Solid Biosciences presents one-year IGNITE DMD data; SGT-001 program concluded

Solid Biosciences Inc.

One-year data: mean 69.8% normal dystrophin by Western blot in 2E14 vg/kg cohort (Pt.5 at 18 months).
Motor function: 6MWT and NSAA showed positive mean change from baseline at 1 year.
Pulmonary function: FVC % predicted and FEV1 % predicted improved from baseline.
Safety: 2/9 SAEs related to SGT-001 (thrombocytopenia, hepatotoxicity, systemic inflammatory response).
SGT-001 program concluded; next-gen SGT-003 with novel capsid in IND-enabling studies.

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