Posted by Michael Wonder on 15 Sep 2022
Aro Biotherapeutics receives FDA rare paediatric drug designation for ABX1100 for the treatment of Pompe disease
15 September 2022 - Aro Biotherapeutics today announced that the US FDA granted rare paediatric drug designation for ABX1100, an investigational centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease.
ABX1100 is a novel approach to treating Pompe disease that aims to reduce glycogen buildup in the muscles.
