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AskBio Licenses GSK Technology To Attack Muscular Dystrophy, Hemophilia B

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CHAPEL HILL, N.C. — Asklepios Biopharmaceutical

, or AskBio, has licensed technology from GlaxoSmithKline that could prove useful in development treatments for muscular dystrophy (MD) and hemophilia B.

Financial terms were not disclosed.

AskBio, which has received funding support from the Muscular Dystrophy Association, will utilize materials from GSK that could lead to development of gene therapy treatments. GSK originally licensed the technology from the University of Pennsylvania.

AskBio will have exclusive access to what it called "recombinant adeno-associated virus vector (rAAV) serotypes". Serotypes are groups of microorganisms or viruses based on antigens. Antigens trigger a response by the body's immune system. Adeno-associated viruses are the smallest known viruses.

AskBio currently has a drug in phase I clinical trial to treat Duchenne Muscular Dystrophy, the most common form of MD. The drug Biostrophin is engineered from rAAV and other materials to deliver what AskBio calls a "genetically re-engineered mini-gene" to treat the disease at a molecular level. The drug "expresses" dystrophin, the gene that is mutated by MD that in turn leads to progressive muscle weakness.

Biostrophin utilizes biological nanoparticle technology developed by AskBio designed to deliver therapeutic genetic material to cells. Biostrophin targets muscle cells, seeking to turn them into what the company calls "bioreactors".

"We are looking forward to obtaining this license to address two complex diseases," said Sheila Mikhail, chief executive officer of AskBio. "This license will bring a combined platform of technologies to the muscular dystrophy and hemophilia communities that will enhance patient care in these two important orphan drug diseases."

Orphan drugs, as determined by the Food and Drug Administration, are for treating diseases that affect fewer than 200,000 people in the U.S.

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