ASH 2020: Late-breaking data on etranacogene dezaparvovec gene therapy

Dutch gene therapy company uniQure (Nasdaq: QURE) today announced the late-breaking presentation of initial data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.

This is the first data set to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of patients receiving a single gene therapy investigational product to be reported to date.

The data will be presented today in an oral session at the virtual 62nd Annual Meeting of the American Society of Hematology (ASH).