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Rare Disease Gene Therapy: Milo Technology’s 1st DMD Patient In Follistatin Trial

April 20, 2015

Milo Biotechnology is a clinical stage startup company developing treatments to strengthen muscle for neuromuscular diseases. The company is developing a muscle-strengthening Follistatin protein gene therapy for Duchenne Muscular Dystrophy (DMD), in collaboration with Nationwide Children’s Hospital (Cleveland).

Milo Biotechnology announces April 9th, that the first DMD patient has been treated in a Follistatin gene therapy trial at Nationwide Children’s Hospital. The therapy is delivered by intramuscular injection. This is a 6 patient clinical trial.

Milo Biotechnology’s main investigational product is AAV1-FS344, which leads to the local expression of Follistatin, a potent TGFb ligand inhibitor inhibitor. The gene therapy technology was developed at and is exclusively licensed from Nationwide Children’s Hospital. AAV1-FS344 is an adeno-associated gene therapy. It receives FDA Orphan Drug Designation (ODD) in November 2012.

Milo Technology’s FDA ODD Database Record For DMD Gene Therapy

Generic Name: Adeno-associated virus transgene of follistatin
Trade Name: n/a
Date Designated: 11-19-2012
Orphan Designation: Treatment of Duchennes and Becker’s muscular dystrophy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Milo Biotechnology 2322 Delaware Drive Cleveland Heights, OH 44106


Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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