Solid Biosciences SGT-001 Gene Therapy Phase 1/2 Clinical Trial (IGNITE DMD)

Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks.

• Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
• Anti-AAV9 antibodies below protocol-specified thresholds
• Stable cardiac and pulmonary function
• Adolescents: non-ambulatory by protocol-specified criteria
• Children: ambulatory by protocol-specified criteria

• Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
• Abnormal liver function
• Abnormal renal function
• Clinically significant coagulation abnormalities
• Impaired cardiovascular function based on cardiac MRI or ECHO
• Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
• Significant spinal deformity or presence of spinal rods
• Body mass index ≥ 95th percentile for age
• Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
• Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening