Solid Biosciences gets FDA IND clearance for Duchenne gene therapy SGT-003; Phase 1/2 trial planned

Solid Biosciences Inc.

FDA clears IND for SGT-003, a next-generation microdystrophin gene therapy for Duchenne muscular dystrophy.
Phase 1/2 trial (SGT-003-101) will be first-in-human, open-label, multicenter; dose 1E14vg/kg as single IV infusion.
Cohort 1 enrolls patients aged 4 to <6 years; minimum 3 patients per cohort with potential expansion; 5-year follow-up.
SGT-003 uses capsid AAV-SLB101 with R16/R17 nNOS-binding domains; preclinical data showed improved biodistribution vs AAV9.
Company expects to submit for IRB approval and commence patient screening shortly.

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