The Muscular Dystrophy Association has awarded $1,195,762 over two years to biotechnology company Valerion Therapeutics (formerly 4s3 Bioscience) for development of a treatment for myotubular myopathy (MTM).
The Concord, Mass., company is developing a therapy for MTM that involves delivering myotubularin — the protein that is deficient in MTM — to muscle fibers using an experimental cell-penetrating strategy.
The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA's translational research program.
It will allow the company to continue testing its experimental compound 3E10FV-MTM1 in mice and to begin producing a pharmaceutical-grade drug that potentially can be tested in people with MTM.
To learn more, read MTM: MDA Funds Development of Myotubularin-Based Treatment.