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The following is an excerpt from a press release from uniQure. Read the full press release here.


uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented data showing successful liver transduction with the AAV5 vector in both non-human primates and humans with pre-existing anti-AAV5 neutralizing antibodies (NABs). In a study re-analyzing pre-treatment sera samples of the ten patients in the Phase I/II clinical trial of AMT-060, no relationship was detected between the presence of pre-treatment anti-AAV5 NABs and clinical outcomes of AMT-060 in patients with hemophilia B.

The data were presented on Saturday, May 19 by Anna Majowicz, Ph.D., a scientist in uniQure’s immunology division, in an oral session at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Chicago, Illinois, the preeminent gene and cell therapy conference in the world.

“These important data presented at ASGCT bolster our confidence that AAV5 gene therapies can provide successful liver transduction in all or nearly all patients,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer of uniQure. “The study suggests that, in contrast to experience with other AAV vectors, detectable pre-existing neutralizing antibodies do not prevent successful gene transfer using AAV5 at clinical doses. In patients pre-exposed to AAV5 who tested positive for anti-AAV5 antibodies, therapeutic transgene expression was established with no cellular immune response observed after systemic administration of AAV5.”

Read the press release in its entirety here.

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