NEW YORK – Solid Biosciences on Tuesday said it has received permission from the US Food and Drug Administration to begin its first clinical trial of SGT-003, the company's gene therapy candidate for Duchenne muscular dystrophy (DMD).
With the FDA's clearance of the company's investigational new drug (IND) application, Charlestown, Massachusetts-based Solid Biosciences plans to initiate a Phase I/II trial in DMD patients, beginning with those aged 4 to 6 years. The Phase I/II trial will be an open-label, multicenter study to determine the safety and tolerability of the drug.
The firm will enroll two cohorts with at least three patients each. Patients enrolled in the trial will receive a one-time intravenous infusion of SGT-003, and investigators will monitor patients for five years following treatment to evaluate long-term safety and efficacy.
The SGT-003 gene therapy is designed to slow or stop progression of DMD, a rare and inherited disorder characterized by progressive muscle weakness. The disorder is caused by mutations in the DMD gene that result in a lack of dystrophin protein. SGT-003 aims to treat the condition by delivering a synthetic form of the DMD gene that encodes for a shortened form of dystrophin.
"We appreciate the FDA's review of the IND and look forward to continuing to work in collaboration with the agency when we initiate dosing of DMD patients," Solid Biosciences Chief Regulatory Officer Jessie Hanrahan said in a statement.