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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 Duchenne Muscular Dystrophy (DMD). It is designed to deliver a functional version of the dystrophin gene to muscle cells to address the underlying cause of the disease. The therapy is currently in clinical development for adolescents and children with DMD.
SGT-001 targets the genetic cause of Duchenne Muscular Dystrophy by delivering a microdystrophin gene. This gene is intended to produce a shortened but functional form of the dystrophin protein, which is missing or defective in people with DMD. The therapy aims to restore muscle function by providing this essential protein.
SGT-001 is being developed by Solid Biosciences Inc., a biopharmaceutical company focused on neuromuscular diseases. The company is listed on the stock exchange under the ticker symbol SLDB. Solid Biosciences is conducting clinical trials to evaluate the safety and efficacy of SGT-001 in patients with Duchenne Muscular Dystrophy.
SGT-001 is currently in Phase 1 clinical development. It is an investigational gene therapy that has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is evaluating the therapy in adolescents and children with Duchenne Muscular Dystrophy to assess its safety and potential benefits.
SGT-001 is being studied in a Phase 1 clinical trial with the identifier NCT03368742, titled "Microdystrophin Gene Transfer Study in Adolescents and Children With DMD." This trial is active but not recruiting participants, and it is enrolling males aged 4 years and older with Duchenne Muscular Dystrophy in the United States.
SGT-001 is a microdystrophin gene therapy, meaning it delivers a microdystrophin gene to muscle cells. The clinical trial for SGT-001 is specifically described as a microdystrophin gene transfer study. This approach is designed to provide a functional version of the dystrophin protein to treat Duchenne Muscular Dystrophy.