cysteamine-pantetheine disulfide

TTI-0102 (cysteamine-pantetheine disulfide) is a novel sulfur-based small molecule disulfide prodrug developed by Thiogenesis Therapeutics for the treatment of mitochondrial and metabolic disorders. The molecule is composed of two cysteamine units and one unit of pantothenic acid (Vitamin B5), designed to release cysteamine through a metabolic 'gating mechanism' upon oral ingestion. This design aims to provide sustained cysteamine exposure and improve gastrointestinal tolerability compared to immediate-release cysteamine formulations. Its primary therapeutic actions include the depletion of intracellular cystine via thiol-disulfide exchange in lysosomes and the enhancement of antioxidant pathways, specifically the synthesis of glutathione and taurine. TTI-0102 is currently being evaluated in Phase 2 clinical trials for Leigh Syndrome Spectrum (LSS) and is in development for other indications including MELAS, pediatric metabolic dysfunction-associated steatohepatitis (MASH), and nephropathic cystinosis.

Other names

cysteamine-pantetheine disulfide