uniQure
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There is hope for uniQure’s HOPE-B trial after all. Shares of uniQure NV are climbing this morning after the company announced the clinical hold on its hemophilia B gene therapy has been lifted by the U.S. Food and Drug Administration.
It was a busy week for clinical trial news. Here’s a look.
An investigation was conducted by an independent laboratory and reviewed by leading external experts in the field.
In a shining moment that signaled hope for hemophilia B patients, uniQure presented data on Tuesday showing that its gene therapy treatment, etranacogene dezaparvovec (AMT-061), substantially increased production of the blood-clotting protein factor IX in nearly all pivotal Phase III HOPE-B trial participants.
In June, it dosed its first two patients using a novel therapeutic, AMT-130, delivered directly to the brain. Initial readouts are expected in a year’s time.
CSL Behring bolstered its growing gene therapy portfolio with the acquisition of global rights to uniQure’s gene therapy program for hemophilia B that has a price tag of up to $2 billion.
uniQure N.V. announced the publication of 26-week interim data from the ongoing Phase IIb clinical trial of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B, in the journal, Blood Advances.
uniQure N.V. announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.
Companies from across Europe and Asia share updates on their business and pipelines.
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