Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy used for the treatment of hemophilia B (congenital factor IX deficiency). Hemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX.
- Mechanism
- Curator reviewed · 2 references
Etranacogene dezaparvovec is a non-replicating recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized DNA sequence of the gain-of-function Padua variant of human Factor IX (variant R338L) controlled by a liver-specific promotor 1. It is administered by a single intravenous infusion and delivers a copy of the gene in which hemophilia B patients are defective, leading to cell transduction and an eventual increase in circulating activity levels of factor IX.
- Primary indication
- Etranacogene dezaparvovec (Hemgenix) is indicated in the United States for the treatment of adults with hemophilia B who fit one of the following criteria: - Currently use factor IX prophylaxis therapy - Have current or...Curator reviewed · 7 structured indications
- First approval
- Canada, 2023 · United States, 2022 · European Union, 2023
- Code names
- AMT-061 · CSL222
Resolves to
What you can answer from here — as of November 21, 2025
Which drugs share a target with Etranacogene dezaparvovec, and which of those have an active Phase 3 trial?
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