Rev1 Ventures Announces Landmark Investment in Nationwide Children’s Hospital Spinout

Investment in Milo Biotechnology marks milestone in strategic relationship.

 Rev1 Ventures and Nationwide Children’s Hospital combine forces to help a promising life science spinout deliver a new gene-based therapy to treat muscular dystrophy and various other muscular disorders to improve the lives of patients with neuromuscular diseases.
Rev1 Ventures will invest in Milo Biotechnology, the exclusive licensee of patented therapy developed at Nationwide Children’s Hospital by Brian Kaspar, PhD and Milo co-founder, and Jerry Mendell, MD and director of Nationwide Children’s Center for Gene Therapy. This is the first Rev1 Ventures investment in a Nationwide Children’s spinout.

“With this milestone investment, we are carrying out the vision that we had when Rev1 Ventures and Nationwide Children’s formed our strategic partnership,” said Tom Walker, Rev1 Ventures CEO.

“Our goal is to provide venture startup services and investment to outstanding entrepreneurs who are commercializing groundbreaking therapies discovered and developed at Nationwide Children’s Hospital, one of the most advanced research institutions in the world,” Mr. Walker said. “With clinical data, significant prior investment from multiple foundations, and Children’s Hospital carrying out further clinical and pivotal trials, Milo Biotechnology is exactly the kind of startup we want to help grow.”

A Phase I/II clinical study in Becker Muscular Dystrophy, completed at Nationwide Children’s this year with Dr. Mendell as chief investigator, established initial safety and efficacy for the follistatin gene therapy. It is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy. Clinical results are reported in the December 2014 online issue of Molecular Therapy.

“We are excited about the [Rev1 Ventures] investment in Milo,” said Dr. Margaret Barkett, senior licensing manager and Nationwide Children’s Hospital lead for Milo Biotechnology. “This company was the first NCH startup based on a technology from our Center for Gene Therapy. The Center has its own Good Manufacturing Production (GMP) facility for manufacturing of clinical-grade product, capable of filing INDs with the FDA and conducting clinical trials. Having all these capabilities enables technologies to be translated from bench to bedside, and thus de-risks the program to licensees, like Milo Biotechnology.”

Two additional trials are underway at Nationwide Children’s in Duchenne Muscular Dystrophy, the most common form of the disease in children and the most debilitating and in inclusion body myositis, an adult onset myopathy. In November, the International Duchenne Alliance, consolidating funding from fifteen foundations from three countries, allocated $500,000 to fund the Duchenne Muscular Dystrophy clinical trial with the potential for additional research funding.

“The strongest validation for the therapy is that we now have clinical data from patients with Becker Muscular Dystrophy,” said Al Hawkins, Milo Biotechnology CEO. “With the combination of foundation funding and resources at National Children’s Hospital, we’ve come far. We are happy and honored that Rev1 Ventures has chosen to become involved and invested.”

Rev1 Ventures’ investment will fund analysis and plans for a path to regulatory and medical reimbursement approvals as clinical trials progress.


About Rev1 Ventures

Rev1 Ventures helps entrepreneurs build great companies. With the capital of a VC and the connections of an accelerator, Rev1 supports start-ups through the first phases of growth to increase their probability of success. Our team of experts leads an ongoing, data-driven process that ensures entrepreneurs develop products people want, and companies people need. Rev1 manages a continuum of investment funds made possible by the participation of Ohio Third Frontier and other strategic partners. For more information, visit

About Nationwide Children’s Hospital

Ranked seventh of only ten children’s hospitals on U.S. News & World Report’s 2014-15 “America’s Best Children’s Hospitals Honor Roll” and among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” list, Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children and adolescents as well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational, and health services research at Nationwide Children’s.

About Milo Biotechnology

Milo Biotechnology is a clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases.  Its lead program, AAV1-FS344, leads to the local expression of follistatin, a potent TGFβ ligand inhibitor. Milo’s core technology was developed at and is exclusively licensed from Nationwide Children’s Hospital in Columbus, Ohio; the company is based in Cleveland, Ohio, and has received funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund and from NIAMS Rare Disease SBIR program.