Steve Bryson, PhD,  science writer—

Eptacog beta was safe and almost 100% effective at stopping bleeds within 24 hours in boys ages 12 and younger with hemophilia A or B, according to final data from the Phase 3 PERSEPT 2 trial. According to researchers, this bypass agent may be an important option for…

Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy Roctavian appear to be influenced by regulatory molecules involved in F8 gene activity and FVIII protein folding and release, a study reveals. Researchers also found a dose-dependent relationship between…

Hemophilia B patients who switched to Rebinyn (nonacog beta pegol), an extended half-life factor IX (FIX) replacement therapy, had similar or better responses than with their previous standard or extended half-life FIX replacement therapies, a real-world study in Canada suggested. The study, “Switching to nonacog beta…

Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…

BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…

Preventive treatment with Esperoct (turoctocog alfa pegol) plus joint surgery significantly decreased annual joint bleeding rates, reduced pain, and improved mobility in hemophilia A patients, according to an analysis of Pathfinder trial data. “Our results therefore support the notion that patients with haemophilia A being treated with [Esperoct]…

Low-dose immune tolerance induction (ITI) — a type of treatment that can be used to eliminate inhibitors that limit the effectiveness of replacement therapies — achieved partial success in 80% of children with severe hemophilia A and high inhibitor levels, according to a recent study. The study, “…

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.

The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s investigational one-time gene therapy BBM-H901. According to the company, BBM-H901 is the first investigational gene therapy given intravenously, or directly into a vein, for hemophilia B or any genetic disease…

When given monthly to people with hemophilia A or B with inhibitors, fitusiran significantly reduced bleeding, and for some, fully eliminated them, according to data from the Phase 3 ATLAS-INH study.