Research Programs

Hero research programs

Exploring new applications for our gene therapy expertise.

As we focus on advancing our potentially transformative gene therapy for Huntington’s disease and enter the clinic in temporal lobe epilepsy, Fabry disease, and SOD1 ALS, we are looking farther out on the horizon for new, life-changing applications of our expertise and technologies.

Amyotrophic Lateral Sclerosis (ALS)

Our gene therapy candidate AMT-161 utilizes miQURE gene silencing technology to target toxic c9orf72 as a treatment for amyotrophic lateral sclerosis (ALS).

The most prevalent genetic defect causing ALS is a mutation in the c9orf72 gene, which acquires toxic properties resulting in degeneration starting in motor neurons in the spinal cord. AMT-161 is a one-time, intrathecally-administered AAV gene therapy using the miQURE silencing technology to target repeat-expanded c9orf72 to lower toxic RNA aggregates and prevent dipeptide protein formation.

Together with our AMT-162 program in ALS (SOD1), these two highly complementary ALS gene therapy candidates have the potential to address most familial forms of ALS and transform the lives of thousands of patients around the world suffering from this disease. 

Alzheimer’s Disease

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.

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