Developing Treatments for Rare Diseases

Using our powerful REVEAL-Tx^TM drug discovery platform, our scientists are identifying and validating promising approaches to develop novel treatments aiming to extend and improve the quality of life of patients with rare diseases. In our most advanced program, the company is conducting a phase 2 clinical trial of HST5040, an oral small molecule investigational drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA). These genetic disorders are caused by deficiencies of certain metabolic enzymes that lead to buildup of life-threatening toxins.