What Is Milo Biotechnology?

Milo Biotechnology is developing therapies for the treatment of muscular dystrophy that increase muscle strength or decrease the rate of muscle atrophy.

The biotechnology company’s core technology, a gene therapy-based up-regulation of the muscle-strengthening follistatin protein, was developed at and is exclusively licensed from Nationwide Children’s Hospital.

Milo Biotechnology is based in Cleveland, Ohio, and has received seed funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund and a grant from NIH-NIAMS rare diseases SBIR program. In late 2012, the company received FDA Orphan Drug Designation for its treatment for Duchenne and Becker Muscular Dystrophy.

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Recent News


Milo Biotechnology’s Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients

Data published today in the journal Molecular Therapy demonstrates initial clinical proof of principle of a gene therapy to increase... Read More →

Preliminary Findings in Milo Follistatin Gene Transfer Study Promising

Preliminary results from a trial to test the safety of injecting follistatin genes into the thigh muscles of adults with... Read More →
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