Opus Genetics announced one-year results from a Phase 1/2 study of its lead gene therapy candidate, OPGx-LCA5, for patients with severe vision impairment due to mutations in the LCA5 gene. The therapy, administered via subretinal injection, showed sustained improvements in vision and was well-tolerated by adult participants. The findings were presented at the 2025 ARVO annual meeting.
This positive one-year data is crucial for individuals suffering from LCA5-associated retinal degeneration, a debilitating condition with limited treatment options. Demonstrating sustained improvement in vision over a year offers hope for a significant and lasting impact on patients’ quality of life, potentially enabling greater independence and participation in daily activities. This success also bolsters confidence in the broader potential of gene therapy to address inherited retinal diseases.
Three adult patients with severe vision impairment received a single low-dose subretinal injection of OPGx-LCA5. At the one-year mark, all patients maintained improvements observed at six months, including enhanced object recognition, better visual acuity (averaging a 3.5-line improvement), and increased retinal sensitivity. Pupillary light reflex data also supports improved cone-mediated (daytime) vision. Importantly, OPGx-LCA5 continued to be well-tolerated, with no serious adverse events reported. Two additional adolescent patients have been treated with promising preliminary results.
The durable positive results from this Phase 1/2 study reinforce the promise of OPGx-LCA5 as a potential treatment for LCA5-associated retinal degeneration. Opus Genetics is engaging with the FDA regarding the design of a registration trial, aiming to initiate the study in 2026. This progress, coupled with plans to advance a BEST-1 gene therapy program into Phase 1/2 trials later this year, signals significant momentum for Opus Genetics and its pipeline of gene therapies targeting various inherited retinal diseases.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
