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ArmaGen Announces Strategic Licensing and Collaboration Agreement with Shire to Develop AGT-182 for Treatment of Hunter Syndrome
Date:7/23/2014

vancing ArmaGen's proprietary pipeline of innovative therapies."

"Our agreement with ArmaGen strengthens our long-standing commitment to the Hunter syndrome community to bring forward novel therapies that have the potential to dramatically redefine the treatment paradigm and address the most critical unmet needs," said Philip J. Vickers, Ph.D., Global Head of Research and Development at Shire. "AGT-182 has the potential to be an important new therapy to our existing portfolio of Hunter syndrome programs. We plan to apply our proven ability to develop therapies for rare genetic diseases to progress AGT-182 as a potential treatment that offers hope to patients with Hunter syndrome and their families."

About Hunter Syndrome

Hunter syndrome, also known as mucopolysaccharidosis type II, or MPS II, is a lysosomal storage disorder caused by inadequate activity of the enzyme iduronate-2-sulfatase (IDS), which is needed to break down complex sugars produced by the body. The buildup of these complex sugars, known as mucopolysaccharides, interferes with functioning of certain cells and organs, leading to serious complications including developmental delays and mental impairment. Symptoms of Hunter syndrome include growth delay, joint stiffness and coarsening of facial features. In severe cases, patients experience respiratory and cardiac problems, enlargement of the liver and spleen, and neurological deficits that can lead to premature death. Hunter syndrome primarily affects males and is almost always severe, progressive and life-limiting. Available treatments for Hunter syndrome are not expected to cross the blood-brain barrier (BBB) in clinically relevant amounts and therefore do not address the progressive neurological complications of the disease.

About AGT-182 

AGT-182 is a novel, investigational ERT that has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the
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