On November 22nd, 2022, the FDA approved the first gene therapy treatment for adults with hemophilia B.
This drug is an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
The introduction of gene therapy treatments is raising many new questions about the future of treatments and the role of Hemophilia Treatment Centers (HTCs).
