Drugs / SGT-003
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT06138639 | May 2024 → May 2027 expected | Duchenne muscular dystrophy | Solid Biosciences Inc. | Recruiting | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT07160634 | Oct 2025 → Jan 2029 expected | Duchenne muscular dystrophy | Solid Biosciences Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2026-04-28 | Solid Biosciences Inc. | Regulatory Solid Biosciences Announces Receipt of European Commission Orphan Drug Designation for SGT-003 for the Treatment of Duchenne Muscular Dystrophy solidbio.com ↗
today announced that the European Commission (EC), acting upon the positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products, has granted Orphan drug designation to SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne). |
| 2026-03-11 | Solid Biosciences Inc. | Results Solid Biosciences Provides Interim Positive Clinical Update on Phase 1/2 INSPIRE DUCHENNE Trial solidbio.com ↗
The new data, including additional muscle biopsy, serum biomarker, cardiac function, and safety analyses, add to a growing dataset that suggests the potential of the biological activity of SGT-003 microdystrophin therapy. |
| 2025-11-06 | Solid Biosciences Inc. | Regulatory Solid Biosciences Awarded Innovation Passport Designation Under the New UK Innovative Licensing and Access Pathway for SGT-003, an Investigational Gene Therapy for Duchenne Muscular Dystrophy solidbio.com ↗
Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that SGT-003, the Company’s investigational gene therapy for Duchenne muscular dystrophy (Duchenne), has been granted an Innovation Passport under the new ILAP. |
| 2025-11-03 | Solid Biosciences Inc. | Results Solid Biosciences Reports Third Quarter 2025 Financial Results and Provides Update on INSPIRE DUCHENNE Clinical Trial Progress and Planned Regulatory Discussions solidbio.com ↗
Day 90 biopsy data from 10 treated participants (ages 5-10) showed all participants responded to treatment with mean microdystrophin expression of 58% by western blot, 58% by mass spectrometry |
| 2025-09-25 | Solid Biosciences Inc. | Results Solid Biosciences to Present at 2025 Neuromuscular Study Group Annual Scientific Meeting solidbio.com ↗ |
| 2025-02-18 | Solid Biosciences Inc. | Results Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 solidbio.com ↗
Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience. |
| 2025-01-15 | Solid Biosciences Inc. | Results Solid Biosciences Outlines Key Priorities for Advancing Diversified Neuromuscular and Cardiac Development Pipeline and Establishing Leadership in Precision Genetic Medicines solidbio.com ↗
Dosed four patients in INSPIRE DUCHENNE clinical trial; SGT-003 has been well tolerated in all patients with no SAEs observed; |
| 2024-05-07 | Solid Biosciences Inc. | Results Solid Biosciences to Present at the American Society of Gene and Cell Therapy Annual Meeting solidbio.com ↗
Systemic Delivery of SGT-003 Microdystrophin Gene Therapy Using the Novel Capsid AAV-SLB101 Ameliorates Muscle Pathology and Rescues Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy |
| 2024-04-01 | Solid Biosciences Inc. | Regulatory Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 solidbio.com ↗
Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate. |
| 2024-01-16 | Solid Biosciences Inc. | Regulatory Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 solidbio.com ↗ |
| 2023-12-07 | Solid Biosciences Inc. | Regulatory Solid Biosciences Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Gene Therapy SGT-003 solidbio.com ↗
today announced that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate. |
| 2023-11-14 | Solid Biosciences Inc. | Regulatory Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 solidbio.com ↗ |
| 2022-04-27 | Solid Biosciences Inc. | Results Solid Biosciences Announces Updated Corporate Strategy to Develop SGT-001 and SGT-003 Pipeline Programs for Patients with Duchenne Muscular Dystrophy solidbio.com ↗
In addition, today we are sharing new preclinical data which suggest that Solid’s novel capsid may offer enhanced muscle tropism compared w |
All press releases naming this drug 15 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | SGT-003 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07160634 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)” NCT06138639 ↗ |
| Route | Intravenous | “single intravenous (IV) infusion of SGT-003” NCT06138639 ↗ |