Gene therapy studies are occurring more and more, and the race to develop the first safe and effective treatment for bleeding disorders is growing in competition. Companies like Uniqure hold the studies and experiments all in the pursuit of a possible treatment.
Clinical trial data has been released from Uniqure's largest study of gene therapy with hemophilia B. Etranacogene Dezaparvovec, an "investigational adeno-associated virus," achieved clinical endpoint (the main result), of non-inferiority (What does non-inferiority mean?), in the annualized bleeding rate of 18-months after application related to baseline prophylactic treatment of Factor IX. Ricardo Dolmetsch, uniQure's president of research and development said about the achievement: “We are very pleased with these top-line results from what is the largest and first pivotal trial of a gene therapy for patients with hemophilia B... The HOPE-B data not only achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate following 12 months or more of stable FIX expression, but also the secondary endpoint of superiority in reduction of annualized bleeding, while continuing to demonstrate durability and stability in FIX levels and other benefits to this point in the study.”
UniQure reported that its improved treatment was better than standard preventive treatments at reducing the number of annual bleeds in patients. You can read the full study if you CLICK HERE.
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