uniQure rolls out gene therapy

Tuesday 7 March 2023


uniQure NV started 2023 with regulatory approvals in both the US and the EU for its gene therapy for haemophilia B as well as a pipeline of seven clinical and pre-clinical gene therapy projects – all in the field of neurology. Hemgenix (etranacogene dezaparvovec) for haemophilia B treats a genetic bleeding disorder and is the first gene therapy for this disease. Until its approval, the standards for haemophilia care were factor replacement therapies as well as a bispecific antibody treatment from Roche specifically developed for haemophilia A.