Huntington's Disease

A promising gene therapy for a rare, devastating disease


Huntington's Disease

Huntington’s disease (HD) is a rare, fatal, neurodegenerative genetic disorder that affects motor function and leads to behavioral symptoms and cognitive decline in young adults, resulting in total physical and mental deterioration over a 12 to 15-year period. Huntington’s disease affects approximately 70,000 people in the U.S. and Europe. 

Huntington’s disease is caused by the expansion of CAG trinucleotide in exon 1 of a multifunctional gene coding for protein called huntingtin. Despite the clear etiology, there are no therapies available to treat the disease, delay onset, or slow progression of a patient's decline.

uniQure is developing AMT-130, a gene therapy for Huntington’s disease (HD) that silences the huntingtin gene, with the goal of inhibiting the production of the mutant protein.  We are very encouraged by the significant reductions in mutant huntingtin protein that we have seen in our preclinical studies.

Read more here about AMT-130 and why we believe it has the potential to significantly alter the course of this disease.  

We are currently enrolling patients in the Phase I/II clinical trial of AMT-130.  Learn more about the Phase I/II study of AMT-130 here.  

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You can read more about uniQure's gene therapy approach to Huntington's disease in this brochure.


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> Gene therapy information (PDF)