Milo Biotechnology Develops Gene Therapy to Treat Muscular Dystrophy

Finding a treatment for an incurable disease is the Holy Grail of bioscience research.

And the modern day quest is every bit as inspiring as the Arthurian legends—especially to families with loved ones who suffer.

One of those quests is the fight against muscular dystrophy (MD).


MUSCULAR DYSTROPHIES are 30+ inherited diseases caused by defects in a person’s genes. Some forms appear in infancy or childhood; others appear in adults. All forms of MD grow worse as a person’s muscles get weaker, causing decreases in mobility and making the tasks of daily living difficult.

There is no known cure for MD, but there is new hope that a promising gene-based therapy from Milo Biotechnology, a life science spinout of Nationwide Children’s Hospital, is delivering treatment for muscular dystrophy and various other muscular disorders.

Gene therapy clinical trial demonstrates first functional improvement in any form of MD

Milo Biotechnology is the exclusive licensee of patented therapy developed at Nationwide Children’s by Brian Kaspar, Ph.D. and Milo co-founder, and Jerry Mendell, MD and director of Nationwide Children’s Center for Gene Therapy.

A Phase I/II clinical study of the therapy in Becker Muscular Dystrophy, completed at Nationwide Children’s in the fall of last year with Dr. Mendell as chief investigator, established initial safety and efficacy for the follistatin gene therapy.

This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy.

Clinical results are reported in the December 2014 online issue of Molecular Therapy.  The results represent a major advance for those suffering from muscle disease.

Fifteen foundations from three continents collaborate to fund first-ever follistatin gene therapy trial for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD), the most common form of the disease in children, is found mainly in young boys; symptoms are capable of appearing before the age of six.

The disease progresses rapidly and focuses mainly on the lower portion of the body. The ability to walk may be lost by age 12. Breathing difficulty and heart disease usually start by age 20.

In an exciting development, the International Duchenne Alliance (DA) announced that it will allocate $500,000 to Milo Biotechnology and Dr. Mendell to support the first ever gene therapy trial to test if children with can also benefit from the follistatin gene therapy.


The Duchenne Alliance is a group of independent non-profits dedicated to defeating Duchenne muscular dystrophy and to improve the lives of those living with this progressive and debilitating muscle wasting condition.


Two additional Duchenne MD trials are underway at Nationwide Children’s.

This is powerful example of how the synergy between an internationally recognized research institution like Nationwide Children’s, the dedication of multiple non-profit organizations to eradicating this disease, and a nimble new biotechnology spinout can accelerate the progress of a new therapy—in this case, one that has shown in trials that it can help people with MD in a way that they couldn’t be helped before.

“The strongest validation for the therapy is that we now have clinical data from patients with Becker Muscular Dystrophy,” said Al Hawkins, Milo Biotechnology CEO. “With the combination of foundation funding and resources at National Children’s Hospital, we’ve come far.

Rev1 Ventures makes landmark investment in Milo Biotechnology

Rev1 Ventures and Nationwide Children’s have joined forces to help propel Milo Biotechnology through this next phase of testing.

Rev1 Ventures announced an investment in the company that will fund analysis and plans for a path to regulatory and medical reimbursement approvals as the clinical trials progress.


Podcasts highlighting the latest in MD and other inherited neuromuscular disease research from Nationwide Children’s.