Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. Applied Therapeutics leverages technological advances to target molecules and pathways with proven roles in disease biology. AT-007 is an investigational, novel Aldose Reductase Inhibitor (ARI) currently in pivotal-stage development as an oral therapy for treatment of Galactosemia. AT-007 is also being developed for the treatment of Sorbitol Dehydrogenase Deficiency (SORD) and Phosphomannomutase 2 Deficiency - a Congenital Disorder of Glycosylation (PMM2-CDG). AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the FDA for the treatment of Galactosemia and PMM2-CDG.
At Applied Therapeutics, our culture is to work collaboratively to develop transformative treatments for patients. We apply the right science and the right development strategies to rapidly develop treatments for people who urgently need them. When someone is fighting a fatal or debilitating disease for which no drugs are available, every day counts.