Etranacogene Dezaparvovec

**Semantic type:** Pharmacologic Substance

**Definition:** A non-replicating, recombinant adeno-associated virus serotype 5 (AAV5) encoding a variant of human coagulation factor IX with a gain-of-function Padua mutation (R338L), under control of a liver-specific promotor 1 (LP1), that may potentially be used to treat hemophilia B (congenital factor IX deficiency; Christmas disease). Upon administration of etranacogene dezaparvovec 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:** - AMT-061 - ETRANACOGENE DEZAPARVOVEC - Etranacogene Dezaparvovec-drlb - Etranacogene dezaparvovec - Hemgenix