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.
"The goal of the MVP program is to help reduce the risk of early-stage therapeutics, so that other investors can bring them into the clinic, and eventually to patients," said Jane Larkindale, MDA's vice president of research.
"Projects like this one fit perfectly into this model. This is an early-stage therapeutic for a rare disorder, where the return on investment may not be as large as for other disorders. MVP's funding allows other investors to come in later in development, when there is a greater chance of success due to the work supported by MVP.
"Furthermore, this project is also great proof of concept of the company's technology, which allows delivery of proteins to muscle — in this case myotubularin, but it also may be able to be applied to other proteins for other diseases in MDA's program."
MDA began supporting development of this therapy in 2010, through a grant to Dustin Armstrong at 4s3.
In January 2013, a research group that included Armstrong and other MDA grantees published a paper showing myotubularin-deficient mice with a disorder mimicking human MTM showed improvements in strength and movement in response to injections of 3E10Fv-MTM1.
The compound is a combination of the myobutularin protein and the cell-penetrating molecule 3E10.
For more information, see MTM Mice Respond to Modified Myotubularin Protein Injections.