Etranacogene Dezaparvovec

ATC Code B02BD16

Etranacogene Dezaparvovec

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.

Also known as AMT-061, ETRANACOGENE DEZAPARVOVEC, Etranacogene Dezaparvovec-drlb, Hemgenix — per NCIT

Also identified as