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MDA Awards Valerion Therapeutics $1.2 Million to Continue Myotubular Myopathy Research
Date:5/30/2013

Tucson, Ariz. (PRWEB) May 30, 2013

The Muscular Dystrophy Association has awarded Valerion Therapeutics $1,195,762 to continue development of an experimental cell-penetrating treatment that may represent a unique approach to treating myotubular myopathy (MTM).

The experimental treatment, 3E10Fv-MTM1, uses a cell-penetrating antibody to deliver myotubularin to muscle fibers. Myotubularin is the protein that is deficient in MTM1.

Researchers at the Concord, Mass., biotech have taken a different approach to creating an effective enzyme-replacement therapy by utilizing an antibody-protein fusion that can penetrate the muscle cell. After receiving injections of 3E10Fv-MTM1 for only two weeks, mice showed improved muscle strength, function and structure.

“We are interested in this technology because it may provide a novel method of delivering proteins to muscle,” said MDA Vice President of Research Jane Larkindale. “In January, the company published data that showed that the technology allowed the protein to get into the muscles of a mouse model of the disease, where it had a positive effect on the disease course. It also may be able to be applied to other proteins for other diseases in MDA's program."

MTM is an inherited muscle disease that can cause severe, general muscle weakness complicating basic activities such as breathing and swallowing.

The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA. It will allow the company to continue testing 3E10Fv-MTM1 in mice and develop a pharmaceutical grade treatment that eventually m
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