TUCSON, Ariz. and PROVIDENCE, R.I., Jan. 5, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) today announced it has awarded $1.0 million to Tivorsan Pharmaceuticals to help speed pre-clinical work vital to a filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for a recombinant humanized form of biglycan as a potential muscular dystrophy treatment. The new funding to the Providence-based biotechnology firm comes from MDA Venture Philanthropy (MVP), a part of MDA's translational research program.
The project being advanced by Tivorsan is focused on treating Duchenne muscular dystrophy (DMD) by up-regulating the utrophin protein that's been shown to provide some compensation for disease-causing dystrophin deficiency in animals with DMD-like muscular dystrophy. The company's TVN-102 experimental drug is based on biglycan, a naturally occurring protein found on the membrane surrounding each muscle fiber. In the standard mouse model of DMD, biglycan reduced muscle damage, reduced muscle degeneration and improved muscle function.
DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily affecting voluntary muscles. Caused by mutations in the gene that makes dystrophin, a protein that normally protects muscle cells and keeps them intact, DMD eventually weakens all voluntary muscles, and the heart and breathing muscles. DMD affects 1 in 3,500 boys with an estimated patient population exceeding 50,000 worldwide.
"We are delighted to have a productive collaboration with Dr. Justin Fallon's laboratory at Brown University to bring such a promising potential muscular dystrophy treatment to market," said Joel B. Braunste