Opus Genetics announced positive one-month data from the first pediatric patient in its Phase 1/2 trial of OPGx-LCA5, a gene therapy for LCA5-related inherited retinal disease. The patient showed clinically meaningful vision improvement with no reported drug-related adverse events. A second pediatric patient has been dosed, with full pediatric cohort data expected in Q3 2025.
This positive pediatric data strengthens the therapeutic potential of OPGx-LCA5, especially given the progressive nature of LCA5 and the potential benefits of early intervention. Currently, no approved treatments exist for this severe, early-onset form of vision loss. Successful results in pediatric patients would significantly expand the addressable patient population and underscore the value of gene therapy for this debilitating condition. This could position Opus as a leader in treating inherited retinal diseases and potentially change the standard of care for LCA5.
Technically, the therapy utilizes an AAV8 vector to deliver a functional LCA5 gene, addressing the underlying genetic defect. Opus has also had discussions with the FDA regarding a registrational trial, proposing a single-arm, adaptive pivotal study with the Multi-Luminance Orientation and Mobility Test (MLoMT) as the primary endpoint. The FDA has requested further information, but if approved, this streamlined trial design could accelerate the path to regulatory approval and potentially lead to market entry sooner than anticipated. The company anticipates initiating the pivotal trial in Q1 2026.
This early pediatric data combined with the planned pivotal trial discussions with the FDA, signifies considerable momentum for OPGx-LCA5. Positive data from the remaining pediatric patients and a clear regulatory pathway from the FDA could substantially increase investor confidence and position Opus for future growth. This also supports the broader potential of gene therapy to address unmet medical needs in ophthalmology and beyond.
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.
