Galibra Neuroscience is a Boston Children’s Hospital-affiliated biotechnology company co-founded by Henry Lee, MPhil, PhD, and Alexander Rotenberg, MD, PhD, dedicated to developing gene therapies for disorders disrupting γ-aminobutyric acid (GABA) signaling, the brain’s primary inhibitory neurotransmitter system.  The company’s initial focus is succinic semialdehyde dehydrogenase deficiency (SSADHD), a rare genetic disorder caused by ALDH5A1 mutations that lead to toxic GABA metabolite accumulation, resulting in intellectual disability, autism features, drug-resistant epilepsy, and other neurodevelopmental issues.  Galibra’s lead candidate, GAL005, is an AAV-based gene replacement therapy delivering a functional human ALDH5A1 gene to the brain, with preclinical studies in mouse models and patient-derived neurons demonstrating efficacy in reversing SSADHD symptoms.  Backed by strong support from the SSADHD patient community, families, and clinicians, Galibra benefits from unique access to natural history data, biomarkers, patient cohorts, and clinical infrastructure to accelerate translation from bench to bedside.
The broader pipeline targets related GABA disorders, including SLC6A1-related conditions, GABA transaminase deficiency, GABA receptor variants, and select drug-resistant epilepsies, Galibra aims to leverage an advanced AAV platform with brain-penetrant capsids (such as Apertura’s TfR1 CapX technology) and shared manufacturing expertise to expand impact across these debilitating conditions.