Expert Interview
A look at uniQure’s Phase I/IIa GenTLE trial evaluating AMT-260, an investigational gene therapy candidate for refractory mesial temporal lobe epilepsy
Ticker(s): QUREInstitution: Johns Hopkins Bayview Medical Center
Professor of Neurology and Director of Epilepsy and EEG, specializing in epilepsy, clinical neurophysiology, and nonconvulsive status epilepticus
Clinical and research interests include eclampsia, neurologic disease in women, and the diagnosis and management of complex seizure disorders
Completed neurology residency and fellowships in epilepsy and clinical neurophysiology at Duke University Medical Center; trained at St. Bartholomew’s Medical School, University of London, and is a member of the Royal College of Physicians
What are your thoughts on the safety and preliminary efficacy data from the GenTLE trial evaluating AMT-260 in patients with refractory mesial temporal lobe epilepsy?
Added By: sara_adminGiven that AMT-260 uses an AAV9 vector to deliver miRNAs targeting GRIK2, do you see potential for off-target effects or long-term concerns with gene silencing in the hippocampus?
Added By: sara_adminIf these early findings are validated in larger trials, how might gene therapy like AMT-260 reshape treatment pathways for patients with drug-resistant MTLE?
Added By: sara_adminAre You Interested In These Questions?
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