FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapy
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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 the therapy in 15 patients.
“The designation is for the LCA5 program, which is a rare, very severe, blinding pediatric genetic disease for which there are no treatments available,” Opus president and CEO Ben Yerxa, PhD, told Healio in an email. “The next steps are to enroll subjects in the next cohort at the medium dose and to add younger subjects down to the age of 13 years of age.”
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