We are continuously advancing our broad pipeline of innovative gene therapies to bring new treatments to patients with severe genetic diseases.
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. There are an estimated 28,000 HD patients in the U.S. and approx.. 35,000 Huntington’s disease patients in the EU.
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 devastating disease.
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