GT0006X

GT0006X is an adeno-associated virus (AAV)-based gene therapy candidate developed by ONODERA GT Pharma for the treatment of GLUT1 deficiency syndrome (GLUT1DS). The condition is a rare genetic metabolic disorder caused by mutations in the SLC2A1 gene, which encodes the glucose transporter protein type 1 (GLUT1). This protein is responsible for transporting glucose across the blood-brain barrier; its deficiency leads to neurodevelopmental impairment, seizures, and movement disorders due to insufficient brain energy supply. GT0006X is designed to deliver a functional copy of the SLC2A1 gene to restore GLUT1 expression and normalize glucose transport into the central nervous system. The program entered Phase 1/2 clinical development in July 2025.