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uniQure reports on progress made in developing AMT-260
The Fly

uniQure reports on progress made in developing AMT-260

uniQure hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy, or rTLE, and other focal epilepsies in an event that featured a presentation from rTLE clinical expert Ellen Bubrick, MD, associate chair of Neurology at Harvard Medical School and director of the Epilepsy Surgery Program at Brigham and Women’s Hospital in Boston, MA. "The presentation highlighted preclinical data that supports the safety and tolerability of AMT-260 and plans for its clinical development, as well as uniQure’s miQURE and linQURE technology platforms that allow the Company to use miRNAs to safely reduce the expression of genes in the brain. The company also highlighted its progress in developing a commercial-scale AAV manufacturing platform," the company stated. "One week after the landmark FDA approval of the uniQure-developed world’s first gene therapy for hemophilia B, we are pleased to share the progress that we have made in developing AMT-260, a first-in-class gene therapy for temporal lobe epilepsy. Today’s event presented the preclinical data and the innovative technologies that we hope will allow us to develop AMT-260 and other gene therapies that can locally regulate neuronal activity for diseases of the central nervous system. We look forward to continuing to work toward submitting an investigational new drug application for AMT-260 and advancing it into the clinic in 2023," stated Ricardo Dolmetsch, Ph.D., president of research & development at uniQure.

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