Solid Biosciences receives Rae Pediatric Disease designation for SGT-003

Solid Biosciences announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy 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 less than 6 and cohort 2 will study patients with DMD ages 6 to less than 8. We anticipate providing an initial safety update for the first three to four patients enrolled in the INSPIRE Duchenne trial in mid-2024, and we anticipate providing initial expression and functional data from those patients in the fourth quarter of 2024.