AAV9-SynI-WWOX

AAV9-SynI-WWOX (MZ-9138) is an investigational adeno-associated virus serotype 9 (AAV9) gene therapy designed to treat WWOX-related neurodevelopmental disorders, including WWOX-related epileptic encephalopathy (WOREE) syndrome (also known as developmental epileptic encephalopathy-28) and spinocerebellar ataxia 12 (SCAR12). Originally developed by the Hebrew University of Jerusalem and licensed to Mahzi Therapeutics, the therapy delivers a functional copy of the human WW domain-containing oxidoreductase (WWOX) cDNA driven by the neuron-specific human Synapsin I (hSynI) promoter. By targeting mature neurons, AAV9-SynI-WWOX aims to restore WWOX expression, rescue myelination defects, suppress neuroinflammation, and reduce neuronal hyperexcitability and seizures. The therapy has demonstrated robust efficacy in preclinical models and was first administered to an infant under a compassionate-use program in 2026.

Other names

AAV9-WWOXAAV-9-WWOXAAV 9-WWOXAAV9-WWOX gene therapyAAV-9-WWOX gene therapyAAV 9-WWOX gene therapyAAV9-hSynI-WWOX gene therapyAAV-9-hSynI-WWOX gene therapyAAV 9-hSynI-WWOX gene therapy