GAL301

GAL301 is an adeno-associated virus (AAV)-based gene therapy being developed by Galibra Neuroscience for the treatment of GABA-T deficiency. The therapy is designed to deliver a functional copy of the *ABAT* gene, which encodes the enzyme 4-aminobutyrate aminotransferase (GABA transaminase or GABA-T). GABA-T is responsible for the catabolism of gamma-aminobutyric acid (GABA). In patients with GABA-T deficiency, mutations in the *ABAT* gene lead to excessive accumulation of GABA, resulting in severe neurological symptoms such as psychomotor retardation, hypotonia, and seizures. GAL301 aims to restore functional GABA-T activity to normalize GABA levels. It is currently in the discovery stage, undergoing validation in mouse models.