The company specializes in creating disease-modifying therapy to treat rare and ultra-rare diseases, specifically targeting the root cause of the disease. Palvella Therapeutics was founded in 2015 and headquartered in Wayne, Pennsylvania, with a focus on treating pachyonychia congenital, a chronically debilitating genetic disease. Their mission is to serve individuals suffering from serious rare genetic diseases with no approved treatments, by developing pathogenetically targeted therapies that enhance quality of life.