Solid Biosciences Says FDA Grants Fast Track Designation For Gene Therapy Candidate SGT-003

(RTTNews) - Life sciences company Solid Biosciences Inc. (SLDB) announced Thursday that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for SGT-003, the company's next-generation Duchenne muscular dystrophy gene therapy candidate.

IND clearance by the FDA for SGT-003 was received in November 2023. The planned Phase 1/2 trial, SGT-003-101, 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 aged 4 to < 6 years of age with DMD. Long-term safety and efficacy will be evaluated for a total of 5 years following treatment.