Atidarsagene autotemcel is a Gene-Modified Cell Therapy owned by Orchard Therapeutics, and is involved in 8 clinical trials, of which 2 were completed, 5 are ongoing, and 1 is planned.

OTL-200 (GSK-2696274) exerts neuroregenerative effects. Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA includes taking cells called CD34+ cells from the blood of the affected patient and introducing the gene for the lacking enzyme into these cells, outside of the body. To introduce the gene into the cells, a man-made virus (lentiviral vector) is used. The cells are then infused back into the patient, where they are expected to produce and replace the lacking enzyme arylsulfatase A. The replacement of the lacking enzyme reduces the accumulated sulfatides and alleviates the condition.

The revenue for Atidarsagene autotemcel is expected to reach a total of $3bn 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 Atidarsagene autotemcel NPV Report.

Atidarsagene autotemcel was originated by IRCCS San Raffaele Hospital and Fondazione Telethon and is currently owned by Orchard Therapeutics.

Atidarsagene autotemcel Overview

Atidarsagene autotemcel (libmeldy) is a gene therapy containing an autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene. It is formulated as dispersion concentrate solution for intravenous route of administration. Libmeldy is indicated  treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. OTL-200 (GSK-2696274) is under development for the treatment of metachromatic leukodystrophy. It is an ex-vivo stem cell gene therapy administered intravenously as an infusion. It consists of autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA.

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 – Atidarsagene autotemcel

Report Segments
  • Innovator (NME)
Drug Name
  • Atidarsagene autotemcel
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Central Nervous System
Key Companies
Highest Development Stage
  • Marketed

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.