HCB-101, hC Bioscience’s lead candidate to overwrite certain mutations that lead to hemophilia A, can reach the liver cells of mice, where it should then be able to drive the production of a working version of the blood-clotting protein that is faulty or missing in the disease. Such…

Using a baby’s own umbilical cells as vehicles to deliver factor VIII (FVIII) — the missing or defective clotting protein in hemophilia A — may be an effective, long-lasting, and more affordable therapeutic approach for this blood disorder, an early study suggests. The findings “lay the groundwork for future…

2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia A. Under this agreement, Novo Nordisk has the option to exclusively license 2seventy Bio’s proprietary mRNA-based megaTAL technology — customizable enzymes designed to recognize and cut specific DNA regions.

Switching to Jivi (damoctocog alfa pegol), an extended half-life replacement therapy, from Kovaltry (octocog alfa), may help adults and children with severe hemophilia A experience fewer or no bleeds, according to a Canadian study. “Data from this study provide routine clinical evidence supporting the use of [Jivi]…

Prophylactic, or preventive, treatment with factor VIII (FVIII) replacement therapy for four years reduced annual bleeding rates by 91% for people with severe hemophilia A, a study from Taiwan reported. After switching from on-demand treatment to prophylaxis, patients also experienced a 90.8% reduction in annual joint bleed rates and reported…

The U.S. Food and Drug Administration (FDA) has cleared for laboratory use CRYOcheck Factor VIII Deficient Plasma with VWF, a way of detecting factor VIII (FVIII) deficiency in blood samples. Now available in the U.S., Precision Biologic’s manufactured plasma — the liquid part of blood that…

Most people with hemophilia A on preventive treatment with Hemlibra (emicizumab) will experience spontaneous or traumatic bleeds at some point, according to a study using real-world data from Israel. Results also indicated the risk of spontaneous bleeds while on Hemlibra is higher for older individuals. “Our findings indicate that…

With 2019 behind us, it’s time for everyone to start anew and welcome 2020 into their lives. It’s hard to be fond of 2019 considering the impact my chronic illness had on my life. I spent practically a third of the year on bed rest because of severe…

European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…

A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with severe hemophilia A, with a planned advisory committee meeting having been canceled. The U.S. Food and Drug Administration (FDA) had requested, but now no longer intends, to meet with…