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Myonexus Therapeutics Receives Orphan Drug Designation for MYO-102, an Investigational Gene Therapy for Alpha-sarcoglycanopathy (LGMD2D)

January 2, 2019 GMT

NEW ALBANY, Ohio--(BUSINESS WIRE)--Jan 2, 2019--Myonexus Therapeutics, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation for MYO-102, a novel gene therapy candidate for alpha-sarcoglycanopathy, also known as Limb Girdle Muscular Dystrophy Type 2D (LGMD2D). Myonexus Therapeutics is a clinical-stage gene therapy company developing first-ever corrective gene therapies for limb girdle muscular dystrophies (LGMD) and has licensed the technology from Nationwide Children’s. Nationwide Children’s holds the Investigational New Drug (IND) application for MYO-102.


LGMD2D is a severe, debilitating condition caused by a defect in the gene that produces the alpha-sarcoglycan protein. Lack of function of this protein causes inflammation and progressive loss of muscle fiber, which is replaced with fat and fibrotic scars. LGMD2D is believed to be the most common sarcoglycanopathy, with conservative estimates indicating it affects approximately three per million people, regardless of gender, race, or ethnicity. There is no treatment or cure for LGMD2D. Experimental gene therapy treatment aims to deliver alpha-sarcoglycan genes to permanently restore protein expression, which could significantly improve symptoms and functional ability for patients.

“The Orphan Drug Designation from the FDA is an important milestone in the development pathway for MYO-102 and reflects its potential to address a considerable unmet medical need in treating LGMD2D,” said Michael Triplett, Ph.D., President and Chief Executive Officer of Myonexus. “As we plan for our first systemic human clinical trial, we aim to build on the promising results of our research to date and offer hope that our gene therapy candidates may one day be able to transform the lives of patients and the families who care for them.”

The FDA’s designation is granted to applications for new therapies that offer potentially significant benefit over current options for rare diseases. As part of the development process, this designation provides for incentives for research in rare disease, including eligibility for a period of market exclusivity in the U.S. following product approval.

“With ongoing research, we have increasing evidence demonstrating the potential for gene therapy. We believe this technology holds promise to address a critical unmet need in treating LGMD,” said Dr. Jerry Mendell, Curran-Peters Chair of Pediatric Research at Nationwide Children’s Hospital. “The FDA’s orphan status represents an appreciation of the need and the potential of this technology to introduce the first major advance for the LGMD community.”

Early clinical studies of MYO-102 have demonstrated safety and expression of alpha-sarcoglycan protein using the gene therapy candidate. After treatment with MYO-102, alpha-sarcoglycan expression increased versus baseline and the protein was present in muscles, as shown in biopsies six months after treatment.

As part of an ongoing collaboration with Sarepta Therapeutics (see announcement ), Myonexus is developing five gene therapy candidates, all using the same AAVrh.74 vector, designed to offer the first-ever corrective treatment for five distinct forms of LGMD. In addition to the clinical research underway on MYO-101 (see announcement ), and this designation for MYO-102, the other candidates include MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. For more information on the MYO-101 trial, please visit (study identifier NCT03492346).

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 The Research Institute at Nationwide Children’s Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report’s 2018-19 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative 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 behavioral health research. The institute houses a Good Manufacturing Practices (GMP) facility for producing gene- and cell-based therapies; one of the largest biorepositories in North America; and comprehensive genome sequencing and analysis capabilities. More information is available at

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CONTACT: Media and Investors:

Myonexus Therapeutics

Laureen Cassidy,media@myonexustx.comMedia Contact for Nationwide Children’s Hospital:

Gina Bericchia




SOURCE: Myonexus Therapeutics

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PUB: 01/02/2019 08:30 AM/DISC: 01/02/2019 08:30 AM