EDIT-301 is a gene-modified cell therapy commercialized by Editas Medicine, with a leading Phase II program in Sickle Cell Disease. According to Globaldata, it is involved in 2 clinical trials, which are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of EDIT-301’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for EDIT-301 is expected to reach an annual total of $138 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.

EDIT-301 Overview

EDIT-301 is under development for the treatment of beta-thalassemia and sickle cell disease. It is developed based on CRISPR/Cas12a genome editing technology. The therapeutic candidate constitutes ex vivo treated stem cells for the B-cell CLL/lymphoma 11 A (BCL11A) gene. The cells are modified for the expression of BCL11A gene using CRISPR/Cas12a ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus and guide RNA molecules to eliminate the mutation in the BCL11A gene. The gene is delivered using adeno-associated virus (AAV) vector to introduce normal copies of gene into the cells. The drug candidate is administered through intravenous route.

Editas Medicine Overview

Editas Medicine formerly Gengine, is a clinical stage biotechnology company focused on the development of genome treatments for rare diseases. The company is developing a proprietary genome editing platform based on its in-licensed clustered regularly interspaced short palindromic repeats (CRISPR) technology. The company’s pre-clinical pipeline spans investigational candidates for the treatment of ocular and blood diseases, cancer, and diseases of other organs and tissues. The company works in collaboration with Allergan/ AbbVie Inc, AskBio, Bayer AG, BlueRock Therapeutics, The Broad Institute Inc, Massachusetts Institute of Technology, Harvard Institute, The Rockefeller University, Celgene Corp and Bristol Myers Squibb to discover and develop its pipeline products. Editas Medicine is headquartered in Cambridge, Massachusetts, the US.

The company reported revenues of (US Dollars) US$25.5 million for the fiscal year ended December 2021 (FY2021), a decrease of 71.8% over FY2020. The operating loss of the company was US$193.2 million in FY2021, compared to an operating loss of US$134.8 million in FY2020. The net loss of the company was US$192.5 million in FY2021, compared to a net loss of US$116 million in FY2020.

For a complete picture of EDIT-301’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

GlobalData

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.