uniQure's has demonstrated preclinical proof-of-concept and initiated IND-enabling studies in Huntington's Disease.
Huntington’s Disease (HD)
uniQure is developing a gene therapy for Huntington’s disease, a severe genetic neurodegenerative disorder causing loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration over a 12 to 15 year period of time. The disease is caused by an autosomal dominant mutation, a cytosine-adenine-guanine (CAG) expansion, in the first exon of the Huntingtin gene leading to a non-functional, aggregation-prone mutated protein. Our product candidate AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA which silences the Huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. uniQure achieved preclinical proof-of-concept in early 2016 with the publication of data demonstrating the potential of a one-time administration of AAV5-delivered gene therapy to successfully silence the Huntingtin gene (view publication).