AAV-SSADH

AAV-SSADH (also known as GAL005 or AAV-FLnP-hALDH5A1) is an investigational adeno-associated virus (AAV)-based gene replacement therapy designed to treat succinic semialdehyde dehydrogenase deficiency (SSADHD), an ultra-rare autosomal recessive neurometabolic disorder. SSADHD is caused by loss-of-function mutations in the ALDH5A1 gene, which encodes succinate-semialdehyde dehydrogenase (SSADH), an enzyme critical for catabolizing the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). The therapy utilizes a blood-brain barrier (BBB)-penetrating capsid (such as AAV-PHP.eB or CapX/AAV-BI-hTFR1) to deliver a functional copy of the human ALDH5A1 coding sequence under the control of a native human ALDH5A1 promoter. Preclinical studies conducted at Boston Children's Hospital, Harvard Medical School, and UMass Chan Medical School demonstrated that AAV-SSADH restores SSADH protein expression, reduces pathological accumulation of GABA and gamma-hydroxybutyrate (GHB), and significantly improves survival and reduces epilepsy-related mortality in mouse models. Galibra Neuroscience is advancing the program (as GAL005) toward clinical trials, with manufacturing support from Genezen.

Other names

AAV-PHP.eB-ALDH5A1AAV-PHP.eB-hALDH5A1