Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026.
23 Pediatric participants have been dosed (1E14vg/kg) so far in the INSPIRE DUCHENNE trial (data cutoff 31st October). SGT-003 was generally well tolerated, there were no cases of drug-induced liver injury observed (n = 23) and day-90 biopsy data from 10 treated participants (ages 5-10) showed that all responded to treatment with mean muscle microdystrophin expression of 58% by both western blot and mass spectrometry.
The company expects to have dosed 30 participants by early 2026, and plans to meet with the FDA to discuss a potential registrational pathway in the first half of 2026.
Solid Bio also reported interim clinical data that included correlations observed between SGT-003 microdystrophin therapy and improvements in several biomarkers of muscle integrity, including serum creatine kinase, lactate dehydrogenase, aspartate aminotransferase and embryonic myosin heavy chain, that suggested an effect of treatment with SGT-003. The company plans to host a number of educational events/webinars in the coming weeks to communicate todays results to the broader Duchenne community.
Link to press release: https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-reports-third-quarter-2025-financial-results.
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