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MDA Awards Valerion Therapeutics $1.2 Million to Continue Myotubular Myopathy Research

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 may be tested in individuals with MTM.

"We are extremely grateful for the continued support of MDA,” said Dustin Armstrong, Ph.D., vice president, research at Valerion Therapeutics. “This preclinical funding will advance testing of our myotubular myopathy product candidate and further validate the potential of our therapeutic platform. Valerion is committed to utilizing our antibody-based delivery system to target genetic diseases, and our partnership with MDA provides additional encouragement for addressing these neuromuscular disorders with high unmet need."

MDA began supporting development of this therapy in 2010 through a grant to Armstrong, who was chief scientific officer at 4s3 Bioscience at the time. 4s3 is now Valerion Therapeutics, taking its name from the Latin term valere meaning “to flourish, be strong and healthy.”

To learn more, read "MTM: MDA Funds Development of Myotubularin-Based Treatment."

About Valerion
Valerion Therapeutics (formerly 4s3 Bioscience) is a biotechnology company focused on developing therapies for orphan genetic diseases using a novel antibody-based targeting system.

Valerion takes its name from the Latin word valere meaning “to flourish, to be strong and healthy.”
Valerion’s platform technology allows for enhanced intracellular delivery of active biological therapeutics for neuromuscular diseases, including enzyme-deficiency disorders and muscular dystrophies. Currently, the company’s lead preclinical programs focus on the unmet medical needs of myoytubular myopathy and myotonic dystrophy. Valerion completed a $20 Million Series A investment with Alopexx Enterprises and Genzyme Ventures in 2012. In addition to this venture funding, the company has obtained competitive grant support from the Massachusetts Life Sciences Center (MLSC), the NIH and the Muscular Dystrophy Association.
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About MDA

MDA is the nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, ALS and related diseases today affecting more than 1 million Americans. It does so by funding research, providing comprehensive health care and support services, and through advocacy and education.

In addition to funding more than 250 research projects worldwide, MDA maintains a national network of 200 medical clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides local summer camp opportunities for thousands of youngsters living with progressive muscle diseases.

For more information, visit and follow us on Facebook ( and Twitter @MDAnews.

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