uniQure N.V. announced on November 21, 2024, that the first patient has been dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE). This marks the third clinical trial initiation for uniQure within the past six months, demonstrating active pipeline progression.
AMT-260 is an investigational gene therapy utilizing an AAV9 vector to locally deliver two engineered miRNAs. These miRNAs are designed to reduce the expression of GluK2 protein subunits, which are believed to trigger seizure activity in patients with refractory mTLE. Preclinical studies showed AMT-260 reduced seizure frequency in a dose-dependent manner.
The GenTLE trial is a multi-center, open-label study in the U.S. evaluating the safety, tolerability, and exploratory efficacy of two doses of AMT-260. Refractory mTLE affects a significant portion of the over 600,000 individuals with temporal lobe epilepsy in the United States, representing a high unmet medical need for new treatment options.
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