Headquartered in Paasheuvelweg 25, Amsterdam Fiscal year ends December NASDAQ: QURE uniqure.com
uniQure N.V. (NASDAQ: QURE) is a gene therapy company developing one-time treatments intended to be potentially curative for severe diseases. Its gene therapy for hemophilia B has received regulatory approvals, and the company earns license revenue tied to that product. Its lead proprietary candidate is ifezuntirgene inilparvovec (AMT-130) for Huntington's disease. The FDA has accepted a BLA for AMT-130 under the Accelerated Approval pathway with Priority Review and a PDUFA date of May 1, 2027, and the U.K. MHRA has validated a Marketing Authorization Application. The company is building commercial infrastructure, including field medical, market access and strategic account teams, and is preparing a roughly 200-patient randomized confirmatory study. Its pipeline also includes AMT-260 for refractory mesial temporal lobe epilepsy and AMT-191 for Fabry disease, along with preclinical and technology innovation projects. Updated
The wider loss is the cost of getting ready to sell a Huntington's disease gene therapy: SG&A climbed to $23.9 million from $19.4 million on commercial hiring ahead of the May 1, 2027 FDA decision date on AMT-130. Both lines came in below estimates, but $768.4 million in cash and investments funds the company into 2030.
uniQure lost more than a third of its value in a single session after Huntington's data spooked investors, but Wall Street's price target still points to triple-digit upside. Whether that gap represents…
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uniQure N.V. (NASDAQ: QURE) is a gene therapy company developing one-time treatments intended to be potentially curative for severe diseases. Its gene therapy for hemophilia B has received regulatory approvals, and the company earns license revenue tied to that product.
The Wall Street consensus 12-month price target for QURE is $60.03.
