Developing thiol-active drugs for critical unmet medical needs of pediatric patients with a metabolic component

Sulfur is the seventh most abundant element in the human body and plays a central role in the structure and function of thousands of biologically important molecules.

Thiols are a class of organic compounds and are one of the most biologically important subcategories of sulfur-containing molecules; this is largely due to their versatile chemistry and contribution to protein structure. Also known for their strong odor, many thiols have a smell similar to that of garlic or rotten eggs.

Thiols have a functional group of the form R−SH, where S is a sulfur atom, H is a hydrogen atom and R represents an alkyl or other organic substituent; the functional group is responsible for chemical reactions independent of the overall compound. Thiols have been limited in their commercial development because they have strong gastrointestinal side effects that have limited dosing flexibility and compliance.

Thiogenesis Therapeutics is a clinical-stage biotechnology company developing novel compounds, that are prodrugs and that act as precursors to certain thiols, with the intention of reducing their side effects and increasing dosing flexibility.

Prodrugs can benefit from the expedited 505(b)(2) regulatory pathway in the US and its equivalent in Europe. Initial therapeutic targets are pediatric indications in Rett syndrome, mitochondrial disease (“MELAS”), and non-alcoholic steatohepatitis (“NASH”).