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SGT-003
SGT-003 is a gene therapy that aims to deliver a functional dystrophin gene to muscle cells.
SGT-003 is a gene therapy that aims to deliver a functional dystrophin gene to muscle cells. Used for Duchenne muscular dystrophy.
At a glance
| Generic name | SGT-003 |
|---|---|
| Sponsor | Solid Biosciences Inc. |
| Drug class | Gene therapy |
| Target | Dystrophin gene |
| Modality | Small molecule |
| Therapeutic area | Muscular dystrophy |
| Phase | Phase 3 |
Mechanism of action
This is achieved through the use of adeno-associated virus (AAV) vectors to deliver the dystrophin gene to the muscle cells, where it can be expressed and help restore muscle function in patients with Duchenne muscular dystrophy.
Approved indications
- Duchenne muscular dystrophy
Common side effects
- Muscle pain
- Muscle weakness
Key clinical trials
- A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) (PHASE3)
- A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |