Myonexus Poised to Unlock Gene Therapy for Limb-Girdle Muscular Dystrophy

Myonexus Therapeutics is a clinical stage gene therapy company developing first-ever gene treatments for multiple types of limb-girdle muscular dystrophy (LGMD), a group of progressive, debilitating musculoskeletal genetic diseases that cause wasting of the muscles in the arms and legs.

Myonexus is a spinout from the Research Institute at Nationwide Children’s Hospital, one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. Myonexus also recently secured $2.5 million seed financing from Rev1 Ventures, CincyTech, The Jain Foundation and GFB ONLUS.

“Nationwide Children’s is one of the preeminent global leaders in gene therapy,” said Michael Triplett, Ph.D. and Myonexus president and CEO. “The Center for Gene Therapy team is brilliant, and Nationwide Children’s leadership boldly has provided the resources to translate the expertise into potentially transformative therapies.”

Triplett, formerly an executive at Battelle and then CEO of N8 Medical, Inc., served on the technical funding committee at Nationwide and had observed over time the institution’s leadership and progress in gene therapy.

“I am fascinated by the intersection of breakthrough science and technology research, venture, and innovation,” he said. “Rare for academic research medical institutions, Nationwide Children’s can advance therapeutic candidates into clinical trials. In our case, two LGMD gene therapy candidates were in clinical trials with a third ready to start clinical studies. Surprisingly, the portfolio was unlicensed. Given the unmet clinical need and the advanced stage of development, we formed a company earlier this year.”

Myonexus: A company formed less than a year ago moves to clinical stage.

LGMD commonly results in wheelchair dependency around the age of 20. Weakening of the heart muscle occurs in some forms of LGMD. Some affected individuals experience mild to severe breathing problems. In severe cases, death may occur by the early thirties. There are currently no cures or effective therapies.

With three clinical stage gene programs and two more gene therapy programs in clinical studies, Myonexus is advancing therapies that offer the potential to transform the quality of life for LGMD patients.

Each LGMD subtype results from a single mutation in genes that affect proteins necessary for normal muscle function. “The therapies leverage the natural efficiency of a virus to deliver correct genes to replace defective genes that cause limb-girdle muscular dystrophy disease,” said Triplett, who has extensive experience in drug delivery technology that enables therapeutics.

The therapies in development are intended as one-time, systemic administrations of the corrective gene.

“The correct genes are delivered by adenoviral particles,” he said. “Scientists recognized that viruses are prolific in their ability to deliver new DNA to humans and other animals. Once the scientists understood which genes were causing the problem, they could build a research agenda to test and develop therapeutic candidates. This is where Nationwide Children’s Center for Gene Therapy excels. The replacement genes produce healthy muscle instead of diseased muscle.”

Strong patient advocacy support is critical to the success of rare disease companies.

The Limb-girdle muscular dystrophy global community is providing Myonexus with initial financing, scientific contributions, and patient engagement. Bryan Barber, the president of the LGMD2D Foundation and serial entrepreneur, is co-founder of Myonexus and the parent of an LGMD patient.

“Bryan provides a drive and human element that keeps us very mission focused. His son played tee ball as a young child and at 12 is non-ambulatory in a wheelchair. It’s for children like this that we do what we do,” said Triplett.

“People from all over the world are looking to Columbus as the beacon of light for this disease,” he said. “I am overwhelmed by the outpouring of desperate and hopeful messages from around the globe. It is inspiring and shows that you can transcend ethnicity, geography, and language. There is no doubt about our motivation.”