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Also known as AAV9-hSyn1-miGRIK2
AMT-260 · 1 trial · 1 indication
Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.
| Arm | Type | Description |
|---|---|---|
| AMT-260 | EXPERIMENTAL | Cohort 1: AMT-260 starting dose (1.0x 10E12 gc/mL). Cohort 2: AMT-260 adapted dose (6.0x 10E11 gc/mL or 3.0x 10E12 gc/mL). |
| Name | Type | Description |
|---|---|---|
| AAV9-hSyn1-miGRIK2 | GENETIC | 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. Intervention will be a one-time intracerebral administration of AMT-260. |
Inclusion Criteria: * Diagnosis of unilateral refractory MTLE * History of seizures with an average of ≥ 2 documented focal impaired awareness seizures or focal to bilateral tonic-clonic seizures per 30-day period over the 3 month Retrospective Period. * On a stable type and dose regimen of up to a...
AMT-260 is an investigational gene therapy being developed by uniQure N.V. for mesial temporal lobe epilepsy, a form of focal epilepsy that is often refractory to standard anti-seizure medications. It is designed as a one-time treatment approach and is currently in Phase 1 clinical development.
AMT-260 is being studied in adults with unilateral refractory mesial temporal lobe epilepsy, meaning seizures that continue despite treatment and originate from one side of the temporal lobe. It is an investigational therapy and has not been approved for any indication.
AMT-260 is developed by uniQure N.V., a gene therapy company whose shares trade on Nasdaq under the ticker QURE. uniQure is the sponsor of the Phase 1 study evaluating AMT-260 in adults with unilateral refractory mesial temporal lobe epilepsy.
AMT-260 is in Phase 1 clinical development. It is an investigational gene therapy and is not FDA approved. The single ongoing Phase 1 trial is currently recruiting participants and is expected to enroll 12 adults with unilateral refractory mesial temporal lobe epilepsy.
AMT-260 is being evaluated in the Phase 1 trial NCT06063850, titled AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy. The trial is recruiting in the United States, is uncontrolled, is not randomized or double blinded, and has a planned enrollment of 12 participants aged 18 years and older.
Yes, AAV9-hSyn1-miGRIK2 is another name for AMT-260. It describes the construct: an AAV9 viral vector carrying a human synapsin 1 promoter and a microRNA targeting GRIK2. Readers searching for AAV9-hSyn1-miGRIK2 are looking at the same uniQure gene therapy candidate for mesial temporal lobe epilepsy.