uniQure Reports Early Clinical Data for Gene Therapy Candidate AMT-260 in Refractory Epilepsy

uniQure NV has announced initial six-month follow-up results from the first patient group enrolled in its ongoing Phase I/IIa clinical trial evaluating AMT-260, an investigational gene therapy for refractory mesial temporal lobe epilepsy (MTLE).

The data were presented at the Epilepsy Foundation Pipeline Conference held in Leesburg, Virginia, and provide an early look at the therapy’s safety and potential effectiveness in patients living with a severe form of epilepsy that often does not respond to currently available treatments.

Three Patients Achieved Significant Seizure Reduction

According to data collected through May 29, 2026, six patients received the low-dose version of AMT-260 at a concentration of 1x10¹² gc/mL.

Among these patients, three experienced meaningful reductions in disabling seizures during months four through six after treatment. The reduction ranged from 79% to 100% compared with their baseline seizure rates.

The remaining three patients showed mixed results. Changes in disabling seizures ranged from a 33% reduction to a 36% increase when compared with baseline levels.

While patient responses varied, the company believes the findings provide early signs that AMT-260 may be biologically active in treating refractory MTLE.

Favorable Safety Profile Reported

Safety remains a key focus in this early-stage study, and the results reported so far have been encouraging.

As of the presentation date, no serious adverse events related to AMT-260 or the surgical delivery procedure had been reported.

All adverse events observed in the low-dose group were classified as mild or moderate in severity. Headache was the most commonly reported side effect, occurring in two patients.

The company also reported that none of the treated patients required immunosuppressive therapy following treatment.

Company Sees Early Signs of Therapeutic Activity

Walid Abi-Saab, Chief Medical Officer of uniQure, said the results provide preliminary evidence that AMT-260 may be having a biological effect in some patients.

He noted that while the current data are based on a small number of participants and require longer follow-up, the combination of observed seizure reductions and favorable tolerability supports continued development of the therapy.

The company plans to gather additional data before drawing broader conclusions about the treatment’s potential.

Higher-Dose Cohort Enrollment Underway

Following the completion of the first low-dose cohort, uniQure is now enrolling patients into a second cohort that will receive a higher dose of AMT-260 at 3x10¹² gc/mL.

The second group is expected to include six patients, with enrollment anticipated to be completed by mid-2026.

Updated results from the ongoing Phase I/IIa study are expected to be presented during the first half of 2027.

Understanding the GenTLE Clinical Trial

The study, known as GenTLE, is a multi-center, open-label Phase I/IIa clinical trial being conducted in the United States.

The trial currently consists of two dosing groups, with up to six patients enrolled in each cohort. Participants receive a one-time intracerebral infusion of AMT-260 directly into the brain.

Patients are monitored during an initial 12-month evaluation period, followed by four years of long-term follow-up.

Researchers are assessing the safety, tolerability, and potential signs of efficacy of AMT-260 in individuals with refractory mesial temporal lobe epilepsy.

What Is AMT-260?

AMT-260 is a one-time gene therapy designed to reduce or potentially eliminate seizures in people with refractory mesial temporal lobe epilepsy.

The therapy works by delivering two engineered microRNAs directly into the affected brain region. These microRNAs are designed to suppress the GRIK2 gene and reduce the abnormal production of GluK2, a component of a kainate glutamate receptor that is believed to play a role in triggering seizures in patients with refractory MTLE.

By targeting one of the potential biological drivers of seizures, AMT-260 aims to provide a long-lasting treatment approach rather than relying on ongoing symptom management.

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