(RTTNews) - Solid Biosciences Inc. (SLDB), a biotechnology company focused on gene therapies for neuromuscular and cardiac diseases, announced the dosing of the first patient in its Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003, an investigational gene therapy designed to treat Duchenne muscular dystrophy (Duchenne).
SGT-003 is a next-generation gene therapy candidate utilizing AAV-SLB101, a proprietary capsid developed by Solid, to deliver a functional copy of the dystrophin gene to muscle cells.
The therapy aims to address the underlying cause of Duchenne, a progressive muscle-wasting disease caused by mutations in the dystrophin gene.
The INSPIRE DUCHENNE trial is a multicenter, open-label study designed to evaluate the safety, tolerability, and preliminary efficacy of SGT-003 in patients with Duchenne.
The trial will assess two dose cohorts (low and high doses) and include multiple clinical sites across North America and the U.K. Approximately 1 in 3,500 boys are affected by Duchenne, a condition for which there are currently limited treatment options.
Solid Biosciences believes that SGT-003 may provide a significant therapeutic benefit for these patients.
Commenting on the trial, Bo Cumbo, President and CEO of Solid Biosciences, said, "We are excited to begin dosing patients in the INSPIRE DUCHENNE trial with SGT-003 and look forward to assessing its safety and potential to improve the lives of patients with Duchenne.
This marks an important step in our mission to bring transformative gene therapies to the Duchenne community."
The company expects to report data from the first three patients enrolled in the trial in Q1 2025. Solid also plans to advance its Duchenne-focused pipeline and explore additional genetic therapies for other rare neuromuscular diseases in the coming years.
As of December 31, 2024, Solid has approximately $148.9 million in cash and investments, which will fund key strategic initiatives through 2026.
Currently SLDB is trading at $3.21 up by 2.39%.
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