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The Muscular Dystrophy Association has granted $2.5 million to a Chapel Hill biopharmaceutical firm to pay for continued trials of a gene therapy targeting Duchenne muscular dystrophy.
Asklepios BioPharmaceutical Inc. said it will match the MDA grant to fund continued trials of Biostrophin, its lead gene therapy for DMD.
The company currently is testing the safety of administering Biostrophin into a single muscle of six human patients with the disease.
The new funds will enable Asklepios to pay for the next stage of trials, which will test the safety and effectiveness of Biostrophin in canine and non-human primate models, according to a written statement released by the company Tuesday.
"The next step in this project is important to establish that we can deliver (Biostrophin) to multiple muscles at once," Sharon Hesterlee, vice president of Translational Research at MDA, said in a written statement.
The $2.5 million grant is the largest grant MDA has awarded to a for-profit company. MDA provided $1.6 million for the first phase of the trial.
There currently is no life-sustaining treatment for the disease, which is genetically inherited.
AskBio is a private development-stage biotechnology company that was spun out from the University of North Carolina at Chapel Hill. It currently has about five full-time employees.