The US Food and Drug Administration (FDA) has granted orphan drug designation for gene therapy to Avrobio for the purpose of treating mucopolysaccharidosis type 2, also known as Hunter syndrome. Hunter Syndrome is a rare disease that mainly affects young boys as a result of lysosomal enzyme iduronate-2-sulfatase (IDS) deficiency. In partnership with the University of Manchester, Avrobio is to initiate a Phase I/II clinical trial for the latter’s gene therapy. The company observed that their gene therapy is one of the four to receive orphan drug designation.
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