uniQure Presents Data

uniQure N.V, a gene therapy company, presented data on successful repeated hepatic gene delivery with its AAV5 vector, following a proprietary immunoadsorption procedure in non-human primates (NHPs). This immune-adsorption procedure shows promising pre-clinical outcomes which can potentially become a successful strategy for readministration of a gene therapy in a therapeutic regimen, the company said in a press release Thursday.

The results describing successful sequential transduction with the two reporter transgenes were presented Thursday in a session at the American Society of Gene and Cell Therapy’s (ASGCT) 20th Annual Meeting in Washington, D.C., and highlight the progress made in readministration protocols for AAV gene transfers, the company said. Circulating anti-AAV neutralizing antibodies, which can pre-exist in patients or develop after a single administration of gene therapy, may prevent successful gene transfer in patients, according to Valerie Sier-Ferreira Ph.D., head of immunology at uniQure.

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