Solid Biosciences announced that its Phase 1/2 INSPIRE DUCHENNE trial has now dosed 40 participants, an increase from 36 dosed as of February 9 2026 and 33 as of January 9 2026. The interim data, released on March 11 2026, show robust microdystrophin expression and restoration of key components of the dystrophin‑associated protein complex (DAPC).
The trial results also demonstrate measurable improvements in muscle integrity and left‑ventricular ejection fraction (LVEF). The safety profile remains encouraging, with no drug‑induced liver injury observed and the therapy administered with a low‑burden, steroid‑only prophylactic immunomodulation regimen.
"These data continue to suggest that SGT‑003 may be having a disease‑relevant treatment effect, with observed robust microdystrophin expression, restoration of key components of the dystrophin‑associated protein complex suggesting a direct biologic correlate of dystrophin activity, and improvements across multiple biomarkers of muscle integrity. Together with the promising safety and tolerability profile, these interim results reinforce our confidence in the potential of SGT‑003 to meaningfully impact the disease course of Duchenne," said Bo Cumbo, President and CEO of Solid Biosciences.
Solid plans additional meetings with the FDA in the first half of 2026 to discuss a possible accelerated approval pathway for SGT‑003 and expects to provide a further update in mid‑2026. The company also announced a $240 million private placement on March 6 2026, extending its cash runway into early 2028 and supporting continued development of its gene‑therapy pipeline, which includes candidates for Friedreich’s ataxia and other rare diseases.
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