Verbrinacogene Setparvovec

**Semantic type:** Pharmacologic Substance

**Definition:** A synthetic, liver-directed adeno-associated virus (AAV) capsid AAVS3 encoding a variant of human coagulation factor IX with a gain-of-function Padua mutation (R338L), that may potentially be used to treat hemophilia B (congenital factor IX deficiency; Christmas disease). Upon administration of verbrinacogene setparvovec and transduction of hepatocytes, human coagulation factor IX is produced. This replaces missing factor IX and may normalize the activated partial thromboplastin time (aPTT), increase blood clotting, and reduce and control bleeding episodes.

**Synonyms:** - FLT-180a - FLT180a - VERBRINACOGENE SETPARVOVEC - Verbrinacogene setparvovec