uniQure updates follow up-data from hemophilia B therapy trials
Category: #health  By Saipriya Iyer  Date: 2019-12-09
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uniQure updates follow up-data from hemophilia B therapy trials

uniQure N.V, a renowned gene therapy firm that advances evolving therapies for all the patients who have severe medical needs, has recently confirmed that it has updated clinical data on three patients who were treated in the company’s current Phase IIb study of etranacogene dezaparvovec. It is an investigational AAV5-based gene therapy that contains a patent-protected variant of FIX-Padua to treat patients with critical or mildly critical hemophilia B.

Furthermore, uniQure also presents a follow-up data of 4 years made on 10 patients in the Phase I/II trial of AMT-060, the company’s first-gen gene therapy to treat hemophilia B. The clinical data is to be presented in Florida, at the 61st Annual Meeting of the American Society of Hematology (ASH) in Orlando.

The etranacogene dezaparvovec Phase IIb study is essentially a single-dose, multi-center, open-label and single-arm trial that has been conducted in the United States. Three patients suffering from severe hemophilia had been enrolled in the study and received a single intravenous infusion. The three patients had shown a low level of the already existing neutralizing antibodies to AAV5 before etranacogene dezaparvovec was administered.

The poster presentation at ASH will feature 52 weeks of follow-up data showing the three patients being stabilized and having sustained FIX activity at various therapeutic levels post one-time etranacogene dezaparvovec administration.

Steven Pipe, M.D., pediatrics professor, pediatric & pathology medical director of hemophilia and coagulation disorders program at University of Michigan as well as a main investigator in the clinical trial HOPE-B, stated that the updated data exhibit one administration of etranacogene dezaparvovec which has been well-taken for 52 weeks and has also accelerated the FIX activity in therapeutic range for people suffering from hemophilia B.

Pipe further added that the data show helpful clinical benefits for every patient  involved in the study over the full year, with no need for FIX replacement outside of surgery as well as not requirement for immunosuppression.

Source Credit- https://tools.eurolandir.com/tools/Pressreleases/GetPressRelease/?ID=3677182&lang=en-GB&companycode=nl-qure&v=



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Saipriya Iyer

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Saipriya Iyer

Saipriya Iyer develops content for Market Size Forecasters, Algosonline, and myriad other platforms. A computer engineer by profession, she ventured into the field of writing for the love of playing with words. Having had a previous experience of 3 years under her bel...

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