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AMT-240

Development stage: Preclinical
Lead developer: uniQure
Modality: Gene Therapies
Administration: Intracranial

Overview

AMT-240 is a one-time-administered investigational gene therapy developed by uniQure for the treatment of autosomal dominant Alzheimer’s disease. It utilizes uniQure’s miQURE gene-silencing technology to selectively silence the toxic APOE4 variant of apolipoprotein E (APOE), while simultaneously overexpressing a protective APOE variant, aiming to modify disease progression in patients at high genetic risk for Alzheimer’s disease[4].

Targets

APOE (Apolipoprotein E)

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