uniQure doses first patient in Phase I/IIa trial of AMT-260 for refractory temporal lobe epilepsy

uniQure N.V.

First patient dosed in GenTLE Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy (MTLE).
AMT-260 uses AAV9 vector to deliver miRNAs that reduce GluK2 protein subunit expression, targeting seizure triggers.
Trial includes two dose cohorts of six patients each across 10 U.S. sites; two more sites expected by end of 2024.
CMO Walid Abi-Saab notes 1/3 of focal seizure patients lack effective treatment, highlighting potential for AMT-260.
Preclinical studies showed dose-dependent seizure reduction; program updates expected in 2025.

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