GAL005

GAL005 is an investigational AAV-mediated gene replacement therapy being developed by Galibra Neuroscience for the treatment of Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD). SSADHD is a rare autosomal recessive GABA metabolic disorder caused by loss-of-function mutations in the ALDH5A1 gene, which leads to the accumulation of GABA and gamma-hydroxybutyric acid (GHB). GAL005 utilizes Apertura Gene Therapy's proprietary TfR1 CapX AAV capsid technology, which is engineered to cross the blood-brain barrier following intravenous administration. The therapy is designed to achieve brain-wide gene replacement with cell-specific expression to restore succinic semialdehyde dehydrogenase (SSADH) enzyme function, thereby normalizing neural function and correcting the underlying metabolic imbalance. It is currently in preclinical development.