AVB-201

AVB-201 is an investigational gene therapy in preclinical development by AviadoBio for the treatment of frontotemporal dementia caused by mutations in the progranulin gene (FTD-GRN). FTD-GRN is a progressive neurodegenerative disease driven by a deficiency in the progranulin (PGRN) protein, which is critical for lysosomal function and neuronal health. AVB-201 utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the *GRN* gene to the central nervous system, aiming to restore PGRN levels and halt disease progression. This program complements AviadoBio's lead candidate, AVB-101, and leverages the company's proprietary delivery platforms designed to achieve optimal distribution within the brain, potentially utilizing blood-brain barrier (BBB)-penetrant vectors or alternative delivery routes.