GT0004X

GT0004X is an adeno-associated virus (AAV) gene therapy candidate developed by ONODERA GT Pharma for the treatment of Spinocerebellar Ataxia Type 1 (SCA1). SCA1 is an autosomal dominant neurodegenerative disease characterized by progressive ataxia and cerebellar atrophy, caused by a CAG trinucleotide repeat expansion in the *ATXN1* gene. This mutation results in an abnormally long polyglutamine (polyQ) tract in the ataxin-1 protein, leading to toxic gain-of-function and neuronal death. GT0004X aims to intervene in this disease process using AAV-mediated delivery to the central nervous system. The program is currently in preclinical development.