OTL-203 is a Gene-Modified Cell Therapy owned by Orchard Therapeutics, and is involved in 2 clinical trials, of which 1 is ongoing, and 1 is planned.

OTL-203 is under investigation for the treatment of Mucopolysaccharidosis type I (MPS I). It acts by targeting a-L-iduronidase (IDUA). The therapy is composed of autologous HSCs genetically modified by LVs to deliver IDUA gene. Lentiviral vectors are used to transduce hematopoietic stem cells and direct IDUA expression.

The revenue for OTL-203 is expected to reach a total of $53m 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 OTL-203 NPV Report.

OTL-203 was originated by San Raffaele Telethon Institute for Gene Therapy and is currently owned by Orchard Therapeutics.

OTL-203 Overview

OTL-203 is under development for the treatment of Mucopolysaccharidosis type I (MPS I). It is administered intravenously. It acts by targetingf a-L-iduronidase (IDUA). The therapy is composed of autologous HSCs genetically modified by LVs to deliver IDUA gene. Lentiviral vectors are used to transduce hematopoietic stem cells and direct IDUA expression.

Orchard Therapeutics Overview

Orchard Therapeutics (Orchard) is a biotechnology company that develops gene therapies for the treatment of serious and life-threatening orphan diseases. Its pipeline products include Strimvelis, an autologous ex vivo gammaretroviral gene therapy for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID); OTL-103 for Wiskott-Aldrich syndrome (WAS); OTL-102 for X-linked chronic granulomatous disease (X-CGD); OTL-300 for transfusion-dependent beta-thalassemia; and OTL-200 for metachromatic leukodystrophy (MLD), among others. It collaborates with institutions that specialize in gene and cell therapy such as The University of Manchester, Boston Children’s Hospital, and The San Raffaele Telethon Institute for Gene Therapy. It operates in France, Germany, Italy, Switzerland, the Netherlands, the US and the UK. Orchard is headquartered in London, Greater London, the UK.

The company reported revenues of (US Dollars) US$1.7 million for the fiscal year ended December 2021 (FY2021), a decrease of 35.5% over FY2020. The operating loss of the company was US$140.4 million in FY2021, compared to an operating loss of US$157 million in FY2020. The net loss of the company was US$144.6 million in FY2021, compared to a net loss of US$152 million in FY2020. The company reported revenues of US$4.4 million for the second quarter ended June 2022, a decrease of 20.9% over the previous quarter.

Quick View – OTL-203

Report Segments
  • Innovator
Drug Name
  • OTL-203
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.