RMAT designation for Opus’ OPGx-LCA5 gene therapy candidate

US ophthalmics-focused biotech Opus Genetics (Nasdaq: IRD) today revealed that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, its investigational gene therapy for the treatment of Leber congenital amaurosis (LCA) due to genetic variations in the LCA5 gene.

The RMAT designation for OPGx-LCA5 is based on early clinical evidence from Opus’s ongoing Phase I/II open-label, dose-escalation trial, which is evaluating the safety and potential efficacy of OPGx-LCA5 in patients with severe vision loss due to confirmed mutations in the LCA5 gene.

The FDA’s decision indicates recognition of the strength of this initial data and the unmet need in this patient population, said Opus, which was created through an all-stock merger with Ocuphire Pharma in October last year, continuing with the Opus name.