News Release

Myonexus Therapeutics receives FDA orphan drug designation for LGMD type 2E treatment

Pioneering, corrective gene therapies have potential to transform quality of life

Business Announcement


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(COLUMBUS, OH-- April 23, 2018 10:00 am EDT) Myonexus Therapeutics, a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children's Hospital announce the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to Myonexus' lead candidate, MYO-101, for the treatment of limb girdle muscular dystrophy (LGMD) type 2E.

Limb girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. In LGMD type 2E, disease onset often occurs before age 10, leads to loss of ambulation in the teen years, and causes death by age 30. There is currently no cure for LGMD type 2E.

"Orphan Drug designation is a milestone in the development of MYO-101. It moves Myonexus a significant step closer to bringing potentially transformative quality of life improvements to children living with limb girdle muscular dystrophy type 2E," said Michael Triplett, President and CEO of Myonexus Therapeutics. "The MYO-101 preclinical efficacy data are compelling, and we look forward to initiating a systemic Phase 1/2a trial of MYO-101 later this year. "

The novel gene therapy is one of five licensed by Myonexus and pioneered within the lab of Louise Rodino-Klapac, Ph.D. and under clinical guidance by Jerry Mendell, M.D., at Nationwide Children's Hospital Center for Gene Therapy.

Orphan Drug designation provides companies working to cure rare diseases, with incentives including tax credits, federal grants, and a waiver of filing fees to trim the cost of development, as well as seven years of market exclusivity.


This milestone comes as Myonexus announces additions to its C-Suite and Board. Peter G. Kleinhenz joins Myonexus as Chief Financial Officer. Kleinhenz brings two decades of experience in commercializing, building, growing, and investing in biotech and biomedical ventures. Kleinhenz was most recently a Venture Partner with Fletcher Spaght Ventures in Boston, and Managing Director/General Partner with CID Capital, Inc.

Robert Beech has joined the Myonexus Board of Directors. Beech brings a wealth of experience leading venture and strategic initiatives in biotech and healthcare IT. Beech served as CEO (2004- 2009) and SVP Corporate Development & Communications for Intrexon Corporation (NYSE: XON), a public biotechnology company focused on creating biologically-based products that improve the quality of life and the health of the planet. Beech is

Executive Chairman at Eccrine Systems, Inc., a venture-backed company pioneering the development of advanced sweat sensors. He previously founded and led Digineer, Inc., an international healthcare IT company.

About Myonexus Therapeutics

Myonexus Therapeutics is a clinical stage, rare disease gene therapy company developing first ever treatments for limb girdle muscular dystrophies (LGMDs) based on research at Nationwide Children's Hospital, a leader in neuromuscular gene therapy discovery and translational research. Myonexus Therapeutics' pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2L). Founded in 2017, Myonexus is headquartered in New Albany, Ohio. More information is available at

About Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report's 2016-17 list of "America's Best Children's Hospitals," Nationwide Children's Hospital is America's largest not-for-profit freestanding pediatric healthcare system providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children's has a staff of more than 11,000 providing state-of-the-art pediatric care during more than 1.2 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children's physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children's Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at

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