Solid Biosciences, a company that aims to solve Duchenne muscular dystrophy (DMD), has initiated clinical trial activities for its leading gene transfer candidate. The Phase I/II trial, called IGNITE MED, will evaluate safety and efficacy in ambulatory and non-ambulatory children and adolescents.
“For more than 20 years, my team has focused our research on understanding the potential of microdystrophin in DMD and optimizing a construct that we believe will provide the most benefit for patients,” said Dr. Jeffrey Chamberlain, professor of Neurology, in a release. “I’m pleased to see Solid Biosciences advance the most compelling of constructs into clinical studies. I believe we are now at an inflection point where, if successful, SGT-001 should represent an innovative new way of treating patients with this devastating disease.”
