GT0005X

GT0005X is an adeno-associated virus (AAV) gene therapy candidate developed by ONODERA GT Pharma for the treatment of Tay-Sachs disease. The therapy is designed to deliver functional copies of the HEXA and HEXB genes, which encode the alpha and beta subunits of the enzyme hexosaminidase A (HexA). Tay-Sachs disease is a fatal neurodegenerative lysosomal storage disorder typically caused by mutations in the HEXA gene, leading to the toxic accumulation of GM2 gangliosides in the central nervous system. By providing both subunits required for the formation of the HexA heterodimer, GT0005X aims to restore enzymatic activity and mitigate disease progression. The program is currently in the preclinical stage of development.