Milo Biotechnology Continues To Make News

In September Rev1 and JumpStart Inc. announced new investment in Milo, and this month Milo has been granted Orphan Drug designation from the FDA for the treatment of inclusion body myositis.

This is the second Orphan Drug designation granted to Milo. Previously the FDA granted designation for treatment of Becker and Duchenne muscular dystrophy. Programs for the three treatments are in Phase I/II trials at Nationwide Children’s Hospital.

  • Milo’s Follistatin Gene Therapy is the first gene therapy to demonstrate functional improvement in any form of muscular dystrophy.
  • Milo’s Phase I/II trials received funding from the International Duchenne Alliance, a group of independent non-profits dedicated to defeating Duchenne Muscular Dystrophy.
  • Nationwide Children’s Hospital holds and has licensed patent number 8,895,309 to Milo Biotechnology for the follistatin gene therapy.
  • With technology that originated at Nationwide Children’s Hospital and company headquarters in Cleveland, and investment by Rev1 and JumpStart Inc., Milo is a great example of cross-region leverage of Ohio Third Frontier’s Entrepreneurial Signature Program.

Visit to learn more about how this Ohio startup is developing therapies to increase muscle strength and prevent muscle atrophy.