ATL-002

ATL-002 is a small-molecule pharmacological chaperone being developed by Atlas Molecular Pharma for the treatment of Tyrosinemia type I. The drug is designed to bind and stabilize the enzyme fumarylacetoacetate hydrolase (FAH), which is deficient in patients with this rare genetic disorder. In Tyrosinemia type I, mutations in the FAH gene lead to enzyme instability or misfolding, resulting in the accumulation of toxic metabolites such as maleylacetoacetate and fumarylacetoacetate, which cause severe liver and kidney damage. By acting as a chaperone, ATL-002 aims to restore or enhance the functional activity of the FAH enzyme, thereby preventing the buildup of toxic precursors and offering a potential therapeutic alternative or adjunct to current standard-of-care treatments.