AAV9-SLC2A1

AAV9-SLC2A1 is an investigational gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector encoding the human SLC2A1 gene. It is designed to treat Glucose Transporter Type 1 Deficiency Syndrome (GLUT1-DS), a rare genetic metabolic disorder caused by mutations in the SLC2A1 gene, which results in insufficient GLUT1 protein at the blood-brain barrier. By delivering a functional copy of the SLC2A1 gene via intrathecal administration, the therapy aims to restore glucose transport into the brain, potentially improving neurological function and reducing seizure burden. The program is currently in early-stage clinical development, with first-in-human studies conducted by researchers at Jichi Medical University.