric disease designation from the FDA for its ocular gene therapy designed to treat patients with a form of Leber congenital amaurosis, according to a press release.
OPGx-LCA5, an adeno-associated virus 8 vector, delivers a functional LCA5 gene to the outer retina, according to the release. The company said there are currently no treatments approved for vision loss related to the early-onset retinal degeneration.
Research on OPGx-LCA5 is underway in a phase 1/2 open-label, dose-escalation clinical trial designed to evaluate the safety and preliminary efficacy of