Glybera
uniQure’s investigational gene therapy shown to slow Huntington’s disease progression
The neurodegenerative disorder affects approximately 70,000 people in Europe and the US
FDA removes clinical hold on uniQure’s haemophilia gene therapy
Clinical hold placed following a case of hepatocellular carcinoma in a patient in the trial
New results back Sangamo, UniQure haemophilia gene therapies
Encouraging results for firms in four-way race
UniQure edges closer to haemophilia B gene therapy OK
The candidate could free patients from regulator injections of FIX products
UniQure cleared to start Huntington gene therapy trial
AMT-130 becomes the first AAV-based therapy approved for trials in Huntington’s disease
UniQure skyrockets on haemophilia B gene therapy data
Competition with Pfizer and Spark rival heats up
UniQure’s starts pivotal haemophilia B gene therapy trial
The treatment could relieve haemophilia B patients from factor IX replacement drugs
EMA hands UniQure orphan status first in Huntington’s
The Dutch biotech expects to file the treatment for approval later this year
FDA grants orphan drug status to UniQure’s Huntington’s Disease treatment
Becomes first investigational gene therapy to receive ODD for the disease
uniQure appoints chief executive officer
Matthew Kapusta takes up the lead role in a permanent capacity