#United States #Rockville #gene therapy #ABBV-RGX-314 #REGENXBIO

REGENXBIO's Breakthrough Presentation at the AAO 2025

On October 17, 2025, REGENXBIO Inc. will unveil exciting interim data from its Phase II ALTITUDE® trial during the American Academy of Ophthalmology 2025 Annual Meeting. The presentation, scheduled for 4:46 p.m. ET, is expected to provide significant insights into the efficacy of a novel suprachoroidal gene therapy known as surabgene lomparvovec (commercially termed ABBV-RGX-314) for the treatment of diabetic retinopathy (DR).

Overview of the Trial

This key presentation will be made by Dr. Charles C. Wykoff from Retina Consultants of Texas, aiming to highlight the first two-year results regarding the treatment's safety and effectiveness specifically for patients with non-proliferative diabetic retinopathy. The ALTITUDE® study, which investigates the novel delivery method of sura-vec into the suprachoroidal space of the eye, is a critical component in understanding how gene therapies might revolutionize the treatment of chronic retinal diseases.

What is Surabgene Lomparvovec?

Surabgene lomparvovec is a pioneering one-time gene therapy designed to target conditions such as DR and wet age-related macular degeneration (wet AMD). Utilizing the NAV® AAV8 vector, the therapy encodes an antibody fragment meant to neutralize vascular endothelial growth factor (VEGF). This action is crucial as VEGF is responsible for the development of leaky blood vessels that lead to fluid build-up in the retina—a common issue in both DR and wet AMD.

The Potential Impact

The implications of REGENXBIO's findings could be profound for the millions affected by diabetic retinopathy and other retinal conditions. Current treatment options are limited and often require multiple interventions over time; however, the potential of a one-time gene therapy could significantly alter patient experience and outcomes. According to Dr. Wykoff, the company is poised to change the landscape of retinal disease treatment, moving towards more innovative, long-lasting solutions.

REGENXBIO's Mission

Founded in 2009, REGENXBIO is dedicated to harnessing the power of gene therapy to improve lives. With a robust pipeline of treatments targeting various genetic disorders and diseases, including Duchenne Muscular Dystrophy, MPS II, and MPS I, the company is on the forefront of biotechnology innovation. Their collaboration with AbbVie on surabgene lomparvovec represents one of several ventures aiming to enhance the effectiveness of treatment approaches in gene therapy.

REGENXBIO emphasizes the need for continued research and diligence as it works to bring its investigational therapies to market. Each new data point shared at events like the AAO is a stepping stone to ensuring that new generations of therapies are backed by solid evidence, ultimately fostering trust in gene therapy as a viable treatment option for numerous conditions.

Conclusion

As anticipation builds for their presentation at the AAO 2025, the developments surrounding REGENXBIO's gene therapy initiatives serve as a promising indicator of the future of medical interventions in ophthalmology. The hope is that with advancements like ABBV-RGX-314, patients grappling with diabetic retinopathy will soon have access to transformative treatment options that offer more than temporary solutions.

For further updates and detailed analysis, attendees and interested parties are encouraged to follow REGENXBIO's announcements and participate in discussions around the impactful changes taking place in retinal therapies.

Stay tuned for more exciting news from the forefront of gene therapy as we approach the AAO conference. Excitement is building around REGENXBIO, as their work could pave the way for groundbreaking changes in eye care treatment protocols.