California, USA-based Armagen says that the US Food and Drug Administration has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II),a rare, genetic lysosomal storage disease caused by a deficient or absent enzyme, iduronate-2-sulfatase. It is a life-threatening disease affecting children as young as two years of age.
AGT-182 is a human insulin receptor monoclonal antibody-fused iduronate 2-sulfate designed to cross the blood brain barrier (BBB) through the insulin receptors present on the BBB. Armagen is currently preparing an IND for AGT-182 in order to begin clinical investigation in the first half of 2014.
Prepares for transition to clinical-stage company
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