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uniQure has announced that the FDA has given clearance to its Investigational New Drug Application for AMT-269 gene therapy for refractory mesial temporal lobe epilepsy with clinical trials expected to commence in the fourth quarter of 2023.
Leading gene therapy company, uniQure, has announced the FDA clearance of its IND application for the company’s gene therapy candidate for refractory mesial temporal epilepsy (MTLE), AMT-260. Temporal lobe epilepsy is a chronic neurologic disorder.
AMT-260 comprises an AAV9 vector that locally delivers two engineered miRNSs designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE.
Walid Abi-Saab, chief medical officer of uniQure stated:
“The clearance of the IND for AMT-260 is an important achievement in advancing our pipeline and is our next program to enter clinical development in an area of high unmet medical need.”
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More than 600,000 individuals suffer from the disorder in the United States with approximately 80% of all temporal lope epilepsy cases being mesial – involving the medial or internal structures. The majority of MTLE cases are refractory to anti-seizure medications. AMT-260 represents a novel potential one-time administered approach to treating refractory MTLE.
Abi-Saab continued:
“There are few treatment options for patients who have refectory MTLE, and we are pleased to soon begin the clinical investigation of this one-time administered gene therapy approach as a potential new treatment.”
The first-in-human Phase I/IIa clinical trial will be conducted in the United States and consist of two parts: a multlicenter, open-label trial with two dosing cohorts of six patients and a randomized controlled trial. The clinical trial is expected to begin patient screening in the fourth quarter of 2023.
uniQure will be leveraging its modular and validate technology and manufacturing platform to advance a pipeline of proprietary gene therapies for treatment of patients with varying conditions such as Huntingdon’s disease, MTLE, amyotrophic lateral sclerosis (ALS), Fabry disease and other severe diseases.
Source: uniQure Press Release
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