A Phase 3 clinical trial investigating gene therapy candidate AMT-061 (etranacogene dezaparvovec) has reached its target of dosing 50 participants with moderate or severe hemophilia B. In fact, according to AMT-061 developer uniQure, 54 patients have been given the one-time dose of AMT-061 in the HOPE-B…

Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional factor VIII were able to ease blood loss in a mouse model of the disease, a study reported. These findings support the potential of induced…

Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ new hemophilia A cell therapy candidate, SIG-001, the company said. The trial is expected to begin by June. Last August, the U.S. Food and Drug Administration (FDA) granted the potential cell therapy…

It’s a struggle to balance life with two disorders. I need to take daily medications for epilepsy while also self-infusing to treat internal bleeds. I recently suffered a horrible epileptic episode. My wife, Cza, and I were walking with our baby, Cittie, when I felt a strong aura warn…

Over the past few years, I have learned that bleeding disorders are complex, particularly for women. Hemophilia is not always easy to understand or treat. It can take women up to 16 years to receive an accurate diagnosis. Then treatment plans are created, which can be challenging…

Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — were successfully grafted into hemophiliac mice, restoring blood levels of FVIII and significantly improving…

Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…

In an agreement expected to exceed $100 million, Medexus Pharmaceuticals has bought global commercial rights to Ixinity, an approved recombinant factor IX therapy for hemophilia B previously marketed by Aptevo Therapeutics. Sale terms include an upfront $30 million payment, plus up to $11 million in prospective…

I do not like to tell people that my sons are hemophiliacs. It is not that I am blind to the reality that they have a bleeding disorder, but my boys are so much more than a diagnosis. Why address them as if they carry a title or wear a…