Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SGT-003 · 2 trials · 1 indication
Microdystrophin expression evaluation in muscle biopsies
| Arm | Type | Description |
|---|---|---|
| SGT-003 in Part 1, Placebo in Part 2 | EXPERIMENTAL | Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria. |
| Placebo in Part 1, SGT-003 in Part 2 | EXPERIMENTAL | Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2 per protocol-defined eligibility criteria. |
| Cohort 1: SGT-003 | EXPERIMENTAL | All ambulatory participants from age 4 to \< 7 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 2: SGT-003 | EXPERIMENTAL | All ambulatory participants from age 7 to \< 12 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 3: SGT-003 | EXPERIMENTAL | All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 4: SGT-003 | EXPERIMENTAL | All ambulatory participants from age 12 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 5: SGT-003 | EXPERIMENTAL | All non-ambulatory participants from age 10 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1. |
| Name | Type | Description |
|---|---|---|
| SGT-003 | DRUG | Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5) |
| Placebo | DRUG | IV infusion |
Inclusion Criteria: * Participant is ambulatory. * Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype. * Negative for antibodies against adeno-associated virus serotype 9 (AAV9). * On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or...
SGT-003 is an investigational gene therapy being developed for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic neuromuscular disorder. It is designed to address the underlying cause of the disease by delivering a functional gene to muscle cells. SGT-003 is currently in clinical development and has not yet been approved by regulatory authorities.
SGT-003 is a gene therapy that targets Duchenne Muscular Dystrophy by delivering a modified version of the dystrophin gene to muscle cells. The therapy aims to restore the production of functional dystrophin protein, which is deficient in patients with DMD. This approach is intended to slow or halt disease progression by addressing the root cause of the condition.
SGT-003 is being developed by Solid Biosciences Inc., a biopharmaceutical company focused on precision genetic medicines. The company's stock is traded under the ticker symbol SLDB. Solid Biosciences is leading the clinical development of SGT-003 for Duchenne Muscular Dystrophy and is responsible for its manufacturing and regulatory strategy.
SGT-003 is currently in Phase 3 clinical development for Duchenne Muscular Dystrophy. It is an investigational gene therapy that has received Fast Track, Rare Pediatric Disease, and Accelerated Approval designations from the FDA. The drug is not yet approved and is being evaluated in ongoing clinical trials to assess its safety and efficacy.
SGT-003 is being studied in two active clinical trials. The INSPIRE DUCHENNE trial (NCT06138639) is a Phase 1 study enrolling 60 male participants in the US, Canada, Italy, and the UK. The IMPACT DUCHENNE trial (NCT07160634) is a Phase 3 study enrolling 80 ambulant males aged 7 years and older in the US, Australia, and Canada. Both trials are recruiting participants.
No, SGT-003 is the investigational drug, while INSPIRE DUCHENNE is the name of one of its clinical trials. The INSPIRE DUCHENNE trial (NCT06138639) is a Phase 1 study evaluating SGT-003 in patients with Duchenne Muscular Dystrophy. The drug is also being studied in a separate Phase 3 trial called IMPACT DUCHENNE (NCT07160634).