AAV gene therapy has proven to be a very successful therapeutic modality, but it is limited by AAV transgene capacity

AAVantgarde’s proprietary platforms enable delivery of large genes to tissue and cells in vivo and this technology could be extended into many different disease areas.

AAVantgarde has two proprietary AAV-based large gene delivery platforms. The first leveraging DNA recombination, named dual hybrid; and the second, based on protein trans-splicing, named AAV intein. 


The company is validating its proprietary platforms in two lead programs in Ophthalmology with a high unmet need: Usher Syndrome Type 1B-associated retinitis pigmentosa (Usher1B), using our dual hybrid platform; and Stargardt disease, using our AAV intein-mediated platform.

Discover more