Posted by Michael Wonder on 23 Nov 2022
FDA approves first gene therapy to treat adults with haemophilia B
22 November 2022 - Today, the US FDA approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with haemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, or have current or historical life-threatening haemorrhage, or have repeated, serious spontaneous bleeding episodes.
Hemgenix is a one time gene therapy product given as a single dose by intravenous infusion.
