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 Rare Pediatric Drug (RPD) designation for ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease.
September 15, 2022
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