Huntington's Disease

A promising gene therapy for a rare, devastating disease

uniQure’s commitment to clinical development of AMT-130 in Huntington’s disease

March 29, 2021 – We understand that recent developments in clinical trials sponsored by other companies have caused a great deal of disappointment and anxiety within the HD community.  There had been significant interest in these trials, not only from participants and those close to them, but from the larger HD community. 

We at uniQure remain fully committed to the clinical development of AMT-130, the first AAV gene therapy to begin clinical development in HD. Our Phase I/II study continues to enroll patients in the first dose cohort, and we expect begin enrollment in the higher-dose second cohort in the second half of this year.  We are deeply appreciative of the support that we’ve received from the community and for the pioneering individuals who have volunteered to be a part of the study. Only through such collaborations can we develop safe and effective treatments to modify the course of HD.

We continue to believe that gene therapy remains a promising approach to treating Huntington’s disease, and look forward to continuing to work closely with the HD community as the program moves forward. 


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)