About AAVantgarde

AAVantgarde develops gene therapies for patients with inherited retinal disorders caused by large gene mutations. The company uses two proprietary AAV-based delivery platforms, Dual Hybrid and Intein-Mediated vectors, engineered to deliver genetic material that exceeds the capacity of standard viral vectors. This approach addresses the root cause of vision loss in conditions like Stargardt disease and Usher 1B syndrome. The lead programs include AAVB-039 for Stargardt disease, which has received FDA Orphan Drug Designation and UK approval, and AAVB-081 for Usher 1B syndrome, currently in Phase 1/2 trials. Both therapies target diseases with high unmet need, offering gene replacement for patients who previously had no options.