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Uniqure, the Dutch gene therapy company, said Monday that its one-time treatment for hemophilia B was not responsible for a previously reported case of liver cancer that put the program on clinical hold.

Findings from the investigation, conducted by an independent lab, were submitted to the Food and Drug Administration last week along with a request to restart the program, the company said.


The FDA placed the clinical hold on Uniqure’s hemophilia B gene therapy treatment, called etranacogene dezaparvovec, in December after a treated patient was diagnosed with liver cancer. At that time, the company had already completed patient dosing in three clinical trials, including its Phase 3 study called HOPE-B. Based on positive results from the HOPE-B study also presented in December, Uniqure had been moving forward with a plan to submit the treatment for approval this year.

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