uniQure Initiates First Patient Dosing in GenTLE Trial for Epilepsy Treatment

uniQure N.V. (NASDAQ: QURE), a prominent firm in the field of gene therapy dedicated to developing innovative treatments for patients facing severe health challenges, has announced that the initial patient has received the dose in the GenTLE Phase I/IIa clinical study of AMT-260 aimed at addressing refractory mesial temporal lobe epilepsy (MTLE).

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"The initiation of dosing for our first patient in the Phase I/II study focused on temporal lobe epilepsy marks a significant achievement for uniQure, particularly as this is our third clinical trial launch within the last six months," mentioned Walid Abi-Saab, M.D., the Chief Medical Officer at uniQure. "Approximately one-third of individuals suffering from focal onset seizures do not find relief with existing therapies, leaving them with few viable treatment alternatives. Our investigational drug, AMT-260, which is administered just once, has the potential to provide a groundbreaking treatment option for these patients. We are actively in the process of screening participants for the trial and anticipate sharing program developments in the upcoming year."

AMT-260 utilizes an AAV9 vector to deliver two modified miRNAs that aim to diminish the expression of GluK2 protein subunits-a variant of the glutamate receptor thought to be inappropriately expressed in the hippocampus of those with refractory MTLE, a condition believed to initiate their seizures. In preclinical studies involving animals, AMT-260 demonstrated a dose-dependent reduction in the frequency of seizures per day. Furthermore, AMT-260 showed a decrease in both GluK2 mRNA and protein levels within the hippocampus of epileptic mice, as well as in resected hippocampal slices from patients suffering from refractory MTLE.

GenTLE is a Phase I/IIa multi-center, open-label study taking place in the U.S., aimed at assessing the safety, tolerability, and initial efficacy signals of two different doses of AMT-260 in participants with refractory MTLE. This trial is structured into two dosing groups, each comprising six patients. Recruitment is currently ongoing at ten sites, with plans to activate two additional sites by the conclusion of 2024. Further information can be found at www.clinicaltrials.gov (NCT06063850).

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According to the data provided by the Synapse Database, As of November 26, 2024, there are 1 investigational drug for the GluK2 x Kainic acid receptor target, including 2 indications, 2 R&D institutions involved, with related clinical trial reaching 1, and as many as 4 patents.

AMT-260 is an AAV-based gene therapy drug developed by uniQure biopharma BV. The drug targets GluK2 x Kainic acid receptor and is being developed for the treatment of epilepsy, specifically temporal lobe epilepsy. It is currently in the Phase 1/2 stage of development, indicating that it is being tested for safety and efficacy in a small group of patients.