Dutch-American gene therapy company uniQure N.V. (NASDAQ: QURE) on Friday released initial six-month follow-up data from the first, low-dose cohort in its Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy, covering six patients.

As of the 29 May 2026 data cut-off, three patients receiving the 1x1012 gc/mL dose recorded reductions in disabling seizures of 79% to 100% during months four to six. The remaining three showed changes ranging from a 33% decrease to a 36% increase over the same period.

No serious adverse events related to AMT-260 or the surgical procedure have been reported to date. All adverse events were mild or moderate, with headache the most common, and no immunosuppression was required.

Enrolment is under way in a second cohort receiving a higher 3x1012 gc/mL dose, with completion expected by mid-2026. uniQure plans to report further Phase I/IIa results in the first half of 2027.

The GenTLE study is a US-based, multi-centre, open-label trial evaluating a one-time intracerebral infusion of AMT-260 in up to 12 patients, with a 12-month assessment period followed by four years of long-term follow-up. AMT-260 is designed to deliver two engineered microRNAs to suppress the GRIK2 gene and reduce aberrant GluK2 expression associated with seizure activity in refractory mesial temporal lobe epilepsy.