Pipeline
AAVantgarde's Pipeline
AAVantgarde 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 in Ophthalmology with a high unmet need: Usher Syndrome Type 1B associated retinitis pigmentosa (Usher1B), using the dual hybrid platform; and Stargardt disease, using our AAV intein-mediated platform.
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 be extended into many different disease areas.
USHER 1B
Usher 1BAAVB-081 is our lead program, an intra-retinal AAV8-based dual hybrid product targeting MYO7A-associated Usher syndrome (USH1B), the most common form of Usher type 1.
STARGARDT
StargardtAAVB-039 is our second program, an intra-retinal AAV8-intein mediated product targeting ABCA4, the gene associated with Stargardt disease, the most common inherited retinal disorder caused by mutations in a single gene.
