Minoryx Therapeutics develops treatments for Inborn Errors of Metabolism, a group of rare diseases of genetic origin affecting 3,700 newborns every year in Europe. Such diseases are life threatening and highly debilitating, usually with neurological manifestations and affect most severely the pediatric population. The social and economic impact of these diseases cannot be overstated and, with 90% of cases lacking curative treatments, there is a high unmet medical need. Minoryx develops new drugs to improve both the quality of life and life expectancy of the patients.
Minoryx has developed SEE-Tx (Site-directed Enzyme Enhancement Therapy), a technological platform that incorporates computational methods developed in the Barril’s lab at Barcelona University. SEE-Tx enables us to develop a new generation of drugs: the non-competitive pharmacological chaperones, which recover the functionality of mutated proteins, thus attacking the root of the disease. We have discovered non-competitive pharmacological chaperones in a range programs, which are at various stages of preclinical research.