Rebisufligene etisparvovec is a Gene Therapy owned by Ultragenyx Pharmaceutical, and is involved in 2 clinical trials, which are ongoing.

Rebisufligene etisparvovec (ABO-102) gene therapy delivers the sulfoglucosamine sulfohydrolase, an enzyme to the CNS where lysosomal storage of heparan sulfate GAGs in cells is disrupted. The enzyme after delivery to the CNS breaks down the heparan sulfate sugar chain whose metabolism is hampered in the sanfilippo (SF) syndrome. The gene therapy corrects of cognitive defects and restores normal cell and organ function.

The revenue for Rebisufligene etisparvovec is expected to reach a total of $1.1bn 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 Rebisufligene etisparvovec NPV Report.

Rebisufligene etisparvovec was originated by Nationwide Children’s Hospital and is currently owned by Ultragenyx Pharmaceutical. Abeona Therapeutics is the other company associated in development or marketing of Rebisufligene etisparvovec.

Rebisufligene etisparvovec Overview

Rebisufligene etisparvovec (ABO-102) is under development for the treatment of Sanfilippo syndrome type A. It is administered through intravenous injection. The therapeutic candidate is a gene therapy that comprises of recombinant adeno-associated virus vector serotype 9 (AAV9) expressing human sulfoglucosamine sulfohydrolase.

Abeona Therapeutics Overview

Abeona Therapeutics (Abeona), formerly PlasmaTech Biopharmaceuticals, Inc., is a biopharmaceutical company that develops and delivers gene therapy and plasma-based products for severe life-threatening rare diseases. The company’s lead programs include ABO-101 and ABO-102 adeno-associated virus. Abeona also develops EB-101 for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), ABO-201 (AAV-CLN3) gene therapy for Juvenile Batten disease (JNCL) and more. The company has partnerships for the commercialization of its products in the US, European Union, Switzerland, Norway, Iceland, Lichtenstein, Australia, New Zealand, China and South Korea. Abeona is headquartered in New York City, the US.

The company reported revenues of (US Dollars) US$3 million for the fiscal year ended December 2021 (FY2021), a decrease of 70% over FY2020. The operating loss of the company was US$88.1 million in FY2021, compared to an operating loss of US$81.4 million in FY2020. The net loss of the company was US$84.9 million in FY2021, compared to a net loss of US$84.2 million in FY2020. The company reported revenues of US$1 million for the second quarter ended June 2022, compared to a revenue of US$0.4 million the previous quarter.

Quick View – Rebisufligene etisparvovec

Report Segments
  • Innovator
Drug Name
  • Rebisufligene etisparvovec
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Genetic Disorders
Key Companies
Highest Development Stage
  • Phase III

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.