uniQure is engaged in the field of gene therapy, focusing to develop single treatments for patients suffering from genetic and other diseases. Co. is developing etranacogene dezaparvovec, a gene therapy for patients with hemophilia B that is designed to restore Factor IX (FIX) activity, a protein for blood clotting. Etranacogene dezaparvovec includes an adeno-associated virus (AAV) 5 vector incorporating the FIX-Padua variant. Co. is also developing AMT-180 for hemophilia A, AMT-190 for fabry disease, and AMT-130 for huntington's disease. Co.'s AMT-150 is a one-time, intrathecally-administered, AAV gene therapy incorporating Co.'s miQURE™ silencing technology.