AMT-260 - (GenTLE)

June 19, 2026: uniQure N.V. (NASDAQ: QURE) reported initial six-month follow-up data from the first (low-dose) cohort in its ongoing Phase I/IIa GenTLE trial of AMT-260, an investigational one-time in vivo gene therapy for refractory mesial temporal lobe epilepsy (MTLE), to be presented at the Epilepsy Foundation Pipeline Conference (Leesburg, VA). As of the May 29, 2026 data cutoff, 3 of 6 patients in the low-dose cohort (1×10^12 gc/mL) had meaningful reductions in disabling seizures during months 4–6, ranging from 79% to 100% declines from baseline; the other 3 patients showed variable changes (33% decrease to 36% increase). Safety was favorable: no serious adverse events related to AMT-260 or the surgical procedure; adverse events were mild/moderate, most commonly headache (n=2); no immunosuppression required. Enrollment is ongoing in a second, higher-dose cohort (3×10^12 gc/mL) of ~6 patients, with enrollment expected to complete mid-2026. Updated trial results are expected in the first half of 2027.