Technology and
Pipeline

AAVantgarde Bio has two proprietary, AAV-based large gene delivery platforms. The first leveraging DNA recombination, named dual hybrid; and the second, a protein trans-splicing, named AAV intein. The company is validating the platforms in two lead programs: Usher Syndrome Type 1 B associated retinitis pigmentosa (Usher1B), using dual hybrid; and Stargardt disease, using AAV intein.

AAV gene therapy has been limited by transgene capacity, and the Company’s platforms enable delivery of large genes to tissue and cells in vivo; something that could extend into many disease areas.

PRE-CLINICALPHASE I/IIPIVOTAL
Usher1B
Stargardt