Business
uniQure Highlights Therapeutic Potential of AMT-260 in Refractory Temporal Lobe Epilepsy (rTLE) at Virtual Research & Development Event
~ IND Submission and Clinical Development Expected to Begin in 2023 ~ ~ Advancements in Manufacturing and Technology Platform Also Highlighted ~ LEXINGTON,
About this update from Uniqure N.v.
[{"type":"text","content":"~ IND Submission and Clinical Development Expected to Begin in 2023 ~ ~ Advancements in Manufacturing and Technology Platform Also Highlighted ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 29, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy (rTLE) and other focal epilepsies. The event 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 on the unmet medical need of patients with rTLE. 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. A replay of the investor event is available here. “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,” stated Ricardo Dolmetsch, Ph.D., president of research & development at uniQure. “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.” AMT-260 in refractory Temporal Lobe Epilepsy (rTLE) AMT-260 is an AAV gene therapy that uses an AAV9 vector and combines uniQure’s miQURE and linQURE technologies to deliver multiple miRNAs that reduce the expression of the GRIK2 gene. The GRIK2 gene encodes a subunit of the kainate glutamate receptor, which plays a key role in neuronal excitability and in the pathogenesis of rTLE. It is the first example of uniQure’s b...