TSCA-001

TSCA-001 is a small molecule drug under development by Everfront Biotech for the treatment of Spinocerebellar Ataxia Type 3 (SCA3), also known as Machado-Joseph disease. The drug is designed to modulate the early metabolic pathway of kynurenine, specifically by reducing the expression of tryptophan 2,3-dioxygenase (TDO2). This action decreases production of the neurotoxic metabolite quinolinic acid and reduces levels of active calpain, an enzyme involved in degrading mutant ATXN3 protein. By targeting these pathways, TSCA-001 aims to decrease generation of toxic protein fragments and associated neurotoxicity in SCA3. The compound demonstrates high permeability and can cross the blood-brain barrier, allowing targeted delivery to brain regions such as the cerebrum and cerebellum[1][2][3].