uniQure N.V. presented new clinical data from the ongoing Phase I-II trial of AMT-060.
uniQure N.V. (NASDAQ:QURE) presented new clinical data from the ongoing Phase I-II trial of AMT-060.
As quoted in the press release:
Clinical data [demonstrates] that the presence of pre-existing anti-AAV5 neutralizing antibodies (NABs) does not predict the potential efficacy of AAV5-mediated gene transfer in patients with hemophilia B. Clinically meaningful factor IX (FIX) activity levels from the ongoing Phase I-II trial of AMT-060 were observed at NAB titers up to 1:341, determined as corresponding up to the 90th percentile of a healthy control population. NABs were quantified in the blood sera of these patients using a highly sensitive assay. These clinical data were presented today in a poster presentation at the 26th Biennial Congress of the International Society on Thrombosis and Hemostasis (ISTH), taking place this week in Berlin, Germany.
The presence of pre-existing NABs to adeno-associated virus (AAV) vectors has long posed a critical challenge for the clinical application of gene therapies, as patients who currently screen positive for NABs are generally excluded from treatment. Researchers from uniQure recently presented data in non-human primates suggesting that AAV5 could successfully mediate gene transfer in the presence of NABs at levels as high as 1:1031.