Opus Genetics Inc. (IRD), a clinical-stage biopharmaceutical company, announced on Monday that the OPGx-LCA5 gene therapy program for treating LCA5 retinal disease had received a rare disease evidence principle (RDEP) designation from the U.S. food and drug administration (FDA).
Leber congenital amaurosis type 5 (LCA5) is a rare inherited retinal disease (IRD) caused by biallelic mutations in the LCA5 gene, which codes for the lebercilin protein. The disease leads to early onset, severe inherited retinal dystrophy, causing severe visual impairment or blindness in childhood. There are no FDA-approved therapies currently available for the treatment of LCA5.
The OPGx-LCA5 gene therapy delivers a functional LCA5 gene to the outer retina by use of an adeno-associated viral vector. The therapy has previously been granted a rare pediatric disease, orphan drug, and regenerative medicine advanced therapy (RMAT) designation by the FDA.
The RDEP is a new FDA initiative to advance therapies for ultra-rare genetic diseases which affect less than 1000 patients in the U.S. The FDA will work closely with Opus to guide the development and clinical trial design for OPGx-LCA5, and to formulate strategies to support clinical benefit demonstrations.
The company is currently evaluating OPGx-LCA5 in the ongoing Phase 1/2 trial and plans to align with the FDA on the pivotal Phase 3 program.
IRD closed Monday at $5.22, up 1.56%. In the pre-market, shares are trading at $5.31, up 1.77%.
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