Recalling the cloud: uniQure announces that gene therapy for hemophilia is "very unlikely" to cause lung cancer and will resume clinical trials

On March 29, local time, after actively cooperating with the U.

Hemophilia is a hereditary bleeding disorder caused by the lack or defect of coagulation factor IX.

AMT-061 is a gene therapy based on adeno-associated virus 5 (AAV-5) by uniQure following AMT-060.

In November last year, uniQure announced the results of a pivotal phase III clinical trial of AMT-061 called "HOPE-B".

One month later, uniQure announced the suspension of its phase III clinical trial for the treatment of hemophilia B.

On March 29, 2021, after nearly four months of investigation, uniQure announced that its AMT-061 gene therapy was "very unlikely" to cause lung cancer symptoms in patients with hemophilia B undergoing treatment.

In early February this year, Bluebird Bio also had such an oolong incident.

Currently, uniQure is preparing to negotiate with the FDA to resume its previously suspended clinical trials for hemophilia B.