Pipeline

AAVB-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.

AAVB-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.

We are working on our next monogenic IRD indications with high unmet need to expand our ophthalmology franchise.

Our platforms can also be used ex-ophthalmology, and we are exploring  different tissues (Inner ear, CNS, etc…) to expand our pipeline.