uniQure N.V., a leading pharmaceutical specialized in human gene therapy, has presented additional data from its clinical trial in hemophilia B patients at the 21st Congress of the European Hematology Association (EHA),  June 9-12, in Copenhagen. The Phase 1/2 clinical trial of AMT-060 titled “Trial of…
Search results for:
A new analysis of the Phase 3 HAVEN 2 study’s results shows that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis (preventive treatment) leads to significant reductions in the number of bleeds among hemophilia A children younger than 12 with factor VIII inhibitors. The results showed that Hemlibra administered on different schedules — once a…
The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion date…
Raising sons with hemophilia is like being a blacksmith forging a sword. The blacksmith shapes a piece of metal that needs careful, skilled effort to become a sharp and strong sword. Each blow of the hammer represents the challenges brought by a bleeding disorder. The process is arduous and…
The Korean Ministry of Food and Drug Safety has agreed to Catalyst Biosciences’ request to add a sixth group to a Phase 1/2 clinical trial of its hemophilia B treatment. The sixth group in the trial (NCT03186677) of the therapy that Catalyst calls CB 2679d (ISU304) will include up…
Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes. Hemgenix is now the first and only gene therapy available to this patient group. “The approval of Hemgenix in Canada…
Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient perceptions. These patients reported that they choose their treatments based mainly on five criteria. Specifically, those are…
Xoma closed a $9.6 million deal to buy from Aptevo Therapeutics the rights to receive a payment stream for sales of Ixinity (trenonacog alfa), an approved medication used to prevent and treat bleeding episodes in people with hemophilia B. Under the terms of the deal, Xoma will…
The hemophilia team and design experts at Nationwide Children’s Hospital partnered with students from Ohio State University’s Advanced Computing Center for the Arts and Design (ACCAD) to develop a virtual reality game that aims to help the hospital’s pediatric hemophilia patients during procedures. Hemophilia patients often have to go through…