Opus Genetics Advances Gene Therapy for Rare Eye Disease, Sets Sights on FDA Talks

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 promising data from its pediatric trials, paving the way for discussions with the FDA about the program's future.

Encouraging Results in Pediatric Population

Opus has expanded its gene therapy trials to include adolescents aged 16 to 17 with severe vision impairment due to LCA5. The treatment, which utilizes an AAV8 vector and the same promoter technology as Roche's Luxturna, aims to deliver a functional LCA5 gene to address photoreceptor impairment.

Initial results from the pediatric cohort show an average improvement of 0.3 logMAR across the group. Two patients demonstrated improvements of 0.2 and 0.7 logMAR three months post-treatment, while the third patient showed a 0.5 logMAR improvement after one month. Notably, the patient with the best baseline vision experienced the smallest improvement.

These results compare favorably to the data from adult trials, where improvements increased to a 0.48 logMAR change by the sixth month. The combined adult data suggest durability of vision improvements through 18 months.

Safety Profile and Next Steps

The safety data from the pediatric trials largely support the encouraging signs reported in adults. No adverse events were related to the study drug itself. However, one pediatric participant experienced a worsening of a preexisting cataract at three months, attributed to the surgical procedure used to deliver the gene therapy.

Opus Genetics plans to meet with the FDA in the fourth quarter to discuss the next steps for the program. The company has indicated that an accelerated clinical development pathway to FDA approval could be possible if the efficacy in children remains comparable to the results seen in adults.

The announcement of these positive results led to a more than 3% increase in Opus Genetics' shares during premarket trading on Tuesday.

As the company moves forward with its gene therapy program, the pharmaceutical industry will be watching closely to see how this potential treatment for an ultrarare eye disease progresses through the regulatory pathway.