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First gene therapy to treat haemophilia B

EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Hemgenix (etranacogene dezaparvovec) for the treatment of severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system which make factor IX medicines less effective).

Hemgenix is the first gene therapy to treat haemophilia B. It is delivered as a single infusion. Etranacogene dezaparvovec, the active substance in Hemgenix, is based on a virus (adeno-associated virus or AAV) which has been modified to not cause disease in humans. The virus contains copies of the gene responsible for producing factor IX. When injected into the patient’s vein, the virus is carried to the liver where the gene will be taken up into the patient’s liver cells and start producing factor IX, thereby limiting bleeding episodes.

Read the news announcement on the EMA Website.


OpenEdition vous propose de citer ce billet de la manière suivante :
vroby (19 avril 2023). First gene therapy to treat haemophilia B. ELSIBI. Consulté le 18 février 2026 à l’adresse https://doi.org/10.58079/o68z


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