Solid Biosciences Inc
NASDAQ:SLDB
Solid Biosciences Inc
Solid Biosciences, Inc. engages in the development of treatments for patients with Duchenne muscular dystrophy (DMD). The company is headquartered in Cambridge, Massachusetts and currently employs 104 full-time employees. The company went IPO on 2018-01-26. Duchenne is a genetic muscle-wasting disease predominantly affecting boys. SGT-001 and SGT-003 are its gene transfer candidates. SGT-001 and SGT-003 are designed to address the underlying genetic cause of Duchenne by delivering a synthetic transgene that produces dystrophin-like protein that is only expressed in muscles of the body, including cardiac and respiratory muscles. SGT-001 is its lead gene transfer candidate and utilizes an adeno-associated virus (AAV)9 vector. In addition to its gene transfer candidates, it has development programs focusing on platform technologies, including dual gene expression, a technology that allows it to package multiple transgenes into one vector, as well as capsids. The company is developing SGT-001 for the treatment of Duchenne through a single intravenous administration.
Solid Biosciences, Inc. engages in the development of treatments for patients with Duchenne muscular dystrophy (DMD). The company is headquartered in Cambridge, Massachusetts and currently employs 104 full-time employees. The company went IPO on 2018-01-26. Duchenne is a genetic muscle-wasting disease predominantly affecting boys. SGT-001 and SGT-003 are its gene transfer candidates. SGT-001 and SGT-003 are designed to address the underlying genetic cause of Duchenne by delivering a synthetic transgene that produces dystrophin-like protein that is only expressed in muscles of the body, including cardiac and respiratory muscles. SGT-001 is its lead gene transfer candidate and utilizes an adeno-associated virus (AAV)9 vector. In addition to its gene transfer candidates, it has development programs focusing on platform technologies, including dual gene expression, a technology that allows it to package multiple transgenes into one vector, as well as capsids. The company is developing SGT-001 for the treatment of Duchenne through a single intravenous administration.
Strategic Refocus: Solid Biosciences is narrowing its focus to two gene therapy programs, SGT-001 and next-generation SGT-003, with both using a single, more efficient manufacturing platform.
Manufacturing Shift: The company is transitioning SGT-001 manufacturing to a transient transfection-based process, expected to streamline operations and secure product supply while maintaining development timelines.
Clinical Progress: SGT-001 continues to show durable functional improvements in patients, with ongoing long-term follow-up and new clinical data expected throughout the year.
SGT-003 Advances: Preclinical studies of SGT-003’s novel capsid show promising muscle targeting and expression, supporting a planned IND submission in early 2023 and clinical proof-of-concept next year.
Financial Runway: The company reported a cash position of approximately $180 million at the end of Q1 2022, providing runway through Q2 2024 and the achievement of key clinical milestones.
Cost Reduction: Organizational restructuring, including a 35% reduction in headcount, aims to extend the company’s cash runway and support the streamlined focus.
Leadership Change: Joel Schneider, COO, will be leaving Solid by the end of May to pursue a CEO role elsewhere.
