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GEN News Highlights : May 31, 2013

MDA Gives Valerion’s Preclinical MTM Pipeline $1.2M Boost

The Muscular Dystrophy Association (MDA)’s Venture Philanthropy arm has injected $1.2 million into Valerion Therapeutics’ myotubular myopathy (MTM) pipeline, funding the firm to continue its development of an experimental treatment, 3E10Fv-MTM1, which uses a cell-penetrating antibody to deliver myotubularin to muscle fibers.

Valerion’s 3E10Fv-MTM1 has shown promise in preclinical studies in which mice administered the experimental drug showed improved muscle strength, function, and structure.

MDA said the funds will allow Valerion to continue testing 3E10Fv-MTM1 in mice and work toward pharmaceutical-grade development.

“We are interested in this technology because it may provide a novel method of delivering proteins to muscle,” Jane Larkindale, vp of research at MDA, said in a statement. “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."

Added Valerion’s Dustin Armstrong, Ph.D., vp of research: “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."