Uniqure's experimental gene therapy called etranacogene dezaparvovec (AMT-061), has entered Phase III of its studies and testing. The study has a goal of treating those with hemophilia B, which is caused by mutations in the Factor 9 gene. The treatment uses a harmless and altered adeno-like virus which consists of an AAV5 viral vector carrying a gene cassette (a type of a mobile genetic element that contains both a gene and a recombination site), with the Padua variant of Factor IX (FIX-Padua).
In June 2021, Uniqure announced positive 52-week data on all participants from the trial, (54 patients), which shows that these attendees proceeded to demonstrate durable and sustained Factor IX (FIX) increases at one year post-infusion.
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Uniqure's HOPE-B pivotal study is described by Uniqure as a "promising clinical program" and the company believes that "etranacogene dezaparvovec has the potential to be a first- and best-in-class gene therapy in hemophilia B, providing durable, functionally curative benefits to nearly all patients with hemophilia B."
Read more about the study here.
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