Thiogenesis Therapeutics

Thiogenesis Therapeutics is a clinical-stage biotech company developing novel thiol-based small molecules designed to address oxidative stress and mitochondrial dysfunction in rare pediatric diseases. Its lead compound, TTI‑0102, has demonstrated good tolerability in human studies and is advancing into Phase 2a trials for Leigh syndrome spectrum after FDA IND clearance. Additional programs are planned or ongoing in MELAS, Rett syndrome, and pediatric MASH. The company’s approach centers on boosting intracellular cysteine to enhance glutathione production—addressing key limitations of previous thiol drugs such as short half-life and GI side effects.