Etranacogene dezaparvovec

Description

Etranacogene dezaparvovec is a gene-therapy medicine for adults with Hemophilia B (a deficiency of blood-clotting factor IX). In the U.S., it is marketed under the name Hemgenix. It uses a modified adeno-associated virus vector (AAV5) to deliver a functional gene for a high-activity variant of factor IX (the “Padua” variant) into liver cells so they can produce factor IX themselves.