Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II or Hunter syndrome. and is expected to initiate a Phase I/II clinical trial for MPS II in partnership with the University of Manchester.
Hunter syndrome primarily affects young boys and is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (IDS).