uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ On Track to Treat First Patient in Phase I/II Study of AMT-130 in 2H19 ~

uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for AMT-130, the Company’s gene therapy candidate for the treatment of Huntington’s disease. AMT-130 comprises 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. AMT-130 also is unique in that it targets the highly toxic exon1 protein fragment that is even more toxic than the mutant huntingtin protein.

“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. “We are nearing the initiation of a Phase I/II study of AMT-130, the first one-time administered AAV gene therapy to enter clinical testing for Huntington’s disease, and are on track to treat the first patient in the second half of 2019.”

The FDA's Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. A therapy granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA) or New Drug Application (NDA).

Source: uniQure

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