Dosing is complete in the first cohort of the phase 1/2 clinical trial of OPGx-LCA5 for the treatment of Leber congenital amaurosis, according to a press release from Opus Genetics.
An adeno-associated virus 8 vector is used to deliver a functional LCA5 gene to the outer retina via subretinal administration in patients with Leber congenital amaurosis resulting from biallelic mutations in the LCA5 gene.
The company plans to advance the gene therapy to the next highest dose following positive safety and efficacy data from the first cohort of three adult (Read more...)