uniQure N.V. announced on May 29, 2025, the presentation of a clinical case study for the first participant dosed in the GenTLE Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy (mTLE). The data were presented at the Epilepsy Therapies & Diagnostics Development Symposium.
The first patient, followed for five months post-administration of AMT-260, experienced a 92% reduction in seizure frequency. Prior to treatment, this participant had an average of seven seizures per month, including five in the 30 days immediately preceding dosing. Since receiving AMT-260, only two seizures were reported over the five-month follow-up period, with no seizures in the last 60 days as of the April 17th cutoff date.
Importantly, no serious adverse events were reported for this participant as of the cutoff date. AMT-260 is a one-time administered gene therapy designed to reduce or eliminate seizures by suppressing the GRIK2 gene and the aberrant expression of GluK2 protein. These early results provide a strong signal of potential therapeutic benefit for patients with drug-resistant mTLE.
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