etranacogene dezaparvovec

Etranacogene dezaparvovec is an adeno-associated virus serotype 5 (AAV5) vector-based gene therapy indicated for the treatment of adults with hemophilia B (congenital Factor IX deficiency). It delivers a codon-optimized DNA sequence encoding the gain-of-function Padua variant of human coagulation Factor IX, controlled by a liver-specific promoter. After intravenous administration, the therapy transduces hepatocytes, resulting in long-term expression of functional Factor IX protein and increased circulating activity levels. This reduces bleeding episodes and decreases or eliminates the need for exogenous factor IX replacement therapy. Etranacogene dezaparvovec is administered as a single intravenous infusion and was approved by the FDA in November 2022 as the first gene therapy for hemophilia B[2][5][6][7].

Brand names

Hemgenix

Other names

etranacogene dezaparvovec-drlb