Opus Genetics Announces Positive Pediatric Data from OPGx-LCA5 Gene Therapy Trial for Leber Congenital Amaurosis Type 5; FDA Meeting Expected in Q4 2025
Reuters
Sep 30, 2025
<a href="https://laohu8.com/S/IRD">Opus Genetics</a> Announces Positive Pediatric Data from OPGx-LCA5 Gene Therapy Trial for Leber Congenital Amaurosis Type 5; FDA Meeting Expected in Q4 2025
Opus Genetics Inc. announced positive three-month data from the pediatric cohort of its ongoing Phase 1/2 clinical trial evaluating OPGx-LCA5, an investigational gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5). According to the company, all three pediatric participants demonstrated improvements in cone-mediated vision, and the therapy was well tolerated without any ocular serious adverse events or dose-limiting toxicities. Durable responses were also observed in adult participants out to 18 months. The results were discussed during a company webcast and conference call. Opus Genetics expects to meet with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2025 to discuss these findings and the next steps for the OPGx-LCA5 program.
Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Opus Genetics Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9537107-en) on September 30, 2025, and is solely responsible for the information contained therein.
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.