uniQure Receives FDA Fast Track Designation for AMT-130

AMT-130 gene therapy consistes of a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that non-selectively lowers or knocks-down human huntingtin protein in Huntington’s disease patients. According to uniyure, AMT-130 is unique in that it targets the exon1 protein fragment that is even more toxic than the mutant huntingtin protein.

Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene, that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington’s disease, there are no therapies to delay the onset or to slow the disease’s progression.

“Achieving Fast Track Designation from the FDA underscores the high unmet medical need for patients suffering from Huntington’s disease, for which there are currently no approved, disease-modifying treatments,” stated Matt Kapusta, chief executive officer of uniQure. .”

An antisense oligonucleotide (IONIS-HTTRx, RG6042), Roche licenced in 2017 from Ionis Pharmaceuticals, has already passed Phase I/II stage. The drug that blocks expression of defective huntingtin protein (mHTT) won PRIME status from the European drug watchdog European Medicines Agency (EMA) in 2018 because it reduced mHTT level by 40% – 60% in about 50 Huntington patients and is currently progressing into Phase III testing.