VCAR33 (Autologous) is a gene-modified cell therapy commercialized by Vor BioPharma, with a leading Phase II program in Refractory Acute Myeloid Leukemia;Relapsed Acute Myeloid Leukemia. 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 VCAR33 (Autologous)’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for VCAR33 (Autologous) is expected to reach an annual total of $49 mn by 2039 globally 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.

VCAR33 (Autologous) Overview

VCAR-33 is under development for the treatment of relapsed and refractory acute myeloid leukemia (AML) in children and adolescents or young adults. The therapeutic candidate comprises of autologous T cells genetically engineered with lentivirus to express chimeric antigen receptors targeting cells expressing CD33. It is administered through intravenous route.

Vor BioPharma Overview

Vor BioPharma, a subsidiary of Puretech Health Plc, is a clinical-stage cell therapy company that develops therapies for the treatment of hematological malignancies. The company’s pipeline products include Trem-cel (formerly VOR33), is an eHSC product candidate created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target protecting them from a targeted therapy post-transplant. Vor BioPharma‘s other pipelines include VCAR33 (Allogeneic), VCAR33 (Autologous), Trem-cel + VCAR33, Treatment System and CD33-CLL1 Treatment System. Vor BioPharma is headquartered in Cambridge, Massachusetts, the US.

The operating loss of the company was US$93.4 million in FY2022, compared to an operating loss of US$69 million in FY2021. The net loss of the company was US$92.1 million in FY2022, compared to a net loss of US$68.9 million in FY2021.

For a complete picture of VCAR33 (Autologous)’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 22 January 2024

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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.