Opus Genetics Advances Gene Therapy for Rare Eye Disease with Promising Pediatric Data

Opus Genetics, a North Carolina-based biotech company, has made significant strides in its gene therapy program for Leber congenital amaurosis type 5 (LCA5), a rare eye disease affecting approximately 200 people in the United States. The company recently shared encouraging data from its pediatric trials, setting the stage for crucial discussions with the FDA.

Positive Results in Pediatric Population

Opus's gene augmentation therapy, which utilizes an AAV8 vector and the same promoter technology as Roche's Luxturna, has shown promising results in adolescent patients aged 16 to 17. The therapy aims to address photoreceptor impairment, the root cause of vision loss in LCA5 patients.

Key findings from the pediatric cohort include:

• An average improvement of 0.3 logMAR across the cohort
• Individual improvements ranging from 0.2 to 0.7 logMAR at three months post-treatment
• One patient showing a 0.5 logMAR improvement after just one month

These results are particularly encouraging when compared to the adult cohort data, which demonstrated increasing improvements over time, reaching a 0.48 logMAR change by the sixth month of the trial.

Safety Profile and Next Steps

The safety data from the pediatric trials has largely supported the positive signals observed in adult patients. No adverse events were directly related to the study drug, although one participant experienced worsening of a preexisting cataract, attributed to the surgical procedure used for gene therapy delivery.

With these promising results in hand, Opus Genetics is preparing for a critical meeting with the FDA in the fourth quarter. The company is optimistic about the possibility of an accelerated clinical development pathway, provided the efficacy in children continues to mirror the results seen in adults.

Market Response and Future Outlook

The announcement of these positive data points has been well-received by the market, with Opus's shares rising more than 3% in premarket trading following the news release. As the company moves forward, the durability of vision improvements, which has been observed for up to 18 months in the combined adult data, will be a key factor in determining the long-term success of this gene therapy approach.

As Opus Genetics continues to advance its LCA5 gene therapy program, the pharmaceutical industry will be watching closely, recognizing the potential impact on patients with this ultrarare eye disease and the broader implications for gene therapy development in ophthalmology.