GCB-002

GCB-002 is an investigational gene therapy developed by Genecombio for the treatment of Rett Syndrome, a rare neurodevelopmental disorder caused by mutations in the *MECP2* gene. The therapy is designed to deliver a functional copy of the human *MECP2* gene directly to the central nervous system via a single intrathecal injection. By restoring the expression of the MECP2 protein in neurons, GCB-002 aims to alleviate the severe neurological and developmental symptoms characteristic of the condition. It is currently being evaluated in dose-escalation clinical trials to determine its safety, tolerability, and preliminary efficacy in pediatric patients.