Solid Biosciences Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company's next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate. The planned Phase 1/2 trial, INSPIRE Duchenne, is a first-in-human, open-label, multicenter trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion.

Cohort 1 will study patients with DMD ages 4 to < 6 and cohort 2 will study patients with DMD ages 6 to < 8. The company anticipated providing an initial safety update for the first three to four patients enrolled in the INSPIRE Duchenne trial in mid-2024, and it anticipate providing initial expression and functional data from those patients in the fourth quarter of 2024.