Innovation
We are focused on addressing the root cause of two IRDs with very high unmet need: Stargardt and Usher 1B
AAVantgarde’s proprietary technology has achieved unprecedented preclinical data demonstrating high transduction, protein expression, and safety in multiple large animal models for both Stargardt and Usher 1B.
The LUCE clinical study ongoing in Usher 1B patients is showing a very good safety profile and early efficacy signal giving hope to this underserved patient population.
Pipeline
Two lead programs in Ophthalmology with a high unmet need: Usher Syndrome Type 1B associated retinitis pigmentosa (Usher1B) and Stargardt disease.
