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AAV9-hSyn1-miGRIK2 · 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...
AAV9-hSyn1-miGRIK2 is an investigational gene therapy being developed for the treatment of mesial temporal lobe epilepsy, a form of epilepsy that originates in the medial part of the temporal lobe. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
AAV9-hSyn1-miGRIK2 targets the GRIK2 gene, which encodes a subunit of kainate-type glutamate receptors. By delivering a microRNA designed to reduce GRIK2 expression, the therapy aims to modulate neuronal excitability in the brain, potentially reducing seizure activity in patients with mesial temporal lobe epilepsy.
AAV9-hSyn1-miGRIK2 is being developed by uniQure N.V., a biotechnology company publicly traded under the ticker symbol QURE. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for mesial temporal lobe epilepsy.
AAV9-hSyn1-miGRIK2 is in Phase 1 clinical development. It is an investigational therapy, meaning it has not yet received regulatory approval and is being studied in a clinical trial to assess its safety and potential efficacy in patients with mesial temporal lobe epilepsy.
AAV9-hSyn1-miGRIK2 is being evaluated in a Phase 1 clinical trial with the identifier NCT06063850. This study, titled 'AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy,' is currently recruiting participants in the United States and aims to enroll 12 adults aged 18 years and older.
Yes, AAV9-hSyn1-miGRIK2 is also known as AMT-260. The clinical trial NCT06063850, which is investigating this gene therapy for mesial temporal lobe epilepsy, uses the name AMT-260 in its title, confirming that both names refer to the same investigational drug.