SGT-001 is under clinical development by Solid Biosciences and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect SGT-001’s likelihood of approval (LoA) and phase transition for Duchenne Muscular Dystrophy took place on 11 Feb 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their SGT-001 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

SGT-001 overview

SGT-001 is under development for the treatment of Duchenne muscular dystrophy (DMD) in adolescents and children. It is administered intravenously in the form of suspension. It is an adeno-associated virus (AAV9) vector-mediated microdystrophin gene therapy that enables the systemic delivery of a synthetic, functional version of the dystrophin gene.

Solid Biosciences overview

Solid Biosciences, operates as a life science company focused on developing therapies for duchenne muscular dystrophy (DMD). Its offers pipeline product candidates such as SGT-001, a microdystrophin gene therapy administered through adeno-associated virus (AAV) stabilizes dystrophin glycoprotein complex and rescue muscle function; and ANTI-LTBP4. Solid Biosciences has development programs across scientific platforms which include corrective therapies, disease-modifying therapies, disease understanding and assistive devices. The company works in collaboration with University of Massachusetts Medical School, The Duchenne Research Fund, University of Washington, Alex’s Wish. It operates across the US. Solid Biosciences is headquartered in Cambridge, Massachusetts, the US.

Quick View SGT-001 LOA Data

Report Segments
  • Innovator
Drug Name
  • SGT-001
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Genetic Disorders
Key Developers
Highest Development Stage
  • Phase II

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.