MZ-9138

MZ-9138 is an investigational gene therapy designed to treat WWOX deficiency disorders such as WOREE syndrome (WWOX-related epileptic encephalopathy). The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human WWOX gene, driven by a neuron-specific Synapsin I promoter, directly to brain cells. By restoring WWOX expression in neurons, this approach aims to address severe neurological symptoms including epileptic encephalopathy and developmental delay. Preclinical studies have shown that intracerebroventricular administration of AAV9-Synapsin I-WWOX can rescue disease phenotypes in mouse models. Developed by Mahzi Therapeutics, MZ-9138 is currently in late preclinical development with plans for first-in-human clinical trials targeting WOREE syndrome[1][2][3][4][5].

Other names

Recombinant AAV9 viral vector containing the transgene encoding the human WW domain-containing oxidoreductase protein under the control of a human Synapsin I promoter