AAV5-hFIX

AAV5-hFIX is a **gene therapy product** consisting of an adeno-associated virus serotype 5 (AAV5) vector carrying a codon-optimized gene for **human factor IX (hFIX)**, enabling expression of FIX in the liver following intravenous infusion. Designed as a one-time treatment for **hemophilia B (congenital Factor IX deficiency)**, its mechanism of action relies on delivery and subsequent expression of the hFIX gene to restore functional FIX protein levels and reduce bleeding episodes. Clinical studies show sustained increases in FIX activity and reduced bleeding rates in patients with severe or moderately severe hemophilia B. The product is a predecessor to later vectors (such as AMT-061/AAV5-hFIX-Padua and etranacogene dezaparvovec).

Other names

AAV5 containing a codon-optimized human factor IX geneAAV-5 containing a codon-optimized human factor IX geneAAV 5 containing a codon-optimized human factor IX gene