Opus Genetics Advances Gene Therapy for Retinal Degeneration: A Study Update

Opus Genetics, Inc. ((IRD)) announced an update on their ongoing clinical study.

Opus Genetics, Inc. is conducting a clinical study titled ‘An Open Label, Dose Exploration, Safety and Tolerability Study of a Subretinal Injection of an OPGx-001 Gene Vector to Participants With LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD).’ The study aims to evaluate the safety and preliminary efficacy of the OPGx-001 gene therapy in patients with retinal degeneration caused by LCA5 gene mutations. This research is significant as it could lead to new treatments for inherited retinal diseases.

The intervention being tested is a biological treatment known as AAV8.hLCA5, which is an adeno-associated virus vector designed to express the human LCA5 gene. This treatment is intended to address the genetic cause of retinal degeneration in affected individuals.

The study is interventional and follows a non-randomized, sequential intervention model with no masking. The primary purpose is treatment-focused, aiming to explore different dosage levels of the gene therapy in both adult and adolescent cohorts.

The study began on November 7, 2022, with primary completion and estimated completion dates not yet specified. The latest update was submitted on June 9, 2025, indicating ongoing recruitment and progress in the study.

Market implications of this study update could be significant for Opus Genetics, potentially influencing stock performance positively if the results show promise. This development is particularly relevant in the competitive field of gene therapy for inherited retinal diseases, where advancements can drive investor interest and confidence.

The study is currently ongoing, and further details are available on the ClinicalTrials portal.