Nick Johnson, M.D. and Melissa Hale, Ph.D. are moving fast to combat a debilitating and incurable disease. 

Their research effort at the Center for Inherited Myology Research targets limb-girdle muscular dystrophy. LGMD is the fourth most common form of muscular dystrophy (MD), a group of genetic diseases causing muscles to progressively weaken and break down. LGMD impacts muscles in the arms, legs and hips. 

“I’m a clinician first, and I can see the urgency at which we need to develop these therapies here in Richmond and around the world,” says Johnson, who directs the Center and is the George Bliley Research Chair in the Department of Neurology at the VCU School of Medicine. Hale is an assistant professor of neurology and one of Johnson’s partners at the Center. 

There are 32 genetic forms (“subtypes”) of LGMD, and about half are amenable to gene-replacement therapy, the team says. The cutting-edge treatment can fix problems caused by faulty or missing genes in the body. Current experimental gene-replacement therapies can be toxic, especially to the liver—and risk of potentially fatal damage increases with age and weight. 

Johnson and Hale are taking an innovative approach, using synthetic viruses to deliver healthy genes into affected muscle cells. Their synthetics are designed to target muscle cells and reduce harmful side effects. Their invention forms the basis of MyoGene Therapies, a startup they hope will help them attract investors and move through FDA clinical trials. 

“I’m very motivated to move our work into the clinic,” Johnson says. “It became clear that the best way to do that is to form a startup to get the necessary investment, to develop the technology into approved clinical therapy.” 

MyoGene is taking advantage of FDA efforts to allow flexibility for ultra-rare diseases. “It’s an efficient approach to get to clinical trials for something like MD,” he says. 

Hale, Johnson’s business and research partner, said her work is focused on “bridging the gap” between lab research and patient impact. “MyoGene is a direct extension of that mission, and we want to create a future where the debilitating effects of muscular dystrophy can be mitigated — or even reversed,” she says. “By leveraging the translational research we perform in the Center, we’re working to develop and deliver safe, effective therapies for patients.” 

Johnson says the support from VCU has been transformative. “They have helped us with our clinical and lab research, the recruitment of faculty, and support of our startup,” he says. “And our intention is to use that support and our expertise to bring our treatments to patients worldwide.”