FDA lifts clinical hold on uniQure's gene therapy amid cancer concerns; shares jump

After four months of hand-wringing and a deep investigation into how a patient was diagnosed with cancer after getting uniQure’s hemophilia gene therapy, the FDA is allowing the trial to continue.

In December, the FDA put uniQure’s hemophilia B gene therapy on clinical hold in response to a case of hepatocellular carcinoma (a form of liver cancer) in a patient in the pivotal trial of etranacogene dezaparvovec.