SGT-001 is a gene therapy commercialized by Solid Biosciences, with a leading Phase II program in Duchenne Muscular Dystrophy. According to Globaldata, it is involved in 1 clinical trial, which is ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of SGT-001’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for SGT-001 is expected to reach an annual total of $718 mn by 2038 in the US based off GlobalData’s Expiry Model. The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). Applying the drug’s phase transition success rate to remaining R&D costs and likelihood of approval (LoA) to sales related costs provides a risk-adjusted NPV model (rNPV). The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress.

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.

For a complete picture of SGT-001’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.


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, drug margins and company expenses. 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 valuation, 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.