Solid Biosciences shares promising early data for Duchenne gene therapy

Massachusetts-based Solid Biosciences announced positive initial data from a phase 1/2 trial evaluating its next-gen gene therapy candidate intended for the treatment of Duchenne muscular dystrophy.

Interim 90-day biopsy data reported in the first three participants of the INSPIRE DUCHENNE trial evaluating SGT-003 showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience.

“Significant reductions observed in all evaluated clinical biomarkers of muscle damage associated with Duchenne provide preliminary evidence of a beneficial effect in muscle integrity, including potential early signals of a positive cardiac benefit of SGT-003 in these young boys. In mid-2025, we plan to request a meeting with the FDA to discuss the potential for an accelerated approval regulatory pathway for SGT-003,” said Bo Cumbo, president and CEO, Solid Biosciences.

Additionally, SGT-003 was well-tolerated in the first six participants dosed as of the data cutoff date of February 11, 2025. Adverse events observed after SGT-003 treatment were typical of those observed in AAV gene therapy, including nausea, vomiting, fever and transient declines in platelets in some participants. No serious adverse events or suspected unexpected serious adverse reactions were observed.

SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, AAV-SLB101, which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies.

This is Solid Bio's second attempt at a Duchenne therapy. The company’s first-generation gene therapy candidate, SGT-001, was hit with two FDA clinical holds in 2019 after trial patients experienced serious adverse events. In September 2022, Solid Bio announced that it had prioritized the development of SGT-003 and would be pausing activities for SGT-001.