uniQure gets FDA clearance for AAV9 vector gene therapy

Published: 7-Sep-2023

The company has received clearance from the FDA for an investigational new drug application for AMT-260, a gene therapy candidate for uniQure

uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-260, the company’s gene therapy candidate for refractory mesial temporal lobe epilepsy (MTLE). 

AMT-260 comprises an AAV9 vector that locally delivers two engineered miRNAs 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.

“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,” said Walid Abi-Saab, Chief Medical Officer of uniQure. “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.”

There are few treatment options for patients who have refectory MTLE, and we are pleased to soon begin the clinical investigation

- Walid Abi-Saab, Chief Medical Officer of uniQure

The first-in-human Phase I/IIa clinical trial will be conducted in the United States and consist of two parts. The first part is a multicentre, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. 

The second part is expected to be a randomised, controlled trial to generate proof of concept (POC) data. The clinical trial is expected to begin patient screening in the fourth quarter of 2023.

AMT-260 is an AAV9 gene therapy product that locally delivers miRNA silencing technology to target the GRIK2 gene and suppress aberrantly expressed GluK2 containing kainate receptors. 

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The therapeutic goal is to lower the expression of GluK2 containing kainate receptors which are believed to trigger epilepsy when aberrantly expressed in the epileptic hippocampus. AMT-260 represents a novel potential one-time administered approach to treating refractory MTLE.

Temporal lobe epilepsy is a chronic neurologic disorder and is the most common form of focal epilepsywith more than 600,000 individuals suffering from the disorder in the United States. Approximately 80% of all temporal lobe epilepsy cases are mesial, which involves the medial or internal structures. The majority of MTLE cases are refractory to anti-seizure medications which severely limits treatment options.

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