In June 2021, uniQure introduced new candidate AMT-240, a new treatment modality for autosomal dominant Alzheimer’s disease.
Alzheimer’s disease is the most prevalent neurodegenerative disease, causing dementia and subsequent gradual loss of ability to function with disease progression. Apolipoprotein E (APOE) has been shown to be a key player in the pathogenesis of Alzheimer’s disease. APOE consists of 3 major isoforms that are structurally and functionally different. The APOE4 isoform is found to be the largest risk factor to develop Alzheimer’s. In contrast to the toxic properties of APOE4, clinical case studies have shown the protective role of other APOE variants.
AMT-240 is a one-time-administered gene therapy using uniQure’s miQURE gene-silencing technology to silence the toxic APOE variant, in combination with overexpressing a protective APOE variant as treatment for autosomal dominant Alzheimer’s disease patients.
