Contact SCGE




Gene Therapy Trial Report

Summary

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

NCTID NCT06063850 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Mesial Temporal Lobe Epilepsy
Disease Ontology Term DOID:3328
Compound Name AMT-260
Sponsor UniQure Biopharma B.V.
Funder Type Industry
Recruitment Status
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information

Target Gene/Variant MiGRIK2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2023-09-01
Completion Date 2031-12
Last Update 2025-11-20

Participation Criteria

Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 18
Locations United States

Regulatory Information

Has US IND True
FDA Designations
Recent Updates First 3 patients in first cohort enrolled September 2025

Resources/Links

News and Press Releases
Preclinical Publications