(RTTNews) - Opus Genetics, Inc. (IRD), a clinical-stage ophthalmic biotechnology company, on Tuesday announced that the first pediatric patient has been dosed in its global Phase 1/2 clinical trial of OPGx-LCA5, an investigational gene therapy for Leber congenital amaurosis or LCA caused by mutations in the LCA5 gene.
OPGx-LCA5 is designed to address inherited retinal degeneration in LCA5 patients, offering potential for significant visual improvements.
The ongoing trial has already shown promising early results in adult patients, with positive visual improvements seen as early as one month post-treatment.
The company expects to share initial data from the pediatric cohort by the third-quarter of 2025.
Opus is also set to present 12-month data from the first three adult patients at a major medical conference in Q2 2025.
Furthermore, the company has scheduled a Type D FDA meeting in March 2025 to discuss the Phase 3 trial design and registrational endpoints for OPGx-LCA5.
This marks a significant step in Opus Genetics' efforts to bring potentially life-changing gene therapy to patients with inherited retinal diseases.
Currently, IRD is trading at $1.14 up by 1.32%.
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