SGT-001 is a Gene Therapy owned by Solid Biosciences, and is involved in 1 clinical trial, which is ongoing.

SGT-001 acts by activating dystrophin. Dystrophin is a structural protein found in small amounts in normal muscle. Due to the mutations in the dystrophin gene, the levels of dystrophin gets reduced or completely become zero starting a chain reaction that eventually leads to muscle cell degeneration and death. The therapeutic candidate by delivering the gene activates it and increases the levels of dystrophin and alleviates the condition.

The revenue for SGT-001 is expected to reach a total of $6.1bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the SGT-001 NPV Report.

SGT-001 was originated by University of Missouri and is currently owned by Solid Biosciences.

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.

The company reported revenues of (US Dollars) US$13.6 million for the fiscal year ended December 2021 (FY2021). The operating loss of the company was US$72.3 million in FY2021, compared to an operating loss of US$88.4 million in FY2020. The net loss of the company was US$72.2 million in FY2021, compared to a net loss of US$88.3 million in FY2020.

Quick View – SGT-001

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

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

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.