Solid Biosciences reports 110% avg microdystrophin expression from SGT-003 Phase 1/2 trial

Solid Biosciences Inc.

First three participants showed average 110% microdystrophin expression by western blot and 78% dystrophin-positive fibers at Day 90.
Mean reductions in muscle injury biomarkers: CK -57%, AST -45%, ALT -54%, LDH -60% at Day 90.
Two participants at Day 180 showed mean LVEF increase of 8% from baseline; no serious adverse events or TMA observed.
Preliminary cash, cash equivalents and available-for-sale securities of ~$148.9M as of Dec 31, 2024.
Company plans mid-2025 FDA meeting to discuss accelerated approval for SGT-003; enrollment ongoing with 7th dosed Feb 17.

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