Muscular dystrophy startup secures SBIR grant to continue developing myostatin inhibitor

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muscular dystrophy

With a new $300,000 Phase I SBIR grant from the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases, biotech startup Milo Biotechnology will march forward with development and testing of its follistatin therapy for muscular dystrophy.

Its lead candidate, AAV1-FS344, is a gene therapy intended to up-regulate follistatin, a protein that blocks myostatin, which is another protein that reduces muscle strength. In nature, myostatin deficiency produces dramatic increases in strength. The idea is that the drug will increase muscle strength and decrease the rate of muscle atrophy in humans.

The technology was developed at Nationwide Children’s Hospital in Columbus and is currently in a Phase 1/2 safety and initial efficacy clinical trial there in patients with Becker muscular dystrophy and inclusion body myositis.


This approach to treating muscular dystrophy isn’t new, but thus far pharmaceutical companies haven’t had success with it. Wyeth Pharmaceuticals quietly halted the development of its myostatin inhibitor in 2008 after the showed no statistically significant muscle growth in a Phase 1/2 safety and tolerability trial. Acceleron Pharma and Shire also stopped a Phase 2 dose-escalation study of its myostatin inhibitor in 2011 based on preliminary safety data.

Milo CEO Al Hawkins said treatments in the Phase 1/2 study of AAV1-FS344, which was granted orphan drug status in December, should wrap up by the end of this year.

Cleveland-based Milo has received prior funding from JumpStart Inc. and Cuyahoga County North Coast Opportunities Fund.

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Deanna Pogorelc

By Deanna Pogorelc MedCity News

Deanna Pogorelc is a Cleveland-based reporter who writes obsessively about life science startups across the country, looking to technology transfer offices, startup incubators and investment funds to see what’s next in healthcare. She has a bachelor’s degree in journalism from Ball State University and previously covered business and education for a northeast Indiana newspaper.
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