ANB-002

Originator investigational product
List of clinical studies

ANB-002 is an investigational gene therapy for the treatment of hemophilia B.

ANB-002 is an adeno-associated virus (AAV51)-based gene-therapy vector which is expected to induce FIX2 expression by delivering FIX gene copies into liver cells.

Hemophilia B is a hereditary blood clotting disorder due to a mutation in the coagulation factor IX gene causing frequent episodes of prolonged bleeding and hemarthroses.3. A single dose of the gene therapy is expected to restore the FIX to levels sufficient to relieve or control clinical symptoms.4, 5.

The most common adverse events associated with the gene therapy may include:6:

  • hepatic adverse events,
  • immune reactions,
  • risk of hypersensitivity reactions.

In the ANB-002-1/SAFRAN clinical trial, ANB-002 will be administered as a single intravenous infusion.

The research physician closely monitors the status of the patient participating in the study and takes all necessary actions to manage adverse events if they occur.

  1. A virus vector is a non-pathogenic engineered virus delivering a desired gene into cells.
  2. FIX is blood clotting factor IX.
  3. Schramm W. The history of haemophilia - a short review. Thromb Res. 2014;134 Suppl 1:S4-9. doi: 10.1016/j.thromres.2013.10.020.
  4. Arruda VR, Doshi BS. Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century. Mediterr J Hematol Infect Dis. 2020;12(1):e2020069. doi: 10.4084/mjhid.2020.069.
  5. Leebeek FWG, Meijer K, Coppens M, Kampmann P, Klamroth R, Schutgens R, et al. AMT-060 gene therapy in adults with severe or moderate-severe hemophilia B confirm stable FIX expression and durable reductions in bleeding and factor IX consumption for up to 5 years. Blood. 2020;136:26. doi: 10.1182/blood-2020-139225.
  6. Monahan PE, Négrier C, Tarantino M, Valentino LA, Mingozzi F. Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia. J Clin Med. 2021;10(11). doi: 10.3390/jcm10112471.

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