uniQure N.V. has received a fast track designation from the FDA for AMT-130, a gene therapy candidate for the treatment of Huntington’s disease. The agent comprises a recombinant AAV5 vector with a DNA cassette encoding a microRNA that non-selectively reduces human huntingtin protein in patients with Huntington’s disease. AMT-130 is unique as it targets the highly toxic exon1 protein fragment, which is more toxic than the mutant huntingtin protein.
Huntington’s disease affects an estimated three to seven per 100,000 people of European ancestry. Adult-onset Huntington’s disease, the most common form, usually appears during the ages of 30 to 40 years. The rarer juvenile form begins in childhood or adolescence, and typically progresses at a faster rate than the adult-onset form. Affected individuals usually live 10 to 15 years after signs and symptoms appear. There are no approved therapies to delay the onset or slow the progression of the disease.
uniQure is poised to begin the phase 1/2 study of AMT-130, which is the first, one-time administered adeno-associated viral gene therapy to enter clinical testing for Huntington’s disease. The company is on track to treat the first patient in the second half of 2019.
Source: uniQure N.V., April 8, 2019