EXG116

EXG116 is an investigational gene therapy being developed by Exegenesis Bio for the treatment of Rett Syndrome. Rett Syndrome is a rare neurodevelopmental disorder primarily caused by mutations in the MECP2 (methyl-CpG binding protein 2) gene, which is critical for normal brain function. EXG116 is designed to restore or modulate MECP2 function, likely through the delivery of a functional copy of the MECP2 gene or a related genetic intervention. The program is currently in the IND-enabling phase and is being evaluated for administration via intravenous (IV) or intrathecal (IT) routes to ensure broad delivery to the central nervous system.