Milo Treats First DMD Patients with Novel Follistatin Gene Therapy

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Clinical stage startup company Milo Biotechnology has begun treating the first Duchenne muscular dystrophy (DMD) patients with follistatin gene therapy. The treatment is being provided through intramuscular injections and its effects in maintaining or restoring muscle function are being studied at Nationwide Children’s Hospital.

The research team, which is led by Jerry Mendell at Nationwide Children’s Hospital, had already evaluated the safety and efficacy of follistatin gene therapy in a previous clinical trial with patients suffering from Becker muscular dystrophy, results of which were released last October.

The results from Milo Biotechnology’s follistatin gene therapy study entitled “A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy” were published in the Molecular Therapy journal, revealing the capabilities of a modified version of follistatin, an alternatively spliced FS344, to prevent potential off target-binding. “The study in Becker was 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,” explained Mendell in a press release.

The follistatin gene therapy, which was created by Mendell in collaboration with Brian Kaspar at the Hospital, is based on adeno-associated virus delivery of follistatin 344 and is expected to improve muscle strength, as well as prevent wasting and fibrosis of the muscles. Since the mechanism of action of the therapy is not specific to any mutation, the researchers believe that the novel treatment may be used on other types of muscular dystrophy.

“We are excited about the broad potential for this therapy and are working with foundations and patient advocates to launch additional studies,” said the CEO of Milo Biotechnology, Al Hawkins, as another clinical trial is taking place at Nationwide Children’s Hospital to evaluate the effectiveness of the follistatin gene therapy in patients with inclusion body myositis.

The novel investigational treatment is now being supported by the Duchenne Alliance, a union of independent non-profit organizations focused on supporting promising projects that may improve the treatment of DMD or find a cure for the disease.

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