UniQure rises on orphan drug status for genetic disorder gene therapy

uniQure

23/09/2024 13:03

** U.S.-listed shares of Netherlands-based gene therapy
company UniQure NV  UQ1.F  rise 2.2% to $5.48 premarket   
    ** Co says the U.S. FDA has granted orphan drug designation
to AMT-191, its experimental gene therapy for the treatment of
Fabry disease  
    ** Fabry disease is a rare inherited disorder in which fat
gets deposited in the body's cells, resulting in cell
abnormalities and organ dysfunction
    ** Co plans to provide initial data from an early-mid stage
study in 2025
    ** Orphan drug status is given by the FDA to drugs that
treat rare conditions and are protected through a series of
incentives once in market 
    ** Up to last close, U.S.-listed stock down ~21% YTD 

 (Reporting by Mariam Sunny in Bengaluru)
 ((Mariam.ESunny@thomsonreuters.com;))