MVX-220 is an investigational hu68AAV gene therapy designed to deliver the human UBE3A gene to neurons of the brain with a single intra-cisterna magna (ICM) injection.

It has been shown in preclinical studies to restore UBE3A protein expression and ameliorate symptoms in the AS mouse model.

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MVX-220 will be evaluated in the ASCEND-AS (AAV-mediated expression in neurons for Angelman syndrome) Phase 1/2 clinical trial