On October 2, 2025, Opus Genetics announced positive three‑month pediatric data from its ongoing Phase 1/2 trial (OPGx‑LCA5‑1001) evaluating the AAV‑based gene therapy OPGx‑LCA5 for Leber congenital amaurosis type 5 (LCA5). The study enrolled three pediatric participants aged 16‑17 with severe baseline vision impairment, all of whom received a single subretinal injection of OPGx‑LCA5.
All three participants showed measurable improvements across multiple visual function endpoints, including visual acuity, retinal sensitivity, and real‑world navigation tasks. No ocular serious adverse events or dose‑limiting toxicities were observed, and the therapy was well tolerated in the pediatric cohort.
The data reinforce the durability of the treatment response observed in adult patients, who have maintained visual gains through 18 months, and provide compelling evidence to support the company’s regulatory strategy for this ultra‑rare disease. The announcement marks a key milestone in the development of OPGx‑LCA5 and underscores Opus Genetics’ progress toward a potential curative therapy for LCA5.
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