Prospect of hemophilia gene therapy closer to reality

Dutch gene therapy company uniQure (Nasdaq: QURE) and CSL Behring, part of Australia’s CSL Limited (ASX: CSL), have announced encouraging results on etranacogene dezaparvovec.

The adeno-associated virus five (AAV5)-based gene therapy, which is being investigated for the treatment of severe to moderately severe hemophilia B, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate (ABR) 18-months following administration compared to baseline Factor IX (FIX) prophylactic therapy in the pivotal Phase III HOPE-B gene therapy trial.

"The largest and first pivotal trial of a gene therapy for patients with hemophilia B"This study also successfully achieved a secondary endpoint demonstrating statistical superiority in reduction of ABR compared to baseline FIX prophylactic therapy.