Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in the muscle, for the treatment of Pompe disease.
Aro Biotherapeutics Receives FDA Orphan Drug Designation for ABX1100 for the Treatment of Pompe Disease
