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Milo Biotechnology Announces FDA Orphan Drug Designation for AAV1-FS344 for Treatment of Duchenne and Becker Muscular Dystrophy
Date:12/12/2012

CLEVELAND, Dec. 12, 2012 /PRNewswire/ -- Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

The program is currently in a Phase I/II trial at Nationwide Children's Hospital in adult patients with Becker muscular dystrophy and inclusion body myositis, a trial funded by the foundation Parent Project Muscular Dystrophy. "The Orphan Drug designation will provide incentives for Milo Biotechnology's continued development of the therapy in rare muscular dystrophies with the greatest medical need," said CEO Al Hawkins. "We hope that our potent muscle strengthening approach will someday augment emerging genetic strategies to transform care for these intractable diseases."

Duchenne muscular dystrophy is the most common inherited myopathy, caused by the absence of dystrophin protein. Becker muscular dystrophy, a less severe form of Duchenne muscular dystrophy, is caused by reduced levels of dystrophin protein. With both types, patients experience progressive muscle weakness, and cardiac and respiratory complications.

The Orphan Drug Act (ODA) grants special status for products to treat rare diseases. Orphan designation qualifies the sponsor of the product for a tax credit and seven years of marketing exclusivity via the ODA. A marketing application for a prescription drug product that has been designated as a drug for a rare disease or condition is not subject to a prescription drug user fee. The disease or condition for which the drug is intended must affect fewer than 200,000 people in the United States.   

About Milo Biotechnology Milo Biotechnology is an early clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases.  Its lead program, AAV1-FS344, leads to the local expression of follistatin, a myostatin inhibitor. The company is developing earlier stage programs to increase expression of follistatin regionally to muscle groups and systemically via intravascular injection.  Milo's core technology was developed at and is exclusively licensed from Nationwide Children's Hospital in Columbus, Ohio; the company is based in Cleveland, Ohio, and has received funding from JumpStart Inc. and The North Coast Opportunities Technology Fund.


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