US FDA grants Rare Pediatric Disease Designation to Solid Biosciences' SGT-003 DMD gene therapy for Phase 1/2 trial, INSPIRE Duchenne.

Life sciences company Solid Biosciences has received Rare Pediatric Disease Designation from the US FDA for its next-generation Duchenne muscular dystrophy (DMD) gene therapy candidate, SGT-003. The Phase 1/2 trial, INSPIRE Duchenne, aims to determine the safety and tolerability of SGT-003 in pediatric DMD patients aged 4 to under 8. Solid Biosciences anticipates dosing patients in Q2 of this year.

April 01, 2024
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