uniQure is a leading gene therapy company advancing transformative therapies for patients with severe medical needs.
The company has engineered an artificial microRNA candidate, AMT-150, to target the ataxin-3 gene in a SCA3 knock-in mouse model.
These results were corroborated by preclinical studies in human induced Pluripotent Stem Cell (iPSC)-derived neurons showing a dose-dependent lowering of ataxin-3 mRNA of up to 55%.
These studies further demonstrate the potential utility and safety profile of the miQURE™ technology, the Company’s proprietary gene-silencing platform.
“We believe that the data from these preclinical studies in the knock-in mouse model and in iPSC-derived neurons show the potential of AMT-150 to alter the course of this devastating disease after a single administration,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure.
Press release, 7 May 2019