- Opus Genetics announced **positive three-month data** from the pediatric cohort of its OPGx-LCA5 Phase 1/2 clinical trial for Leber congenital amaurosis type 5 (LCA5).  **[1228627_0001140361-25-036637_ef20056246_ex99-1.htm:1]** 
- The OPGx-LCA5 therapy was **well tolerated** in all six participants (three adults and three pediatric), with **no ocular serious adverse events or dose-limiting toxicities** observed.  **[1228627_0001140361-25-036637_ef20056246_ex99-1.htm:1]** **[1228627_0001140361-25-036637_ef20056246_8k.htm:2]** **[1228627_0001140361-25-036637_ef20056246_ex99-1.htm:2]** 
- Pediatric participants demonstrated **significant improvements in visual function**, including a group average of a **0.3 logMAR improvement in visual acuity** and **greater than one log unit improvement in cone sensitivity**.  **[1228627_0001140361-25-036637_ef20056246_ex99-1.htm:1]** **[1228627_0001140361-25-036637_ef20056246_8k.htm:2]** 
- **Durable improvements** in visual acuity were sustained through **18 months in adult participants**.  **[1228627_0001140361-25-036637_ef20056246_ex99-1.htm:1]** 
- The company expects to **meet with the FDA in Q4 2025** to discuss the trial results and next steps for the LCA5 program.  **[1228627_0001140361-25-036637_ef20056246_ex99-1.htm:1]** **[1228627_0001140361-25-036637_ef20056246_8k.htm:2]**

Opus Genetics (IRD)