The Duchenne Alliance commits $500,000 to Jerry Mendell M.D. and Milo Biotechnology to fund first Duchenne Follistatin Gene Therapy Trial

November 30, 2014

he International Duchenne Alliance announced today that it will allocate $500,000 to Jerry Mendell, M.D. and Milo Biotechnology to support the first ever follistatin gene therapy trial for Duchenne muscular dystrophy. The trial will be conducted by Mendell at Nationwide Children’s Hospital (Columbus, Ohio). Mendell has recently published positive data on follistatin in Becker Muscular Dystrophy.

According to Mendell, “This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease.” Based on that result, Mendell will now test if children with Duchenne Muscular Dystrophy can also benefit from the therapy.

Duchenne Alliance (DA) liaisons for the project – Save Our Sons from Sydney, Australia and Hope for Gus fromNew Hampshire, U.S. – recognized the potential value of follistatin gene therapy. DA liaisons provide support for projects by raising awareness among the foundations and community, collecting funds and managing the milestone-driven disbursements.

The therapy, developed at Nationwide Children’s Hospital by Mendell and Brian Kaspar Ph.D., is based on adeno-associated virus delivery of follistatin 344 to increase muscle strength and prevent muscle wasting and fibrosis. Because follistatin’s mechanism of action is not mutation specific, it could potentially help other forms of muscular dystrophy.

“This has truly been a community effort,” says Steve Dreher of Hope for Gus. “Fifteen foundations from three continents collaborated to fund this project.”

In addition to the initial funding, the DA may provide additional support to Milo Biotechnology for follow-up studies. “With unprecedented speed to funding, our member foundations continue to accelerate the pace of Duchenne biomedical research,” says Carlo Rago Ph.D., Scientific Director of the Duchenne Alliance.

The following foundations contributed to the funding:  Charley’s Fund (US), Duchenne Now (ENG), Harrison’s Fund (ENG), Hope for Gabe (US), Hope for Gus (US), JB’s Keys (US), Jett Foundation (US), Little Hercules Foundation (US), Michael’s Cause (US), Nash Avery Foundation (US), Pietro’s Fight (US), Ryan’s Quest (US), Save Our Sons (AU), Team Joseph (US), Two Smiles One Hope Foundation (US).

About Duchenne Alliance
The Duchenne Alliance is a group of independent non-profit organizations dedicated to defeating Duchenne Muscular Dystrophy. The Duchenne Alliance works to promote better collaboration among the many non-profit organizations around the world committed to conquering Duchenne and improving the lives of those living with this progressive and debilitating muscle wasting condition. The Duchenne Alliance member foundations collaborate to co-identify, co-review and co-fund the most promising biomedical research. The Duchenne Alliance Research Fund was established to streamline their biomedical research programs.  Please to follow and support our progress.

About Milo Biotechnology
Milo Biotechnology is a 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 potent myostatin inhibitor. Milo’s core technology was developed at and is exclusively licensed from Nationwide Children’s Hospital in Columbus, Ohio; the company is based inCleveland, Ohio, and has received funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund and from NIAMS Rare Disease SBIR program. Visit for more information.

SOURCE The Duchenne Alliance


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