AAV2-hFIX16

**AAV2-hFIX16** is a gene therapy product consisting of an **adeno-associated virus serotype 2 (AAV2) vector carrying a modified human factor IX (hFIX16) gene cassette**. It is designed for hepatocyte-specific expression to treat **severe hemophilia B** by facilitating endogenous production of coagulation factor IX. The vector includes an AAV2 capsid, a human α-1 anti-trypsin promoter, APOE enhancer, hepatic control region, and human F9 cDNA interrupted by a 1.4-kb fragment of intron 1. Its therapeutic action is mediated by gene transfer leading to hepatic synthesis of factor IX. Although some subjects experienced transient or limited factor expression due to immune responses, long-term safety over 12–15 years has been demonstrated, with no major vector-related adverse events and no evidence of hepatocellular carcinoma or sustained liver toxicity.

Other names

AAV2-hFIX16AAV-2-hFIX16AAV 2-hFIX16