Solid Biosciences Inc是一家生命科学公司。该公司专注于开发变革性治疗,以改善Duchenne肌营养不良症(Duchenne)患者的生活。Duchenne是一种遗传性肌肉萎缩疾病,主要影响男孩。SGT-001和SGT-003是其基因转移候选产品。SGT-001和SGT-003旨在通过传递一种合成的转基因来解决Duchenne的潜在遗传原因,该转基因可产生仅在身体肌肉(包括心肌和呼吸肌)中表达的肌营养不良蛋白样蛋白。SGT-001是其主要基因转移候选基因,利用腺相关病毒(AAV)9载体。除了候选基因转移外,该公司还制定了专注于平台技术的开发计划,包括双基因表达,这项技术允许它将多个转基因包装到一个载体以及衣壳中。它正在开发SGT-001,用于通过单次静脉给药治疗Duchenne。
名称 | 标题 | 任期 | 年龄 |
|---|---|---|---|
| Ian F. Smith | Executive Chair | 2022 | 60 |
| Alexander G. Cumbo | President, CEO & Director | 2022 | 55 |
| Ilan Ganot | Co-founder, Strategic Advisor to the CEO & Director | 2018 | 51 |
| Adam Leo Stone | Lead Independent Director | 2015 | 46 |
| Lynne Marie Sullivan | Independent Director | 2015 | 59 |
| Jeffrey Chamberlain | Chairman of Scientific Advisory Board | 2018 | - |
| Georgia Keresty | Independent Director | 2021 | 63 |
| Carrie Miceli | Member of Scientific Advisory Board | - | - |
| Ronald D. Cohn | Member of Scientific Advisory Board | - | - |
| Jeffrey A. Bluestone | Member of Scientific Advisory Board | - | 72 |
| Dongsheng Duan | Member of Scientific Advisory Board | - | - |
| Laurence A. Turka | Member of Scientific Advisory Board | - | 68 |
| Geoffrey F. Slaff | Member of Scientific Advisory Board | - | - |
| Michael W. Lawlor | Member of Scientific Advisory Board | - | - |
| Martin I. Freed | Independent Director | 2018 | 65 |
| Sukumar Nagendran | Independent Director | 2018 | 59 |
| Clare Kahn | Independent Director | 2021 | 73 |
| Joe Kornegay | Member of the Scientific Advisory Board | - | - |
| Lee Sweeney | Member of Scientific Advisory Board | - | - |