AAVantgarde Bio is a clinical-stage gene therapy company focused on overcoming AAV vector cargo capacity limitations. Our dual hybrid and AAV intein platforms enable the delivery of large genes that do not fit in standard AAV vectors. We are actively validating these platforms through our two lead programs:

• Usher Syndrome Type 1B (dual hybrid platform): We’ve published safety data from our first two subjects and have dosed additional patients in our low and mid-dose cohorts. With maturing datasets, we plan to present updated clinical data at ARVO in May.
• Stargardt Disease (AAV intein platform): Having completed our IND-enabling studies, we are preparing our IND filing and anticipate treating our first patient in Q3 2025. 

Beyond ophthalmology, we’re expanding into inner ear and CNS indications, as well as other therapeutic areas where large gene delivery is a challenge. Given its versatility, we are also exploring ways to apply our platform to genes of interest for potential partners.