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SGT-001 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| SGT-001 - Dose Level 1 | EXPERIMENTAL | Single IV infusion of SGT-001 at starting dose |
| SGT-001 - Dose Level 2 | EXPERIMENTAL | Single IV infusion of SGT-001 at next ascending dose |
| Untreated Control | NO_INTERVENTION | Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose. |
| Name | Type | Description |
|---|---|---|
| SGT-001 | GENETIC | AAV9 vector containing muscle-specific promoter and microdystrophin construct |
Inclusion Criteria: * Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype * Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants) * Anti-AAV9 antibodies below protocol-specified thresholds * Stable cardiac and ...
SGT-001 is an investigational gene therapy being developed for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is designed to deliver a functional version of the dystrophin gene to muscle cells, potentially addressing the underlying cause of the disease.
SGT-001 targets the dystrophin gene. It is a gene therapy that aims to deliver a functional copy of the dystrophin gene to muscle cells, which is missing or defective in patients with Duchenne Muscular Dystrophy. By restoring dystrophin production, the therapy seeks to stabilize or improve muscle function.
SGT-001 is being developed by Solid Biosciences Inc., a biotechnology company traded on NASDAQ under the ticker symbol SLDB. The company focuses on developing therapies for neuromuscular diseases, with SGT-001 being one of its lead investigational candidates for Duchenne Muscular Dystrophy.
SGT-001 is currently in Phase 1 clinical development. It is an investigational gene therapy, meaning it has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is designed to evaluate the safety and tolerability of the therapy in patients with Duchenne Muscular Dystrophy.
SGT-001 is being evaluated in a Phase 1 clinical trial with the identifier NCT03368742, titled 'Microdystrophin Gene Transfer Study in Adolescents and Children With DMD.' This active but not recruiting study is enrolling 12 male patients aged 4 years and older with Duchenne Muscular Dystrophy in the United States.