New follistatin gene therapy for muscular dystrophy gets orphan drug status

An experimental drug being developed by Milo Biotechnology for the treatment of certain neuromuscular diseases has been given orphan drug designation by the U.S. Food and Drug Administration.

The Cleveland company’s drug candidate, AAV1-FS344, is a gene delivery therapy that’s designed to produce follistatin, a protein associated with muscle size and strength, in patients with Becker and Duchenne muscular dystrophy.

BMD and DMD are similar forms of muscular dystrophy caused by a gene mutation that results in either no or only partial production of the protein dystrophin. Patients with these conditions experience muscle weakness, cardiac complications and respiratory complications that progress over time.


Milo Biotechnology’s drug is based on technology licensed from Nationwide Children’s Hospital in Columbus, where a Phase 1/2 clinical trial is underway in adult patients with BMD and inclusion body myositis.

It’s not the only muscular dystrophy drug in clinical trials. An experimental drug from Sarepta Therapeutics has gotten some attention this year as a small clinical trial produced promising results of improved muscle function in boys with muscular dystrophy. Researchers at Cedars-Sinai Medical Center are also testing the erectile dysfunction drug Cialis, marketed by Eli Lilly & Co., in BMD patients. Last month, researchers reported that it corrected abnormal blood flow in patients with the condition.

With the orphan drug designation, Milo becomes qualified for a tax credit and seven years of marketing exclusivity. The company, which received a $250,000 investment from economic development group JumpStart earlier this year (Disclosure: JumpStart is also an investor in MedCity Media), is led by former BioEnterprise Entrepreneur-in-Residence Al Hawkins.

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