An innovative gel containing two snake venom proteins known to help blood clotting, rapidly and effectively forms stable clots and reduces bleeding even in the presence of a blood-clotting disorder, as is the case in hemophilia, a preclinical study shows. The gel, which transitions to a liquid form below…

My 16-year-old son, Caeleb, is on summer break. He told me that summer is a little too boring and the school year is a little too much. He is definitely a teenager. Next month he begins summer band, and boredom will become exhaustion. Caeleb is already concerned about marching…

ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy for hemophilia A. The agreement expands upon their current collaboration, begun in 2019 that focused on achieving Good Manufacturing Practice (GMP)-virus manufacturing and establishing processes for adeno-associated virus (AAV) production…

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…

Children and young adults with hemophilia A and hemophilia B have several behavioral problems, ranging from depression and anxiety to aggressive behaviors, a small study suggests. Age, disease severity, and joint disease duration were found to be significantly correlated with several of these problems. The findings were reported in the…

Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in which it did so varied, a recent study reported. Researchers noted that while findings support the use of this…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) prevents bleeds and the need for preventive treatment, or prophylaxis, for up to six years in men with severe hemophilia A, according to updated data from a Phase 1/2 trial. “With every year of observation in this…

Caeleb, the child doctors said I’d never have, has changed my life. He is 16 and thinking about his future. That may sound like a normal thing for a 16-year-old, but I was unsure about his future with his hemophilia A. His life with this disease has been…

The effectiveness of liver-directed, viral-based gene transfer — the most used experimental gene therapy approach for hemophilia A — is significantly reduced in mouse models of hemophilia A relative to healthy mice, a study shows. This limitation was associated with a pronounced reduction in fenestrations — cell surface…