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The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.


uniQure N.V.,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive top-line 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. These clinical data were published today as a late-breaking abstract, one of only six accepted for presentation at the 62nd聽Annual Meeting of the American Society of Hematology (ASH) and will be featured as an oral presentation in the conference on Dec. 8, 2020. The abstract is available here.

鈥淲e are extremely pleased that these top-line pivotal data show that a single administration of etranacogene dezaparvovec gene therapy led to sustained increases of Factor IX (FIX) to functionally-curative levels capable of eliminating the need for regular infusions to control and prevent bleeding episodes,鈥 stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. 鈥淢ost impressively, these data also demonstrate the potential to achieve clinical benefit in patients with a range of pre-existing neutralizing antibodies representative of the general population. The ability to dose a gene therapy in patients with pre-existing neutralizing antibodies has not been demonstrated for any other gene therapy and illustrates the potentially unique ability of our AAV5 platform to address the needs of a broad set of patients living with hemophilia B and other disorders.鈥

The pivotal, Phase III HOPE-B clinical trial of etranacogene dezaparvovec is an open-label, single-dose, single-arm, multi-national trial in adult males with severe or moderately severe hemophilia. All patients required prophylactic routine FIX replacement prior to entering the clinical trial, and patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.

Patients in the HOPE-B clinical study were initially enrolled into a prospective, observational lead-in period of at least six months during which bleeding events and FIX replacement therapy usage were monitored. Fifty-four patients received a single intravenous infusion of etranacogene dezaparvovec gene therapy at 2×1013 gc/kg, including 23 patients who had pre-existing NAbs to AAV5. Patients are then evaluated to assess FIX activity determined by a one-stage assay performed at a central laboratory, annualized bleeding rates and usage of Factor IX replacement therapy. Patients will be monitored for five years to evaluate the safety of etranacogene dezaparvovec.

Read the press release in its entirety here.聽


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