uniQure gets FDA fast track designation for Huntington’s disease gene therapy


uniQure (NASDAQ:QURE) received FDA fast track designation for AMT-130, a gene therapy candidate for the treatment of Huntington’s disease (HD).

HD is an inherited neurodegenerative disorder where production and aggregation of an abnormal protein in the brain leads to cognitive decline and loss of muscle coordination. AMT-130 has the potential to lower or knock-down this protein, know as huntingtin, in HD patients.

“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,” Matt Kapusta, uniQure’s CEO, said in a statement.

uniQure plans to initiate a Phase 1/2 study of AMT-130 in the second half of 2019.

Jennifer Poland