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Dec 31, 2020
Solid Biosciences Q4 2020 Earnings Report
Solid Biosciences reported positive interim efficacy and safety data from the ongoing IGNITE DMD clinical trial and the resumption of patient dosing in the 2E14 vg/kg cohort.
Key Takeaways
Solid Biosciences reported encouraging interim functional and biomarker data, and patient reported outcome measures (PROMs) from six patients after treatment in the ongoing IGNITE DMD Phase I/II clinical trial of its lead gene therapy candidate, SGT-001. The Company also announced that patient 7 in IGNITE DMD was safely dosed. The totality of data collected, and the re-initiation of dosing support the continued enrollment of patients into the IGNITE DMD study.
Interim data from six patients provide evidence of a potential benefit of SGT-001 in functional endpoints.
Patient 7 safely dosed with SGT-001 experienced transient and manageable adverse events.
Six patients previously dosed showed no new drug-related safety findings 17-37 months post dosing.
Screening and enrolling of patients into IGNITE DMD continue.