SGT-003 CRISPR-Based Gene Editing for DMD
Not Yet RecruitingPhase 1/2Gene Editing
Study Overview
- Age
- 6–13 years
- Phase
- Phase 1/2
- Sponsor
- Solid Biosciences
- Therapeutic Approach
- Gene Editing
- Variant Requirement
- Any DMD-causing mutation; CRISPR approach targets dystrophin restoration broadly
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Must be on stable corticosteroid therapy for minimum 3 months
Inclusion Criteria
Ambulatory males aged 6–13 with genetically confirmed DMD. North Star Ambulatory Assessment score ≥18. No prior gene editing therapy.
Exclusion Criteria
Prior gene therapy or CRISPR treatment. Active hepatitis B or C. Immunodeficiency. Significant liver enzyme elevation.
Clinical Trial Registry
NCT ID
NCT06138627This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.