Milo Biotechnology


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 […]

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A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.


Muscular dystrophy therapy biotech wins $250K JumpStart investment

A clinical-stage biotechnology startup that’s developing a therapy for neuromuscular diseases such as muscular dystrophy has received a $250,000 investment from nonprofit economic development group JumpStart. Cleveland-based Milo Biotechnology’s drug candidate is based on technology developed at Columbus’ Nationwide Children’s Hospital, and the company has already begun a clinical trial there, according to a statement […]