TUB102

TUB102 is a preclinical antisense oligonucleotide (ASO) being developed by Synaptix Biotherapeutics for the treatment of TUBB4A-related leukodystrophy. While the company's lead program, TUB001, targets the specific H284Y mutation, TUB102 is designed to address other mutations within the TUBB4A gene. The drug utilizes a gene-silencing mechanism to reduce the expression of the mutated tubulin beta-4A chain protein, which is responsible for the progressive loss of myelin in the central nervous system. By lowering the levels of the toxic or dysfunctional protein, TUB102 aims to prevent the neurodegeneration associated with this rare genetic disorder. It is currently in the preclinical stage of development.